Family Mismatched Allogeneic Stem Cell Transplantation for Myelofibrosis: Report from the Chronic Malignancies Working Party of European Society for Blood and Marrow Transplantation
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Family Mismatched Allogeneic Stem Cell Transplantation for Myelofibrosis: Report from the Chronic Malignancies Working Party of European Society for Blood and Marrow Transplantation. / Raj, Kavita; Eikema, Diderik-Jan; McLornan, Donal P; Olavarria, Eduardo; Blok, Henric-Jan; Bregante, Stefania; Ciceri, Fabio; Passweg, Jakob; Ljungman, Per; Schaap, Nicolaas; Carlson, Kristina; Zuckerman, Tsila; de Wreede, Liesbeth C; Volin, Liisa; Koc, Yener; Diez-Martin, Jose Luis; Brossart, Peter; Wolf, Dominik; Blaise, Didier; Bartolomeo, Paolo Di; Vitek, Antonin; Robin, Marie; Yakoub-Agha, Ibrahim; Chalandon, Yves; Kroger, Nicolaus.
In: BIOL BLOOD MARROW TR, Vol. 25, No. 3, 03.2019, p. 522-528.Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review
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TY - JOUR
T1 - Family Mismatched Allogeneic Stem Cell Transplantation for Myelofibrosis: Report from the Chronic Malignancies Working Party of European Society for Blood and Marrow Transplantation
AU - Raj, Kavita
AU - Eikema, Diderik-Jan
AU - McLornan, Donal P
AU - Olavarria, Eduardo
AU - Blok, Henric-Jan
AU - Bregante, Stefania
AU - Ciceri, Fabio
AU - Passweg, Jakob
AU - Ljungman, Per
AU - Schaap, Nicolaas
AU - Carlson, Kristina
AU - Zuckerman, Tsila
AU - de Wreede, Liesbeth C
AU - Volin, Liisa
AU - Koc, Yener
AU - Diez-Martin, Jose Luis
AU - Brossart, Peter
AU - Wolf, Dominik
AU - Blaise, Didier
AU - Bartolomeo, Paolo Di
AU - Vitek, Antonin
AU - Robin, Marie
AU - Yakoub-Agha, Ibrahim
AU - Chalandon, Yves
AU - Kroger, Nicolaus
N1 - Copyright © 2018 Elsevier Ltd. All rights reserved.
PY - 2019/3
Y1 - 2019/3
N2 - This analysis included 56 myelofibrosis (MF) patients transplanted from family mismatched donor between 2009 and 2015 enrolled in the European Society for Blood and Marrow Transplantation database. The median age was 57 years (range, 38 to 72); 75% had primary MF and 25% had secondary MF. JAK2 V617F was mutated in 61%. Donors were HLA mismatched at 2 or more loci. Stem cells were sourced from bone marrow in 66% and peripheral blood in 34%. The median CD34+ cell dose was 4.8 × 106/kg (range, 1.7 to 22.9; n = 43). Conditioning was predominantly myeloablative in 70% and reduced intensity in the remainder. Regimens were heterogeneous with thiotepa, busulfan, fludarabine, and post-transplant cyclophosphamide used in 59%. The incidence of neutrophil engraftment by 28 days was 82% (range, 70% to 93%), at a median of 21 days (range, 19 to 23). At 2 years the cumulative incidence of primary graft failure was 9% (95% CI 1% to 16%) and secondary graft failure was 13% (95% CI 4% to 22%). The cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV was 28% (95% CI 16% to 40%) and 9% (95% CI 2% to 17%) at 100 days. The cumulative incidence of chronic GVHD at 1 year was 45% (95% CI 32% to 58%), but the cumulative incidence of death without chronic GVHD by 1 year was 20% (95% CI 10% to 31%). With a median follow-up of 32 months, the 1- and 2-year overall survival was 61% (95% CI 48% to 74%) and 56% (95% CI 41% to 70%), respectively. The 1- and 2- year progression-free survival was 58% (95% CI 45% to 71%) and 43% (95% CI 28% to 58%), respectively, with a 2-year cumulative incidence of relapse of 19% (95% CI 7% to 31%). The 2-year nonrelapse mortality was 38% (95% CI 24% to 51%). This retrospective study of MF allo-SCT using family mismatched donors demonstrated feasibility of the approach, timely neutrophil engraftment in over 80% of cases, and acceptable overall and progression-free survival rates with relapse rates not dissimilar to the unrelated donor setting. However, strategies to minimize the risk of graft failure and the relatively high nonrelapse mortality need to be used, ideally in a multicenter prospective fashion.
AB - This analysis included 56 myelofibrosis (MF) patients transplanted from family mismatched donor between 2009 and 2015 enrolled in the European Society for Blood and Marrow Transplantation database. The median age was 57 years (range, 38 to 72); 75% had primary MF and 25% had secondary MF. JAK2 V617F was mutated in 61%. Donors were HLA mismatched at 2 or more loci. Stem cells were sourced from bone marrow in 66% and peripheral blood in 34%. The median CD34+ cell dose was 4.8 × 106/kg (range, 1.7 to 22.9; n = 43). Conditioning was predominantly myeloablative in 70% and reduced intensity in the remainder. Regimens were heterogeneous with thiotepa, busulfan, fludarabine, and post-transplant cyclophosphamide used in 59%. The incidence of neutrophil engraftment by 28 days was 82% (range, 70% to 93%), at a median of 21 days (range, 19 to 23). At 2 years the cumulative incidence of primary graft failure was 9% (95% CI 1% to 16%) and secondary graft failure was 13% (95% CI 4% to 22%). The cumulative incidence of acute graft-versus-host disease (GVHD) grades II to IV and III to IV was 28% (95% CI 16% to 40%) and 9% (95% CI 2% to 17%) at 100 days. The cumulative incidence of chronic GVHD at 1 year was 45% (95% CI 32% to 58%), but the cumulative incidence of death without chronic GVHD by 1 year was 20% (95% CI 10% to 31%). With a median follow-up of 32 months, the 1- and 2-year overall survival was 61% (95% CI 48% to 74%) and 56% (95% CI 41% to 70%), respectively. The 1- and 2- year progression-free survival was 58% (95% CI 45% to 71%) and 43% (95% CI 28% to 58%), respectively, with a 2-year cumulative incidence of relapse of 19% (95% CI 7% to 31%). The 2-year nonrelapse mortality was 38% (95% CI 24% to 51%). This retrospective study of MF allo-SCT using family mismatched donors demonstrated feasibility of the approach, timely neutrophil engraftment in over 80% of cases, and acceptable overall and progression-free survival rates with relapse rates not dissimilar to the unrelated donor setting. However, strategies to minimize the risk of graft failure and the relatively high nonrelapse mortality need to be used, ideally in a multicenter prospective fashion.
KW - Journal Article
U2 - 10.1016/j.bbmt.2018.10.017
DO - 10.1016/j.bbmt.2018.10.017
M3 - SCORING: Journal article
C2 - 30408564
VL - 25
SP - 522
EP - 528
JO - BIOL BLOOD MARROW TR
JF - BIOL BLOOD MARROW TR
SN - 1083-8791
IS - 3
ER -