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Combining all-trans retinoic acid with arsenic trioxide cured almost every patient with lower-risk acute promyelocytic leukaemia without any conventional chemotherapy, and did so with fewer complications than the chemotherapy-based standard.
Phase 3 non-inferiority trial of 162 patients with non-high-risk APL randomised to all-trans retinoic acid plus arsenic trioxide or to retinoic acid plus idarubicin chemotherapy (AIDA).
Two-year event-free survival was 97 percent with the chemotherapy-free regimen against 86 percent with AIDA, and overall survival 99 versus 91 percent; the arsenic arm had less haematological toxicity and fewer infections but more liver enzyme rises and QT prolongation.
Low- and intermediate-risk APL is now treated without cytotoxic chemotherapy. The regimen has become the standard worldwide and made APL the most curable adult acute leukaemia.
Shares Acute promyelocytic leukaemia, New England Journal of Medicine.
Shares APL0406, Acute promyelocytic leukaemia.