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

Background: 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. Patients and Methods: 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). Results: 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. Conclusion: 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.