Myelodysplastic Syndromes Highlights From ASCO 2021

Amy DeZern, MD


June 30, 2021

Key studies on myelodysplastic syndromes (MDS) presented at the American Society of Clinical Oncology (ASCO) 2021 annual meeting included clinical trial results on the use of enasidenib in patients with higher-risk MDS and data on the discontinuation of hypomethylating agents (HMA) for higher-risk MDS patients.

Dr Amy DeZern, of Johns Hopkins University School of Medicine, highlights data from a phase 2 clinical trial of enasidenib in higher-risk MDS patients. This IDH2 inhibitor was evaluated as monotherapy for patients with IDH2-mutation whose disease was relapsed or refractory to HMA therapy, and in combination with azacytidine for HMA-naive MDS patients. In both cohorts, enasidenib showed favorable response rates.

She next reports on an observational study of HMA discontinuation, a frequent predictor of suboptimal clinical outcomes. The study found that nearly a third of patients discontinued treatment prior to completion, and it also identified predictors of discontinuation. She then discusses a study of dose-modification strategies for venetoclax to manage hematologic toxicities in treatment-naive, higher-risk MDS patients.

Finally, Dr DeZern reviews an analysis of Medicare data on almost 17,000 MDS patients. The study found that over half of patients with no transfusion dependence received complete diagnostic workups despite a lack of clinical benefit, suggesting that unnecessary diagnostic interventions can be reduced by more careful patient selection.


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