1. Association between the choice of the conditioning regimen and outcomes of allogeneic hematopoietic cell transplantation for myelofibrosis.
- Author
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Murthy GSG, Kim S, Estrada-Merly N, Abid MB, Aljurf M, Assal A, Badar T, Badawy SM, Ballen K, Beitinjaneh A, Cerny J, Chhabra S, DeFilipp Z, Dholaria B, Perez MAD, Farhan S, Freytes CO, Gale RP, Ganguly S, Gupta V, Grunwald MR, Hamad N, Hildebrandt GC, Inamoto Y, Jain T, Jamy O, Juckett M, Kalaycio M, Krem MM, Lazarus HM, Litzow M, Munker R, Murthy HS, Nathan S, Nishihori T, Ortí G, Patel SS, Van der Poel M, Rizzieri DA, Savani BN, Seo S, Solh M, Verdonck LF, Wirk B, Yared JA, Nakamura R, Oran B, Scott B, and Saber W
- Subjects
- Adult, Humans, Adolescent, Busulfan therapeutic use, Melphalan, Retrospective Studies, Cyclophosphamide therapeutic use, Transplantation Conditioning, Vidarabine therapeutic use, Primary Myelofibrosis diagnosis, Primary Myelofibrosis therapy, Hematopoietic Stem Cell Transplantation adverse effects, Graft vs Host Disease etiology
- Abstract
Allogeneic hematopoietic cell transplantation (allo-HCT) remains the only curative treatment for myelofibrosis. However, the optimal conditioning regimen either with reduced-intensity conditioning (RIC) or myeloablative conditioning (MAC) is not well known. Using the Center for International Blood and Marrow Transplant Research database, we identified adults aged ≥18 years with myelofibrosis undergoing allo-HCT between 2008-2019 and analyzed the outcomes separately in the RIC and MAC cohorts based on the conditioning regimens used. Among 872 eligible patients, 493 underwent allo-HCT using RIC (fludarabine/ busulfan n=166, fludarabine/melphalan n=327) and 379 using MAC (fludarabine/busulfan n=247, busulfan/cyclophosphamide n=132). In multivariable analysis with RIC, fludarabine/melphalan was associated with inferior overall survival (hazard ratio [HR]=1.80; 95% confidenec interval [CI]: 1.15-2.81; P=0.009), higher early non-relapse mortality (HR=1.81; 95% CI: 1.12-2.91; P=0.01) and higher acute graft-versus-host disease (GvHD) (grade 2-4 HR=1.45; 95% CI: 1.03-2.03; P=0.03; grade 3-4 HR=2.21; 95%CI: 1.28-3.83; P=0.004) compared to fludarabine/busulfan. In the MAC setting, busulfan/cyclophosphamide was associated with a higher acute GvHD (grade 2-4 HR=2.33; 95% CI: 1.67-3.25; P<0.001; grade 3-4 HR=2.31; 95% CI: 1.52-3.52; P<0.001) and inferior GvHD-free relapse-free survival (GRFS) (HR=1.94; 95% CI: 1.49-2.53; P<0.001) as compared to fludarabine/busulfan. Hence, our study suggests that fludarabine/busulfan is associated with better outcomes in RIC (better overall survival, lower early non-relapse mortality, lower acute GvHD) and MAC (lower acute GvHD and better GRFS) in myelofibrosis.
- Published
- 2023
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