Targeting health-related quality of life in patients with myelodysplastic syndromes - Current knowledge and lessons to be learned

Esther N Oliva; Uwe Platzbecker; Pierre Fenaux; Guillermo Garcia-Manero; Thomas W LeBlanc; Bhumika J Patel; Anne Sophie Kubasch; Mikkael A Sekeres

doi:10.1016/j.blre.2021.100851

Targeting health-related quality of life in patients with myelodysplastic syndromes - Current knowledge and lessons to be learned

Blood Rev. 2021 Nov:50:100851. doi: 10.1016/j.blre.2021.100851. Epub 2021 May 14.

Authors

Esther N Oliva¹, Uwe Platzbecker², Pierre Fenaux³, Guillermo Garcia-Manero⁴, Thomas W LeBlanc⁵, Bhumika J Patel⁶, Anne Sophie Kubasch⁷, Mikkael A Sekeres⁸

Affiliations

¹ Grande Ospedale Metropolitano Bianchi Melacrino Morelli, Reggio Calabria, Italy. Electronic address: [email protected].
² Department of Hematology, Cellular Therapy and Hemostaseology, Leipzig University Hospital, Leipzig, Germany. Electronic address: [email protected].
³ Service d'Hématologie Séniors, Hôpital Saint-Louis, Université Paris 7, Paris, France. Electronic address: [email protected].
⁴ The University of Texas MD Anderson Cancer Center, Houston, TX, USA. Electronic address: [email protected].
⁵ Duke Cancer Institute, Durham, NC, USA. Electronic address: [email protected].
⁶ Leukemia Program, Cleveland Clinic, Cleveland, OH, USA. Electronic address: [email protected].
⁷ Department of Hematology, Cellular Therapy and Hemostaseology, Leipzig University Hospital, Leipzig, Germany. Electronic address: [email protected].
⁸ Sylvester Cancer Center, University of Miami Miller School of Medicine, Miami, FL, USA. Electronic address: [email protected].

PMID: 34088518
DOI: 10.1016/j.blre.2021.100851

Abstract

Using a range of health-related quality of life (HRQoL) instruments, most - but not all - studies of myelodysplastic syndromes (MDS) have reported that lower hemoglobin levels and red blood cell transfusion dependency are associated with worse HRQoL. In addition, some MDS treatments may significantly improve HRQoL, particularly among those patients who respond to therapy; however, the majority of these studies were underpowered for this secondary endpoint. Furthermore, decreased HRQoL has been associated with worse survival outcomes, and HRQoL scores can be used to refine classical prognostic systems. Despite the subjective nature of HRQoL, the importance and validity of measuring it in trials and clinical practice are increasingly being recognized, but properly validated MDS-specific instruments are required. We describe what is currently known about HRQoL in patients with MDS, and the limitations of measuring HRQoL, and we provide some recommendations to improve the measurement of this outcome in future trials.

Keywords: Health-related quality of life; Myelodysplastic syndromes; Patient-reported outcomes.

Publication types

Research Support, Non-U.S. Gov't
Review

MeSH terms

Erythrocyte Transfusion / methods
Humans
Myelodysplastic Syndromes* / diagnosis
Myelodysplastic Syndromes* / therapy
Prognosis
Quality of Life*