Resumen
Different consolidation strategies are available for acute myeloid leukemia (AML) patients fit for intensive treatment. For favorable- or intermediate-risk AML, high-dose chemotherapy (HDCT) followed by autologous stem cell transplantation (ASCT) is one of these options. Busulfan plus melphalan is a frequently used and efficient HDCT regimen, but it bears neurotoxic potential and may cause irreversible alopecia, amongst other toxicities. Thus, improving HDCT regimens with lesser toxicity, albeit at comparable anti-leukemic efficacy, is wishful. We combined treosulfan with its more favorable toxicity profile with melphalan for HDCT and compared these patients with a group receiving busulfan/treosulfan. Whereas disease-free and overall survival did not differ significantly, the treosulfan regimen compared favorably, with the absence of neurotoxicity and irreversibly alopecia. Treosulfan serum levels by mass cytometry demonstrated considerable interindividual biovariability. Further studies should explore treosulfan/melphalan for HDCT/ASCT in AML, aiming to improve the quality of life of AML survivors and offer safer consolidation strategies.