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.
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Acknowledgements
The authors are grateful to the Fondation HPN France—Aplasie Médullaire (Paroxysmal Nocturnal Hemoglobinuria—Aplastic Anemia Foundation in France) for helping fund this study.
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PHP, RPL, CD and AR planned this study. PHP, DJE and RPL wrote the paper with support of GS. PB was responsible for data collection and processing. DJE performed statistical analyses. All authors provided critical feedback and helped shape the research, analyses, and paper.
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Prata, P.H., Eikema, DJ., Afansyev, B. et al. Haploidentical transplantation and posttransplant cyclophosphamide for treating aplastic anemia patients: a report from the EBMT Severe Aplastic Anemia Working Party. Bone Marrow Transplant 55, 1050–1058 (2020). https://doi.org/10.1038/s41409-019-0773-0
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DOI: https://doi.org/10.1038/s41409-019-0773-0
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