eCite Digital Repository

Does knowledge of treatment assignment affect patient report of symptoms, function, and health status? An evaluation using multiple myeloma trials

Citation

Roydhouse, JK and Mishra-Kalyani, PS and Bhatnagar, V and Gutman, R and King-Kallimanis, BL and Sridhara, R and Kluetz, PG, Does knowledge of treatment assignment affect patient report of symptoms, function, and health status? An evaluation using multiple myeloma trials, Value in Health ISSN 1098-3015 (2021) [Refereed Article]

Copyright Statement

Copyright 2021 ISPOR

DOI: doi:10.1016/j.jval.2020.12.015

Abstract

Objectives: Unblinded trials are common in oncology, but patient knowledge of treatment assignment may bias response to questionnaires. We sought to ascertain the extent of possible bias arising from patient knowledge of treatment assignment.

Methods: This is a retrospective analysis of data from 2 randomized trials in multiple myeloma, 1 double-blind and 1 open label. We compared changes in patient reports of symptoms, function, and health status from prerandomization (screening) to baseline (pretreatment but postrandomization) across control and investigational arms in the 2 trials. Changes from prerandomization scores at ~2 and 6 months on treatment were evaluated only across control arms to avoid comparisons between 2 different experimental drugs. All scores were on 0- to 100-point scales. Inverse probability weighting, entropy balancing, and multiple imputation using propensity score splines were used to compare score changes across similar groups of patients.

Results: Minimal changes from screening were seen at baseline in all arms. In the control arm, mean changes of <7 points were seen for all domains at 2 and 6 months. The effect of unblinding at 6 months in social function was a decline of less than 6 points (weighting: −3.09; 95% confidence interval −8.41 to 2.23; balancing: −4.55; 95% confidence interval −9.86 to 0.76; imputation: −5.34; 95% confidence interval −10.64 to −0.04).

Conclusion: In this analysis, we did not find evidence to suggest that there was a meaningful differential effect on how patients reported their symptoms, function or health status after knowing their treatment assignment.

Item Details

Item Type:Refereed Article
Keywords:clinical trial, blinding, outcomes, patient-reported outcomes, bias, cancer
Research Division:Biomedical and Clinical Sciences
Research Group:Oncology and carcinogenesis
Research Field:Oncology and carcinogenesis not elsewhere classified
Objective Division:Health
Objective Group:Evaluation of health and support services
Objective Field:Evaluation of health outcomes
UTAS Author:Roydhouse, JK (Dr Jessica Roydhouse)
ID Code:143565
Year Published:2021
Deposited By:Menzies Institute for Medical Research
Deposited On:2021-03-24
Last Modified:2021-06-18
Downloads:0

Repository Staff Only: item control page