Leiden University Scholarly Publications

Persistent URL of this record https://hdl.handle.net/1887/3181404

In Collections

This item can be found in the following collections:

Prata, P.H.; Eikema, D.J.; Afansyev, B.; Bosman, P.; Smiers, F.; Diez-Martin, J.L.; ... ; SAA WP EBMT (2020)

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

Article / Letter to editor
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...Show moreIn 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.Show less
All authors
Prata, P.H.; Eikema, D.J.; Afansyev, B.; Bosman, P.; Smiers, F.; Diez-Martin, J.L.; Arrais-Rodrigues, C.; Koc, Y.; Poir?, X.; Sirvent, A.; Kr?ger, N.; Porta, F.; Holter, W.; Bloor, A.; Jubert, C.; Ganser, A.; Tanase, A.; M?nard, A.L.; Pioltelli, P.; P?rez-Sim?n, J.A.; Ho, A.; Aljurf, M.; Russell, N.; Labussiere-Wallet, H.; Kerre, T.; Rocha, V.; Soci?, G.; Risitano, A.; Dufour, C.; Latour, R.P. de; SAA WP EBMT
Date
2020-06-01
Journal
Bone Marrow Transplantation
Volume
55
Issue
6
Pages
1050 - 1058
DOI
doi:10.1038/s41409-019-0773-0