Clomiphene as a novel modality for the treatment of acute myeloid leukemia: A pilot phase II study.

Abstract Clomiphene, an antiestrogen clinically used for ovulation induction, kills leukemic cells ex vivo via apoptosis. This study was designed to evaluate the antileukemic effect of clomiphene in patients with AML. Eleven patients with recurrent or chemoresistant AML aged 54–79 years received oral clomiphene (25–50 mg per day), for seven consecutive days per cycle, up to three cycles while concurrent non intravenous chemotherapy was continued. Ten patients showed a partial response or remained stable during therapy; 7 had a rapid increase in disease parameters shortly after cessation of therapy while four patients survived 6–18 months. We believe that clomiphene contributes to stabilizing disease during therapy and appears to prolong survival in a subset of relapsed or refractory patients and may perhaps be considered as a new therapeutic option.