TY - JOUR
T1 - The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe
AU - on behalf of Paediatric Diseases (PDWP) and Inborn Errors Working Parties (IEWP) of the EBMT
AU - Gluckman, Eliane
AU - Fuente, Josu de la
AU - Cappelli, Barbara
AU - Scigliuolo, Graziana M.
AU - Volt, Fernanda
AU - Tozatto-Maio, Karina
AU - Rocha, Vanderson
AU - Tommaso, Mina
AU - O’Boyle, Farah
AU - Smiers, Frans
AU - Cunha-Riehm, Claudia Bettoni Da
AU - Calore, Elisabetta
AU - Bonanomi, Sonia
AU - Graphakos, Stelios
AU - Paisiou, Anna
AU - Albert, Michael H.
AU - Ruggeri, Annalisa
AU - Zecca, Marco
AU - Lankester, Arjan C.
AU - Corbacioglu, Selim
PY - 2020/10/1
Y1 - 2020/10/1
N2 - We report the results of an analysis of unrelated allogeneic hematopoietic stem cell transplantations (HSCT) in 71 patients with sickle cell disease (SCD) transplanted in EBMT centers between 2005 and 2017. Median age was 9.3 years; graft type was bone marrow in 79% and peripheral blood in 21%. Recipient–donor HLA match at high resolution typing was 10/10 in 31, 9/10 in 20, and 8/10 in 4 patients; the other patients had intermediate resolution typing. The most frequent conditioning regimens were fludarabine–thiotepa–treosulfan (64%) or busulfan–cyclophosphamide (12%). Cumulative incidence of neutrophil engraftment was 92%; platelet engraftment was 90%. Eleven patients (15%) experienced graft failure. Grade II–IV acute graft-vs.-host disease (GvHD) was 23%; 3-year chronic GvHD was 23%. Three-year overall survival (OS) was 88 ± 4%. GRFS was 62 ± 6%. HLA matching was the most significant risk factor for OS: 3-year OS was 96 ± 4% in 10/10 group vs. 75 ± 10% in 9–8/10 (p = 0.042); GRFS was 69 ± 9% vs. 50 ± 12% (p = 0.114), respectively. In conclusion, unrelated donor HSCT is a valid option for SCD patients who lack an HLA-identical sibling donor, preferably in the context of clinical trials. Using a 10/10 HLA-matched unrelated donor yields better survival indicating that HLA matching is an important donor selection factor in this nonmalignant disease.
AB - We report the results of an analysis of unrelated allogeneic hematopoietic stem cell transplantations (HSCT) in 71 patients with sickle cell disease (SCD) transplanted in EBMT centers between 2005 and 2017. Median age was 9.3 years; graft type was bone marrow in 79% and peripheral blood in 21%. Recipient–donor HLA match at high resolution typing was 10/10 in 31, 9/10 in 20, and 8/10 in 4 patients; the other patients had intermediate resolution typing. The most frequent conditioning regimens were fludarabine–thiotepa–treosulfan (64%) or busulfan–cyclophosphamide (12%). Cumulative incidence of neutrophil engraftment was 92%; platelet engraftment was 90%. Eleven patients (15%) experienced graft failure. Grade II–IV acute graft-vs.-host disease (GvHD) was 23%; 3-year chronic GvHD was 23%. Three-year overall survival (OS) was 88 ± 4%. GRFS was 62 ± 6%. HLA matching was the most significant risk factor for OS: 3-year OS was 96 ± 4% in 10/10 group vs. 75 ± 10% in 9–8/10 (p = 0.042); GRFS was 69 ± 9% vs. 50 ± 12% (p = 0.114), respectively. In conclusion, unrelated donor HSCT is a valid option for SCD patients who lack an HLA-identical sibling donor, preferably in the context of clinical trials. Using a 10/10 HLA-matched unrelated donor yields better survival indicating that HLA matching is an important donor selection factor in this nonmalignant disease.
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U2 - 10.1038/s41409-020-0847-z
DO - 10.1038/s41409-020-0847-z
M3 - Article
C2 - 32157246
AN - SCOPUS:85081672936
JO - Bone Marrow Transplantation
JF - Bone Marrow Transplantation
SN - 0268-3369
ER -