SAN DIEGO – Combination therapy with the BCL-2 inhibitor venetoclax and low-dose cytarabine (LDAC) achieved a 61% overall response rate in older patients with treatment-naive acute myeloid leukemia, Andrew Wei, MBBS, PhD, reported at the annual meeting of the American Society of Hematology.
That is about three times higher than historically reported response rates for this deadly blood cancer, said Dr. Wei of Alfred Hospital in Melbourne, Australia. He discussed the findings in a video interview.
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The multicenter phase II study evaluated 28-cycles of venetoclax (600 mg, given orally) and LDAC (20 mg/m2 subcutaneously) in 53 treatment-naive AML patients aged 65 years and older, who were ineligible for intensive chemotherapy but had adequate liver and kidney function and an ECOG performance status between 0 and 2.
A total of 21% of patients had a complete remission, 33% had complete remission with incomplete marrow recovery, and 70% reached one of these endpoints during cycles 1 and 2. Common adverse events included vomiting, diarrhea, hypokalemia, and febrile neutropenia. Grade 3-4 adverse events included febrile neutropenia, hypokalemia, hypophosphatemia, and hypertension.
Researchers are planning larger randomized trials of venetoclax/LDAC combination therapy in AML, Dr. Wei said. Larger sample sizes will yield more data on how to best target this regimen based on prognostic indicators, such as gene mutations, he added.
Abbvie is the maker of venetoclax and sponsored the study. Dr. Wei disclosed a consulting relationship with Abbvie and ties to Novartis, Celgene, and several other pharmaceutical companies.