Dr Eckfeldt on the Treatment Paradigm and Current Challenges in MDS

“For lower-risk MDS, [agents] like luspatercept certainly has provided a whole new avenue of treatments in a situation where traditional therapies like erythropoietin may not have been as effective [for MDS-related anemia]. Some of the molecular markers [used] to guide decision-making have been a major advance.”

Craig Eckfeldt, MD, PhD, assistant professor of medicine, faculty, Microbiology, Immunology, and Cancer Biology, PhD Graduate Program, Division of Hematology, Oncology, and Transplantation, Masonic Cancer Center, the University of Minnesota Medical School, discusses the current treatment paradigm and persistent unmet needs in the myelodysplastic syndrome (MDS) landscape.

At an OncLive® State of the Science Summit on hematologic malignancies, Eckfeldt and colleagues discussed changes in care for MDS and acute myeloid leukemia, highlighting the current treatment paradigm for MDS.

Over time, better understanding the pathophysiology of MDS has opened the door to developing new therapies that can specifically target cancer-driving factors, Eckfeldt explains. For lower-risk MDS, he notes that frontline treatment with luspatercept-aamt (Reblozyl) has provided a new option in the landscape; in August 2023, the FDA approved luspatercept for the treatment of anemia without prior erythropoiesis-stimulating agent use in adult patients with very low– to intermediate-risk MDS who may require regular red blood cell transfusions. The approval has provided a new option in a setting where other treatments, such as erythropoietin, have not been as effective in treating MDS-related anemia, he notes. Additionally, Eckfeldt says that identifying the molecular markers that can help guide decision-making has made a significant difference in advancing care for MDS.

However, unmet needs persist in MDS, especially within specific subsets of patients, Eckfeldt emphasizes, noting that a lot is unknown for patients with poor prognostic markers. Previous research has demonstrated specific patient populations who may not respond as well to treatment, but there’s still a need to change the treatment trajectory and outcomes for these patients, he says.

Although there are new therapies and investigational agents displaying encouraging data, subsets of patients with MDS, such as those harboring TP53 mutations, still face treatment disparities, he notes. In the coming years, more meaningful or new approaches could help close the gap with these disparities in MDS, Eckfeldt concludes.