Gluckman, Eliane and de la Fuente, Josu and Cappelli, Barbara and Scigliuolo, Graziana M. and Volt, Fernanda and Tozatto-Maio, Karina and Rocha, Vanderson and Tommaso, Mina and O'Boyle, Farah and Smiers, Frans and Da Cunha-Riehm, Claudia Bettoni and Calore, Elisabetta and Bonanomi, Sonia and Graphakos, Stelios and Paisiou, Anna and Albert, Michael H. and Ruggeri, Annalisa and Zecca, Marco and Lankester, Arjan C. and Corbacioglu, Selim (2020) The role of HLA matching in unrelated donor hematopoietic stem cell transplantation for sickle cell disease in Europe. BONE MARROW TRANSPLANTATION, 55 (10). pp. 1946-1954. ISSN 0268-3369, 1476-5365
Full text not available from this repository. (Request a copy)Abstract
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.
| Item Type: | Article |
|---|---|
| Uncontrolled Keywords: | BONE-MARROW-TRANSPLANTATION; CORD BLOOD TRANSPLANTATION; RELAPSE-FREE SURVIVAL; QUALITY-OF-LIFE; CHILDREN; CHIMERISM; PATIENT; GRAFTS; CURE; |
| Subjects: | 600 Technology > 610 Medical sciences Medicine |
| Divisions: | Medicine > Abteilung für Pädiatrische Hämatologie, Onkologie und Stammzelltransplantation |
| Depositing User: | Dr. Gernot Deinzer |
| Date Deposited: | 29 Mar 2021 08:41 |
| Last Modified: | 29 Mar 2021 08:41 |
| URI: | https://pred.uni-regensburg.de/id/eprint/44951 |
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