Luspatercept Benefits Transfusion-Dependent Patients With MDS and Anemia in Real-World Study

Luspatercept (Reblozyl; Bristol Myers Squibb) can reduce the need for red blood cell transfusions among people with lower-risk myelodysplastic syndromes (LR-MDS), according to a new report.

The study, published in HemaSphere, found the therapy had similar efficacy in real-world usage as it did in its pivotal clinical trials.

Myelodysplastic disease | Image Credit: © ibreakstock - stock.adobe.com

The authors wrote that anemia is the most common cytopenia in patients with LR-MDS, and the majority of those patients require red blood cell transfusions. Severe anemia and transfusion dependency have both been linked with a shorter overall survival, they added.

One therapeutic option for these patients, the authors said, is luspatercept, an erythroid maturation agent that improves both the number and quality of mature red blood cells.

Last year, the FDA approved the therapy for patients with very low- to intermediate-risk MDS who require regular transfusions of red blood cells.

In the new report, investigators decided to analyze real-world data to find out how luspatercept was being used and to characterize its clinical outcomes.

The researchers used an electronic case report database to locate deidentified cases of adult patients who had been diagnosed with very low-, low-, or intermediate-risk MDS confirmed by bone marrow biopsy on or after January 1, 2015, and who had been given at least 12 weeks of luspatercept. The primary outcome was transfusion independence or maintenance of non–transfusion-dependent status for at least 8 weeks during the first 24 weeks on luspatercept. The authors also tracked whether patients had a decrease in the number of transfusion sessions they needed.

They identified a total of 253 cases that met the study’s inclusion criteria. Most of those patients (82.2%) had a low transfusion burden at baseline, meaning they required 1 to 3 red blood cell sessions and/or platelet sessions during the 8 weeks prior to initiation of luspatercept. A similar majority of the patients (86.6%) had already received 1 line of treatment, and 12.3% had received 2 or 3 lines of treatment prior to luspatercept initiation. The most common prior therapy utilized by the patients was erythropoiesis stimulating agents.

All patients started luspatercept at the recommended dose of 1 mg/kg every 3 weeks. The majority of patients (61.3%) had no dose escalations, and most (93.7%) had no dose reductions.

The authors noted that one-third of patients discontinued luspatercept, but 57 of those 84 patients did so when they were receiving less than the maximum recommended dose.

At a median follow-up of 5.7 months, most of the patients (87.4%) achieved transfusion independence or maintained non–transfusion-dependent status for at least 8 weeks.

Broken up by baseline transfusion burden, the authors found that 89.9% of the 208 patients who had a low baseline transfusion burden achieved treatment independence for at least 8 weeks, and the remaining 21 patients maintained the low transfusion burden status for at least 8 weeks. Among the 24 patients with moderate transfusion burden (4-5 red blood cell sessions and/or platelet sessions during the 8 weeks prior to initiation of luspatercept), the transfusion independence rate was slightly lower, at 58.3%, with all of the remaining patients achieving low transfusion burden for the first 8 weeks of luspatercept.

The authors noted that their findings line up with the results of the trials upon which the FDA’s approvals were based, showing that luspatercept can promote transfusion independence among people with low or moderate transfusion dependence at baseline.

“These data extend evidence from the MEDALIST trialand COMMANDS trial in support of the continued, robust clinical benefit of luspatercept in patients with LR‐MDS who are [transfusion dependent],” they wrote.

Reference:

Mukherjee S, Brown-Bickerstaff C, Falkenstein A, et al. Treatment patterns and outcomes with luspatercept in patients with lower-risk myelodysplastic syndromes: a retrospective US cohort analysis. Hemasphere. 2024;8(1):e38. doi:10.1002/hem3.38