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Autor
Ansari, Marc 1 Bader, Peter 1 Beguin, Yves 1 Bertrand, Yves 1 Bodova, Ivana 1 Bonanomi, Sonia 1 Bosman, Paul 1 Bruno, Benedicte 1 Bykova, Tatiana A 1 Dalle, Jean Hugues 1 Diallo, Safiatou 1 Dufour, Carlo 1 Díaz Pérez, Miguel Ángel 1 Eikema, Dirk-Jan 1 Faraci, Maura 1 Formankova, Renata 1 Ghavamzadeh, Ardeshir 1 Giardino, Stefano 1 Gozdzik, Jolanta 1 Holter, Wolfgang 1
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Pracoviště
Armed Forces Bone Marrow Transplant C... 1 BMT Unit Istituto Giannina Gaslini Ge... 1 Bone Marrow Transplantation Unit Sain... 1 CHU Lille Hématogie pédiatrique Lille... 1 CHU de Liège University of Liège Lièg... 1 Center for Pediatric Oncology and Hem... 1 Centre for Haematology Imperial Colle... 1 Churchill Hospital Oxford United Kingdom 1 Department of Clinical Medicine and S... 1 Department of Hematology Jules Bordet... 1 Department of Paediatric Haematology ... 1 Department of Pediatrics Hematology O... 1 Department of Pediatrics Leiden Unive... 1 Division for Stem Cell Transplantatio... 1 Division of Pediatric Hematology and ... 1 Division of Pediatric Stem Cell Thera... 1 EBMT Statistics EBMT Data Office Leid... 1 Haematology Unit IRCCS Istituto Giann... 1 Haematology Unit Ospedale Civile Pesc... 1 Hematology Oncology and Stem Cell Tra... 1
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Autor
Ansari, Marc 1 Bader, Peter 1 Beguin, Yves 1 Bertrand, Yves 1 Bodova, Ivana 1 Bonanomi, Sonia 1 Bosman, Paul 1 Bruno, Benedicte 1 Bykova, Tatiana A 1 Dalle, Jean Hugues 1 Diallo, Safiatou 1 Dufour, Carlo 1 Díaz Pérez, Miguel Ángel 1 Eikema, Dirk-Jan 1 Faraci, Maura 1 Formankova, Renata 1 Ghavamzadeh, Ardeshir 1 Giardino, Stefano 1 Gozdzik, Jolanta 1 Holter, Wolfgang 1
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Pracoviště
Armed Forces Bone Marrow Transplant C... 1 BMT Unit Istituto Giannina Gaslini Ge... 1 Bone Marrow Transplantation Unit Sain... 1 CHU Lille Hématogie pédiatrique Lille... 1 CHU de Liège University of Liège Lièg... 1 Center for Pediatric Oncology and Hem... 1 Centre for Haematology Imperial Colle... 1 Churchill Hospital Oxford United Kingdom 1 Department of Clinical Medicine and S... 1 Department of Hematology Jules Bordet... 1 Department of Paediatric Haematology ... 1 Department of Pediatrics Hematology O... 1 Department of Pediatrics Leiden Unive... 1 Division for Stem Cell Transplantatio... 1 Division of Pediatric Hematology and ... 1 Division of Pediatric Stem Cell Thera... 1 EBMT Statistics EBMT Data Office Leid... 1 Haematology Unit IRCCS Istituto Giann... 1 Haematology Unit Ospedale Civile Pesc... 1 Hematology Oncology and Stem Cell Tra... 1
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NLK
Elsevier Open Access Journals
od 2021-01-01 do Před 1 rokem
PubMed
33781541
DOI
10.1016/j.jtct.2020.12.024
Knihovny.cz E-zdroje
Data on stem cell transplantation (SCT) for Diamond-Blackfan Anemia (DBA) is limited. We studied patients transplanted for DBA and registered in the EBMT database. Between 1985 and 2016, 106 DBA patients (median age, 6.8 years) underwent hematopoietic stem cell transplantation from matched-sibling donors (57%), unrelated donors (36%), or other related donors (7%), using marrow (68%), peripheral blood stem cells (20%), both marrow and peripheral blood stem cells (1%), or cord blood (11%). The cumulative incidence of engraftment was 86% (80% to 93%), and neutrophil recovery and platelet recovery were achieved on day +18 (range, 16 to 20) and +36 (range, 32 to 43), respectively. Three-year overall survival and event-free survival were 84% (77% to 91%) and 81% (74% to 89%), respectively. Older patients were significantly more likely to die (hazard ratio, 1.4; 95% confidence interval, 1.06 to 1.23; P < .001). Outcomes were similar between sibling compared to unrelated-donor transplants. The incidence of acute grades II to IV of graft-versus-host disease (GVHD) was 30% (21% to 39%), and the incidence of extensive chronic GVHD was 15% (7% to 22%). This study shows that SCT may represent an alternative therapeutic option for transfusion-dependent younger patients.
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