The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe: Bone Marrow Transplantation

E. Gluckman, J. Fuente, B. Cappelli, G.M. Scigliuolo, F. Volt, K. Tozatto-Maio, V. Rocha, M. Tommaso, F. O’Boyle, F. Smiers, C.B.D. Cunha-Riehm, E. Calore, S. Bonanomi, S. Graphakos, A. Paisiou, M.H. Albert, A. Ruggeri, M. Zecca, A.C. Lankester, S. Corbaciogluon behalf of Paediatric Diseases (PDWP) and Inborn Errors Working Parties (IEWP) of the EBMT

Research output: Contribution to journalArticlepeer-review


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. © 2020, The Author(s), under exclusive licence to Springer Nature Limited.
Original languageEnglish
Pages (from-to)1946-1954
Number of pages9
JournalBone Marrow Transplant.
Issue number10
Publication statusPublished - 2020


  • busulfan
  • cyclophosphamide
  • fludarabine
  • thiotepa
  • treosulfan
  • acute graft versus host disease
  • adolescent
  • adult
  • Article
  • child
  • chronic graft versus host disease
  • engraftment
  • Europe
  • female
  • graft failure
  • hematopoietic stem cell transplantation
  • HLA matching
  • human
  • major clinical study
  • male
  • neutrophil
  • overall survival
  • priority journal
  • recipient
  • retrospective study
  • sickle cell anemia
  • thrombocyte
  • transplantation conditioning
  • unrelated donor


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