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The current standard of care for patients with high-risk myelodysplastic syndromes (MDS) is treatment with the hypomethylating agents (HMAs) azacitidine or decitabine. However, this is associated with low rates of response. If venetoclax, an inhibitor of the BCL-2 protein, is combined with an HMA, high response rates are seen in unfit and previously untreated acute myeloid leukemia.1
Armon Azizia et al.1 performed a retrospective study of patients with high risk-MDS receiving the combination of HMA plus venetoclax to assess its effectiveness in this setting. The study was published in Leukemia & Lymphoma, and concluded that the combination of venetoclax and an HMA results in high response rates in patients with high-risk MDS, but at the cost of high frequency of myelosuppression, warranting further prospective evaluation in clinical trials.
This was a retrospective analysis of 20 patients with high-risk MDS who received an HMA plus venetoclax. Researchers retrospectively reviewed patients with MDS who were assessed and/or received treatment at Stanford Cancer Institute (Stanford, CA) between June 2017 and September 2019 or the University of Massachusetts Memorial Medical Center (Worcester, MA) between July 2019 and December 2019. A total of 20 patients (18 patients from Stanford Cancer Institute and 2 patients from the University of Massachusetts Memorial Medical Center) were included in the final analysis.
Overall response rate (ORR) was defined as the percentage of patients achieving complete response (CR), maintained complete response (mCR), or partial response (PR). Duration of response (DOR) was defined as the time from when a patient achieved a CR/mCR until disease progression, relapse, or death. HMA failure was defined as having received at least four cycles of decitabine or six cycles of azacitidine prior to starting venetoclax. Log-rank tests were performed on censored OS using response to therapy as the sole predictor to compare the OS of responders and non-responders.
In conclusion, this retrospective analysis of 20 patients with high-risk MDS, who received an HMA plus venetoclax, reports an ORR of 75%. Venetoclax plus HMA results in high response rates in patients with high-risk MDS but is associated with toxicities and myelosuppression, which necessitate extra investigation of this combination therapy in clinical trials.
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