Abstract
The safety and efficacy of a 4-day myeloablative conditioning (MAC) regimen consisting of Bu 3.2 mg/kg and fludarabine 40 mg/m2/day for HLA-identical sibling allogeneic hematopoietic cell transplantation (HCT) in myeloid malignancies was investigated in 133 patients (median age, 47 years; range 19–74 years) with de novo AML (60%), secondary AML (20%) or myelodysplastic syndrome (20%). All patients engrafted. Hepatic veno-occlusive disease occurred in five patients (4%), and severe toxicities, mostly mucositis, occurred in twenty-three (17%) patients. The non-relapse mortality (NRM) at 100 days was 1.5%. The incidences of acute GVHD grade 2–4 and grade 3–4 were 32 and 13%, respectively. At a median follow-up of 38 months, the cumulative incidence of chronic GVHD was 67%. The relapse incidence was 30% (27 and 31%, respectively, in patients with early- and late-stage disease), and the overall NRM was 15%. The actuarial 4-year disease-free survival (DFS) and overall survival (OS) were 54 and 62%, respectively. Patients aged <50 years had better outcomes compared with older patients (DFS 64 vs 42%, P=0.006; OS 73 vs 47%, P<0.001, respectively).
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Acknowledgements
We greatly appreciate the participation of all the patients and their families. We are indebted to the contributing members of the GETH at the participating institutions, and nurses, data managers and physicians of the HCT teams at contributing sites. We specially thank Mr Luis Benlloch, GETH Data Manager, for data collection and database assembly. We also thank Dr Gregory Morley for editorial assistance. This clinical trial was funded in part by an educational grant from Pierre-Fabre Iberica Pharmaceutical. This company was not involved in the design and running of the trial, nor in the collection and analysis of the clinical data, and the final manuscript.
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De La Serna, J., Sanz, J., Bermúdez, A. et al. Toxicity and efficacy of busulfan and fludarabine myeloablative conditioning for HLA-identical sibling allogeneic hematopoietic cell transplantation in AML and MDS. Bone Marrow Transplant 51, 961–966 (2016). https://doi.org/10.1038/bmt.2016.42
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DOI: https://doi.org/10.1038/bmt.2016.42
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