Abstract
A total of 21 patients with severe aplastic anemia (SAA) underwent marrow transplantation from HLA-identical siblings following a standard conditioning regimen with cyclophosphamide (50 mg/kg/day × 4 days) and horse antithymocyte globulin (30 mg/kg/day × 3 days). Post-grafting immunosuppression consisted of a short course of methotrexate (MTX) combined with cyclosporine (CSP). The transplant protocol tested the hypothesis that the incidence of chronic GvHD could be reduced by limiting the marrow grafts to ⩽2.5 × 108 nucleated marrow cells/kg. None of the patients rejected the graft, all had sustained engraftment and all are surviving at a median of 4 (range 1–8) years after transplantation. Chronic GvHD developed in 16% of patients given ⩽2.5 × 108 nucleated marrow cells/kg. Post-grafting immunosuppression has been discontinued in 20 of the 21 patients. In conclusion, limiting the number of transplanted marrow cells may have resulted in minimal improvement in the incidence and severity of chronic GvHD.
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Acknowledgements
We are grateful to all research nurses and data coordinators for implementation of protocols. We also thank our administrative staff for their assistance with manuscript preparation. We are grateful to the many physicians, nurses, physician assistants, nurse practitioners, pharmacists and support staff who cared for our patients, and to the patients who allowed us to care for them and who participated in our ongoing clinical research. This study was supported by Grants HL122173, HL036444, DK106829, CA018029 and CA015704 from the National Institutes of Health, Bethesda, MD, USA. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health nor its subsidiary Institutes and Centers.
Author contributions
SG collected and analyzed the data and assisted in writing the paper; AEW, LMB, MEDF, PH, MAP, H-PK and BMS entered patients on the trial and critically reviewed and revised the paper; BES performed statistical analyses and critically reviewed the paper; SH performed flow cytometry analyses on the marrow cells and critically reviewed the paper; RS designed the study, entered patients on the trial, analyzed the data and wrote the paper.
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Gallo, S., Woolfrey, A., Burroughs, L. et al. Marrow grafts from HLA-identical siblings for severe aplastic anemia: does limiting the number of transplanted marrow cells reduce the risk of chronic GvHD?. Bone Marrow Transplant 51, 1573–1578 (2016). https://doi.org/10.1038/bmt.2016.198
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DOI: https://doi.org/10.1038/bmt.2016.198
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