Differences in Patient-Reported Outcomes That Are Most Frequently Detected in Randomized Controlled Trials in Patients With Solid Tumors: A Pooled Analysis of 229 Trials

Johannes M Giesinger; Jane Blazeby; Neil K Aaronson; Mirjam Sprangers; Peter Fayers; Francesco Sparano; Jonathan Rees; Amelie Anota; Chonghua Wan; Mike Pezold; Sumit Isharwal; Francesco Cottone; Fabio Efficace; EORTC Quality of Life Group

doi:10.1016/j.jval.2020.02.007

Differences in Patient-Reported Outcomes That Are Most Frequently Detected in Randomized Controlled Trials in Patients With Solid Tumors: A Pooled Analysis of 229 Trials

Value Health. 2020 May;23(5):666-673. doi: 10.1016/j.jval.2020.02.007. Epub 2020 Apr 25.

Authors

Affiliations

¹ Medical University of Innsbruck, University Hospital of Psychiatry II, Innsbruck, Austria.
² Bristol Centre for Surgical Research and Bristol Biomedical Research Centre, Population Health Sciences, University of Bristol, Bristol, England, UK.
³ Division of Psychosocial Research and Epidemiology, The Netherlands Cancer Institute, Amsterdam, The Netherlands.
⁴ Department of Medical Psychology, Amsterdam University Medical Centers, Amsterdam, The Netherlands.
⁵ Institute of Applied Health Sciences, University of Aberdeen, Aberdeen, Scotland, England, UK.
⁶ Italian Group for Adult Hematologic Diseases, Data Center and Health Outcomes Research Unit, Rome, Italy.
⁷ Methodology and Quality of Life in Oncology Unit (INSERM UMR 1098), University Hospital of Besançon, Besançon, France; French National Platform Quality of Life and Cancer, Besançon, France.
⁸ Guangdong Medical University, School of Humanities and Management, Research Center for Quality of Life and Applied Psychology, Dongguan, China.
⁹ Division of Vascular Surgery, Department of Surgery, New York University Langone Medical Center, New York, NY, USA.
¹⁰ Department of Urology, University of Virginia, Charlottesville, VA, USA.
¹¹ Italian Group for Adult Hematologic Diseases, Data Center and Health Outcomes Research Unit, Rome, Italy. Electronic address: f.efficace@gimema.it.

PMID: 32389233
DOI: 10.1016/j.jval.2020.02.007

Abstract

Objectives: Patient-reported outcome (PRO) measurements used in cancer research can assess a number of health domains. Our primary objective was to investigate which broad types of PRO domains (namely, functional health, symptoms, and global quality of life [QoL]) most frequently yielded significant differences between treatments in randomized controlled trials (RCTs).

Methods: A total of 229 RCTs published between January 2004 and February 2019, conducted on patients diagnosed with the most common solid malignancies and assessed using the European Organization for the Research and Treatment of Cancer (EORTC) Quality of Life Questionnaire (QLQ)-C30, were considered. Studies were identified systematically using literature searches in key electronic databases. Unlike other PRO measurements typically used in RCTs, the scoring algorithm of the multidimensional EORTC QLQ-C30 allowed us to clearly distinguish the 3 broad types of PRO domains.

Results: In total, 134 RCTs (58.5%) reported statistically significant differences between treatment arms for at least 1 of the QLQ-C30 domains. Most frequently, differences were reported for 2 or all 3 broad types of PRO domains (78 of 134 trials; 58.2%). In particular, 35 trials (26.1%) found significant differences for symptoms, functional health, and global QoL, 24 trials (17.9%) for symptoms and functional health, 11 trials (8.2%) for functional health and global QoL, and 8 trials (6.0%) for symptoms and global QoL. The likelihood of finding a statistically significant difference between treatment arms was not associated with key study characteristics, such as study design (ie, open-label vs blinded trials) and industry support.

Conclusions: Our findings emphasize the importance of a multidimensional PRO assessment to most comprehensively capture the overall burden of therapy from the patients' standpoint.

Keywords: endpoint; functional health; patient-reported outcomes; quality of life; randomized controlled trials; symptoms.

Publication types

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

MeSH terms

Adult
Female
Health Status Indicators*
Humans
Male
Middle Aged
Neoplasms*
Patient Reported Outcome Measures*
Quality of Life*
Randomized Controlled Trials as Topic*

Grants and funding

MR/K025643/1/MRC_/Medical Research Council/United Kingdom