Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party
Standard
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, Jahrgang 55, Nr. 6, 06.2020, S. 1050-1058.Publikationen: SCORING: Beitrag in Fachzeitschrift/Zeitung › SCORING: Zeitschriftenaufsatz › Forschung › Begutachtung
Harvard
APA
Vancouver
Bibtex
}
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 -