Emerging Treatments and Clinical Challenges in Myelodysplastic Syndromes: ASCO 2021 and EHA 2021 Updates

Dr. Sekeres discusses the current unmet needs of patients with higher-risk myelodysplastic syndromes receiving hypomethylating agents, the potential utility of novel combinations in development, and the incorporation of other end points beyond overall survival in clinical trials.

Joshua F. Zeidner, MD, discusses the risk of transformation to acute myeloid leukemia in higher-risk myelodysplastic syndromes.

Mikkael A. Sekeres, MD, discusses the goal of transfusion independence in higher-risk myelodysplastic syndromes.

Joshua F. Zeidner, MD, discusses the role that HMAs have played in the MDS treatment landscape and shared data reported with promising emerging agents like pevonedistat that can be combined with HMAs to improve outcomes for certain patient subsets.

Treatment with imetelstat demonstrated clinical efficacy across molecularly defined subgroups of patients with heavily transfused, lower-risk myelodysplastic syndrome who did not harbor 5q deletions and were relapsed/refractory to erythropoiesis-stimulating agents.

The IDH2 inhibitor enasidenib demonstrated promising efficacy in combination with azacitidine and as a single agent in patients with high-risk, IDH2-mutated myelodysplastic syndrome.

Novel combinations with agents, such as pevonedistat, eprenetapopt, venetoclax or magrolimab, plus hypomethylating agents like azacitidine are being evaluated to determine whether they can improve upon the lackluster median survival for patients with higher-risk myelodysplastic syndrome.

David Sallman, MD, discusses the efficacy of pevonedistat plus azacitidine in higher-risk myelodysplastic syndrome.

Daniel Pollyea, MD, MS, discusses emerging therapies in higher-risk myelodysplastic syndrome.

Daniel Pollyea, MD, MS, discusses the current management of patients with higher-risk myelodysplastic syndrome, the data noted so far with pevonedistat in this paradigm, and other therapies positioned to improve outcomes for this patient population.

David Sallman, MD, discusses the role of hypomethylating agents in the treatment of patients with higher-risk myelodysplastic syndrome, the promise of pevonedistat in this population, the hunt for biomarkers of response to treatment, and remaining areas of unmet need to address with future efforts.

David Sallman, MD, discusses unmet needs in the treatment of patients with higher-risk myelodysplastic syndromes.

Pevonedistat demonstrated a favorable risk-benefit profile at a dose of 20 mg/m2 in combination with 75 mg/m2 of azacitidine in patients with higher-risk myelodysplastic syndromes/chronic myelomonocytic leukemia and acute myeloid leukemia.