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Most patients with acute myeloid leukaemia (AML) are older, with many unsuitable for conventional chemotherapy. Low-dose Ara-C (LDAC) is superior to best supportive care but is still inadequate. The combination of arsenic trioxide (ATO) and LDAC showed promise in an unrandomised study. We report a randomised trial of LDAC versus LDAC+ATO. Patients with AML according to WHO criteria or myelodysplastic syndrome with >10% blasts, considered as unfit for conventional chemotherapy, were randomised between subcutaneous Ara-C (20 mg b.d. for 10 days) and the same LDAC schedule with ATO (0.25 mg/kg) on days 1-5, 9 and 11, for at least four courses every 4 to 6 weeks. Overall 166 patients were entered; the trial was terminated on the advice of the DMC, as the projected benefit was not observed. Overall 14% of patients achieved complete remission (CR) and 7% CRi. Median survival was 5.5 months and 19 months for responders (CR: not reached; CRi: 14 months; non-responders: 4 months). There were no differences in response or survival between the arms. Grade 3/4 cardiac and liver toxicity, and supportive care requirements were greater in the ATO arm. This randomised comparison demonstrates that adding ATO to LDAC provides no benefit for older patients with AML.

Original publication




Journal article



Publication Date





1122 - 1127


Aged, Aged, 80 and over, Antineoplastic Combined Chemotherapy Protocols, Arsenic Trioxide, Arsenicals, Chemical and Drug Induced Liver Injury, Comorbidity, Cytarabine, Disease-Free Survival, Early Termination of Clinical Trials, Female, Gastrointestinal Diseases, Heart Diseases, Humans, Kaplan-Meier Estimate, Leukemia, Myeloid, Acute, Leukemia, Myelomonocytic, Acute, Male, Middle Aged, Oxides, Remission Induction, Treatment Outcome