Does Knowledge of Treatment Assignment Affect Patient Report of Symptoms, Function, and Health Status? An Evaluation Using Multiple Myeloma Trials

Jun 1, 2021, 00:00
10.1016/j.jval.2020.12.015
https://www.valueinhealthjournal.com/article/S1098-3015(21)00058-9/fulltext
Title : Does Knowledge of Treatment Assignment Affect Patient Report of Symptoms, Function, and Health Status? An Evaluation Using Multiple Myeloma Trials
Citation : https://www.valueinhealthjournal.com/action/showCitFormats?pii=S1098-3015(21)00058-9&doi=10.1016/j.jval.2020.12.015
First page : 822
Section Title : METHODOLOGY
Open access? : No
Section Order : 822

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 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.

Categories :
  • Health State Utilities
  • Methodological & Statistical Research
  • Oncology
  • Patient Behavior and Incentives
  • Patient-Centered Research
  • Patient-reported Outcomes & Quality of Life Outcomes
  • PRO & Related Methods
  • Specific Diseases & Conditions
  • Stated Preference & Patient Satisfaction
Tags :
  • bias
  • cancer
  • clinical trial
  • open-label
  • patient-reported outcomes
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