The Lancet. Haematology
Decitabine plus busulfan-cyclophosphamide versus busulfan-cyclophosphamide myeloablative conditioning before allogeneic haematopoietic stem cell transplantation for reducing risk of relapse in adult KMT2A‑rearranged leukaemia in China: an open-label, multicentre, randomised phase 3 trial
By Hu Z, Fan Z, Dai M and 15 others.
Abstract
Background
An unmet need persists for optimal myeloablative conditioning before allogeneic haematopoietic stem cell transplantation (HSCT) in adult KMT2A‑rearranged leukaemia. We investigated whether decitabine plus busulfan-cyclophosphamide (DAC-BuCy) conditioning reduced the risk of relapse compared with busulfan-cyclophosphamide (BuCy) conditioning.
Methods
In this open-label, randomised, phase 3 trial at eight transplantation centres in China, eligible patients were aged 14-65 years, had morphologically confirmed KMT2A-rearranged leukaemia, and had an Eastern Cooperative Oncology Group performance status of 0-2. Patients were randomly assigned (1:1) before HSCT to DAC-BuCy (intravenous decitabine 20 mg/m2 once a day on days -14 to -10, intravenous busulfan 3·2 mg/kg once a day on days -7 to -4, and intravenous cyclophosphamide 60 mg/kg once a day on days -3 to -2) or BuCy (same dose and duration of busulfan and cyclophosphamide). Randomisation was done with permuted blocks (block size four) with no stratification and was implemented through an independent, interactive, web-based response system. The primary endpoint was 2-year cumulative incidence of relapse in the intention-to-treat population. Safety was assessed in all patients who received treatment. This trial is registered with ClinicalTrials.gov (NCT03596892) and is complete.