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Blood, 15 January 2007, Vol. 109, No. 2, pp. 465-470.
Prepublished online as a Blood First Edition Paper on September 28, 2006; DOI 10.1182/blood-2006-07-032987.
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Submitted July 5, 2006
Accepted August 31, 2006
The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis
Angela Dispenzieri*, Martha Q. Lacy, Steven R. Zeldenrust, Suzanne R. Hayman, Shaji K. Kumar, Susan M. Geyer, John A. Lust, Jacob B. Allred, Thomas E. Witzig, S. Vincent Rajkumar, Philip R. Greipp, Stephen J. Russell, Brian Kabat, and Morie A. Gertz
Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN
Department of Biostatistics, Mayo Clinic, Rochester, MN
Department of Laboratory Medicine, Mayo Clinic, Rochester, MN
Department of Molecular Medicine, Mayo Clinic, Rochester, MN
* Corresponding author; email: dispenzieri.angela{at}mayo.edu.
Primary systemic amyloidosis (AL) is an incurable plasma cell disorder. Lenalidomide, especially in conjunction with dexamethasone, is highly active in patients with multiple myeloma. We studied the toxicity and efficacy of lenalidomide in patients with AL. Patients with symptomatic AL, a measurable plasma cell disorder, and adequate hematologic and renal reserve were eligible. Patients received single agent lenalidomide. If progression by 3 months or no evidence of hematologic response after 3 cycles, dexamethasone was added. Twenty-three patients were enrolled. Thirteen were previously treated. Organ involvement was cardiac (64%), renal (73%), hepatic (23%), nerve (14%). Within the first 3 cycles of therapy, ten patients discontinued treatment: 4 early deaths, 3 adverse events, and 3 other causes. With a median follow-up of 17 months, ten patients responded to treatment. In these patients, responses included 9 hematologic, four renal, two cardiac and two hepatic. All but one of the responders had dexamethasone added to their treatment program. The most common grade 3-4 adverse advents at least possibly attributable to lenalidomide were neutropenia (45%), thrombocytopenia (27%), rash (18%), and fatigue (18%). In AL patients we saw limited activity of single agent lenalidomide, but significant activity of the combination with dexamethasone, which warrants further investigation.

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