REAL-WORLD outcomes for individuals who received the standard treatment regimens for multiple myeloma were significantly poorer than outcomes reported in clinical trials. Lead author of the research, Alissa Visram, Ottawa Hospital Research Institute, Ontario, Canada, and colleagues, launched the study to try to better comprehend the potential differences between clinical trial and real-world outcomes.
The retrospective study comprised 3,951 patients with either newly diagnosed or refractory multiple myeloma, who had received treatment between 2007–2020 in Ontario. Researchers compared real-world outcomes amongst patients receiving standard multiple myeloma treatments with patient outcomes reported in Phase III randomised controlled trials. Treatment regimens for newly diagnosed individuals included lenalidomide with dexamethasone, as well as triple combination therapy with bortezomib, lenalidomide, and dexamethasone. Treatment regimens for patients with relapsed myeloma included pomalidomide with dexamethasone, carfilzomib with dexamethasone, or triple combination therapy including carfilzomib, lenalidomide, and dexamethasone.
Results indicated that patients in a real-world setting have demonstrably worse overall survival outcomes for six out of the seven treatment regimens (pooled hazard ratio: 1.75; P=0.010). Real-world patients also had a poorer progression-free survival outcome for six out of the seven treatment regimens (pooled hazard ratio: 1.44; P=0.034). Progression-free survival was noted to be at least 3–18 months longer in clinical trial patients, with a median survival of at least 19 months longer than the real-world patient cohort. The only treatment with comparable outcomes in both cohorts was pomalidomide and dexamethasone. Adverse effects were also reported to be similar between the real-world and clinical trial patient groups.
Visram commented that the results will likely influence how they speak to patients about their outcomes, saying: “I’ll probably present both numbers [from real-life and clinical-trial data] and give them a sense of the best-case scenario.” The next step is to explore what is causing these differences in outcomes.