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Allogeneic hematopoietic stem cell transplantation with fludarabine, busulfan, and thiotepa conditioning is associated with favorable outcomes in myelofibrosis

Bone Marrow Transplantation volume 55, pages 147–156 (2020)Cite this article

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Abstract

Allogeneic stem cell transplantation is a curative therapy for myelofibrosis. The optimal conditioning regimen has not been well defined. We retrospectively compared transplantation outcomes in patients with myelofibrosis (n = 67) conditioned with myeloablative (MAC, 36%) and reduced-intensity (RIC, 46%) regimens, and more recently with the combination of thiotepa, busulfan, and fludarabine (TBF, 18%). Patients were transplanted from HLA-matched sibling (n = 26) or unrelated donors (n = 41) between the years 2003 and 2018. The median follow-up was 2.9 years for all patients but shorter in the TBF group (1.1 years). The probability of 3-year progression-free survival (PFS) was 43%. At 1 year, the rate of PFS was 80%, 54%, and 45% with TBF, MAC, and RIC, respectively (p = 0.031). In a multivariable model, there was a greater risk for death with MAC (hazard ratio [HR] 12.26, p = 0.026) and lower PFS with both MAC (hazard ratio [HR] 7.78, p = 0.017) and RIC (HR 5.43, p = 0.027) compared with TBF. Relapse was higher with RIC (HR 8.20, p = 0.043) while nonrelapse mortality was increased with MAC (HR 9.63 p = 0.049). Our results indicate that TBF is a promising preparative regimen in myelofibrosis patients transplanted from matched sibling or unrelated donors, and should be further explored.

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Acknowledgements

This study was supported by The Varda and Boaz Dotan Research Center in Hemato-Oncology affiliated with the Cancer Biology Research Center—Tel Aviv University.

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  1. These authors contributed equally: Roni Shouval, Yakov Vega

Authors and Affiliations

  1. Division of Hematology and Bone Marrow Transplantation, Chaim Sheba Medical Center, Tel Aviv University, Ramat Gan, Israel

    Roni Shouval, Yakov Vega, Joshua A. Fein, Ivetta Danylesko, Noga Shem Tov, Ronit Yerushalmi, Arnon Nagler & Avichai Shimoni

  2. Dr. Pinchas Bornstein Talpiot Medical Leadership Program, Chaim Sheba Medical Center, Ramat-Gan, Israel

    Roni Shouval

  3. Department of Hematology, Blood Neoplasms and Bone Marrow Transplantation, Wroclaw Medical University, Wroclaw, Poland

    Marta Sobas & Anna Czyż

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Shouval, R., Vega, Y., Fein, J.A. et al. Allogeneic hematopoietic stem cell transplantation with fludarabine, busulfan, and thiotepa conditioning is associated with favorable outcomes in myelofibrosis. Bone Marrow Transplant 55, 147–156 (2020). https://doi.org/10.1038/s41409-019-0653-7

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