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Posttransplant cyclophosphamide in allogeneic bone marrow transplantation for the treatment of nonmalignant hematological diseases

Bone Marrow Transplantation volume 55, pages 758–762 (2020)Cite this article

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Abstract

We present a single-center retrospective series of allogeneic bone marrow transplantation (BMT) with the use of posttransplant cyclophosphamide (PTCy) in the setting of nonmalignant hematological conditions. Nine patients were treated between 2013 and 2019. Nonmyeloablative conditioning consisted of antithymocyte globulin, fludarabine, low-dose cyclophosphamide, and total body irradiation (200cGy) followed by allogeneic bone marrow infusion. Post-BMT GVHD prophylaxis was with PTCy, tacrolimus, and mycophenolate mofetil. At a median follow-up of 24 months (range 4, 63), all patients are alive, with donor-derived hematopoiesis and free of significant acute or chronic GVHD. Donors were haploidentical (n = 6), fully matched unrelated (n = 2), and fully matched sibling (n = 1). Neutrophil and platelet engraftment occurred at a median of 21 days and 33 days, respectively, after transplantation. Three patients (3/9, 33%) experienced stage 1–2 acute skin GVHD. The only cases of chronic GVHD are in three patients (3/9, 33%) with ocular disease (two mild, one moderate). No patient has required systemic immunosuppression beyond 12 months after BMT. PTCy-based nonmyeloablative allogeneic BMT is safe and effective for nonmalignant hematologic conditions and should be prospectively compared with historical regimens.

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Authors and Affiliations

  1. Blood and Marrow Transplant Program, Massachusetts General Hospital, Boston, MA, USA

    Mark Leick, Bradley Hunter, Zachariah DeFilipp, Bimalangshu R. Dey, Areej El-Jawahri, Matthew Frigault, Steven McAfee, Thomas R. Spitzer, Paul O’Donnell & Yi-Bin Chen

  2. Harvard Medical School, Boston, MA, USA

    Mark Leick, Bradley Hunter, Zachariah DeFilipp, Bimalangshu R. Dey, Areej El-Jawahri, Matthew Frigault, Steven McAfee, Thomas R. Spitzer, Paul O’Donnell & Yi-Bin Chen

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  1. Mark Leick
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Correspondence to Yi-Bin Chen.

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Leick, M., Hunter, B., DeFilipp, Z. et al. Posttransplant cyclophosphamide in allogeneic bone marrow transplantation for the treatment of nonmalignant hematological diseases. Bone Marrow Transplant 55, 758–762 (2020). https://doi.org/10.1038/s41409-019-0725-8

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