Treatment for systemic amyloidosis targets the aberrant plasma cell clone to prevent further synthesis and deposition of the amyloid protein. Conventional therapy usually combines oral melphalan with prednisone (MP), shown to yield higher response rates and longer survival than colchicine or prior therapies.
A recent Phase II trial in 23 patients whose primary objective was hematologic response rate of single-agent lenalidomide in patients with primary systemic amyloidosis was reported this year. Secondary objectives were the hematologic response rate of the lenalidomide-dexamethasone combination as well as toxicity and rate of organ response for single-agent lenalidomide or the 2-drug combination. With a median follow-up of 17 months, 10 patients responded to treatment. In these patients, responses included 9 hematologic, 4 renal, 2 cardiac, and 2 hepatic. All but one of the responders had dexamethasone added to their treatment program. Another Phase II trial is referenced below.
NCCN 2011 guideline lists lenalidomide and dexamethasone.
Guidelines on the diagnosis and management of AL amyloidosis.
British Journal of Haematology 125 (6), 681-70, 2004
Hans P. Sviggum, BA; Mark D. P. Davis, MD; S. Vincent Rajkumar, MD; Angela Dispenzieri, MD Dermatologic Adverse Effects of Lenalidomide Therapy for Amyloidosis and Multiple Myeloma Arch Dermatol. 2006;142:1298-1302
Dispenzieri et al.The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis Blood 2007;109:465-470.
G. Merlini Refining therapy for AL amyloidosis
Blood, December 1, 2006; 108(12): 3632 – 3633
Vaishali Sanchorawala et al,Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial Blood, 15 January 2007, Vol. 109, No. 2, pp. 492-496.