title: Lenalidomide and dexamethasone in the treatment of AL amyloidosis: results of a phase 2 trial.
abstract: In immunoglobulin light chain (AL) amyloidosis, amyloid fibril deposits derived from immunoglobulin light chains produced by a clonal plasma cell dyscrasia accumulate in tissues and damage vital organs. Treatment regimens used in multiple myeloma can be effective in AL amyloidosis; however, patients with this disease often tolerate these regimens poorly because of multisystem organ dysfunction. Thalidomide and lenalidomide have both been shown to be effective in myeloma. In this report, we describe results of a phase 2 trial of the use of lenalidomide, as a single agent and in combination with dexamethasone, for the treatment of AL amyloidosis. Thirty-four patients with AL amyloidosis, most with prior therapies, were enrolled in the trial. The initial dose of lenalidomide used (25 mg/d) was poorly tolerated; however, a reduced dose of 15 mg/d was generally well tolerated. Of 24 evaluable patients, 7 (29%) achieved a hematologic complete response and 9 (38%) achieved a partial hematologic response, for an overall hematologic response rate of 67%. Hematologic responses were also associated with clinical responses. Fatigue and myelosuppression were the most common treatment-related adverse events (35%), while thromboembolic complications (9%) were the most serious. Findings from this trial indicate that lenalidomide can be effective in treating AL amyloidosis.
authors.affiliation: Department of Medicine, F 302, 732 Harrison Ave, Boston University Medical Center, MA 02118, USA. vaishali.sanchorawala@bmc.org
authors.forename: Vaishali
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: Daniel G
authors.initials: DG
authors.lastname: Wright
authors.forename: Michael
authors.initials: M
authors.lastname: Rosenzweig
authors.forename: Kathleen T
authors.initials: KT
authors.lastname: Finn
authors.forename: Salli
authors.initials: S
authors.lastname: Fennessey
authors.forename: Jerome B
authors.initials: JB
authors.lastname: Zeldis
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
authors.forename: David C
authors.initials: DC
authors.lastname: Seldin
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000097:blood / Q000175:diagnosis / Q000188:drug therapy*; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D004359:Drug Therapy, Combination; D004361:Drug Tolerance; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains / Q000097:blood*; 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