Abstract
Patients with refractory severe aplastic anemia (SAA) who lack a matched sibling or unrelated donor need new therapeutic approaches. Hematopoietic SCT (HSCT) using mismatched or haploidentical related donors has been used in the past, but was associated with a significant risk of GVHD and mortality. Recently, the use of post-transplant cyclophosphamide (Cy) has been shown to be an effective strategy to prevent GVHD in recipients of haploidentical HSCT, but the majority of reports have focused on patients with hematology malignancies. We describe the outcome of 16 patients who underwent haploidentical transplantation using a reduced-intensity conditioning regimen with post-transplant Cy. Stem cell sources were BM (N=13) or PBSCs (N=3). The rate of neutrophil engraftment was 94% and of platelet engraftment was 75%. Two patients had secondary graft failure and were successfully salvaged with another transplant. Three patients developed acute GVHD being grades 2–4 in two. Five patients have died and the 1-year OS was 67.1% (95% confidence interval: 36.5–86.4%). In our small series, the use of a reduced-intensity conditioning with post-transplant Cy in haploidentical BMT was associated with high rates of engraftment and low risk of GVHD in patients with relapsed/refractory SAA.
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Acknowledgements
We thank Phillip Scheinberg for critically reading the manuscript.
Author Contributions
IE designed the study, collected data, analyzed data and wrote the paper; CB, VR and NH designed the study, collected data and reviewed the paper; FPSS designed the study, analyzed data and wrote the paper; all the other authors contributed to data collection, analysis and critically reviewed the paper.
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Esteves, I., Bonfim, C., Pasquini, R. et al. Haploidentical BMT and post-transplant Cy for severe aplastic anemia: a multicenter retrospective study. Bone Marrow Transplant 50, 685–689 (2015). https://doi.org/10.1038/bmt.2015.20
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DOI: https://doi.org/10.1038/bmt.2015.20
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