Real-World Evidence Supports Luspatercept in Lower-Risk MDS

Idoroenyi Amanam, MD assistant professor in the Division of Leukemia at City of Hope, discusses the significant impact of luspatercept in the treatment of patients with lower-risk myelodysplastic syndromes (MDS), particularly those who are transfusion dependent.

Dr. Amanam emphasizes the real-world challenges faced by patients with low-risk MDS, most notably, the burden of frequent transfusions. Many patients must spend hours, or even days, at hospitals or infusion centers each week to receive blood transfusions, which significantly impacts their quality of life. For these patients, reducing the frequency and dependency on transfusions is not just a clinical goal, but a matter of restoring time and dignity to their lives.

He highlights that while phase 3 clinical trials are essential for bringing therapies to market, real-world studies are equally important for understanding how treatments perform across more diverse populations. The COMMANDS trial and subsequent real-world evidence support the use of luspatercept as a meaningful alternative to erythropoiesis-stimulating agents.

The unique mechanism of action of luspatercept allows for faster, more durable responses, leading to higher rates of transfusion independence—even in patients who are not RS-positive, a group traditionally thought to be less responsive. This, Dr. Amanam notes, reinforces the therapy’s effectiveness and broadens its relevance.

The takeaway from this research is that luspatercept should be considered the frontline therapy for lower-risk MDS patients who are transfusion dependent. By focusing on patient-centered outcome such as reducing transfusion burden and improving quality of life, luspatercept represents a significant advancement in MDS treatment.