PMID- 32201153 OWN - NLM STAT- MEDLINE DCOM- 20210326 LR - 20210326 IS - 2589-0646 (Electronic) IS - 2589-0646 (Linking) VI - 13 IP - 4 DP - 2020 Dec TI - Alternative donor hematopoietic stem cell transplantation for sickle cell disease in Europe. PG - 181-188 LID - S1658-3876(20)30034-0 [pii] LID - 10.1016/j.hemonc.2019.12.011 [doi] AB - Allogeneic hematopoietic stem cell transplantation (HSCT) is, to date, the only curative treatment for sickle cell disease (SCD). Because an human leukocyte antigen (HLA)-matched sibling donor is not always available, alternative stem cell sources such as unrelated or haploidentical related donors have been explored. To date, few series of SCD patients transplanted with an unrelated donor, cord blood, and haploidentical related donor have been reported, but the high rates of rejection and chronic graft versus host disease have limited their widespread application. We describe the outcomes of a retrospective, registry-based, survey on 144 alternative donor HSCT performed for SCD in 30 European Society for Blood and Marrow Transplantation centers between 1999 and 2017. Data on 70 unrelated adult donors (49%), six cord blood (4%), and 68 haploidentical donors (47%; including post-HSCT Cy, ex vivo T-cell depleted, and other haplo-HSCTs) were reported and missing information was updated by the centers. Overall, 16% patients experienced graft failure, Grade II-IV acute GVHD at 100 days was 24%, whereas Grade III-IV was 10%. Chronic GVHD was observed in 24% (limited for 13 patients and extensive for 18 patients). Overall, the 3-year overall survival (OS) was 86% +/- 3% and 3-year event-free survival (EFS; considering death and graft failure as events) was 72% +/- 4%. We therefore conclude that alternative donor HSCT for SCD can be feasible but efforts in decreasing relapse and GVHD should be promoted to increase its safe and successful utilization. Moreover, a better knowledge of HLA matching and the tailoring of conditioning could help improve EFS and OS. CI - Copyright (c) 2020 King Faisal Specialist Hospital & Research Centre. Published by Elsevier Ltd. All rights reserved. FAU - Gluckman, Eliane AU - Gluckman E AD - Monacord, Centre Scientifique de Monaco, Monaco; Eurocord, Hopital Saint Louis, Universite Paris Diderot, Paris, France. Electronic address: eliane.gluckman@aphp.fr. FAU - Cappelli, Barbara AU - Cappelli B AD - Monacord, Centre Scientifique de Monaco, Monaco; Eurocord, Hopital Saint Louis, Universite Paris Diderot, Paris, France. FAU - Scigliuolo, Graziana Maria AU - Scigliuolo GM AD - Monacord, Centre Scientifique de Monaco, Monaco; Eurocord, Hopital Saint Louis, Universite Paris Diderot, Paris, France. FAU - De la Fuente, Josu AU - De la Fuente J AD - Department of Pediatrics, St Mary's Hospital, Imperial College, London, UK. FAU - Corbacioglu, Selim AU - Corbacioglu S AD - Department of Pediatric Hematology, Oncology and Stem Cell Transplantation, University of Regensburg, Regensburg, Germany. LA - eng PT - Journal Article PT - Review DEP - 20200316 PL - Saudi Arabia TA - Hematol Oncol Stem Cell Ther JT - Hematology/oncology and stem cell therapy JID - 101468532 SB - IM MH - Adolescent MH - Adult MH - Aged MH - Anemia, Sickle Cell/*mortality/*therapy MH - Child MH - Child, Preschool MH - Disease-Free Survival MH - Europe/epidemiology MH - Female MH - Graft vs Host Disease/etiology/*mortality/prevention & control MH - *Hematopoietic Stem Cell Transplantation MH - Humans MH - Lymphocyte Depletion MH - Male MH - Middle Aged MH - Survival Rate MH - *Transplantation Conditioning MH - *Unrelated Donors OTO - NOTNLM OT - Alternative donor HSCT OT - Haploidentical HSCT OT - Sickle cell disease OT - Unrelated donor HSCT COIS- Declaration of Competing Interest The authors declare that they have no known competing financial interests or personal relationships that could have appeared to influence the work reported in this paper. EDAT- 2020/03/24 06:00 MHDA- 2021/03/27 06:00 CRDT- 2020/03/24 06:00 PHST- 2019/10/31 00:00 [received] PHST- 2019/12/11 00:00 [accepted] PHST- 2020/03/24 06:00 [pubmed] PHST- 2021/03/27 06:00 [medline] PHST- 2020/03/24 06:00 [entrez] AID - S1658-3876(20)30034-0 [pii] AID - 10.1016/j.hemonc.2019.12.011 [doi] PST - ppublish SO - Hematol Oncol Stem Cell Ther. 2020 Dec;13(4):181-188. doi: 10.1016/j.hemonc.2019.12.011. Epub 2020 Mar 16.