Real-world data highlights effectiveness of luspatercept in treating anemic patients with MDS

Luspatercept, especially when combined with erythropoiesis-stimulating agents (ESA) and administered at higher doses, is highly effective in achieving transfusion independence and hematologic improvement in lower-risk patients with myelodysplastic syndrome (MDS), according to a study.

The study evaluated 54 patients with lower-risk MDS receiving luspatercept, with or without ESA.

Key Findings

• Transfusion Independence (TI):
  • 62.7% achieved TI for at least 8 weeks.
  • 49% maintained TI for 24 weeks or longer.
• Hematologic Improvement (HI):
  • HI without TI was achieved in 11.7%.
  • Overall, 74.5% of patients achieved either TI or HI.
• Concurrent ESA Use:
  • Epoetin alfa was used alongside luspatercept in 60.7% of cases.
  • Among these, 55.2% saw improved responses, with 16 patients achieving TI.
• Dose Escalation:
  • Optimal response required increasing the dose to 1.75 mg/kg in 35 patients.
• Response Variability:
  • TI rates were higher in patients with low transfusion burden (79%) compared to those with high burden (50%).
  • SF3B1-positive patients had a TI rate of 61.6%.
  • Higher response rates (86%) were observed in the low and very low IPSS-M risk groups.

Luspatercept was generally well-tolerated, with no serious adverse events beyond grade II toxicity. However, 13 patients (25.5%) did not respond, and 21% relapsed into transfusion dependence.

Reference
Jonasova A, Sotakova S, Belohlavkova P, et al. Experience with luspatercept therapy in patients with transfusion-dependent low-risk myelodysplastic syndromes in real-world clinical practice: exploring the positive effect of combination with erythropoietin alfa. Front Oncol. 2024;14:1398331. doi: 10.3389/fonc.2024.1398331. PMID: 39416466; PMCID: PMC11480566.