Purpose : Many clinical trials have been conducted to prove the efficiacy of the new agrent or treatment regimen in patients with multiple myeloma (MM), but there was no analysis of the effect of enrollment in clinical trials on survival in patients with MM. Therefore, we evaluate the survival outcome in patients with multiple myeloma who participated in clinical trial.
Method: Retrospective data of 446 patients with newly diagnosed MM between January 2003 and December 2016 in Chonnam National University Hwasun Hospital were analyzed. Patients diagnosed with monoclonal gammopathy of undetermined significance, asymptomatic MM, and plasma cell leukemia were excluded. In addition, non-interventional study was not included as a clinical trial in this study.
Results: The median overall survival (OS) for the entire cohort was 62.5 months (95%CI 52.4-72), and median OS has gradually improved (46.5 months in 2003-2008 vs. 64.9 months in 2009-2014 vs. not reached in 2015-2016, P = 0.007). A total of 197 patients (44.2%) participated in the clinical trial and 7.4% participated in more than two clinical trials during the entire treatment period. Participation rates in clinical trials were significantly higher in transplant-eligible patients than transplant-ineligible patients (55.7% vs. 34.8%, P <0.001). There was no significant difference in OS according to the enrollment of clinical trial in entire patients (62.9 months vs. 64.9 months, P = 0.265). In transplant-eligible patients, patients who participated clinical trial showed improved OS than patients who did not participate clinical trial (77.0 months vs. 52.1 months, P = 0.058). However, in transplant-ineligible patients, no survival benefits were showed from participation in clinical trial (50.3 months vs. 66.0 months, P = 0.811)
Conclusion: This study showed survival benefit of enrollment of clinical trial in transplant-eligible patients. Further analysis is needed to analysis the effects of clinical trial in elderly patients.
No relevant conflicts of interest to declare.
Asterisk with author names denotes non-ASH members.