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Aspacytarabine for Patients With AML Who Are Unfit for Standard Chemotherapy

By: Joseph Fanelli
Posted: Monday, April 20, 2020

The novel antimetabolite aspacytarabine may prove to be a safe and effective treatment for patients diagnosed with acute myeloid leukemia (AML) who are considered to be unfit for standard induction chemotherapy, according to clinical data presented in the journal Blood. Jessica K. Altman, MD, of Northwestern University, Evanston, Illinois, and colleagues, found that aspacytarabine seemed to be active even in patients with AML who had prior exposure to hypomethylating agents. The authors added that their clinical data in this patient population may establish aspacytarabine as a “new therapeutic backbone for AML, either as a single agent or in combination with targeted therapy.”

In this phase 1/2a and ongoing phase IIb study, the authors enrolled 34 patients with AML and treated each with at least one dose of aspacytarabine. Of the patients, 30 had been unable to receive standard induction therapy because of age or comorbidities. Ultimately, 25 patients completed one course of aspacytarabine, 4 patients received two courses, and one patient each completed three and four courses.

Among the patients who received more than 4.5 g/m2 per day of aspacytarabine, the 30-day mortality rate was 7%. The combined complete remission rate for patients receiving all doses was 33%, including one patient who achieved a complete response with partial platelet recovery. The complete remission rate for those patients treated with at least 4.5 g/m2 per day was 36%, with a median time for complete hematologic recovery of 27 days. The authors noted that for the seven patients who had a complete response with partial platelet recovery, three had secondary AML, including two patients with prior exposure to hypomethylating agents and one with prior exposure to radiotherapy.

Adverse events among the population were mostly hematologic “on-target” events. No cases of drug-related mucositis or cerebellar toxicity were reported.

Disclosure: For full disclosures of the study authors, visit

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