Review of patient-reported outcomes in multiple myeloma registrational trials: highlighting areas for improvement.
Blood Cancer J
; 11(8): 148, 2021 08 31.
Article
in En
| MEDLINE
| ID: mdl-34465728
ABSTRACT
Over the past 13 years, there have been advances in characterizing the patient experience in oncology trials, primarily using patient-reported outcomes (PROs). This review aims to provide details on the PRO measures and analyses used in multiple myeloma (MM) registrational trials. We identified registrational trials supporting MM indications from 2007 to 2020 from FDA databases. Trial protocols, statistical analysis plans, and clinical study reports were reviewed for PRO measures used, collection methods, statistical analyses, baseline and instrument completion definitions, and thresholds for clinical meaningfulness. Twenty-five trials supporting 20 MM indications were identified; 17 (68%) contained submitted PRO data. Of the 17 trials, 14 were randomized controlled trials and the remainder were single-arm trials. All but one trial were open label trials. Seven trials collected data electronically and five in paper format. The majority of trials evaluated at least two PRO measures (82%) with two trials (12%) utilizing four measures. Nine unique PRO measures were used, most commonly the EORTC QLQ-30 (87%), EQ-5D (65%), and QLQ-MY20 (47%). All 17 (100%) trials provided descriptive summaries, 10 (59%) carried out longitudinal mixed model analysis, 9 (53%) conducted responder analysis, and 2 (12%) did a basic inferential test. We noted substantial heterogeneity in terms of PRO collection methods, measures, definitions, and analyses, which may hinder the ability to effectively capture and interpret patient experience in future MM clinical trials. Further research is needed to determine the most appropriate approaches for statistical and analytical methodologies for PRO data in MM trials.
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Quality of Life
/
Patient Reported Outcome Measures
/
Multiple Myeloma
Type of study:
Clinical_trials
/
Guideline
/
Prognostic_studies
Aspects:
Patient_preference
Limits:
Humans
Language:
En
Journal:
Blood Cancer J
Year:
2021
Document type:
Article
Affiliation country:
United States