Dimopoulos MA, et al. - Experts ascertained the safety and efficacy of ixazomib as maintenance therapy following autologous stem cell transplantation (ASCT). Progression-free survival (PFS) was prolonged by ixazomib maintenance and it represents an additional option for post-transplant maintenance therapy in patients with newly diagnosed multiple myeloma.

• Authors conducted a phase 3, double-blind, placebo-controlled TOURMALINE-MM3 study in 167 clinical or hospital sites in 30 countries in Europe, the Middle East, Africa, Asia, and North and South America.
• Adults with a confirmed diagnosis of symptomatic multiple myeloma according to International Myeloma Working Group criteria who had achieved at least a partial response after undergoing standard-of-care induction therapy followed by high-dose melphalan (200 mg/m ²) conditioning and single ASCT within 12 months of diagnosis were the eligible participants.
• They randomly assigned the patients in a 3:2 ratio to oral ixazomib or matching placebo on days 1, 8, and 15 in 28-day cycles for 2 years following induction, high-dose therapy, and transplantation.
• They increased the initial 3 mg dose to 4 mg from cycle 5 if tolerated during cycles 1–4.
• They stratified the randomisation by induction regimen, pre-induction disease stage, and response post-transplantation.
• Progression-free survival (PFS) by intention-to-treat analysis was the primary endpoint.
• They evaluated safety in all patients who received at least one dose of ixazomib or placebo, according to treatment actually received.

• They enrolled 656 patients between July 31, 2014, and March 14, 2016 and randomly assigned to receive ixazomib maintenance therapy (n=395) or placebo (n=261).
• As per data, with a median follow-up of 31 months (IQR 27.3–35.7), they observed a 28% reduction in the risk of progression or death with ixazomib vs placebo (median PFS 26.5 months [95% CI 23.7–33.8] vs 21.3 months [18.0–24.7]; hazard ratio 0.72, 95% CI 0.58–0.89; p=0.0023).
• At the time of this analysis, no increase in second malignancies was noted with ixazomib therapy (12 [3%] patients) vs placebo (eight [3%] patients).
• Results demonstrated that 108 (27%) of 394 patients in the ixazomib group and 51 (20%) of 259 patients in the placebo group experienced serious adverse events.
• In the ixazomib group one patient died during the treatment period, and none died in the placebo group.