CPX‑351 in Down Syndrome–Associated Myeloid Leukemia: a dose‑sensitivity mismatch that reduced event‑free survival in the ML‑DS 2018 trial
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– Replacing the ML‑DS 2006 reduced‑intensity induction with CPX‑351 (liposomal cytarabine:daunorubicin/cytarabine formulation) in the ML‑DS 2018 trial produced a significantly lower 24‑month event‑free survival (EFS) — 69% versus 90% (P <.001) — leading to early trial suspension.
– Despite higher relapse frequency, most relapsed patients achieved remission with salvage therapy and overall survival (OS) at 24 months remained similar to historical controls (88% vs 92%, P = .612).
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