TY - JOUR
T1 - Targeting health-related quality of life in patients with myelodysplastic syndromes – Current knowledge and lessons to be learned
AU - Oliva, Esther N.
AU - Platzbecker, Uwe
AU - Fenaux, Pierre
AU - Garcia-Manero, Guillermo
AU - LeBlanc, Thomas W.
AU - Patel, Bhumika J.
AU - Kubasch, Anne Sophie
AU - Sekeres, Mikkael A.
N1 - Funding Information:
ENO has received honoraria, provided consultancy, participated in a speakers' bureau and has patents (for the QOL-E instrument) and royalties (for use of the QOL-E instrument in the BMS-sponsored MEDALIST clinical trial) with BMS; and provided consultancy to AbbVie, Alexion, Amgen, Apellis, and Novartis. UP has provided consultancy to AbbVie, BMS, Geron, Janssen, Novartis, and Takeda. PF has received research funding and honoraria from AbbVie, BMS/Celgene, Jazz Pharmaceuticals, and Novartis. GG-M has no conflicts of interest to disclose. TWL has received royalties from UptoDate; provided consultancy, received honoraria, and been a member of a board of directors or advisory committee for AbbVie, Agios, Amgen, AstraZeneca, BMS, Carevive, Daiichi-Sankyo, Flatiron, Helsinn, Heron, Otsuka, Medtronic, Pfizer, Seattle Genetics, and Welvie; received research funding from American Cancer Society, BMS, Duke University, Jazz Pharmaceuticals, NINR/NIH, and Seattle Genetics; and participated in a speakers' bureau for AbbVie, Agios, and BMS. BJP has participated in a speakers' bureau for Alexion. ASK has provided consultancy to Amgen, Novartis, and Takeda. MAS has been a member of a board of directors or advisory committee for BMS, Pfizer, and Takeda/Millennium.
Publisher Copyright:
© 2021 The Authors
PY - 2021/11
Y1 - 2021/11
N2 - 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.
AB - 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.
KW - Health-related quality of life
KW - Myelodysplastic syndromes
KW - Patient-reported outcomes
UR - http://www.scopus.com/inward/record.url?scp=85107278829&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85107278829&partnerID=8YFLogxK
U2 - 10.1016/j.blre.2021.100851
DO - 10.1016/j.blre.2021.100851
M3 - Review article
C2 - 34088518
AN - SCOPUS:85107278829
SN - 0268-960X
VL - 50
JO - Blood Reviews
JF - Blood Reviews
M1 - 100851
ER -