1. Busulfan-fludarabine versus busulfan-cyclophosphamide for allogeneic transplant in acute myeloid leukemia: long term analysis of GITMO AML-R2 trial.
- Author
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Cavallaro G, Grassi A, Pavoni C, Micò MC, Busca A, Cavattoni IM, Santarone S, Borghero C, Olivieri A, Milone G, Chiusolo P, Musto P, Saccardi R, Patriarca F, Pane F, Saporiti G, Rivela P, Terruzzi E, Cerretti R, Marotta G, Carella AM, Nagler A, Russo D, Corradini P, Bernasconi P, Iori AP, Castagna L, Mordini N, Oldani E, Di Grazia C, Bacigalupo A, and Rambaldi A
- Subjects
- Humans, Middle Aged, Adult, Female, Male, Aged, Follow-Up Studies, Busulfan administration & dosage, Busulfan therapeutic use, Leukemia, Myeloid, Acute therapy, Leukemia, Myeloid, Acute mortality, Leukemia, Myeloid, Acute drug therapy, Vidarabine analogs & derivatives, Vidarabine administration & dosage, Vidarabine therapeutic use, Cyclophosphamide therapeutic use, Cyclophosphamide administration & dosage, Hematopoietic Stem Cell Transplantation methods, Transplantation Conditioning methods, Antineoplastic Combined Chemotherapy Protocols therapeutic use, Transplantation, Homologous
- Abstract
We report the long-term results of a randomized trial (GITMO, AML-R2), comparing 1:1 the combination of busulfan and cyclophosphamide (BuCy2, n = 125) and the combination of busulfan and fludarabine (BuFlu, n = 127) as conditioning regimen in acute myeloid leukemia patients (median age 51 years, range 40-65) undergoing allogeneic hematopoietic stem cell transplantation. With a median follow-up of 6 years, significantly better non-relapse mortality (NRM) was confirmed in BuFlu recipients, which is sustained up to 4 years after transplant (10% vs. 20%, p = 0.0388). This difference was higher in patients older than 51 years (11% in BuFlu vs. 27% in BuCy2, p = 0.0262). The cumulative incidence of relapse, which was the first cause of death in the entire study population, did not differ between the two randomized arms. Similarly, the leukemia-free survival (LFS) and overall survival (OS) were not different in the two cohorts, even when stratifying patients per median age. Graft-and relapse-free survival (GRFS) in BuFlu arm vs. the BuCy2 arm was 25% vs. 20% at 4 years and 20% vs. 17% at 10 years. Hence, the benefit gained by NRM reduction is not offsets by an increased relapse. Leukemia relapse remains a major concern, urging the development of new therapeutic approaches., (© 2024. The Author(s).)
- Published
- 2024
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