title: The activity of lenalidomide with or without dexamethasone in patients with primary systemic amyloidosis.
abstract: 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 there was no evidence of progression after 3 months or 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%), and nerve (14%). Within the first 3 cycles of therapy, 10 patients discontinued treatment: 4 early deaths, 3 adverse events, and 3 other causes. 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. The most common grade 3 or 4 adverse events 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.
authors.affiliation: Department of Biostatistics, Mayo Clinic, Rochester, MN 55905, USA. dispenzieri.angela@mayo.edu
authors.forename: Angela
authors.initials: A
authors.lastname: Dispenzieri
authors.forename: Martha Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: Steven R
authors.initials: SR
authors.lastname: Zeldenrust
authors.forename: Suzanne R
authors.initials: SR
authors.lastname: Hayman
authors.forename: Shaji K
authors.initials: SK
authors.lastname: Kumar
authors.forename: Susan M
authors.initials: SM
authors.lastname: Geyer
authors.forename: John A
authors.initials: JA
authors.lastname: Lust
authors.forename: Jacob B
authors.initials: JB
authors.lastname: Allred
authors.forename: Thomas E
authors.initials: TE
authors.lastname: Witzig
authors.forename: S Vincent
authors.initials: SV
authors.lastname: Rajkumar
authors.forename: Philip R
authors.initials: PR
authors.lastname: Greipp
authors.forename: Stephen J
authors.initials: SJ
authors.lastname: Russell
authors.forename: Brian
authors.initials: B
authors.lastname: Kabat
authors.forename: Morie A
authors.initials: MA
authors.lastname: Gertz
mesh_terms: D000284:Administration, Oral; D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology*; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D005260:Female; D005500:Follow-Up Studies; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000276:immunology*; D000077269:Lenalidomide; D008297:Male; D008875:Middle Aged; D011237:Predictive Value of Tests; D015996:Survival Rate; D013792:Thalidomide / Q000008:administration & dosage / Q000009:adverse effects / Q000031:analogs & derivatives*; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains; D013792:Thalidomide; D003907:Dexamethasone; D000077269:Lenalidomide