Abstract
PT-CY use in T cell-replete haploidentical HCT has significantly improved outcomes. However, hyperhydration with MESNA in CY administration poses a challenge, in patients with cardiac/ renal problems. PT-CY also increases VOD risk with prior exposure to hepatotoxic drugs. Katsanis et al. in a phase Ia trial in patients undergoing HCT for hematological malignancies showed that partially replacing PT-CY with PT-BEN had comparable outcomes to conventional PT-CY. We conducted an ambispective study in 54 patients [haplo (39), MSD(14), and MUD(1)] with nonmalignant hematological disorders and hematological malignancies in pediatric and adult patients undergoing HCT (MAC/RIC) from February 2019 to May 2024. GvHD prophylaxis comprised of PT-CY/BEN (PT-CY 50 mg/kg Day +3; PT-BEN 90 mg/m2 Day +4) in a prospective arm (n = 21) and PT-CY/CY (50 mg/kg on Days +3, +4; comparator arm) in ambispective (prospective 12; retrospective 21) arm. In both groups, immunosuppression with CNI and MMF was also given. PT-CY/BEN was comparable to PT-CY/CY in terms of safety, efficacy, and GVHD prevention. In the PT-CY/BEN group, there was earlier neutrophil (0.008) and platelet (0.0057) engraftment with significantly lower BK viremia. Incidence of bacterial infection, TRM, EFS, and OS were comparable in both groups.
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The datasets generated during and/or analysed during the current study are available from the corresponding author on reasonable request.
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VN conceptualized, designed the study, and primarily reviewed the manuscript. MK was involved with manuscript writing, patient management, data collection, entry, and analysis. SP was responsible for statistical analysis. VS, SB, SK, JP, NM, DS, GS, CP and were involved with patient management and final manuscript review. UY reviewed the final manuscript and helped in the statistical analysis. Vivek N, AW provided dermatology consultations.
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Nair, V., Kathrotiya, M., Shirure, V. et al. Feasibility and efficacy of partial replacement of post transplantation cyclophosphamide with bendamustine on day +4 for graft versus host disease prophylaxis in patients undergoing allogeneic hematopoietic cell transplantation. Bone Marrow Transplant 60, 994–1001 (2025). https://doi.org/10.1038/s41409-025-02581-7
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DOI: https://doi.org/10.1038/s41409-025-02581-7


