Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.
Identifieur interne : 000618 ( Main/Exploration ); précédent : 000617; suivant : 000619Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.
Auteurs : Mark W. Kieran [France] ; Julia Chisholm [France] ; Michela Casanova [France] ; Alba A. Brandes [France] ; Isabelle Aerts [France] ; Eric Bouffet [France] ; Simon Bailey [France] ; Sarah Leary [France] ; Tobey J. Macdonald [France] ; Francoise Mechinaud [France] ; Kenneth J. Cohen [France] ; Riccardo Riccardi [France] ; Warren Mason [France] ; Darren Hargrave [France] ; Stacey Kalambakas ; Priya Deshpande [France] ; Feng Tai [France] ; Eunju Hurh ; Birgit Geoerger [France]Source :
- Neuro-oncology [ 1523-5866 ] ; 2017.
Abstract
Sonidegib (LDE225) is a potent, selective hedgehog (Hh) inhibitor of Smoothened. This study explored the safety and pharmacokinetics of sonidegib in children with relapsed/recurrent tumors followed by a phase II trial in pediatric and adult patients with relapsed medulloblastoma (MB) to assess tumor response.
DOI: 10.1093/neuonc/nox109
PubMed: 28605510
Affiliations:
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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.</title><author><name sortKey="Kieran, Mark W" sort="Kieran, Mark W" uniqKey="Kieran M" first="Mark W" last="Kieran">Mark W. Kieran</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Chisholm, Julia" sort="Chisholm, Julia" uniqKey="Chisholm J" first="Julia" last="Chisholm">Julia Chisholm</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Casanova, Michela" sort="Casanova, Michela" uniqKey="Casanova M" first="Michela" last="Casanova">Michela Casanova</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Brandes, Alba A" sort="Brandes, Alba A" uniqKey="Brandes A" first="Alba A" last="Brandes">Alba A. Brandes</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Aerts, Isabelle" sort="Aerts, Isabelle" uniqKey="Aerts I" first="Isabelle" last="Aerts">Isabelle Aerts</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Bouffet, Eric" sort="Bouffet, Eric" uniqKey="Bouffet E" first="Eric" last="Bouffet">Eric Bouffet</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Bailey, Simon" sort="Bailey, Simon" uniqKey="Bailey S" first="Simon" last="Bailey">Simon Bailey</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Leary, Sarah" sort="Leary, Sarah" uniqKey="Leary S" first="Sarah" last="Leary">Sarah Leary</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Macdonald, Tobey J" sort="Macdonald, Tobey J" uniqKey="Macdonald T" first="Tobey J" last="Macdonald">Tobey J. Macdonald</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Mechinaud, Francoise" sort="Mechinaud, Francoise" uniqKey="Mechinaud F" first="Francoise" last="Mechinaud">Francoise Mechinaud</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Cohen, Kenneth J" sort="Cohen, Kenneth J" uniqKey="Cohen K" first="Kenneth J" last="Cohen">Kenneth J. Cohen</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Riccardi, Riccardo" sort="Riccardi, Riccardo" uniqKey="Riccardi R" first="Riccardo" last="Riccardi">Riccardo Riccardi</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Mason, Warren" sort="Mason, Warren" uniqKey="Mason W" first="Warren" last="Mason">Warren Mason</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Hargrave, Darren" sort="Hargrave, Darren" uniqKey="Hargrave D" first="Darren" last="Hargrave">Darren Hargrave</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Kalambakas, Stacey" sort="Kalambakas, Stacey" uniqKey="Kalambakas S" first="Stacey" last="Kalambakas">Stacey Kalambakas</name></author><author><name sortKey="Deshpande, Priya" sort="Deshpande, Priya" uniqKey="Deshpande P" first="Priya" last="Deshpande">Priya Deshpande</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Tai, Feng" sort="Tai, Feng" uniqKey="Tai F" first="Feng" last="Tai">Feng Tai</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Hurh, Eunju" sort="Hurh, Eunju" uniqKey="Hurh E" first="Eunju" last="Hurh">Eunju Hurh</name></author><author><name sortKey="Geoerger, Birgit" sort="Geoerger, Birgit" uniqKey="Geoerger B" first="Birgit" last="Geoerger">Birgit Geoerger</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PubMed</idno><date when="2017">2017</date><idno type="RBID">pubmed:28605510</idno><idno type="pmid">28605510</idno><idno type="doi">10.1093/neuonc/nox109</idno><idno type="wicri:Area/PubMed/Corpus">000433</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Corpus" wicri:corpus="PubMed">000433</idno><idno type="wicri:Area/PubMed/Curation">000431</idno><idno type="wicri:explorRef" wicri:stream="PubMed" wicri:step="Curation">000431</idno><idno type="wicri:Area/PubMed/Checkpoint">000431</idno><idno type="wicri:explorRef" wicri:stream="Checkpoint" wicri:step="PubMed">000431</idno><idno type="wicri:Area/Ncbi/Merge">004966</idno><idno type="wicri:Area/Ncbi/Curation">004966</idno><idno type="wicri:Area/Ncbi/Checkpoint">004966</idno><idno type="wicri:Area/Main/Merge">000613</idno><idno type="wicri:Area/Main/Curation">000618</idno><idno type="wicri:Area/Main/Exploration">000618</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en">Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.</title><author><name sortKey="Kieran, Mark W" sort="Kieran, Mark W" uniqKey="Kieran M" first="Mark W" last="Kieran">Mark W. Kieran</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Chisholm, Julia" sort="Chisholm, Julia" uniqKey="Chisholm J" first="Julia" last="Chisholm">Julia Chisholm</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Casanova, Michela" sort="Casanova, Michela" uniqKey="Casanova M" first="Michela" last="Casanova">Michela Casanova</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Brandes, Alba A" sort="Brandes, Alba A" uniqKey="Brandes A" first="Alba A" last="Brandes">Alba A. Brandes</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Aerts, Isabelle" sort="Aerts, Isabelle" uniqKey="Aerts I" first="Isabelle" last="Aerts">Isabelle Aerts</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Bouffet, Eric" sort="Bouffet, Eric" uniqKey="Bouffet E" first="Eric" last="Bouffet">Eric Bouffet</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Bailey, Simon" sort="Bailey, Simon" uniqKey="Bailey S" first="Simon" last="Bailey">Simon Bailey</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Leary, Sarah" sort="Leary, Sarah" uniqKey="Leary S" first="Sarah" last="Leary">Sarah Leary</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Macdonald, Tobey J" sort="Macdonald, Tobey J" uniqKey="Macdonald T" first="Tobey J" last="Macdonald">Tobey J. Macdonald</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Mechinaud, Francoise" sort="Mechinaud, Francoise" uniqKey="Mechinaud F" first="Francoise" last="Mechinaud">Francoise Mechinaud</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Cohen, Kenneth J" sort="Cohen, Kenneth J" uniqKey="Cohen K" first="Kenneth J" last="Cohen">Kenneth J. Cohen</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Riccardi, Riccardo" sort="Riccardi, Riccardo" uniqKey="Riccardi R" first="Riccardo" last="Riccardi">Riccardo Riccardi</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Mason, Warren" sort="Mason, Warren" uniqKey="Mason W" first="Warren" last="Mason">Warren Mason</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Hargrave, Darren" sort="Hargrave, Darren" uniqKey="Hargrave D" first="Darren" last="Hargrave">Darren Hargrave</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Kalambakas, Stacey" sort="Kalambakas, Stacey" uniqKey="Kalambakas S" first="Stacey" last="Kalambakas">Stacey Kalambakas</name></author><author><name sortKey="Deshpande, Priya" sort="Deshpande, Priya" uniqKey="Deshpande P" first="Priya" last="Deshpande">Priya Deshpande</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Tai, Feng" sort="Tai, Feng" uniqKey="Tai F" first="Feng" last="Tai">Feng Tai</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author><author><name sortKey="Hurh, Eunju" sort="Hurh, Eunju" uniqKey="Hurh E" first="Eunju" last="Hurh">Eunju Hurh</name></author><author><name sortKey="Geoerger, Birgit" sort="Geoerger, Birgit" uniqKey="Geoerger B" first="Birgit" last="Geoerger">Birgit Geoerger</name><affiliation wicri:level="1"><nlm:affiliation>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif, France.</nlm:affiliation><country xml:lang="fr">France</country><wicri:regionArea>Pediatric Neuro-Oncology, Dana-Farber Boston Children's Cancer Blood Disorders Center, Harvard Medical School, Boston, Massachusetts, USA; The Royal Marsden Hospital, Sutton, Surrey, UK; Fondazione IRCCS Istituto Nazionale dei Tumori, Milan, Italy; Department of Medical Oncology, Azienda USL-IRCCS Institute of Neurological Science, Bologna, Italy; Institut Curie and University Paris Descartes, Paris, France; Division of Haematology/Oncology at The Hospital for Sick Children, Toronto, Ontario, Canada; Sir James Spence Institute of Child Health Royal Victoria Infirmary, Newcastle, UK; Seattle Children's Hospital, University of Washington and Fred Hutchinson Cancer Research Center, Seattle, Washington, USA; Aflac Cancer and Blood Disorders Center, Children's Healthcare of Atlanta, Emory University School of Medicine, Atlanta, Georgia, USA; The Royal Children's Hospital, Children's Cancer Center, Melbourne, Australia; The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins, Baltimore, Maryland, USA; Catholic University of the Sacred Heart, Rome, Italy; Cancer Clinical Research Unit, Princess Margaret Cancer Centre, Toronto, Ontario, Canada; Paediatric Oncology Unit, Great Ormond Street Hospital, London, UK; Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA; Gustave Roussy, Department of Pediatric and Adolescent Oncology, University Paris-Sud, Université Paris-Saclay, Villejuif</wicri:regionArea><wicri:noRegion>Villejuif</wicri:noRegion><wicri:noRegion>Villejuif</wicri:noRegion></affiliation></author></analytic><series><title level="j">Neuro-oncology</title><idno type="eISSN">1523-5866</idno><imprint><date when="2017" type="published">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">Sonidegib (LDE225) is a potent, selective hedgehog (Hh) inhibitor of Smoothened. This study explored the safety and pharmacokinetics of sonidegib in children with relapsed/recurrent tumors followed by a phase II trial in pediatric and adult patients with relapsed medulloblastoma (MB) to assess tumor response.</div></front></TEI><affiliations><list><country><li>France</li></country></list><tree><noCountry><name sortKey="Hurh, Eunju" sort="Hurh, Eunju" uniqKey="Hurh E" first="Eunju" last="Hurh">Eunju Hurh</name><name sortKey="Kalambakas, Stacey" sort="Kalambakas, Stacey" uniqKey="Kalambakas S" first="Stacey" last="Kalambakas">Stacey Kalambakas</name></noCountry><country name="France"><noRegion><name sortKey="Kieran, Mark W" sort="Kieran, Mark W" uniqKey="Kieran M" first="Mark W" last="Kieran">Mark W. Kieran</name></noRegion><name sortKey="Aerts, Isabelle" sort="Aerts, Isabelle" uniqKey="Aerts I" first="Isabelle" last="Aerts">Isabelle Aerts</name><name sortKey="Bailey, Simon" sort="Bailey, Simon" uniqKey="Bailey S" first="Simon" last="Bailey">Simon Bailey</name><name sortKey="Bouffet, Eric" sort="Bouffet, Eric" uniqKey="Bouffet E" first="Eric" last="Bouffet">Eric Bouffet</name><name sortKey="Brandes, Alba A" sort="Brandes, Alba A" uniqKey="Brandes A" first="Alba A" last="Brandes">Alba A. Brandes</name><name sortKey="Casanova, Michela" sort="Casanova, Michela" uniqKey="Casanova M" first="Michela" last="Casanova">Michela Casanova</name><name sortKey="Chisholm, Julia" sort="Chisholm, Julia" uniqKey="Chisholm J" first="Julia" last="Chisholm">Julia Chisholm</name><name sortKey="Cohen, Kenneth J" sort="Cohen, Kenneth J" uniqKey="Cohen K" first="Kenneth J" last="Cohen">Kenneth J. Cohen</name><name sortKey="Deshpande, Priya" sort="Deshpande, Priya" uniqKey="Deshpande P" first="Priya" last="Deshpande">Priya Deshpande</name><name sortKey="Geoerger, Birgit" sort="Geoerger, Birgit" uniqKey="Geoerger B" first="Birgit" last="Geoerger">Birgit Geoerger</name><name sortKey="Hargrave, Darren" sort="Hargrave, Darren" uniqKey="Hargrave D" first="Darren" last="Hargrave">Darren Hargrave</name><name sortKey="Leary, Sarah" sort="Leary, Sarah" uniqKey="Leary S" first="Sarah" last="Leary">Sarah Leary</name><name sortKey="Macdonald, Tobey J" sort="Macdonald, Tobey J" uniqKey="Macdonald T" first="Tobey J" last="Macdonald">Tobey J. Macdonald</name><name sortKey="Mason, Warren" sort="Mason, Warren" uniqKey="Mason W" first="Warren" last="Mason">Warren Mason</name><name sortKey="Mechinaud, Francoise" sort="Mechinaud, Francoise" uniqKey="Mechinaud F" first="Francoise" last="Mechinaud">Francoise Mechinaud</name><name sortKey="Riccardi, Riccardo" sort="Riccardi, Riccardo" uniqKey="Riccardi R" first="Riccardo" last="Riccardi">Riccardo Riccardi</name><name sortKey="Tai, Feng" sort="Tai, Feng" uniqKey="Tai F" first="Feng" last="Tai">Feng Tai</name></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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|texte= Phase I study of oral sonidegib (LDE225) in pediatric brain and solid tumors and a phase II study in children and adults with relapsed medulloblastoma.
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