Patient-reported outcome domains in multiple myeloma randomized controlled trials and association with survival outcomes
Contact us if these videos are not relevant.
Contact us if these videos are not relevant.
View abstract on PubMed
Summary
This summary is machine-generated.Patient-reported outcomes (PROs) in multiple myeloma clinical trials show significant improvements, especially in quality of life and pain. These PROs demonstrate a notable association with overall survival, highlighting their importance in treatment efficacy.
Area Of Science
- Oncology
- Clinical Trials Methodology
- Patient-Reported Outcomes
Background
- Patient-reported outcomes (PROs) are vital in multiple myeloma (MM) randomized controlled trials (RCTs).
- Current methodology and reporting of PROs in MM RCTs exhibit considerable variability.
- Standardization is needed to fully leverage PRO data in evaluating treatment efficacy.
Purpose Of The Study
- To identify frequently pre-specified PRO domains in MM RCTs.
- To determine which PRO domains are most responsive to contemporary MM therapies.
- To investigate the correlation between PROs and progression-free survival (PFS) or overall survival (OS).
Main Methods
- Systematic review of MM RCTs utilizing the EORTC QLQ-C30 questionnaire.
- Studies published between January 2014 and June 2023 were included.
- Association between PROs and PFS/OS analyzed using Fisher's exact test or Pearson's Chi-squared test.
Main Results
- Thirty-five RCTs were reviewed; PROs were secondary/exploratory endpoints in all.
- Global health status/Quality of Life, Physical Functioning, Fatigue, and Pain were the most common pre-specified PRO domains.
- Over 65% of trials showed significant improvements in at least one PRO domain, notably GHS/QoL, Pain, Fatigue, and Physical Functioning.
- PROs showed concordance with PFS in 57.6% of trials and with OS in 71% of trials (p=0.018).
Conclusions
- Key PRO domains responsive to modern therapies were identified for potential use as primary endpoints in MM RCTs.
- The significant association between PROs and OS underscores their value in assessing treatment effectiveness.
- Integrating PROs more effectively can provide a comprehensive understanding of treatment benefits in multiple myeloma.
Contact us if these videos are not relevant.
Contact us if these videos are not relevant.
Related Concept Videos
Cancer survival analysis focuses on quantifying and interpreting the time from a key starting point, such as diagnosis or the initiation of treatment, to a specific endpoint, such as remission or death. This analysis provides critical insights into treatment effectiveness and factors that influence patient outcomes, helping to shape clinical decisions and guide prognostic evaluations. A cornerstone of oncology research, survival analysis tackles the challenges of skewed, non-normally...
The Kaplan-Meier estimator is a non-parametric method used to estimate the survival function from time-to-event data. In medical research, it is frequently employed to measure the proportion of patients surviving for a certain period after treatment. This estimator is fundamental in analyzing time-to-event data, making it indispensable in clinical trials, epidemiological studies, and reliability engineering. By estimating survival probabilities, researchers can evaluate treatment effectiveness,...
Survival analysis is a cornerstone of medical research, used to evaluate the time until an event of interest occurs, such as death, disease recurrence, or recovery. Unlike standard statistical methods, survival analysis is particularly adept at handling censored data—instances where the event has not occurred for some participants by the end of the study or remains unobserved. To address these unique challenges, specialized techniques like the Kaplan-Meier estimator, log-rank test, and...