Haematopoietic stem cell transplantation for shwachman- diamond disease : a study from the European group for blood and marrow transplantation
Identifieur interne : 00A609 ( Main/Exploration ); précédent : 00A608; suivant : 00A610Haematopoietic stem cell transplantation for shwachman- diamond disease : a study from the European group for blood and marrow transplantation
Auteurs : Simone Cesaro [Italie] ; Rosi Oneto [Italie] ; Chiara Messina [Italie] ; Brenda E. Gibson [Royaume-Uni] ; Agnes Buzyn [France] ; Colin Steward [Royaume-Uni] ; Eliane Gluckman [France] ; Robbert Breddius [Pays-Bas] ; Marc Boogaerts [Belgique] ; Christiane Vermylen [Belgique] ; Paul Veys [Royaume-Uni] ; Judith Marsh [Royaume-Uni] ; Isabel Badell [Espagne] ; Gerard Michel [France] ; Tayfun Güngör [Suisse] ; Dietrich Niethammer [Allemagne] ; Pierre Bordigoni [France] ; Cecilia Oswald [Australie] ; Claudio Favre [Italie] ; Jakob Passweg [Suisse] ; Giorgio Dini [Italie]Source :
- British journal of haematology [ 0007-1048 ] ; 2005.
Descripteurs français
- Pascal (Inist)
- Wicri :
- topic : Cellule souche.
English descriptors
- KwdEn :
Abstract
This report assessed the results of allogeneic stem cell transplantation (allo-SCT) in 26 patients with Shwachman-Diamond disease (SDS) and severe bone marrow abnormalities. The conditioning regimen was based on busulphan (54%), total body irradiation (23%), fludarabine (15%) or other chemotherapy combinations (8%). Standard prevention of graft versus host disease (GVHD) with ciclosporin ± methotrexate was adopted in 54% of the patients whilst in vivo or in vitro T-cell depletion was used in 17 and four patients respectively. Neutrophil and platelet engraftment were achieved in 21 (81%) and 17 (65%) of 26 patients after a median time of 18 days and 29 days respectively. The incidence of grade III and IV acute GVHD was 24% and of chronic GVHD 29%. Nine patients died after a median time of 70 d, post-SCT. After a median follow-up of 1.1 years, the transplant-related mortality was 35.5% (95% CI 17-54) whilst the overall survival was 64.5% (95% CI 45.7-83.2). Allo-SCT was found to be successful in more than half of SDS patients with severe bone marrow dysfunction. Further improvements would be anticipated by a better definition of the optimum time in the course of disease to transplant and by the adoption of less toxic conditioning regimens.
Affiliations:
- Allemagne, Australie, Belgique, Espagne, France, Italie, Pays-Bas, Royaume-Uni, Suisse
- Angleterre, Canton de Zurich, Catalogne, Grand Est, Grand Londres, Hollande-Méridionale, Lorraine (région), Provence-Alpes-Côte d'Azur, Région de Bruxelles-Capitale, Toscane, Île-de-France
- Barcelone, Bruxelles, Leyde, Londres, Marseille, Nancy, Paris, Pise, Zurich
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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en" level="a">Haematopoietic stem cell transplantation for shwachman- diamond disease : a study from the European group for blood and marrow transplantation</title><author><name sortKey="Cesaro, Simone" sort="Cesaro, Simone" uniqKey="Cesaro S" first="Simone" last="Cesaro">Simone Cesaro</name><affiliation wicri:level="1"><inist:fA14 i1="01"><s1>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</s1><s3>ITA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</wicri:noRegion></affiliation></author><author><name sortKey="Oneto, Rosi" sort="Oneto, Rosi" uniqKey="Oneto R" first="Rosi" last="Oneto">Rosi Oneto</name><affiliation wicri:level="1"><inist:fA14 i1="02"><s1>Division of Haematology, San Martino Hospital</s1><s2>Genova</s2><s3>ITA</s3><sZ>2 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Genova</wicri:noRegion></affiliation></author><author><name sortKey="Messina, Chiara" sort="Messina, Chiara" uniqKey="Messina C" first="Chiara" last="Messina">Chiara Messina</name><affiliation wicri:level="1"><inist:fA14 i1="01"><s1>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</s1><s3>ITA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</wicri:noRegion></affiliation></author><author><name sortKey="Gibson, Brenda E" sort="Gibson, Brenda E" uniqKey="Gibson B" first="Brenda E." last="Gibson">Brenda E. Gibson</name><affiliation wicri:level="1"><inist:fA14 i1="03"><s1>Department of Haematology, Royal Hospital for Sick Children</s1><s2>Glasgow</s2><s3>GBR</s3><sZ>4 aut.</sZ></inist:fA14><country>Royaume-Uni</country><wicri:noRegion>Glasgow</wicri:noRegion></affiliation></author><author><name sortKey="Buzyn, Agnes" sort="Buzyn, Agnes" uniqKey="Buzyn A" first="Agnes" last="Buzyn">Agnes Buzyn</name><affiliation wicri:level="3"><inist:fA14 i1="04"><s1>Unité d'lmmunologie et d'Hématologie and INSERM U 2 - Hôpital Necker</s1><s2>Paris</s2><s3>FRA</s3><sZ>5 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Steward, Colin" sort="Steward, Colin" uniqKey="Steward C" first="Colin" last="Steward">Colin Steward</name><affiliation wicri:level="1"><inist:fA14 i1="05"><s1>Department of Paediatric Oncology/BMT, Bristol Royal Hospital for Children</s1><s2>Bristol</s2><s3>GBR</s3><sZ>6 aut.</sZ></inist:fA14><country>Royaume-Uni</country><wicri:noRegion>Bristol</wicri:noRegion></affiliation></author><author><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name><affiliation wicri:level="3"><inist:fA14 i1="06"><s1>Department of Haematology - BMT, Hopital St Louis</s1><s2>Paris</s2><s3>FRA</s3><sZ>7 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Breddius, Robbert" sort="Breddius, Robbert" uniqKey="Breddius R" first="Robbert" last="Breddius">Robbert Breddius</name><affiliation wicri:level="3"><inist:fA14 i1="07"><s1>Department of Paediatric Immunology/Haematology and Stem Cell Transplantation, Leiden University Medical Centre</s1><s2>Leiden</s2><s3>NLD</s3><sZ>8 aut.</sZ></inist:fA14><country>Pays-Bas</country><placeName><settlement type="city">Leyde</settlement><region nuts="2" type="province">Hollande-Méridionale</region></placeName></affiliation></author><author><name sortKey="Boogaerts, Marc" sort="Boogaerts, Marc" uniqKey="Boogaerts M" first="Marc" last="Boogaerts">Marc Boogaerts</name><affiliation wicri:level="1"><inist:fA14 i1="08"><s1>Department of Haematology, University Hospital Gasthuisberg</s1><s2>Leuven</s2><s3>BEL</s3><sZ>9 aut.</sZ></inist:fA14><country>Belgique</country><wicri:noRegion>Leuven</wicri:noRegion></affiliation></author><author><name sortKey="Vermylen, Christiane" sort="Vermylen, Christiane" uniqKey="Vermylen C" first="Christiane" last="Vermylen">Christiane Vermylen</name><affiliation wicri:level="3"><inist:fA14 i1="09"><s1>Department of Haematology, Cliniques Universitaires St Luc</s1><s2>Brussels</s2><s3>BEL</s3><sZ>10 aut.</sZ></inist:fA14><country>Belgique</country><placeName><settlement type="city">Bruxelles</settlement><region nuts="2">Région de Bruxelles-Capitale</region></placeName></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="3"><inist:fA14 i1="10"><s1>Great Ormond Street Hospital for Children NHS Trust</s1><s2>London</s2><s3>GBR</s3><sZ>11 aut.</sZ></inist:fA14><country>Royaume-Uni</country><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Marsh, Judith" sort="Marsh, Judith" uniqKey="Marsh J" first="Judith" last="Marsh">Judith Marsh</name><affiliation wicri:level="3"><inist:fA14 i1="11"><s1>Department of Haematology, St. George's Hospital</s1><s2>London</s2><s3>GBR</s3><sZ>12 aut.</sZ></inist:fA14><country>Royaume-Uni</country><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Badell, Isabel" sort="Badell, Isabel" uniqKey="Badell I" first="Isabel" last="Badell">Isabel Badell</name><affiliation wicri:level="3"><inist:fA14 i1="12"><s1>Department of Paediatrics, Hospital de la Santa Creu i Sant Pau</s1><s2>Barcelona</s2><s3>ESP</s3><sZ>13 aut.</sZ></inist:fA14><country>Espagne</country><placeName><settlement type="city">Barcelone</settlement><region nuts="2" type="region">Catalogne</region></placeName></affiliation></author><author><name sortKey="Michel, Gerard" sort="Michel, Gerard" uniqKey="Michel G" first="Gerard" last="Michel">Gerard Michel</name><affiliation wicri:level="3"><inist:fA14 i1="13"><s1>Service d'Oncologie Pédiatrique, Hopital d'Enfants de la Timone</s1><s2>Marseille</s2><s3>FRA</s3><sZ>14 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Güngör">Tayfun Güngör</name><affiliation wicri:level="3"><inist:fA14 i1="14"><s1>Division of Immunology /Haematology /Oncology, University Children's Hospital</s1><s2>Zürich</s2><s3>CHE</s3><sZ>15 aut.</sZ></inist:fA14><country>Suisse</country><placeName><settlement type="city">Zurich</settlement><region nuts="3" type="region">Canton de Zurich</region></placeName></affiliation></author><author><name sortKey="Niethammer, Dietrich" sort="Niethammer, Dietrich" uniqKey="Niethammer D" first="Dietrich" last="Niethammer">Dietrich Niethammer</name><affiliation wicri:level="1"><inist:fA14 i1="15"><s1>Department of Paediatrics, University Hospital</s1><s2>Tubingen</s2><s3>DEU</s3><sZ>16 aut.</sZ></inist:fA14><country>Allemagne</country><wicri:noRegion>Tubingen</wicri:noRegion><wicri:noRegion>University Hospital</wicri:noRegion><wicri:noRegion>Tubingen</wicri:noRegion></affiliation></author><author><name sortKey="Bordigoni, Pierre" sort="Bordigoni, Pierre" uniqKey="Bordigoni P" first="Pierre" last="Bordigoni">Pierre Bordigoni</name><affiliation wicri:level="3"><inist:fA14 i1="16"><s1>Department of Paediatrics, Hopital d'Enfants, Vandeouvre Les Nancy</s1><s2>Nancy</s2><s3>FRA</s3><sZ>17 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Grand Est</region><region type="old region">Lorraine (région)</region><settlement type="city">Nancy</settlement></placeName></affiliation></author><author><name sortKey="Oswald, Cecilia" sort="Oswald, Cecilia" uniqKey="Oswald C" first="Cecilia" last="Oswald">Cecilia Oswald</name><affiliation wicri:level="1"><inist:fA14 i1="17"><s1>Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital</s1><s2>Randwick, NSW</s2><s3>AUS</s3><sZ>18 aut.</sZ></inist:fA14><country>Australie</country><wicri:noRegion>Randwick, NSW</wicri:noRegion></affiliation></author><author><name sortKey="Favre, Claudio" sort="Favre, Claudio" uniqKey="Favre C" first="Claudio" last="Favre">Claudio Favre</name><affiliation wicri:level="3"><inist:fA14 i1="18"><s1>Unità Operativa Oncoematologia Pediatrica, Azienda Ospedaliera Universitaria Pisa</s1><s2>Pisa</s2><s3>ITA</s3><sZ>19 aut.</sZ></inist:fA14><country>Italie</country><placeName><settlement type="city">Pise</settlement><region nuts="2">Toscane</region></placeName></affiliation></author><author><name sortKey="Passweg, Jakob" sort="Passweg, Jakob" uniqKey="Passweg J" first="Jakob" last="Passweg">Jakob Passweg</name><affiliation wicri:level="1"><inist:fA14 i1="19"><s1>Division of Haematology, Basel University Hospital</s1><s2>Basel</s2><s3>CHE</s3><sZ>20 aut.</sZ></inist:fA14><country>Suisse</country><wicri:noRegion>Basel</wicri:noRegion></affiliation></author><author><name sortKey="Dini, Giorgio" sort="Dini, Giorgio" uniqKey="Dini G" first="Giorgio" last="Dini">Giorgio Dini</name><affiliation wicri:level="1"><inist:fA14 i1="20"><s1>Department of Paediatric Haematology and Oncology, IRCCS 'G.Gaslini'</s1><s2>Genoa</s2><s3>ITA</s3><sZ>21 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Genoa</wicri:noRegion></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">INIST</idno><idno type="inist">05-0459610</idno><date when="2005">2005</date><idno type="stanalyst">PASCAL 05-0459610 INIST</idno><idno type="RBID">Pascal:05-0459610</idno><idno type="wicri:Area/PascalFrancis/Corpus">004713</idno><idno type="wicri:Area/PascalFrancis/Curation">001990</idno><idno type="wicri:Area/PascalFrancis/Checkpoint">004577</idno><idno type="wicri:explorRef" wicri:stream="PascalFrancis" 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Padova</wicri:noRegion></affiliation></author><author><name sortKey="Oneto, Rosi" sort="Oneto, Rosi" uniqKey="Oneto R" first="Rosi" last="Oneto">Rosi Oneto</name><affiliation wicri:level="1"><inist:fA14 i1="02"><s1>Division of Haematology, San Martino Hospital</s1><s2>Genova</s2><s3>ITA</s3><sZ>2 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Genova</wicri:noRegion></affiliation></author><author><name sortKey="Messina, Chiara" sort="Messina, Chiara" uniqKey="Messina C" first="Chiara" last="Messina">Chiara Messina</name><affiliation wicri:level="1"><inist:fA14 i1="01"><s1>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</s1><s3>ITA</s3><sZ>1 aut.</sZ><sZ>3 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Department of Pediatrics, Clinic of Paediatric Haematology Oncology, University of Padova</wicri:noRegion></affiliation></author><author><name sortKey="Gibson, Brenda E" sort="Gibson, Brenda E" uniqKey="Gibson B" first="Brenda E." last="Gibson">Brenda E. Gibson</name><affiliation wicri:level="1"><inist:fA14 i1="03"><s1>Department of Haematology, Royal Hospital for Sick Children</s1><s2>Glasgow</s2><s3>GBR</s3><sZ>4 aut.</sZ></inist:fA14><country>Royaume-Uni</country><wicri:noRegion>Glasgow</wicri:noRegion></affiliation></author><author><name sortKey="Buzyn, Agnes" sort="Buzyn, Agnes" uniqKey="Buzyn A" first="Agnes" last="Buzyn">Agnes Buzyn</name><affiliation wicri:level="3"><inist:fA14 i1="04"><s1>Unité d'lmmunologie et d'Hématologie and INSERM U 2 - Hôpital Necker</s1><s2>Paris</s2><s3>FRA</s3><sZ>5 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Steward, Colin" sort="Steward, Colin" uniqKey="Steward C" first="Colin" last="Steward">Colin Steward</name><affiliation wicri:level="1"><inist:fA14 i1="05"><s1>Department of Paediatric Oncology/BMT, Bristol Royal Hospital for Children</s1><s2>Bristol</s2><s3>GBR</s3><sZ>6 aut.</sZ></inist:fA14><country>Royaume-Uni</country><wicri:noRegion>Bristol</wicri:noRegion></affiliation></author><author><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name><affiliation wicri:level="3"><inist:fA14 i1="06"><s1>Department of Haematology - BMT, Hopital St Louis</s1><s2>Paris</s2><s3>FRA</s3><sZ>7 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Île-de-France</region><region type="old region">Île-de-France</region><settlement type="city">Paris</settlement></placeName></affiliation></author><author><name sortKey="Breddius, Robbert" sort="Breddius, Robbert" uniqKey="Breddius R" first="Robbert" last="Breddius">Robbert Breddius</name><affiliation wicri:level="3"><inist:fA14 i1="07"><s1>Department of Paediatric Immunology/Haematology and Stem Cell Transplantation, Leiden University Medical Centre</s1><s2>Leiden</s2><s3>NLD</s3><sZ>8 aut.</sZ></inist:fA14><country>Pays-Bas</country><placeName><settlement type="city">Leyde</settlement><region nuts="2" type="province">Hollande-Méridionale</region></placeName></affiliation></author><author><name sortKey="Boogaerts, Marc" sort="Boogaerts, Marc" uniqKey="Boogaerts M" first="Marc" last="Boogaerts">Marc Boogaerts</name><affiliation wicri:level="1"><inist:fA14 i1="08"><s1>Department of Haematology, University Hospital Gasthuisberg</s1><s2>Leuven</s2><s3>BEL</s3><sZ>9 aut.</sZ></inist:fA14><country>Belgique</country><wicri:noRegion>Leuven</wicri:noRegion></affiliation></author><author><name sortKey="Vermylen, Christiane" sort="Vermylen, Christiane" uniqKey="Vermylen C" first="Christiane" last="Vermylen">Christiane Vermylen</name><affiliation wicri:level="3"><inist:fA14 i1="09"><s1>Department of Haematology, Cliniques Universitaires St Luc</s1><s2>Brussels</s2><s3>BEL</s3><sZ>10 aut.</sZ></inist:fA14><country>Belgique</country><placeName><settlement type="city">Bruxelles</settlement><region nuts="2">Région de Bruxelles-Capitale</region></placeName></affiliation></author><author><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name><affiliation wicri:level="3"><inist:fA14 i1="10"><s1>Great Ormond Street Hospital for Children NHS Trust</s1><s2>London</s2><s3>GBR</s3><sZ>11 aut.</sZ></inist:fA14><country>Royaume-Uni</country><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Marsh, Judith" sort="Marsh, Judith" uniqKey="Marsh J" first="Judith" last="Marsh">Judith Marsh</name><affiliation wicri:level="3"><inist:fA14 i1="11"><s1>Department of Haematology, St. George's Hospital</s1><s2>London</s2><s3>GBR</s3><sZ>12 aut.</sZ></inist:fA14><country>Royaume-Uni</country><placeName><settlement type="city">Londres</settlement><region type="country">Angleterre</region><region type="région" nuts="1">Grand Londres</region></placeName></affiliation></author><author><name sortKey="Badell, Isabel" sort="Badell, Isabel" uniqKey="Badell I" first="Isabel" last="Badell">Isabel Badell</name><affiliation wicri:level="3"><inist:fA14 i1="12"><s1>Department of Paediatrics, Hospital de la Santa Creu i Sant Pau</s1><s2>Barcelona</s2><s3>ESP</s3><sZ>13 aut.</sZ></inist:fA14><country>Espagne</country><placeName><settlement type="city">Barcelone</settlement><region nuts="2" type="region">Catalogne</region></placeName></affiliation></author><author><name sortKey="Michel, Gerard" sort="Michel, Gerard" uniqKey="Michel G" first="Gerard" last="Michel">Gerard Michel</name><affiliation wicri:level="3"><inist:fA14 i1="13"><s1>Service d'Oncologie Pédiatrique, Hopital d'Enfants de la Timone</s1><s2>Marseille</s2><s3>FRA</s3><sZ>14 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Provence-Alpes-Côte d'Azur</region><region type="old region">Provence-Alpes-Côte d'Azur</region><settlement type="city">Marseille</settlement></placeName></affiliation></author><author><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Güngör">Tayfun Güngör</name><affiliation wicri:level="3"><inist:fA14 i1="14"><s1>Division of Immunology /Haematology /Oncology, University Children's Hospital</s1><s2>Zürich</s2><s3>CHE</s3><sZ>15 aut.</sZ></inist:fA14><country>Suisse</country><placeName><settlement type="city">Zurich</settlement><region nuts="3" type="region">Canton de Zurich</region></placeName></affiliation></author><author><name sortKey="Niethammer, Dietrich" sort="Niethammer, Dietrich" uniqKey="Niethammer D" first="Dietrich" last="Niethammer">Dietrich Niethammer</name><affiliation wicri:level="1"><inist:fA14 i1="15"><s1>Department of Paediatrics, University Hospital</s1><s2>Tubingen</s2><s3>DEU</s3><sZ>16 aut.</sZ></inist:fA14><country>Allemagne</country><wicri:noRegion>Tubingen</wicri:noRegion><wicri:noRegion>University Hospital</wicri:noRegion><wicri:noRegion>Tubingen</wicri:noRegion></affiliation></author><author><name sortKey="Bordigoni, Pierre" sort="Bordigoni, Pierre" uniqKey="Bordigoni P" first="Pierre" last="Bordigoni">Pierre Bordigoni</name><affiliation wicri:level="3"><inist:fA14 i1="16"><s1>Department of Paediatrics, Hopital d'Enfants, Vandeouvre Les Nancy</s1><s2>Nancy</s2><s3>FRA</s3><sZ>17 aut.</sZ></inist:fA14><country>France</country><placeName><region type="region">Grand Est</region><region type="old region">Lorraine (région)</region><settlement type="city">Nancy</settlement></placeName></affiliation></author><author><name sortKey="Oswald, Cecilia" sort="Oswald, Cecilia" uniqKey="Oswald C" first="Cecilia" last="Oswald">Cecilia Oswald</name><affiliation wicri:level="1"><inist:fA14 i1="17"><s1>Centre for Children's Cancer and Blood Disorders, Sydney Children's Hospital</s1><s2>Randwick, NSW</s2><s3>AUS</s3><sZ>18 aut.</sZ></inist:fA14><country>Australie</country><wicri:noRegion>Randwick, NSW</wicri:noRegion></affiliation></author><author><name sortKey="Favre, Claudio" sort="Favre, Claudio" uniqKey="Favre C" first="Claudio" last="Favre">Claudio Favre</name><affiliation wicri:level="3"><inist:fA14 i1="18"><s1>Unità Operativa Oncoematologia Pediatrica, Azienda Ospedaliera Universitaria Pisa</s1><s2>Pisa</s2><s3>ITA</s3><sZ>19 aut.</sZ></inist:fA14><country>Italie</country><placeName><settlement type="city">Pise</settlement><region nuts="2">Toscane</region></placeName></affiliation></author><author><name sortKey="Passweg, Jakob" sort="Passweg, Jakob" uniqKey="Passweg J" first="Jakob" last="Passweg">Jakob Passweg</name><affiliation wicri:level="1"><inist:fA14 i1="19"><s1>Division of Haematology, Basel University Hospital</s1><s2>Basel</s2><s3>CHE</s3><sZ>20 aut.</sZ></inist:fA14><country>Suisse</country><wicri:noRegion>Basel</wicri:noRegion></affiliation></author><author><name sortKey="Dini, Giorgio" sort="Dini, Giorgio" uniqKey="Dini G" first="Giorgio" last="Dini">Giorgio Dini</name><affiliation wicri:level="1"><inist:fA14 i1="20"><s1>Department of Paediatric Haematology and Oncology, IRCCS 'G.Gaslini'</s1><s2>Genoa</s2><s3>ITA</s3><sZ>21 aut.</sZ></inist:fA14><country>Italie</country><wicri:noRegion>Genoa</wicri:noRegion></affiliation></author></analytic><series><title level="j" type="main">British journal of haematology</title><title level="j" type="abbreviated">Br. j. haematol.</title><idno type="ISSN">0007-1048</idno><imprint><date when="2005">2005</date></imprint></series></biblStruct></sourceDesc><seriesStmt><title level="j" type="main">British journal of haematology</title><title level="j" type="abbreviated">Br. j. haematol.</title><idno type="ISSN">0007-1048</idno></seriesStmt></fileDesc><profileDesc><textClass><keywords scheme="KwdEn" xml:lang="en"><term>Acute</term><term>Aplastic anemia</term><term>Bone marrow failure</term><term>European group for blood and marrow transplantation</term><term>Graft</term><term>Hematology</term><term>Hematopoietic cell</term><term>Leukemia</term><term>Myelodysplastic syndrome</term><term>Stem cell</term></keywords><keywords scheme="Pascal" xml:lang="fr"><term>Greffe</term><term>Cellule hématopoïétique</term><term>Cellule souche</term><term>Myélodysplasique syndrome</term><term>Aigu</term><term>Hématologie</term><term>Leucémie</term><term>Anémie aplasique</term><term>Insuffisance médullaire</term><term>EBMT</term></keywords><keywords scheme="Wicri" type="topic" xml:lang="fr"><term>Cellule souche</term></keywords></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en">This report assessed the results of allogeneic stem cell transplantation (allo-SCT) in 26 patients with Shwachman-Diamond disease (SDS) and severe bone marrow abnormalities. The conditioning regimen was based on busulphan (54%), total body irradiation (23%), fludarabine (15%) or other chemotherapy combinations (8%). Standard prevention of graft versus host disease (GVHD) with ciclosporin ± methotrexate was adopted in 54% of the patients whilst in vivo or in vitro T-cell depletion was used in 17 and four patients respectively. Neutrophil and platelet engraftment were achieved in 21 (81%) and 17 (65%) of 26 patients after a median time of 18 days and 29 days respectively. The incidence of grade III and IV acute GVHD was 24% and of chronic GVHD 29%. Nine patients died after a median time of 70 d, post-SCT. After a median follow-up of 1.1 years, the transplant-related mortality was 35.5% (95% CI 17-54) whilst the overall survival was 64.5% (95% CI 45.7-83.2). Allo-SCT was found to be successful in more than half of SDS patients with severe bone marrow dysfunction. Further improvements would be anticipated by a better definition of the optimum time in the course of disease to transplant and by the adoption of less toxic conditioning regimens.</div></front></TEI><affiliations><list><country><li>Allemagne</li><li>Australie</li><li>Belgique</li><li>Espagne</li><li>France</li><li>Italie</li><li>Pays-Bas</li><li>Royaume-Uni</li><li>Suisse</li></country><region><li>Angleterre</li><li>Canton de Zurich</li><li>Catalogne</li><li>Grand Est</li><li>Grand Londres</li><li>Hollande-Méridionale</li><li>Lorraine (région)</li><li>Provence-Alpes-Côte d'Azur</li><li>Région de Bruxelles-Capitale</li><li>Toscane</li><li>Île-de-France</li></region><settlement><li>Barcelone</li><li>Bruxelles</li><li>Leyde</li><li>Londres</li><li>Marseille</li><li>Nancy</li><li>Paris</li><li>Pise</li><li>Zurich</li></settlement></list><tree><country name="Italie"><noRegion><name sortKey="Cesaro, Simone" sort="Cesaro, Simone" uniqKey="Cesaro S" first="Simone" last="Cesaro">Simone Cesaro</name></noRegion><name sortKey="Dini, Giorgio" sort="Dini, Giorgio" uniqKey="Dini G" first="Giorgio" last="Dini">Giorgio Dini</name><name sortKey="Favre, Claudio" sort="Favre, Claudio" uniqKey="Favre C" first="Claudio" last="Favre">Claudio Favre</name><name sortKey="Messina, Chiara" sort="Messina, Chiara" uniqKey="Messina C" first="Chiara" last="Messina">Chiara Messina</name><name sortKey="Oneto, Rosi" sort="Oneto, Rosi" uniqKey="Oneto R" first="Rosi" last="Oneto">Rosi Oneto</name></country><country name="Royaume-Uni"><noRegion><name sortKey="Gibson, Brenda E" sort="Gibson, Brenda E" uniqKey="Gibson B" first="Brenda E." last="Gibson">Brenda E. Gibson</name></noRegion><name sortKey="Marsh, Judith" sort="Marsh, Judith" uniqKey="Marsh J" first="Judith" last="Marsh">Judith Marsh</name><name sortKey="Steward, Colin" sort="Steward, Colin" uniqKey="Steward C" first="Colin" last="Steward">Colin Steward</name><name sortKey="Veys, Paul" sort="Veys, Paul" uniqKey="Veys P" first="Paul" last="Veys">Paul Veys</name></country><country name="France"><region name="Île-de-France"><name sortKey="Buzyn, Agnes" sort="Buzyn, Agnes" uniqKey="Buzyn A" first="Agnes" last="Buzyn">Agnes Buzyn</name></region><name sortKey="Bordigoni, Pierre" sort="Bordigoni, Pierre" uniqKey="Bordigoni P" first="Pierre" last="Bordigoni">Pierre Bordigoni</name><name sortKey="Gluckman, Eliane" sort="Gluckman, Eliane" uniqKey="Gluckman E" first="Eliane" last="Gluckman">Eliane Gluckman</name><name sortKey="Michel, Gerard" sort="Michel, Gerard" uniqKey="Michel G" first="Gerard" last="Michel">Gerard Michel</name></country><country name="Pays-Bas"><region name="Hollande-Méridionale"><name sortKey="Breddius, Robbert" sort="Breddius, Robbert" uniqKey="Breddius R" first="Robbert" last="Breddius">Robbert Breddius</name></region></country><country name="Belgique"><noRegion><name sortKey="Boogaerts, Marc" sort="Boogaerts, Marc" uniqKey="Boogaerts M" first="Marc" last="Boogaerts">Marc Boogaerts</name></noRegion><name sortKey="Vermylen, Christiane" sort="Vermylen, Christiane" uniqKey="Vermylen C" first="Christiane" last="Vermylen">Christiane Vermylen</name></country><country name="Espagne"><region name="Catalogne"><name sortKey="Badell, Isabel" sort="Badell, Isabel" uniqKey="Badell I" first="Isabel" last="Badell">Isabel Badell</name></region></country><country name="Suisse"><region name="Canton de Zurich"><name sortKey="Gungor, Tayfun" sort="Gungor, Tayfun" uniqKey="Gungor T" first="Tayfun" last="Güngör">Tayfun Güngör</name></region><name sortKey="Passweg, Jakob" sort="Passweg, Jakob" uniqKey="Passweg J" first="Jakob" last="Passweg">Jakob Passweg</name></country><country name="Allemagne"><noRegion><name sortKey="Niethammer, Dietrich" sort="Niethammer, Dietrich" uniqKey="Niethammer D" first="Dietrich" last="Niethammer">Dietrich Niethammer</name></noRegion></country><country name="Australie"><noRegion><name sortKey="Oswald, Cecilia" sort="Oswald, Cecilia" uniqKey="Oswald C" first="Cecilia" last="Oswald">Cecilia Oswald</name></noRegion></country></tree></affiliations></record>Pour manipuler ce document sous Unix (Dilib)
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