Low-dose ruxolitinib for graft-versus-host disease prevention in haploidentical haematopoietic stem-cell transplantation: a multicentre, open-label, randomised, controlled, phase 3 trial.
Low-dose ruxolitinib dramatically reduced severe graft rejection after transplant, dropping dangerous complications from 37% to 7% with manageable side effects.
Low-dose ruxolitinib replacing MMF within ATG+CNI+methotrexate backbone reduced grade II-IV acute GVHD by day 100 to 6.8% vs 36.9% in standard prophylaxis arm (SHR 0.15, 95% CI 0.07-0.34; p<0.0001) in haploidentical HCT, with manageable safety profile. This record was retained from the prior triage attempt for PubMed pipeline handoff.
What the study was
- Study design
- Phase 3 multicenter open-label randomized controlled trial
- Category
- sentinel
- Maturity
- Potentially Practice-Changing
- Journal
- Lancet Haematol
Why it surfaced
Phase 3 RCT in Lancet Haematol with a dramatically positive primary endpoint. Acute GVHD is a leading cause of transplant-related morbidity and mortality; a ~5-fold reduction in grade II-IV GVHD with a JAK inhibitor is a paradigm-shifting finding. Implications for global haploidentical HCT practice are immediate.
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