Aspacytarabine for the treatment of patients with AML unfit for intensive chemotherapy: a phase 2 study

Jessica K. Altman*, Tsila Zuckerman, Jamie Koprivnikar, James McCloskey, Vamsi Kota, Michael Keng, Olga Frankfurt, Yasmin Abaza, Dale L. Bixby, Ashkan Emadi, Micah Burch, Bhavana Bhatnagar, Selina M. Luger, Mary Elizabeth Percival, Ofir Wolach, Michael Craig, Chezi Ganzel, Gail Roboz, Itai Levi, Anna GourevitchLiat Flaishon, Shoshi Tessler, Chen Blumberg, Stela Gengrinovitch, Ruth Ben Yakar, Jacob M. Rowe

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


High-dose cytarabine is associated with gastrointestinal and cerebellar toxicity, precluding its use for older or unfit patients with acute myeloid leukemia (AML). Aspacytarabine, an inactive prodrug of cytarabine, was evaluated as monotherapy in a phase 2b study of patients unfit for intensive chemotherapy (NCT03435848). Sixty-five patients with AML were treated with aspacytarabine 4.5 g/m2 per day (equimolar to 3 g/m2 per day cytarabine) for 6 doses per treatment. The median age was 75 years; 60.6% of patients had de novo AML, 28.8% had AML secondary to myelodysplastic syndrome, and 10.6% had therapy-related AML. Overall, 36.9% achieved complete remission (CR) with full count recovery. CR rates in patients with secondary AML, patients with prior treatment with hypomethylating agents, and patients with TP53 mutation were 26.7%, 25%, and 36%, respectively. Median overall survival was 9 months (range, 6-15.9) and was not reached among responders. Hematologic recovery was observed in all responding patients by day 26 without prolonged cytopenias. Adverse events typically precluding the use of high-dose cytarabine in older or unfit patients were not observed. These data suggest that aspacytarabine may be an effective regimen with a reduction in the attendant toxicities associated with high-dose cytarabine, an important consideration when treating AML and other hematologic disorders that use high-dose cytarabine. This trial was registered at as #NCT03435848.

Original languageEnglish
Pages (from-to)7494-7500
Number of pages7
JournalBlood advances
Issue number24
StatePublished - 26 Dec 2023
Externally publishedYes


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