Abstract
Introduction: Patient-reported outcomes (PROs) are valuable tools in clinical trials, providing crucial insights into treatment impact from the patient's perspective. However, PRO reporting in oncology trials lacks consistency. Patients with multiple myeloma are often treated with triple or quadruple drug regimens, which may reduce disease progression but negatively impact their quality of life. Therefore, there is a need to assess the PROs in phase 3 trials for multiple myeloma.
Objective:
To assess the PROs and their pattern in phase 3 trials in multiple myeloma published between 2020 and 2024.
Methods: Literature Search Strategy:
We searched for “Multiple Myeloma” as MeSH term in PubMed, Cochrane, and as Emtree term in Embase database for clinical trials published between January 1, 2020, and December 31, 2024. These results were exported to Covidence software, filtered by “phase III” or “phase 3” study, and screened by two independent reviewers. We excluded all abstracts, preprint articles, subgroup analyses, long-term follow-up results, and exploratory results, and included the first analysis published in a peer-reviewed journal.
We aimed to evaluate;
What proportion of these trials have included PRO in their study?
Are PROs reported in the main text or the supplement?
Are PRO results reported in the main text or the supplement?
What PROs are included, and is there consistency?
When reported, is there a significant difference in PRO between the intervention and control arms?
Is there an association between the characteristics of clinical trials (Funding, Type of Masking, National/Multinational, Type of treatment, New/Relapse Disease) with the PRO inclusion?
Results: Our preliminary search identified 5226 articles across the three databases, out of which 21 studies met our inclusion criteria.
Fifteen trials (71%) included PRO, while six did not include PRO as an outcome. Out of the 15 trials that included PRO, the majority (14/15) included it in the main text, while only one reported it in the supplement. Of the 15 trials that included PRO, 13 trials included results, whereas two trials did not report results in either the main text or the supplement. Of the 13 trials with results, eight included them in the main text, three had data results in the supplement with a result conclusion in the main article, and two included results solely in the supplement. Among the 13 trials, PRO was not statistically significant between the intervention and control arm in 10 trials, was better in the intervention group in 2 trials, and the result was unclear in 1 trial. Most trials (14/15) included PRO as a secondary outcome, and only one included it as both a coprimary and secondary endpoint.
There was significant heterogeneity in the PROs used across these trials. Eight different types of PROs were utilized, with the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core-30 (EORTC-30) and Multiple Myeloma (MY20) modules being the most frequently used.
We utilized the Chi-Square test to identify the association between the type of studies (Funding, Type of Masking, National/Multinational, Type of treatment, New/Relapse Disease) and the inclusion of the PRO in these studies. The p-value for none of these associations was statistically significant, which is most likely due to the small sample size.
Conclusion: This systematic review revealed that the majority of published clinical trials in multiple myeloma include PROs as secondary endpoints. However, inconsistent reporting of PRO measures and their results was common, potentially hindering readers' ability to locate and interpret these findings effectively. These findings highlight the need for standardized reporting guidelines to ensure clarity and consistency in presenting PRO data in future clinical trials.
