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Article

Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial

1
Grande Ospedale Metropolitano Bianchi Melacrino Morelli, 89124 Reggio Calabria, Italy
2
Medical Clinic and Policlinic 1, Hematology and Cellular Therapy, Leipzig University Hospital, 04103 Leipzig, Germany
3
The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA
4
Department of Haemato-Oncology, King’s College London, London SE5 9RS, UK
5
MDS Unit, Azienda Ospedaliero Universitaria Careggi, University of Florence, 50134 Florence, Italy
6
Sylvester Comprehensive Cancer Center, University of Miami, Miami, FL 33136, USA
7
Moffitt Cancer Center, Tampa, FL 33612, USA
8
Bristol Myers Squibb, Princeton, NJ 08540, USA
9
Evidera, Waltham, MA 02451, USA
10
Acceleron Pharma, Cambridge, MA 02139, USA
11
Service d’Hématologie Séniors, Hôpital Saint-Louis, Assistance Publique des Hôpitaux de Paris (AP-HP), 75010 Paris, France
12
Senior Hematology Department, Saint Louis Hospital, Paris 7 University, 75013 Paris, France
*
Author to whom correspondence should be addressed.
These authors contributed equally to the work.
Academic Editor: Alicia Rovo
J. Clin. Med. 2022, 11(1), 27; https://doi.org/10.3390/jcm11010027
Received: 10 November 2021 / Revised: 10 December 2021 / Accepted: 18 December 2021 / Published: 22 December 2021
(This article belongs to the Special Issue Myelodysplastic Syndrome: Recent Advances and Future Directions)
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. View Full-Text
Keywords: transfusion dependence; quality of life; myelodysplastic syndromes; luspatercept transfusion dependence; quality of life; myelodysplastic syndromes; luspatercept
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MDPI and ACS Style

Oliva, E.N.; Platzbecker, U.; Garcia-Manero, G.; Mufti, G.J.; Santini, V.; Sekeres, M.A.; Komrokji, R.S.; Shetty, J.K.; Tang, D.; Guo, S.; Liao, W.; Zhang, G.; Ha, X.; Ito, R.; Lord-Bessen, J.; Backstrom, J.T.; Fenaux, P. Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial. J. Clin. Med. 2022, 11, 27. https://doi.org/10.3390/jcm11010027

AMA Style

Oliva EN, Platzbecker U, Garcia-Manero G, Mufti GJ, Santini V, Sekeres MA, Komrokji RS, Shetty JK, Tang D, Guo S, Liao W, Zhang G, Ha X, Ito R, Lord-Bessen J, Backstrom JT, Fenaux P. Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial. Journal of Clinical Medicine. 2022; 11(1):27. https://doi.org/10.3390/jcm11010027

Chicago/Turabian Style

Oliva, Esther Natalie, 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, and Pierre Fenaux. 2022. "Health-Related Quality of Life Outcomes in Patients with Myelodysplastic Syndromes with Ring Sideroblasts Treated with Luspatercept in the MEDALIST Phase 3 Trial" Journal of Clinical Medicine 11, no. 1: 27. https://doi.org/10.3390/jcm11010027

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