A phase II study of bisantrene in patients with relapsed/refractory acute myeloid leukemia.

Objectives: To determine the current role of bisantrene, an anthracene with anthracycline‐like activity which was shown in earlier studies to be effective therapy in relapsed/refractory acute myeloid leukemia with no discernible cardiotoxicity, in the treatment of patients with R/R AML. Methods: This phase 2, single‐center study (NCT03820908) enrolled adult R/R AML to receive bisantrene (250 mg/m2 daily for 7 days) which was administered via an intravenous infusion over 2 hours on days 1‐7. Disease assessment included routine blood work and bone marrow studies. Results: In all, 10 patients were enrolled with a median of 3 lines of prior therapy including seven patients who had relapsed following allogeneic stem cell transplantation. The most frequently reported grade ≥3 treatment‐attributed hematologic AE was thrombocytopenia, whereas the most frequently reported grade ≥3 treatment‐attributed non‐hematologic AE was mucositis. Of the 10 patients, one (10%) achieved a complete remission and three patients achieved a partial remission resulting in an overall response rate of 40%. Next‐generation sequencing of patient samples identified a wide array of mutations associated with activated signaling, splicing, and epigenetic modification. Conclusions: In view of the observed low toxicity, a follow‐up study combining bisantrene with complementary anti‐leukemic therapy is planned. [ABSTRACT FROM AUTHOR]