Luspatercept Yields Transfusion Independence in ESA-Naïve Lower-Risk MDS

Investigators of this retrospective, observational cohort study assessed the outcomes of patients treated with luspatercept for lower-risk MDS in clinical practices across the United States.

Almost all patients with lower-risk myelodysplastic syndrome (MDS) who were naïve to treatment with erythropoietin-stimulating agents (ESAs) and transfusion-dependent (TD) achieved transfusion independence (TI) following therapy with luspatercept-aamt (Reblozyl), according to findings from a retrospective cohort study.¹

Overall, 80.0% (n = 8/10) of patients who are ESA-naïve with available data achieved physician-reported modified hematologic response-erythroid (mHI-E) status, and 20.0% (n = 2/10) did not. Among patients who were TD at baseline, 96.9% (n = 31/32) reached TI for at least 8 weeks, and 93.8% (n = 30/32) achieved TI for 12 weeks or longer. Investigators highlighted that 1 patient (3.0%) who was non-transfusion dependent (NTD) prior to beginning study treatment remained NTD for at least 12 weeks.

Investigators of this retrospective, observational cohort study assessed the outcomes of patients treated with luspatercept for lower-risk MDS in clinical practices across the United States. Twenty-four participating physicians from the Cardinal Health Oncology Provider Extended Network abstracted data on eligible patients from medical records between May 2022 and July 2022.

Investigators conducted a subgroup analysis of those who did not receive prior treatment with ESAs and defined baseline transfusion burden (TB) based on the lowest number of transfusion sessions. Patients who were NTD had 0 prior transfusion sessions, those with low TB had 1 to 3, those with moderate TB had 4 to 5, and those with high TB had 6 or more sessions.

Patients 18 years and older diagnosed with lower-risk MDS, lower-risk MDS with ring sideroblasts (RS), or MDS or myeloproliferative neoplasm with RS and thrombocytosis on or after January 1, 2015, were included in the study. Additional eligibility criteria included receiving treatment from an abstracting physician or another physician in the same practice for at least 1 year before initiating treatment with luspatercept. Those who previously received luspatercept in a clinical trial, any treatment for MDS in a clinical trial prior to beginning luspatercept, or prior therapy with luspatercept for β-thalassemia were not included.

The study included a total of 33 patients who were naïve to ESAs. The mean age was 68.8 years (standard deviation [SD], 8.3) at baseline and 70.1 years (SD, 8.2) at the time of beginning treatment with luspatercept. Most of the study population were men (51.5%), White (78.8%), non-Hispanic/Latino (87.9%), from the Southern US (84.9%), and had low TB (81.8%). Additionally, most patients were alive at the time of data collection (97.0%) and insured with Medicare (60.6%). The median duration of follow-up was 4.1 months (range, 2.1-13.4).

Overall, 90.9% of patients received 1 or more prior lines of therapy before undergoing treatment with luspatercept, and 9.1% received the agent as frontline therapy. Investigators reported that 93.9% of the population remained on study treatment. Two patients (6.1%) discontinued therapy during follow-up, which included 1 due to a lack of hematologic improvement and another due to hemoglobin normalization. According to the investigators, the data were not sufficiently mature to estimate the median duration of study treatment, as 93.9% of patients did not discontinue treatment or were censored during follow-up.

Investigators highlighted that 69.7% of patients had no dose reductions. Additionally, 93.9% had no dose escalations, and 97.0% had no dose holds or interruptions.

These data support the interim results from the phase 3 COMMANDS trial (NCT03682536), which helped lead to the FDA approval of luspatercept for patients with lower-risk MDS with anemia in the first-line setting.²

These data were presented as part of a poster session at the 2023 Society of Hematologic Oncology (SOHO) Annual Meeting.

References

1. Mukherjee S, Brown-Bickerstaff C, Huggar D, et al. Treatment patterns and transfusion outcomes among erythropoietin-stimulating agent-naïve patients with lower-risk myelodysplastic syndromes receiving luspatercept in routine clinical practice in the United States. Presented at: 2023 Society of Hematologic Oncology (SOHO). Annual Meeting; September 6-9, 2023; Houston, TX. Abstract MDS-065.
2. U.S. FDA approves Bristol Myers Squibb’s Reblozyl® (luspatercept-aamt) as first-line treatment of anemia in adults with lower-risk myelodysplastic syndromes (MDS) who may require transfusions. News release. Bristol Myers Squibb. August 28, 2023. Accessed October 17, 2023. https://bit.ly/3qH7YEQ