Use Via Early Access to Ixazomib (UVEA-IXA) Study: Effectiveness and Safety of Ixazomib-based Therapy in Relapsed/Refractory Multiple Myeloma Outside of the Clinical Trial Setting

In multiple myeloma (MM), improving our understanding of routine clinical practice and the effectiveness of agents outside of clinical trials is important. TOURMALINE-MM1 data resulted in approval of ixazomib for MM patients who have received ≥1 prior therapy. UVEA-IXA comprised a retrospective chart review in the early access program, and a prospective 1-year follow-up period. Eligible patients had had a biochemical and/or symptomatic relapse after 1-3 prior lines of therapy; no anti-MM therapy for >3 cycles at the start of ixazomib therapy; and an Eastern Cooperative Oncology Group performance score of 0-2. Lenalidomide- or proteasome inhibitor (PI)-refractory patients were ineligible. Primary endpoints were response and progression-free survival (PFS). Of 357 enrolled patients, 309 were evaluable; most patients received ixazomib alongside lenalidomide (98%) and dexamethasone (97%); 61% had received 2-3 prior lines of therapy. Median PFS was 15.6 months (95% confidence interval [CI]: 12.0-20.6) in all evaluable patients, and 19.6 (95% CI: 12.1-27.0) and 13.9 (95% CI: 10.1-18.1) months in patients who received 1 and ≥2 prior lines of therapy, respectively. The overall response rate was 67% in all evaluable patients, and 72% and 63%, respectively, in patients who received 1 and ≥2 prior lines of therapy. Median overall survival was 35.5 months. The ixazomib safety profile was consistent with previous reports. This study supports ixazomib-based therapy as an effective and tolerable treatment in the real-world. Outcomes were favorable in patients with 1 or ≥2 prior lines of therapy who were not lenalidomide- or PI-refractory. UXEA-IXA reports treatment patterns and clinical outcomes for patients with relapsed and/or refractory multiple myeloma who received ixazomib-based therapy within the context of an early access program, prior to its approval in Europe. Results of the final analysis support ixazomib-based therapy as an effective and tolerable option for previously treated patients with multiple myeloma outside the clinical trial setting.