Dr Jain on the Long-Term Investigation of Luspatercept in Anemic MDS

Akriti Jain, MD, Department of Hematology and Medical Oncology, Cleveland Clinic, discusses updated data from the phase 3 MEDALIST trial (NCT02631070) evaluating luspatercept-aamt (Reblozyl) for the treatment of anemia following progression on an erythropoiesis stimulating agent (ESA) in adult patients with very low– to intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts (MDS-RS) or myelodysplastic/myeloproliferative neoplasms with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).

The MEDALIST trial stands as a pivotal study that led to the FDA approval of luspatercept for the treatment of patients with MDS-RS or MDS/MPN-RS-T, which came as a breakthrough for patients who have experienced ESA progression or intolerance, Jain begins. Recently, updated results from the trial, extending until a data cutoff of January 2nd, 2023, were disclosed during the 2023 ASH Annual Meeting. Notably, the data revealed a significant improvement in red blood cell (RBC) transfusion independence lasting at least 8 weeks, with rates climbing from what has been previously reported she notes, saying that this enhancement underscores the therapeutic efficacy of luspatercept in addressing transfusion dependency in patients with MDS.

Another noteworthy aspect of the updated findings was the prolonged duration of response observed among patients who achieved transfusion independence, Jain expands. This sustained benefit highlights the enduring impact of luspatercept therapy in managing RBC transfusion needs over an extended period, according to Jain. Furthermore, regarding safety outcomes, fatigue is an important consideration in the management of MDS. Despite achieving RBC transfusion independence, many patients continue to experience fatigue, Jain elucidates.

The discourse on fatigue in patients with MDS at the 2023 ASH Annual Meeting seamlessly segued into an intriguing avenue of exploration involving a TGF- β inhibitor, she continues. Promising reports indicate that this inhibitor holds potential in alleviating fatigue, Jain says. Anticipation is high for further insights and clinical data on the efficacy of TGF-βinhibitors, with hopes that upcoming conferences will shed more light on this promising therapeutic avenue, Jain concludes.