Health-related quality of life outcomes in patients with myelodysplastic syndromes with ring sideroblasts treated with luspatercept in the MEDALIST phase 3 trial

Esther Natalie Oliva*, Uwe Platzbecker, Guillermo Garcia-Manero, Ghulam J. Mufti, Valeria Santini, Mikkael A. Sekeres, Rami S. Komrokji, Jeevan K. Shetty, Derek Tang, Shien Guo, Weiqin Liao, George Zhang, Xianwei Ha, Rodrigo Ito, Jennifer Lord-Bessen, Jay T. Backstrom, Pierre Fenaux

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

12 Citations (Scopus)

Abstract

Patients with myelodysplastic syndromes (MDS) often experience chronic anemia and long-term red blood cell transfusion dependence associated with significant burden on clinical and health-related quality of life (HRQoL) outcomes. In the MEDALIST trial (NCT02631070), luspatercept significantly reduced transfusion burden in patients with lower-risk MDS who had ring sideroblasts and were refractory to, intolerant to, or ineligible for prior treatment with erythropoiesis-stimulating agents. We evaluated the effect of luspatercept on HRQoL in patients enrolled in MEDALIST using the EORTC QLQ-C30 and the QOL-E questionnaire. Change in HRQoL was assessed every 6 weeks in patients receiving luspatercept with best supportive care (+ BSC) and placebo + BSC from baseline through week 25. No clinically meaningful within-group changes and between-group differences across all domains of the EORTC QLQ-C30 and QOL-E were observed. On one item of the QOL-E MDS-specific disturbances domain, patients treated with luspatercept reported marked improvements in their daily life owing to the reduced transfusion burden, relative to placebo. Taken together with previous reports of luspatercept + BSC reducing transfusion burden in patients from baseline through week 25 in MEDALIST, these results suggest luspatercept may offer a treatment option for patients that reduces transfusion burden while providing stability in HRQoL.

Original languageEnglish
Article number27
JournalJournal of Clinical Medicine
Volume11
Issue number1
Early online date22 Dec 2021
DOIs
Publication statusPublished - 1 Jan 2022

Keywords

  • Luspatercept
  • Myelodysplastic syndromes
  • Quality of life
  • Transfusion dependence

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