Abstract
We report the results from a multicenter retrospective study of 69 adult patients who underwent haploidentical blood or marrow transplantation (haplo-BMT) with post-transplantation cyclophosphamide (PTCy) for chronic phase myelofibrosis. The median age at BMT was 63 years (range, 41–74). Conditioning regimens were reduced intensity in 54% and nonmyeloablative in 39%. Peripheral blood grafts were used in 86%. The median follow-up was 23.1 months (range, 1.6–75.7). At 3 years, the overall survival, relapse-free survival (RFS), and graft-versus-host-disease (GVHD)-free-RFS were 72% (95% CI 59–81), 44% (95% CI 29–59), and 30% (95% CI 17–43). Cumulative incidences of non-relapse mortality and relapse were 23% (95% CI 14–34) and 31% (95% CI 17–47) at 3 years. Spleen size ≥22 cm or prior splenectomy (HR 6.37, 95% CI 2.02–20.1, P = 0.002), and bone marrow grafts (HR 4.92, 95% CI 1.68–14.4, P = 0.004) were associated with increased incidence of relapse. Cumulative incidence of acute GVHD grade 3–4 was 10% at 3 months and extensive chronic GVHD was 8%. Neutrophil engraftment was reported in 94% patients, at a median of 20 days (range, 14–70). In conclusion, haplo-BMT with PTCy is feasible in patients with myelofibrosis. Splenomegaly ≥22 cm and bone marrow grafts were associated with a higher incidence of relapse in this study.
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S.K., L.R., and T.J. wrote the first draft of the manuscript and were responsible for acquiring, analyzing, and interpreting the data. S.K., A.T.G. and T.J. conceptualized the project. L.R. conducted the statistical analysis. All other authors contributed to the collection of data, revising the manuscript critically for important intellectual content. All authors approved the final version of the manuscript. S.K. and T.J. are accountable for all aspects of the work related to accuracy and integrity.
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M.R.G. has received consulting fees from Abbvie, Agios, Amgen, Astellas, Blueprint Medicines, Bristol Myers Squibb, Cardinal Health, Daiichi Sankyo, Gilead, Incyte, Karius, Pfizer, Premier, Sierra Oncology, Stemline, and Trovagene; research support from Incyte, Genentech/Roche, and Janssen; and owns stock in Medtronic. B.D. reports institutional research support from Takeda, Janssen, Angiocrine, Pfizer, and Poseida, and serves on the advisory board of Jazz. S.A. reports research funding through Helsinn Healthcare, Actinium Pharmaceuticals, and Pfizer and has received consulting fees from Abbvie and Agios. A.D. reports honoraria through Abbvie, Taiho, and Novartis. V.G. reports institutional research funding through Novartis and honoraria through Novartis, BMS-Celgene, Abbvie, Constellation Pharmaceuticals, and Sierra Oncology. A.T.G. reports research funding through Sierra Oncology, Pfizer, Celgene, CTI Biopharma, Incyte Corporation, Roche/Genentech, Imago Biosciences, and Gilead Sciences, and has received consulting fees from Celgene, CTI Biopharma, AstraZeneca/MedImmune, Incyte Corporation, and Apexx Oncology. T.J. reports institutional research support from CTI Biopharma, Incyte and Syneos Health, Consultancy with Targeted Healthcare Communications, advisory board with Care Dx, and Bristol Myers Squibb. The remaining authors do not have any conflicts of interest.
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Kunte, S., Rybicki, L., Viswabandya, A. et al. Allogeneic blood or marrow transplantation with haploidentical donor and post-transplantation cyclophosphamide in patients with myelofibrosis: a multicenter study. Leukemia 36, 856–864 (2022). https://doi.org/10.1038/s41375-021-01449-1
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DOI: https://doi.org/10.1038/s41375-021-01449-1
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