Haploidentical transplant in patients with myelodysplastic syndrome

Marie Robin, Raphael Porcher, Fabio Ciceri, Maria Teresa van Lint, Stella Santarone, Gerhard Ehninger, Didier Blaise, Zafer Güllbas, Soledad Gonzáles Muñiz, Mauricette Michallet, Andrea Velardi, Linda Koster, Johan Maertens, Jorge Sierra, Dominik Selleslag, Aleksandar Radujkovic, José L. Díez-Martin, Lothar Kanz, Concepcion Herrera Arroyo, Dietger Niederwieser & 5 others He Huang, Andrew McDonald, Theo de Witte, Yener Koc, Nicolaus Kröger

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Abstract

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.

Original languageEnglish
Pages (from-to)1876-1883
Number of pages8
JournalBlood advances
Volume1
Issue number22
DOIs
Publication statusPublished - Oct 10 2017

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Transplants
Myelodysplastic Syndromes
Cyclophosphamide
T-cells
Grafts
Tissue Donors
Transplantation (surgical)
Stem cells
Graft vs Host Disease
Transplantation
T-Lymphocytes
Hematopoietic Stem Cell Transplantation
Incidence
Acute Myeloid Leukemia

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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 advances, Vol. 1, No. 22, 10.10.2017, p. 1876-1883.

Research output: Contribution to journalArticle

Robin, M, Porcher, R, Ciceri, F, van Lint, MT, Santarone, S, Ehninger, G, Blaise, D, Güllbas, Z, Gonzáles Muñiz, S, Michallet, M, Velardi, A, Koster, L, Maertens, J, Sierra, J, Selleslag, D, Radujkovic, A, Díez-Martin, JL, Kanz, L, Arroyo, CH, Niederwieser, D, Huang, H, McDonald, A, de Witte, T, Koc, Y & Kröger, N 2017, 'Haploidentical transplant in patients with myelodysplastic syndrome', Blood advances, vol. 1, no. 22, pp. 1876-1883. https://doi.org/10.1182/bloodadvances.2017007146
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. / Haploidentical transplant in patients with myelodysplastic syndrome. In: Blood advances. 2017 ; Vol. 1, No. 22. pp. 1876-1883.
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abstract = "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.",
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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.

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