The FDA's approval of luspatercept-aamt (Reblozyl) in August 2023 has expanded the first-line treatment options for anemia in adult patients with very low- to intermediate-risk myelodysplastic syndrome (MDS) who may require regular red blood cell transfusions. This approval marks a significant step forward, offering an efficacious and well-tolerated treatment option for this patient population.
Luspatercept's Impact on Anemia and Quality of Life
In an interview with OncLive ®, Jorge Cortes, MD, emphasized the importance of considering the overall health and quality of life (QOL) of patients when treating anemia in lower-risk MDS. Patients with anemia often experience fatigue, reduced ability to perform daily activities, and mental fogginess, all of which significantly impact their QOL. Luspatercept has shown a significant difference compared to erythropoiesis-stimulating agents (ESAs) in the phase 3 COMMANDS trial (NCT03682536).
"We need to think about improving the overall health of the patient [when treating for anemia in lower-risk MDS, including] the QOL of the patient," Cortes stated.
Balancing Efficacy and Tolerability
According to Cortes, the impact of treating anemia extends beyond merely increasing hemoglobin levels. The QOL impact is substantial, especially with a drug like luspatercept that is generally well-tolerated. This means patients can experience the benefits of improved hemoglobin levels without the burden of significant adverse effects.
Risk Stratification and Treatment Goals
Risk stratification for treatment in patients with lower-risk MDS involves considering factors such as gene mutations, cytogenetics, and the severity of anemia symptoms. The primary goal when selecting a therapy is to improve symptoms and QOL, allowing patients to better manage their daily activities and enjoy life.
While luspatercept is not a curative approach, it effectively addresses the symptoms of anemia and improves QOL in patients with lower-risk MDS. Curative strategies like stem cell transplants are generally not considered for this patient population due to their invasive nature and the older age of many MDS patients.
Addressing Remaining Challenges and Future Research
Despite the advancements in treatment options, the main challenge remains the potential for disease progression and eventual fatality. Future research should focus on developing transformative approaches that can change the natural history of the disease and provide a potential cure for MDS. While stem cell transplants can be an option, they are often not feasible for many patients with MDS.
"Although we have some good treatments and developments, we're still lacking a transformative approach that could be accessible to most patients that can eradicate the disease and provide a cure, which has been elusive," Cortes concluded.
