OBJECTIVE: Melphalan flufenamide (melflufen) plus dexamethasone is fully approved in Europe for patients with relapsed/refractory multiple myeloma (RRMM) with ≥ 3 prior lines of therapy. We analyzed the efficacy and safety of melflufen in the real-world setting. METHODS: In this retrospective analysis, we examined baseline features, efficacy, and safety outcomes with melflufen plus dexamethasone in a cohort of 12 patients with heavily pre-treated RRMM at the Dana-Farber Cancer Institute, USA. RESULTS: Patients had received a median of 5.5 prior lines of therapy. Three patients (25%) had extramedullary disease, three (25%) cytogenetically high-risk features, and five (42%) had received prior autologous stem cell transplantation. The overall response rate was 55% (complete response: three [27%], very good partial response: one [9%], partial response: two [18%] patients). Five patients (42%) had stable disease
one was non-evaluable. Adverse events (AEs) were mostly hematologic and proved manageable
two patients had Grade 2 infections. Reasons for melflufen discontinuation were progressive disease (42%), drug withdrawal from the United States market (33%), AEs (17%), and sudden death (8%) unrelated to treatment. CONCLUSIONS: Consistent with clinical trial data, melflufen had an expected safety profile with manageable toxicity and clinically meaningful efficacy in patients with RRMM treated in the real-world setting.