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

Research output: Contribution to journalArticlepeer-review

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 (US)
Article number27
JournalJournal of Clinical Medicine
Volume11
Issue number1
DOIs
StatePublished - Jan 1 2022

Keywords

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

ASJC Scopus subject areas

  • Medicine(all)

Fingerprint

Dive into the research topics of 'Health-related quality of life outcomes in patients with myelodysplastic syndromes with ring sideroblasts treated with luspatercept in the MEDALIST phase 3 trial'. Together they form a unique fingerprint.

Cite this