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Amyloid light-chain (AL) amyloidosis is initially treated with melphalan and dexamethasone or cyclophosphamide, dexamethasone, and bortezomib. Immunomodulatory agents such as lenalidomide and pomalidomide also have activity but substantial toxicity (NEJM JW Oncol Hematol Mar 2017 and Blood 2017; 129:2120)
To examine the safety and effectiveness in this setting of ixazomib, an oral proteasome inhibitor recently approved for the treatment of relapsed myeloma, investigators conducted an industry-funded phase I/II trial in 27 patients with relapsed or refractory AL amyloidosis. Patients had received a median of three agents, and 67% had undergone autologous stem cell transplant. Ixazomib was given weekly for up to 12 cycles.
At a median follow-up…