Abstract
In this report, a panel of multiple myeloma experts assesses the role of melflufen (melphalan flufenamide) in the treatment of patients with relapsed or refractory multiple myeloma (RRMM). Based on available data, recommendations for administration of melflufen, management of adverse events and optimal patient selection are given. Melflufen, a first-in-class peptide-drug conjugate, is approved in the European Union and the United Kingdom in combination with dexamethasone for the treatment of adult patients with multiple myeloma (MM) who have received at least three prior lines of therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least three years from transplantation. Melflufen has demonstrated sustained and durable responses in single-arm and randomized trials. The recommended starting is 40 mg intravenously every 28 days in combination with dexamethasone. Adverse events are mainly dose-related haematological toxicities and infections, which are manageable according to standard guidelines. Melflufen is recommended in patients with no prior high-dose melphalan therapy or in those with a long treatment-free interval after such treatment (> 36 months). In high-risk patients, including patients with del(17p) and TP53 gene mutations, melflufen may be considered as a treatment option, especially if patients have not previously been treated with high-dose melphalan.