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Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party

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Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. / Prata, Pedro H; Eikema, Dirk-Jan; Afansyev, Boris; Bosman, Paul; Smiers, Frans; Diez-Martin, José L; Arrais-Rodrigues, Celso; Koc, Yener; Poiré, Xavier; Sirvent, Anne; Kröger, Nicolaus; Porta, Fulvio; Holter, Wolfgang; Bloor, Adrian; Jubert, Charlotte; Ganser, Arnold; Tanase, Alina; Ménard, Anne-Lise; Pioltelli, Pietro; Pérez-Simón, José A; Ho, Aloysius; Aljurf, Mahmoud; Russell, Nigel; Labussiere-Wallet, Helene; Kerre, Tessa; Rocha, Vanderson; Socié, Gérard; Risitano, Antonio; Dufour, Carlo; Peffault de Latour, Régis; SAA WP of the EBMT.

In: BONE MARROW TRANSPL, Vol. 55, No. 6, 06.2020, p. 1050-1058.

Research output: SCORING: Contribution to journal › SCORING: Journal article › Research › peer-review

Harvard

Prata, PH, Eikema, D-J, Afansyev, B, Bosman, P, Smiers, F, Diez-Martin, JL, Arrais-Rodrigues, C, Koc, Y, Poiré, X, Sirvent, A, Kröger, N, Porta, F, Holter, W, Bloor, A, Jubert, C, Ganser, A, Tanase, A, Ménard, A-L, Pioltelli, P, Pérez-Simón, JA, Ho, A, Aljurf, M, Russell, N, Labussiere-Wallet, H, Kerre, T, Rocha, V, Socié, G, Risitano, A, Dufour, C, Peffault de Latour, R & SAA WP of the EBMT 2020, 'Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party', BONE MARROW TRANSPL, vol. 55, no. 6, pp. 1050-1058. https://doi.org/10.1038/s41409-019-0773-0

APA

Prata, P. H., Eikema, D-J., Afansyev, B., Bosman, P., Smiers, F., Diez-Martin, J. L., Arrais-Rodrigues, C., Koc, Y., Poiré, X., Sirvent, A., Kröger, N., Porta, F., Holter, W., Bloor, A., Jubert, C., Ganser, A., Tanase, A., Ménard, A-L., Pioltelli, P., ... SAA WP of the EBMT (2020). Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. BONE MARROW TRANSPL, 55(6), 1050-1058. https://doi.org/10.1038/s41409-019-0773-0

Vancouver

Prata PH, Eikema D-J, Afansyev B, Bosman P, Smiers F, Diez-Martin JL et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. BONE MARROW TRANSPL. 2020 Jun;55(6):1050-1058. https://doi.org/10.1038/s41409-019-0773-0

Bibtex

@article{ebfd2d4f64074ae5ac01c4d12d75d8ed,
title = "Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party",
abstract = "In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51-83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II-III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0-20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64-93), and 2-year graft-versus-host disease-free survival was 63% (46-81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81-100) versus 64% (41-87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.",
author = "Prata, {Pedro H} and Dirk-Jan Eikema and Boris Afansyev and Paul Bosman and Frans Smiers and Diez-Martin, {Jos{\'e} L} and Celso Arrais-Rodrigues and Yener Koc and Xavier Poir{\'e} and Anne Sirvent and Nicolaus Kr{\"o}ger and Fulvio Porta and Wolfgang Holter and Adrian Bloor and Charlotte Jubert and Arnold Ganser and Alina Tanase and Anne-Lise M{\'e}nard and Pietro Pioltelli and P{\'e}rez-Sim{\'o}n, {Jos{\'e} A} and Aloysius Ho and Mahmoud Aljurf and Nigel Russell and Helene Labussiere-Wallet and Tessa Kerre and Vanderson Rocha and G{\'e}rard Soci{\'e} and Antonio Risitano and Carlo Dufour and {Peffault de Latour}, R{\'e}gis and {SAA WP of the EBMT}",
year = "2020",
month = jun,
doi = "10.1038/s41409-019-0773-0",
language = "English",
volume = "55",
pages = "1050--1058",
journal = "BONE MARROW TRANSPL",
issn = "0268-3369",
publisher = "NATURE PUBLISHING GROUP",
number = "6",

}

RIS

TY - JOUR

T1 - Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party

AU - Prata, Pedro H

AU - Eikema, Dirk-Jan

AU - Afansyev, Boris

AU - Bosman, Paul

AU - Smiers, Frans

AU - Diez-Martin, José L

AU - Arrais-Rodrigues, Celso

AU - Koc, Yener

AU - Poiré, Xavier

AU - Sirvent, Anne

AU - Kröger, Nicolaus

AU - Porta, Fulvio

AU - Holter, Wolfgang

AU - Bloor, Adrian

AU - Jubert, Charlotte

AU - Ganser, Arnold

AU - Tanase, Alina

AU - Ménard, Anne-Lise

AU - Pioltelli, Pietro

AU - Pérez-Simón, José A

AU - Ho, Aloysius

AU - Aljurf, Mahmoud

AU - Russell, Nigel

AU - Labussiere-Wallet, Helene

AU - Kerre, Tessa

AU - Rocha, Vanderson

AU - Socié, Gérard

AU - Risitano, Antonio

AU - Dufour, Carlo

AU - Peffault de Latour, Régis

AU - SAA WP of the EBMT

PY - 2020/6

Y1 - 2020/6

N2 - In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51-83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II-III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0-20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64-93), and 2-year graft-versus-host disease-free survival was 63% (46-81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81-100) versus 64% (41-87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.

AB - In the absence of an HLA-matched donor, the best treatment for acquired aplastic anemia patients refractory to immunosuppression is unclear. We collected and analyzed data from all acquired aplastic anemia patients who underwent a haploidentical transplantation with posttransplant cyclophosphamide in Europe from 2011 to 2017 (n = 33). The cumulative incidence of neutrophil engraftment was 67% (CI95%: 51-83%) at D +28 and was unaffected by age group, stem cell source, ATG use, or Baltimore conditioning regimen. The cumulative incidence of grades II-III acute GvHD was 23% at D +100, and limited chronic GvHD was 10% (0-20) at 2 years, without cases of grade IV acute or extensive chronic GvHD. Two-year overall survival was 78% (64-93), and 2-year graft-versus-host disease-free survival was 63% (46-81). In univariate analysis, the 2-year OS was higher among patients who received the Baltimore conditioning regimen (93% (81-100) versus 64% (41-87), p = 0.03), whereas age group, stem cell source, and ATG use had no effect. Our results using unmanipulated haploidentical transplantation and posttransplant cyclophosphamide for treating refractory AA patients are encouraging, but warrant confirmation in a prospective study with a larger number of patients and longer follow-up.

U2 - 10.1038/s41409-019-0773-0

DO - 10.1038/s41409-019-0773-0

M3 - SCORING: Journal article

C2 - 31844137

VL - 55

SP - 1050

EP - 1058

JO - BONE MARROW TRANSPL

JF - BONE MARROW TRANSPL

SN - 0268-3369

IS - 6

ER -