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

TY - JOUR

T1 - Health-related quality of life outcomes in patients with myelodysplastic syndromes with ring sideroblasts treated with luspatercept in the MEDALIST phase 3 trial

AU - Oliva, Esther Natalie

AU - Platzbecker, Uwe

AU - Garcia-Manero, Guillermo

AU - Mufti, Ghulam J.

AU - Santini, Valeria

AU - Sekeres, Mikkael A.

AU - Komrokji, Rami S.

AU - Shetty, Jeevan K.

AU - Tang, Derek

AU - Guo, Shien

AU - Liao, Weiqin

AU - Zhang, George

AU - Ha, Xianwei

AU - Ito, Rodrigo

AU - Lord-Bessen, Jennifer

AU - Backstrom, Jay T.

AU - Fenaux, Pierre

N1 - Funding Information: Funding: This study was supported by Celgene, a Bristol-Myers Squibb Company, in collaboration with Acceleron Pharma. Funding Information: Conflicts of Interest: Ester N. Oliva: consultancy, honoraria for/from Amgen, BMS, and Novar-tis; honoraria, patents, royalties, speakers’ bureau from/for BMS. Uwe Platzbecker: consultancy, honoraria for/from BMS, Janssen, and Novartis; research funding from Amgen, Janssen, Merck, and Novartis. Guillermo Garcia-Manero: consultancy for Astex Pharmaceuticals, BMS, Genentech, Helsinn Therapeutics; research funding from AbbVie, Amphivena Therapeutics, Aprea, Astex Pharmaceuticals, BMS, Curis, Forty Seven, Genentech, Helsinn Therapeutics, H3 Biomedicine, Janssen, Merck, Novartis, Onconova. Ghulam J. Mufti: research funding from Aplastic Anaemia Trust, Blood-wise UK/Leukaemia and Lymphoma Research Programme, BMS, Cancer Research UK, Life Arc, and Novartis. Valeria Santini: research funding from Janssen; honoraria from BMS, Johnson & Johnson, and Novartis; consultancy for Acceleron Pharma, BMS, Menarini, and Novartis; membership on an entity’s board of directors or advisory committees for Pfizer and Takeda. Mikkael A. Sekeres: membership on an entity’s board of directors or advisory committees for BMS, Millennium/Takeda, and Novartis. Rami S. Komrokji: honoraria from AbbVie, Acceleron Pharma, Agios, BMS, Geron, Jazz Pharmaceuticals, and Novartis; speakers’ bureau for BMS and Jazz Pharmaceuticals. Jeevan K. Shetty, George Zhang, and Xianwei Ha: employment at BMS. Derek Tang and Jennifer Lord-Bessen: employment, equity ownership at BMS. Rodrigo Ito: former employment at BMS during time of study; current employment at Eli Lilly and Company; equity ownership at BMS, Eli Lilly and Company. Shien Guo: consultancy for BMS, Gilead, Janssen. Weigin Liao: consultancy for BMS. Jay T. Backstrom: employment, equity ownership at Acceleron Pharma; equity ownership at BMS. Pierre Fenaux: honoraria from BMS. Publisher Copyright: © 2021 by the authors. Licensee MDPI, Basel, Switzerland.

PY - 2022/1/1

Y1 - 2022/1/1

N2 - 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.

AB - 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.

KW - Luspatercept

KW - Myelodysplastic syndromes

KW - Quality of life

KW - Transfusion dependence

UR - https://www.scopus.com/pages/publications/85121431318

U2 - 10.3390/jcm11010027

DO - 10.3390/jcm11010027

M3 - Article

AN - SCOPUS:85121431318

SN - 2077-0383

VL - 11

JO - Journal of Clinical Medicine

JF - Journal of Clinical Medicine

IS - 1

M1 - 27

ER -