Haploidentical transplant in patients with myelodysplastic syndrome
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Haploidentical transplant in patients with myelodysplastic syndrome. / Robin, Marie; Porcher, Raphael; Ciceri, Fabio; van Lint, Maria Teresa; Santarone, Stella; Ehninger, Gerhard; Blaise, Didier; Güllbas, Zafer; Gonzáles Muñiz, Soledad; Michallet, Mauricette; Velardi, Andrea; Koster, Linda; Maertens, Johan; Sierra, Jorge; Selleslag, Dominik; Radujkovic, Aleksandar; Díez-Martin, José L; Kanz, Lothar; Arroyo, Concepcion Herrera; Niederwieser, Dietger; Huang, He; McDonald, Andrew; de Witte, Theo; Koc, Yener; Kröger, Nicolaus.
in: BLOOD ADV, Jahrgang 1, Nr. 22, 10.10.2017, S. 1876-1883.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
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TY - JOUR
T1 - Haploidentical transplant in patients with myelodysplastic syndrome
AU - Robin, Marie
AU - Porcher, Raphael
AU - Ciceri, Fabio
AU - van Lint, Maria Teresa
AU - Santarone, Stella
AU - Ehninger, Gerhard
AU - Blaise, Didier
AU - Güllbas, Zafer
AU - Gonzáles Muñiz, Soledad
AU - Michallet, Mauricette
AU - Velardi, Andrea
AU - Koster, Linda
AU - Maertens, Johan
AU - Sierra, Jorge
AU - Selleslag, Dominik
AU - Radujkovic, Aleksandar
AU - Díez-Martin, José L
AU - Kanz, Lothar
AU - Arroyo, Concepcion Herrera
AU - Niederwieser, Dietger
AU - Huang, He
AU - McDonald, Andrew
AU - de Witte, Theo
AU - Koc, Yener
AU - Kröger, Nicolaus
PY - 2017/10/10
Y1 - 2017/10/10
N2 - 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.
AB - 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.
KW - Journal Article
U2 - 10.1182/bloodadvances.2017007146
DO - 10.1182/bloodadvances.2017007146
M3 - SCORING: Journal article
C2 - 29296834
VL - 1
SP - 1876
EP - 1883
JO - BLOOD ADV
JF - BLOOD ADV
SN - 2473-9529
IS - 22
ER -