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Blood Adv. 2017 Sep 27;1(22):1876-1883. doi: 10.1182/bloodadvances.2017007146. eCollection 2017 Oct 10.

Haploidentical transplant in patients with myelodysplastic syndrome.

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Hospital Saint-Louis, Assistance Publique Hôpitaux de Paris, INSERM 1131, University Paris 7, Paris, France.
Team methods, Epidemiology and Statistics Sorbonne Paris Cité Research Centre, Unité Mixte de Recherche 1153, INSERM, Paris Descartes University, Paris, France.
Epidemiology Center, Assistance Publique Hôpitaux de Paris, Hôpital Hôtel-Dieu, Paris, France.
Division of Hematology/Oncology, Department of Internal Medicine, San Raffaele Hospital, Milan, Italy.
Department of Hematology, San Martino Hospital, Genoa, Italy.
Department of Hematology, Civil Hospital, Pescara, Italy.
Division of Hematology, Department of Internal Medicine, Dresden University Hospital, Dresden, Germany.
Department Hematology, Paoli Calmettes Institute, Marseille, France.
Hematologic Oncology and Bone Marrow Transplantation Unit, Anadolu Medical Center Hospital, Kocaeli, Turkey.
Division of Hematology/Oncology, Asturias Central University Hospital, Oviedo, Spain.
Department of Hematology, Lyon Sud Hospital, Lyon, France.
Division of Hematology and Clinical Immunology, Perugia University, Perugia, Italy.
European Group for Blood and Marrow Transplantation Data Office, Leiden, The Netherlands.
Department of Hematology, University Hospital Gasthuisberg, Leuven, Belgium.
Department of Internal Medicine, Hospital Santa Creu i Sant Pau, Barcelona, Spain.
A.Z. Sint-Jan, Brugge, Belgium.
Division of Hematology/Oncology, Department of Internal Medicine, University of Heidelberg, Heidelberg, Germany.
Department of Hematology, Gregorio Marañon General University Hospital, Madrid, Spain.
Department of Hematology/Immunology/Oncology, Universität Tübingen, Tübingen, Germany.
Department of Hematology, Hospital Reina Sofia Córdoba Hospital, Córdoba, Spain.
Department of Hematology and Oncology, University Hospital Leipzig, Leipzig, Germany.
Department of Hematology, First Affiliated Hospital, Zhejiang University, Hangzhou Zhejiang, China.
Department of Hematology, Netcare Pretoria East Hospital, Pretoria Gauteng, South Africa.
Department of Hematopoietic Diseases, Radboud University Medical Center, Nijmegen, The Netherlands.
Stem Cell Transplant Unit, Medical Park Hospitals, Antalya, Turkey; and.
Department of Hematology/Oncology, University Hospital Eppendorf, Hamburg, Germany.


The only curative treatment in patients with intermediate or high-risk myelodysplastic syndrome (MDS) is allogeneic hematopoietic stem cell transplantation (HSCT), which usually results in a long-term, disease-free survival rate of between 30% and 50%, depending on the disease risk and the type of donor. In patients without an HLA-matched sibling donor, a family haploidentical donor is an alternative option. The present study reports the European Group for Blood and Marrow Transplantation activity for haploidentical transplantation in MDS patients. A total of 228 patients transplanted from a mismatched HLA-related donor between 2007 and 2014 were studied. The median age at transplant was 56 years. Eighty-four (37%) patients had MDS transformed into acute myeloid leukemia at the time of transplant. Ex vivo T-cell depletion was used in 34 patients. One hundred ninety-four patients received a T-cell replete transplant and 102 patients received posttransplant cyclophosphamide (PT-CY) as graft-versus-host disease (GVHD) prophylaxis. The cumulative incidences of acute and chronic GVHD in PT-CY vs other patients were 25% vs 37% and 37% vs 24%, respectively. The cumulative incidence of nonrelapse mortality was 55% in patients who did not receive PT-CY (no PT-CY) and 41% in patients who did receive PT-CY. Three-year overall survival was 28% in no PT-CY patients and 38% in PT-CY patients. In multivariable analysis, the main risk factors were the intensity of the conditioning regimen and the use of PT-CY. In conclusion, the outcomes of MDS patients who received an haploidentical transplant are close to the results other transplantations from HLA-mismatched donors with approximately one-third of patients alive and free of disease 3 years after transplant, and the use of PT-CY may improve their outcomes.

Conflict of interest statement

Conflict-of-interest disclosure: The authors declare no competing financial interests.

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