Patient-Reported Outcomes in Randomized Controlled Trials of Patients with Multiple Myeloma: A Systematic Literature Review of Studies Published Between 2014 and 2021

Background: We performed a systematic literature review to identify the most recently published randomized controlled trials (RCTs) in multiple myeloma (MM) with a patient-reported outcome (PRO) endpoint, and to summarize both clinical and PRO results, as well as to examine the quality of reporting by phase of disease. We also aimed to describe main type of PRO analysis used and interpretation of clinical significance of PRO findings. Materials and Methods: We searched PubMed and the Cochrane Central Register of Controlled Trials to identify RCTs of cancer-directed therapy in patients with MM published between January 2014 and April 2021. Results: Thirty-two RCTs with a total of 19,798 patients enrolled were identified in our review. In all studies, PROs were secondary or exploratory endpoints. Half of the studies (n = 16) included newly diagnosed patients, 15 RCTs included patients with relapsed/refractory MM, and one study included patients with smoldering MM. Progression-free survival was the most frequently used primary endpoint. All studies provided unique PRO information that could be used to more comprehensively assess the risk/benefit of the newly tested drugs. However, the identified RCTs were heterogeneous regarding the presentation, and interpretation of PRO results. Conclusion: The number of RCTs including PROs in MM research has notably increased in recent years. However, more consistency in the methodological approach to PRO assessment, and interpretation of outcomes is needed to ensure that PRO findings will be more impactful on patient care.