A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome
Identifieur interne : 002419 ( Pmc/Curation ); précédent : 002418; suivant : 002420A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome
Auteurs : Zhanna Shekhovtsova [France, Russie] ; Carmem Bonfim [Brésil] ; Annalisa Ruggeri [France] ; Samantha Nichele [Brésil] ; Kristin Page [États-Unis] ; Amal Alseraihy [Arabie saoudite] ; Francisco Barriga [Chili] ; José Sánchez De Toledo Codina [Espagne] ; Paul Veys [Royaume-Uni] ; Jaap Jan Boelens [Pays-Bas] ; Karin Mellgren [Suède] ; Henrique Bittencourt [Canada] ; Tracey O Rien [Australie] ; Peter J. Shaw [Australie] ; Alicja Chybicka [Pologne] ; Fernanda Volt [France] ; Federica Giannotti [France] ; Eliane Gluckman [France, Monaco] ; Joanne Kurtzberg [États-Unis] ; Andrew R. Gennery [Royaume-Uni] ; Vanderson Rocha [France, Royaume-Uni]Source :
- Haematologica [ 0390-6078 ] ; 2017.
Abstract
Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5–5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II–IV acute graft-
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DOI: 10.3324/haematol.2016.158808
PubMed: 28255019
PubMed Central: 5451344
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Chile</nlm:aff><country xml:lang="fr">Chili</country><wicri:regionArea>Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago</wicri:regionArea></affiliation></author><author><name sortKey="De Toledo Codina, Jose Sanchez" sort="De Toledo Codina, Jose Sanchez" uniqKey="De Toledo Codina J" first="José Sánchez" last="De Toledo Codina">José Sánchez De Toledo Codina</name><affiliation wicri:level="1"><nlm:aff id="af8-1021112">Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d’Hebron, Barcelona, Spain</nlm:aff><country xml:lang="fr">Espagne</country><wicri:regionArea>Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d’Hebron, Barcelona</wicri:regionArea></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="1"><nlm:aff id="af9-1021112">Great Ormond Street Hospital Children’s Charity, London, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Great Ormond Street Hospital Children’s Charity, London</wicri:regionArea></affiliation></author><author><name sortKey="Boelens, Jaap Jan" sort="Boelens, Jaap Jan" uniqKey="Boelens J" first="Jaap Jan" last="Boelens">Jaap Jan Boelens</name><affiliation wicri:level="1"><nlm:aff id="af10-1021112">Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht, the Netherlands</nlm:aff><country xml:lang="fr" wicri:curation="lc">Pays-Bas</country><wicri:regionArea>Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht</wicri:regionArea></affiliation></author><author><name sortKey="Mellgren, Karin" sort="Mellgren, Karin" uniqKey="Mellgren K" first="Karin" last="Mellgren">Karin Mellgren</name><affiliation wicri:level="1"><nlm:aff id="af11-1021112">Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children’s Hospital Gothenburg, Sweden</nlm:aff><country xml:lang="fr">Suède</country><wicri:regionArea>Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children’s Hospital Gothenburg</wicri:regionArea></affiliation></author><author><name sortKey="Bittencourt, Henrique" sort="Bittencourt, Henrique" uniqKey="Bittencourt H" first="Henrique" last="Bittencourt">Henrique Bittencourt</name><affiliation wicri:level="1"><nlm:aff id="af12-1021112">Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC, Canada</nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC</wicri:regionArea></affiliation></author><author><name sortKey="O Rien, Tracey" sort="O Rien, Tracey" uniqKey="O Rien T" first="Tracey" last="O Rien">Tracey O Rien</name><affiliation wicri:level="1"><nlm:aff id="af13-1021112">Sydney Children’s Hospital Kids Cancer Centre, Randwick, Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Sydney Children’s Hospital Kids Cancer Centre, Randwick</wicri:regionArea></affiliation></author><author><name sortKey="Shaw, Peter J" sort="Shaw, Peter J" uniqKey="Shaw P" first="Peter J." last="Shaw">Peter J. Shaw</name><affiliation wicri:level="1"><nlm:aff id="af14-1021112">The Children’s Hospital at Westmead, Sydney, Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>The Children’s Hospital at Westmead, Sydney</wicri:regionArea></affiliation></author><author><name sortKey="Chybicka, Alicja" sort="Chybicka, Alicja" uniqKey="Chybicka A" first="Alicja" last="Chybicka">Alicja Chybicka</name><affiliation wicri:level="1"><nlm:aff id="af15-1021112">Wroclaw Medical University, Poland</nlm:aff><country xml:lang="fr">Pologne</country><wicri:regionArea>Wroclaw Medical University</wicri:regionArea></affiliation></author><author><name sortKey="Volt, Fernanda" sort="Volt, Fernanda" uniqKey="Volt F" first="Fernanda" last="Volt">Fernanda Volt</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation></author><author><name sortKey="Giannotti, Federica" sort="Giannotti, Federica" uniqKey="Giannotti F" first="Federica" last="Giannotti">Federica Giannotti</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af4-1021112">Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris</wicri:regionArea></affiliation></author><author><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af16-1021112">Centre Scientifique de Monaco, Monaco</nlm:aff><country xml:lang="fr">Monaco</country><wicri:regionArea>Centre Scientifique de Monaco</wicri:regionArea></affiliation></author><author><name sortKey="Kurtzberg, Joanne" sort="Kurtzberg, Joanne" uniqKey="Kurtzberg J" first="Joanne" last="Kurtzberg">Joanne Kurtzberg</name><affiliation wicri:level="1"><nlm:aff id="af5-1021112">Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC</wicri:regionArea></affiliation></author><author><name sortKey="Gennery, Andrew R" sort="Gennery, Andrew R" uniqKey="Gennery A" first="Andrew R." last="Gennery">Andrew R. Gennery</name><affiliation wicri:level="1"><nlm:aff id="af17-1021112">Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne</wicri:regionArea></affiliation></author><author><name sortKey="Rocha, Vanderson" sort="Rocha, Vanderson" uniqKey="Rocha V" first="Vanderson" last="Rocha">Vanderson Rocha</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af18-1021112">Oxford University Hospitals NHS Trust, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Oxford University Hospitals NHS Trust</wicri:regionArea></affiliation></author></titleStmt><publicationStmt><idno 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id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af2-1021112">Dmitry Rogachev National Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation</nlm:aff><country xml:lang="fr">Russie</country><wicri:regionArea>Dmitry Rogachev National Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow</wicri:regionArea></affiliation></author><author><name sortKey="Bonfim, Carmem" sort="Bonfim, Carmem" uniqKey="Bonfim C" first="Carmem" last="Bonfim">Carmem Bonfim</name><affiliation wicri:level="1"><nlm:aff id="af3-1021112">Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil</nlm:aff><country xml:lang="fr">Brésil</country><wicri:regionArea>Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba</wicri:regionArea></affiliation></author><author><name sortKey="Ruggeri, Annalisa" sort="Ruggeri, Annalisa" uniqKey="Ruggeri A" first="Annalisa" last="Ruggeri">Annalisa Ruggeri</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af4-1021112">Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris</wicri:regionArea></affiliation></author><author><name sortKey="Nichele, Samantha" sort="Nichele, Samantha" uniqKey="Nichele S" first="Samantha" last="Nichele">Samantha Nichele</name><affiliation wicri:level="1"><nlm:aff id="af3-1021112">Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil</nlm:aff><country xml:lang="fr">Brésil</country><wicri:regionArea>Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba</wicri:regionArea></affiliation></author><author><name sortKey="Page, Kristin" sort="Page, Kristin" uniqKey="Page K" first="Kristin" last="Page">Kristin Page</name><affiliation wicri:level="1"><nlm:aff id="af5-1021112">Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC</wicri:regionArea></affiliation></author><author><name sortKey="Alseraihy, Amal" sort="Alseraihy, Amal" uniqKey="Alseraihy A" first="Amal" last="Alseraihy">Amal Alseraihy</name><affiliation wicri:level="1"><nlm:aff id="af6-1021112">Section of Pediatric SCT, King Faisal Specialist Hospital & Research Centre-Riyadh, Saudi Arabia</nlm:aff><country xml:lang="fr">Arabie saoudite</country><wicri:regionArea>Section of Pediatric SCT, King Faisal Specialist Hospital & Research Centre-Riyadh</wicri:regionArea></affiliation></author><author><name sortKey="Barriga, Francisco" sort="Barriga, Francisco" uniqKey="Barriga F" first="Francisco" last="Barriga">Francisco Barriga</name><affiliation wicri:level="1"><nlm:aff id="af7-1021112">Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago, Chile</nlm:aff><country xml:lang="fr">Chili</country><wicri:regionArea>Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago</wicri:regionArea></affiliation></author><author><name sortKey="De Toledo Codina, Jose Sanchez" sort="De Toledo Codina, Jose Sanchez" uniqKey="De Toledo Codina J" first="José Sánchez" last="De Toledo Codina">José Sánchez De Toledo Codina</name><affiliation wicri:level="1"><nlm:aff id="af8-1021112">Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d’Hebron, Barcelona, Spain</nlm:aff><country xml:lang="fr">Espagne</country><wicri:regionArea>Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d’Hebron, Barcelona</wicri:regionArea></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="1"><nlm:aff id="af9-1021112">Great Ormond Street Hospital Children’s Charity, London, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Great Ormond Street Hospital Children’s Charity, London</wicri:regionArea></affiliation></author><author><name sortKey="Boelens, Jaap Jan" sort="Boelens, Jaap Jan" uniqKey="Boelens J" first="Jaap Jan" last="Boelens">Jaap Jan Boelens</name><affiliation wicri:level="1"><nlm:aff id="af10-1021112">Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht, the Netherlands</nlm:aff><country xml:lang="fr" wicri:curation="lc">Pays-Bas</country><wicri:regionArea>Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht</wicri:regionArea></affiliation></author><author><name sortKey="Mellgren, Karin" sort="Mellgren, Karin" uniqKey="Mellgren K" first="Karin" last="Mellgren">Karin Mellgren</name><affiliation wicri:level="1"><nlm:aff id="af11-1021112">Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children’s Hospital Gothenburg, Sweden</nlm:aff><country xml:lang="fr">Suède</country><wicri:regionArea>Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children’s Hospital Gothenburg</wicri:regionArea></affiliation></author><author><name sortKey="Bittencourt, Henrique" sort="Bittencourt, Henrique" uniqKey="Bittencourt H" first="Henrique" last="Bittencourt">Henrique Bittencourt</name><affiliation wicri:level="1"><nlm:aff id="af12-1021112">Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC, Canada</nlm:aff><country xml:lang="fr">Canada</country><wicri:regionArea>Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC</wicri:regionArea></affiliation></author><author><name sortKey="O Rien, Tracey" sort="O Rien, Tracey" uniqKey="O Rien T" first="Tracey" last="O Rien">Tracey O Rien</name><affiliation wicri:level="1"><nlm:aff id="af13-1021112">Sydney Children’s Hospital Kids Cancer Centre, Randwick, Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>Sydney Children’s Hospital Kids Cancer Centre, Randwick</wicri:regionArea></affiliation></author><author><name sortKey="Shaw, Peter J" sort="Shaw, Peter J" uniqKey="Shaw P" first="Peter J." last="Shaw">Peter J. Shaw</name><affiliation wicri:level="1"><nlm:aff id="af14-1021112">The Children’s Hospital at Westmead, Sydney, Australia</nlm:aff><country xml:lang="fr">Australie</country><wicri:regionArea>The Children’s Hospital at Westmead, Sydney</wicri:regionArea></affiliation></author><author><name sortKey="Chybicka, Alicja" sort="Chybicka, Alicja" uniqKey="Chybicka A" first="Alicja" last="Chybicka">Alicja Chybicka</name><affiliation wicri:level="1"><nlm:aff id="af15-1021112">Wroclaw Medical University, Poland</nlm:aff><country xml:lang="fr">Pologne</country><wicri:regionArea>Wroclaw Medical University</wicri:regionArea></affiliation></author><author><name sortKey="Volt, Fernanda" sort="Volt, Fernanda" uniqKey="Volt F" first="Fernanda" last="Volt">Fernanda Volt</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation></author><author><name sortKey="Giannotti, Federica" sort="Giannotti, Federica" uniqKey="Giannotti F" first="Federica" last="Giannotti">Federica Giannotti</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af4-1021112">Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris</wicri:regionArea></affiliation></author><author><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af16-1021112">Centre Scientifique de Monaco, Monaco</nlm:aff><country xml:lang="fr">Monaco</country><wicri:regionArea>Centre Scientifique de Monaco</wicri:regionArea></affiliation></author><author><name sortKey="Kurtzberg, Joanne" sort="Kurtzberg, Joanne" uniqKey="Kurtzberg J" first="Joanne" last="Kurtzberg">Joanne Kurtzberg</name><affiliation wicri:level="1"><nlm:aff id="af5-1021112">Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA</nlm:aff><country xml:lang="fr">États-Unis</country><wicri:regionArea>Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC</wicri:regionArea></affiliation></author><author><name sortKey="Gennery, Andrew R" sort="Gennery, Andrew R" uniqKey="Gennery A" first="Andrew R." last="Gennery">Andrew R. Gennery</name><affiliation wicri:level="1"><nlm:aff id="af17-1021112">Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne</wicri:regionArea></affiliation></author><author><name sortKey="Rocha, Vanderson" sort="Rocha, Vanderson" uniqKey="Rocha V" first="Vanderson" last="Rocha">Vanderson Rocha</name><affiliation wicri:level="1"><nlm:aff id="af1-1021112">Hôpital Saint Louis, Eurocord, Paris, France</nlm:aff><country xml:lang="fr">France</country><wicri:regionArea>Hôpital Saint Louis, Eurocord, Paris</wicri:regionArea></affiliation><affiliation wicri:level="1"><nlm:aff id="af18-1021112">Oxford University Hospitals NHS Trust, UK</nlm:aff><country xml:lang="fr">Royaume-Uni</country><wicri:regionArea>Oxford University Hospitals NHS Trust</wicri:regionArea></affiliation></author></analytic><series><title level="j">Haematologica</title><idno type="ISSN">0390-6078</idno><idno type="eISSN">1592-8721</idno><imprint><date when="2017">2017</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><p>Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5–5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II–IV acute graft-<italic>versus</italic>-host disease at day 100 was 38%. The use of methotrexate for graft-<italic>versus</italic>-host disease prophylaxis delayed engraftment (<italic>P</italic>=0.02), but decreased acute graft-<italic>versus</italic>-host disease (<italic>P</italic>=0.03). At 5 years, overall survival and event-free survival rates were 75% and 70%, respectively. The estimated 5-year event-free survival rates were 83%, 73% and 55% for patients with a clinical score of 2, 4–5 and 3, respectively. In multivariate analysis, age <2 years at the time of the umbilical cord blood transplant and a clinical phenotype of X-linked thrombocytopenia were associated with improved event-free survival. Overall survival tended to be better in patients transplanted after 2007 (<italic>P</italic>=0.09). In conclusion, umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor.</p></div></front><back><div1 type="bibliography"><listBibl><biblStruct><analytic><author><name sortKey="Jin, Y" uniqKey="Jin Y">Y Jin</name></author><author><name sortKey="Mazza, C" uniqKey="Mazza C">C Mazza</name></author><author><name sortKey="Christie, Jr" uniqKey="Christie J">JR Christie</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Ochs, Hd" uniqKey="Ochs H">HD Ochs</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Massaad, Mj" uniqKey="Massaad M">MJ Massaad</name></author><author><name sortKey="Ramesh, N" uniqKey="Ramesh N">N Ramesh</name></author><author><name sortKey="Geha, Rs" uniqKey="Geha R">RS Geha</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Bosticardo, M" uniqKey="Bosticardo M">M Bosticardo</name></author><author><name sortKey="Marangoni, F" uniqKey="Marangoni F">F Marangoni</name></author><author><name sortKey="Aiuti, A" uniqKey="Aiuti A">A Aiuti</name></author><author><name sortKey="Villa, A" uniqKey="Villa A">A Villa</name></author><author><name sortKey="Grazia Roncarolo, M" uniqKey="Grazia Roncarolo M">M Grazia Roncarolo</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Imai, K" uniqKey="Imai K">K Imai</name></author><author><name sortKey="Nonoyama, S" uniqKey="Nonoyama S">S Nonoyama</name></author><author><name sortKey="Ochs, Hd" uniqKey="Ochs H">HD Ochs</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Ochs, Hd" uniqKey="Ochs H">HD Ochs</name></author><author><name sortKey="Filipovich, Ah" uniqKey="Filipovich A">AH Filipovich</name></author><author><name sortKey="Veys, P" uniqKey="Veys P">P Veys</name></author><author><name sortKey="Cowan, Mj" uniqKey="Cowan M">MJ Cowan</name></author><author><name sortKey="Kapoor, N" uniqKey="Kapoor N">N Kapoor</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Albert, Mh" uniqKey="Albert M">MH Albert</name></author><author><name sortKey="Bittner, Tc" uniqKey="Bittner T">TC Bittner</name></author><author><name sortKey="Nonoyama, S" uniqKey="Nonoyama S">S Nonoyama</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Notarangelo, Ld" uniqKey="Notarangelo L">LD Notarangelo</name></author><author><name sortKey="Miao, Ch" uniqKey="Miao C">CH Miao</name></author><author><name sortKey="Ochs, Hd" uniqKey="Ochs H">HD Ochs</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Sullivan, Ke" uniqKey="Sullivan K">KE Sullivan</name></author><author><name sortKey="Mullen, Ca" uniqKey="Mullen C">CA Mullen</name></author><author><name sortKey="Blaese, Rm" uniqKey="Blaese R">RM Blaese</name></author><author><name sortKey="Winkelstein, Ja" uniqKey="Winkelstein J">JA Winkelstein</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Hacein Bey Abina, S" uniqKey="Hacein Bey Abina S">S Hacein-Bey Abina</name></author><author><name sortKey="Gaspar, Hb" uniqKey="Gaspar H">HB Gaspar</name></author><author><name sortKey="Blondeau, J" uniqKey="Blondeau J">J Blondeau</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Filipovich, Ah" uniqKey="Filipovich A">AH Filipovich</name></author><author><name sortKey="Stone, Jv" uniqKey="Stone J">JV Stone</name></author><author><name sortKey="Tomany, Sc" uniqKey="Tomany S">SC Tomany</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Kobayashi, R" uniqKey="Kobayashi R">R Kobayashi</name></author><author><name sortKey="Ariga, T" uniqKey="Ariga T">T Ariga</name></author><author><name sortKey="Nonoyama, S" uniqKey="Nonoyama S">S Nonoyama</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Buckley, Rh" uniqKey="Buckley R">RH Buckley</name></author><author><name sortKey="Schiff, Se" uniqKey="Schiff S">SE Schiff</name></author><author><name sortKey="Schiff, Ri" uniqKey="Schiff R">RI Schiff</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Ozsahin, H" uniqKey="Ozsahin H">H Ozsahin</name></author><author><name sortKey="Cavazzana Calvo, M" uniqKey="Cavazzana Calvo M">M Cavazzana-Calvo</name></author><author><name sortKey="Notarangelo, Ld" uniqKey="Notarangelo L">LD Notarangelo</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Shin, Cr" uniqKey="Shin C">CR Shin</name></author><author><name sortKey="Kim, Mo" uniqKey="Kim M">MO Kim</name></author><author><name sortKey="Li, D" uniqKey="Li D">D Li</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Moratto, D" uniqKey="Moratto D">D Moratto</name></author><author><name sortKey="Giliani, S" uniqKey="Giliani S">S Giliani</name></author><author><name sortKey="Bonfim, C" uniqKey="Bonfim C">C Bonfim</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Knutsen, Ap" uniqKey="Knutsen A">AP Knutsen</name></author><author><name sortKey="Steffen, M" uniqKey="Steffen M">M Steffen</name></author><author><name sortKey="Wassmer, K" uniqKey="Wassmer K">K Wassmer</name></author><author><name sortKey="Wall, Da" uniqKey="Wall D">DA Wall</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Knutsen, Ap" uniqKey="Knutsen A">AP Knutsen</name></author><author><name sortKey="Wall, Da" uniqKey="Wall D">DA Wall</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Diaz De Heredia, C" uniqKey="Diaz De Heredia C">C Diaz de Heredia</name></author><author><name sortKey="Ortega, Jj" uniqKey="Ortega J">JJ Ortega</name></author><author><name sortKey="Diaz, Ma" uniqKey="Diaz M">MA Diaz</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Bhattacharya, A" uniqKey="Bhattacharya A">A Bhattacharya</name></author><author><name sortKey="Slatter, Ma" uniqKey="Slatter M">MA Slatter</name></author><author><name sortKey="Chapman, Ce" uniqKey="Chapman C">CE Chapman</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Jaing, Th" uniqKey="Jaing T">TH Jaing</name></author><author><name sortKey="Tsai, By" uniqKey="Tsai B">BY Tsai</name></author><author><name sortKey="Chen, Sh" uniqKey="Chen S">SH Chen</name></author><author><name sortKey="Lee, Wi" uniqKey="Lee W">WI Lee</name></author><author><name sortKey="Chang, Kw" uniqKey="Chang K">KW Chang</name></author><author><name sortKey="Chu, Sm" uniqKey="Chu S">SM Chu</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Kaneko, M" uniqKey="Kaneko M">M Kaneko</name></author><author><name sortKey="Watanabe, T" uniqKey="Watanabe T">T Watanabe</name></author><author><name sortKey="Watanabe, H" uniqKey="Watanabe H">H Watanabe</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Przepiorka, D" uniqKey="Przepiorka D">D Przepiorka</name></author><author><name sortKey="Weisdorf, D" uniqKey="Weisdorf D">D Weisdorf</name></author><author><name sortKey="Martin, P" uniqKey="Martin P">P Martin</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Shulman, Hm" uniqKey="Shulman H">HM Shulman</name></author><author><name sortKey="Sullivan, Km" uniqKey="Sullivan K">KM Sullivan</name></author><author><name sortKey="Weiden, Pl" uniqKey="Weiden P">PL Weiden</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Comans Bitter, Wm" uniqKey="Comans Bitter W">WM Comans-Bitter</name></author><author><name sortKey="De Groot, R" uniqKey="De Groot R">R de Groot</name></author><author><name sortKey="Van Den Beemd, R" uniqKey="Van Den Beemd R">R van den Beemd</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Niehues, T" uniqKey="Niehues T">T Niehues</name></author><author><name sortKey="Rocha, V" uniqKey="Rocha V">V Rocha</name></author><author><name sortKey="Filipovich, Ah" uniqKey="Filipovich A">AH Filipovich</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Gooley, Ta" uniqKey="Gooley T">TA Gooley</name></author><author><name sortKey="Leisenring, W" uniqKey="Leisenring W">W Leisenring</name></author><author><name sortKey="Crowley, J" uniqKey="Crowley J">J Crowley</name></author><author><name sortKey="Storer, Be" uniqKey="Storer B">BE Storer</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Cox" uniqKey="Cox">Cox</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Filipovich, Ah" uniqKey="Filipovich A">AH Filipovich</name></author><author><name sortKey="Shapiro, Rs" uniqKey="Shapiro R">RS Shapiro</name></author><author><name sortKey="Ramsay, Nk" uniqKey="Ramsay N">NK Ramsay</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Lenarsky, C" uniqKey="Lenarsky C">C Lenarsky</name></author><author><name sortKey="Weinberg, K" uniqKey="Weinberg K">K Weinberg</name></author><author><name sortKey="Kohn, Db" uniqKey="Kohn D">DB Kohn</name></author><author><name sortKey="Parkman, R" uniqKey="Parkman R">R Parkman</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Balashov, D" uniqKey="Balashov D">D Balashov</name></author><author><name sortKey="Shcherbina, A" uniqKey="Shcherbina A">A Shcherbina</name></author><author><name sortKey="Maschan, M" uniqKey="Maschan M">M Maschan</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Gluckman, E" uniqKey="Gluckman E">E Gluckman</name></author><author><name sortKey="Rocha, V" uniqKey="Rocha V">V Rocha</name></author><author><name sortKey="Arcese, W" uniqKey="Arcese W">W Arcese</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Albert, Mh" uniqKey="Albert M">MH Albert</name></author><author><name sortKey="Bittner, T" uniqKey="Bittner T">T Bittner</name></author><author><name sortKey="Stachel, D" uniqKey="Stachel D">D Stachel</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Buchbinder, D" uniqKey="Buchbinder D">D Buchbinder</name></author><author><name sortKey="Nugent, Dj" uniqKey="Nugent D">DJ Nugent</name></author><author><name sortKey="Fillipovich, Ah" uniqKey="Fillipovich A">AH Fillipovich</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Rocha, V" uniqKey="Rocha V">V Rocha</name></author><author><name sortKey="Gluckman, E" uniqKey="Gluckman E">E Gluckman</name></author></analytic></biblStruct><biblStruct><analytic><author><name sortKey="Ballen, Kk" uniqKey="Ballen K">KK Ballen</name></author></analytic></biblStruct></listBibl></div1></back></TEI><pmc article-type="research-article"><pmc-dir>properties open_access</pmc-dir>
<front><journal-meta><journal-id journal-id-type="nlm-ta">Haematologica</journal-id><journal-id journal-id-type="iso-abbrev">Haematologica</journal-id><journal-id journal-id-type="hwp">haematol</journal-id><journal-id journal-id-type="publisher-id">Haematologica</journal-id><journal-title-group><journal-title>Haematologica</journal-title></journal-title-group><issn pub-type="ppub">0390-6078</issn><issn pub-type="epub">1592-8721</issn><publisher><publisher-name>Ferrata Storti Foundation</publisher-name></publisher></journal-meta><article-meta><article-id pub-id-type="pmid">28255019</article-id><article-id pub-id-type="pmc">5451344</article-id><article-id pub-id-type="doi">10.3324/haematol.2016.158808</article-id><article-id pub-id-type="publisher-id">1021112</article-id><article-categories><subj-group subj-group-type="heading"><subject>Article</subject><subj-group subj-group-type="heading"><subject>Cell Therapy & Immunotherapy</subject></subj-group></subj-group></article-categories><title-group><article-title>A risk factor analysis of outcomes after unrelated cord blood transplantation for children with Wiskott-Aldrich syndrome</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Shekhovtsova</surname><given-names>Zhanna</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref><xref ref-type="aff" rid="af2-1021112">2</xref><xref ref-type="corresp" rid="c1-1021112"></xref></contrib><contrib contrib-type="author"><name><surname>Bonfim</surname><given-names>Carmem</given-names></name><xref ref-type="aff" rid="af3-1021112">3</xref></contrib><contrib contrib-type="author"><name><surname>Ruggeri</surname><given-names>Annalisa</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref><xref ref-type="aff" rid="af4-1021112">4</xref></contrib><contrib contrib-type="author"><name><surname>Nichele</surname><given-names>Samantha</given-names></name><xref ref-type="aff" rid="af3-1021112">3</xref></contrib><contrib contrib-type="author"><name><surname>Page</surname><given-names>Kristin</given-names></name><xref ref-type="aff" rid="af5-1021112">5</xref></contrib><contrib contrib-type="author"><name><surname>AlSeraihy</surname><given-names>Amal</given-names></name><xref ref-type="aff" rid="af6-1021112">6</xref></contrib><contrib contrib-type="author"><name><surname>Barriga</surname><given-names>Francisco</given-names></name><xref ref-type="aff" rid="af7-1021112">7</xref></contrib><contrib contrib-type="author"><name><surname>de Toledo Codina</surname><given-names>José Sánchez</given-names></name><xref ref-type="aff" rid="af8-1021112">8</xref></contrib><contrib contrib-type="author"><name><surname>Veys</surname><given-names>Paul</given-names></name><xref ref-type="aff" rid="af9-1021112">9</xref></contrib><contrib contrib-type="author"><name><surname>Boelens</surname><given-names>Jaap Jan</given-names></name><xref ref-type="aff" rid="af10-1021112">10</xref></contrib><contrib contrib-type="author"><name><surname>Mellgren</surname><given-names>Karin</given-names></name><xref ref-type="aff" rid="af11-1021112">11</xref></contrib><contrib contrib-type="author"><name><surname>Bittencourt</surname><given-names>Henrique</given-names></name><xref ref-type="aff" rid="af12-1021112">12</xref></contrib><contrib contrib-type="author"><name><surname>O’Brien</surname><given-names>Tracey</given-names></name><xref ref-type="aff" rid="af13-1021112">13</xref></contrib><contrib contrib-type="author"><name><surname>Shaw</surname><given-names>Peter J.</given-names></name><xref ref-type="aff" rid="af14-1021112">14</xref></contrib><contrib contrib-type="author"><name><surname>Chybicka</surname><given-names>Alicja</given-names></name><xref ref-type="aff" rid="af15-1021112">15</xref></contrib><contrib contrib-type="author"><name><surname>Volt</surname><given-names>Fernanda</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref></contrib><contrib contrib-type="author"><name><surname>Giannotti</surname><given-names>Federica</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref><xref ref-type="aff" rid="af4-1021112">4</xref></contrib><contrib contrib-type="author"><name><surname>Gluckman</surname><given-names>Eliane</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref><xref ref-type="aff" rid="af16-1021112">16</xref></contrib><contrib contrib-type="author"><name><surname>Kurtzberg</surname><given-names>Joanne</given-names></name><xref ref-type="aff" rid="af5-1021112">5</xref></contrib><contrib contrib-type="author"><name><surname>Gennery</surname><given-names>Andrew R.</given-names></name><xref ref-type="aff" rid="af17-1021112">17</xref></contrib><contrib contrib-type="author"><name><surname>Rocha</surname><given-names>Vanderson</given-names></name><xref ref-type="aff" rid="af1-1021112">1</xref><xref ref-type="aff" rid="af18-1021112">18</xref></contrib><on-behalf-of>on behalf of Eurocord, Cord Blood Committee of Cellular Therapy and Immunobiology Working Party of the EBMT, Federal University of Parana, Duke University Medical Center and Inborn Errors Working Party of the EBMT</on-behalf-of></contrib-group><aff id="af1-1021112"><label>1</label>Hôpital Saint Louis, Eurocord, Paris, France</aff><aff id="af2-1021112"><label>2</label>Dmitry Rogachev National Research Centre of Pediatric Hematology, Oncology and Immunology, Moscow, Russian Federation</aff><aff id="af3-1021112"><label>3</label>Bone Marrow Transplantation Service, Hospital de Clínicas, Universidade Federal do Paraná, Curitiba, Brazil</aff><aff id="af4-1021112"><label>4</label>Service d’Hematologie et Therapie Cellulaire, Hôpital Saint Antoine, Paris, France</aff><aff id="af5-1021112"><label>5</label>Pediatric Blood and Marrow Transplantation Program, Duke University Medical Center, Durham, NC, USA</aff><aff id="af6-1021112"><label>6</label>Section of Pediatric SCT, King Faisal Specialist Hospital & Research Centre-Riyadh, Saudi Arabia</aff><aff id="af7-1021112"><label>7</label>Programa de Hematologia Oncologia Departamento de Pediatria, Pontificia Universidad Catolica de Chile, Santiago, Chile</aff><aff id="af8-1021112"><label>8</label>Servicio de Hematologia y Oncologia Pediatrica, Hospital Vall d’Hebron, Barcelona, Spain</aff><aff id="af9-1021112"><label>9</label>Great Ormond Street Hospital Children’s Charity, London, UK</aff><aff id="af10-1021112"><label>10</label>Pediatric Blood and Marrow Transplantation Program, University Hospital Utrecht, the Netherlands</aff><aff id="af11-1021112"><label>11</label>Department of Oncology, Hematology and Stem Cell Transplantation, The Queen Silvia Children’s Hospital Gothenburg, Sweden</aff><aff id="af12-1021112"><label>12</label>Hematology-Oncology Division, Centre Hospitalier Universitaire Sainte-Justine, Montréal, QC, Canada</aff><aff id="af13-1021112"><label>13</label>Sydney Children’s Hospital Kids Cancer Centre, Randwick, Australia</aff><aff id="af14-1021112"><label>14</label>The Children’s Hospital at Westmead, Sydney, Australia</aff><aff id="af15-1021112"><label>15</label>Wroclaw Medical University, Poland</aff><aff id="af16-1021112"><label>16</label>Centre Scientifique de Monaco, Monaco</aff><aff id="af17-1021112"><label>17</label>Institute of Cellular Medicine, Newcastle University, Newcastle-Upon-Tyne, UK</aff><aff id="af18-1021112"><label>18</label>Oxford University Hospitals NHS Trust, UK</aff><author-notes><corresp id="c1-1021112"><bold>Correspondence:</bold><email>zhanna.shekhovtsova@fccho-moscow.ru</email></corresp></author-notes><pub-date pub-type="ppub"><month>6</month><year>2017</year></pub-date><volume>102</volume><issue>6</issue><fpage>1112</fpage><lpage>1119</lpage><history><date date-type="received"><day>27</day><month>10</month><year>2016</year></date><date date-type="accepted"><day>28</day><month>2</month><year>2017</year></date></history><permissions><copyright-statement>Copyright© Ferrata Storti Foundation</copyright-statement><copyright-year>2017</copyright-year><license license-type="open-access"><license-p>Material published in Haematologica is covered by copyright. All rights are reserved to the Ferrata Storti Foundation. Use of published material is allowed under the following terms and conditions:</license-p><license-p><ext-link ext-link-type="uri" xlink:href="https://creativecommons.org/licenses/by-nc/4.0/legalcode">https://creativecommons.org/licenses/by-nc/4.0/legalcode</ext-link>. Copies of published material are allowed for personal or internal use. Sharing published material for non-commercial purposes is subject to the following conditions:</license-p><license-p><ext-link ext-link-type="uri" xlink:href="https://creativecommons.org/licenses/by-nc/4.0/legalcode">https://creativecommons.org/licenses/by-nc/4.0/legalcode</ext-link>, sect. 3. Reproducing and sharing published material for commercial purposes is not allowed without permission in writing from the publisher.</license-p></license></permissions><self-uri content-type="pdf" xlink:type="simple" xlink:href="1021112.pdf"></self-uri><abstract><p>Wiskott-Aldrich syndrome is a severe X-linked recessive immune deficiency disorder. A scoring system of Wiskott-Aldrich syndrome severity (0.5–5) distinguishes two phenotypes: X-linked thrombocytopenia and classic Wiskott-Aldrich syndrome. Hematopoietic cell transplantation is curative for Wiskott-Aldrich syndrome; however, the use of unrelated umbilical cord blood transplantation has seldom been described. We analyzed umbilical cord blood transplantation outcomes for 90 patients. The median age at umbilical cord blood transplantation was 1.5 years. Patients were classified according to clinical scores [2 (23%), 3 (30%), 4 (23%) and 5 (19%)]. Most patients underwent HLA-mismatched umbilical cord blood transplantation and myeloablative conditioning with anti-thymocyte globulin. The cumulative incidence of neutrophil recovery at day 60 was 89% and that of grade II–IV acute graft-<italic>versus</italic>-host disease at day 100 was 38%. The use of methotrexate for graft-<italic>versus</italic>-host disease prophylaxis delayed engraftment (<italic>P</italic>=0.02), but decreased acute graft-<italic>versus</italic>-host disease (<italic>P</italic>=0.03). At 5 years, overall survival and event-free survival rates were 75% and 70%, respectively. The estimated 5-year event-free survival rates were 83%, 73% and 55% for patients with a clinical score of 2, 4–5 and 3, respectively. In multivariate analysis, age <2 years at the time of the umbilical cord blood transplant and a clinical phenotype of X-linked thrombocytopenia were associated with improved event-free survival. Overall survival tended to be better in patients transplanted after 2007 (<italic>P</italic>=0.09). In conclusion, umbilical cord blood transplantation is a good alternative option for young children with Wiskott-Aldrich syndrome lacking an HLA identical stem cell donor.</p></abstract></article-meta></front></pmc></record>Pour manipuler ce document sous Unix (Dilib)
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