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.
Zhongli Hu, Zhiping Fan, Min Dai et al.
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In 30 seconds
This open-label, multicentre, randomised phase 3 trial compared decitabine plus busulfan-cyclophosphamide (DAC-BuCy) with BuCy conditioning before allogeneic HSCT in 130 patients aged 14–65 years with KMT2A-rearranged leukaemia at eight centres in China. At a median follow-up of 34·7 months, the 2-year cumulative incidence of relapse was 12% (95% CI 6–22) with DAC-BuCy and 29% (19–41) with BuCy (hazard ratio 0·41 [0·20–0·87]; p=0·017). The authors reported an acceptable safety profile in both groups.
Key findings
- Two-year cumulative relapse incidence was 12% (95% CI 6–22) with DAC-BuCy versus 29% (19–41) with BuCy.
- The relapse hazard ratio for DAC-BuCy versus BuCy was 0·41 (95% CI 0·20–0·87; p=0·017).
- Grade 3–5 infections or infestations occurred in 27 (42%) DAC-BuCy recipients and 25 (38%) BuCy recipients.
- Adverse-event deaths occurred in four (6%) DAC-BuCy recipients and five (8%) BuCy recipients.
Why it matters
Relapse after allogeneic HSCT remains an important concern in KMT2A-rearranged leukaemia. In this randomised trial, adding decitabine to myeloablative BuCy conditioning was associated with a lower 2-year relapse incidence, while reported severe adverse-event patterns and deaths were broadly similar between groups. These results provide comparative evidence for conditioning selection in the studied population.
What to keep in mind
The trial was open-label and conducted at eight transplantation centres in China; applicability beyond the studied population and setting is uncertain.