source: Amyloid: The International Journal of Experimental and Clinical Investigation

year: 2017

authors: Phillips EH, Nash S, Adedayo T, Whelan CJ, Fontana M, Mahmood S, Lachmann HJ, Gillmore JD, Smith P, Clifton-Hadley L, Hawkins PN, Wechalekar AD

Very few prospective clinical trials have been conducted in advanced cardiac AL amyloidosis due to the rarity of the disease and challenging clinical course of the condition. With a median survival of only 7 months, cardiac AL represents a major area of unmet need. 

The introduction of proteasome inhibitors and immunomodulatory agents over the last decade has marked a new era in the treatment of plasma cell dyscrasias. The phase 1 CAN2007 study demonstrated the feasibility of single-agent bortezomib in relapsed/ refractory AL up to a dose of 1.3 mg/m2 twice weekly. The REsponse to VElcade combination chemotherapy in AL amyloidosis (REVEAL) trial was conceived in 2008 to test the efficacy and toxicity of bortezomib-based triplet therapy in AL amyloidosis. Here, we report the course of the REVEAL trial, which illustrates the challenges in conducting trials where high mortality is part of the disease course.

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