title: Melphalan, lenalidomide and dexamethasone for the treatment of immunoglobulin light chain amyloidosis: results of a phase II trial.
abstract: We report results of a phase II trial of combination of melphalan, lenalidomide, and dexamethasone for the treatment of immunoglobulin light chain (AL) amyloidosis. The primary objectives were tolerability and hematologic response rate; secondary objectives were organ responses and survival. Treatment protocol consisted of melphalan 5 mg/m(2)/day for four days, lenalidomide 10 mg/day for 21 days and dexamethasone 20-40 mg once a week every 28 days for a total of 12 cycles. Sixteen subjects were enrolled of whom 14 completed at least 3 cycles and were evaluable for response. Grade 3/4 toxicities were experienced by 88% (n=14), the most common being myelosuppression (n=7). Dose reductions occurred in 85% (n=12 of 14) of subjects. Hematologic partial and complete responses were achieved by 43% (n=6 of 14) and 7% (n=1 of 14), respectively. The median overall survival has not been reached and median progression-free survival is 24 months. In conclusion, this combination is associated with significant myelosuppression leading to dose modifications and producing minor hematologic responses in AL amyloidosis. http://clinicaltrials.gov/ct2/show/NCT00679367.
authors.affiliation: Amyloid Treatment and Research Program, Section of Hematology and Oncology, Boston University School of Medicine, Boston Medical Center, Boston, MA, USA. Vaishali.Sanchorawala@bmc.org
authors.forename: Vaishali
authors.initials: V
authors.lastname: Sanchorawala
authors.forename: Jaymin M
authors.initials: JM
authors.lastname: Patel
authors.forename: J Mark
authors.initials: JM
authors.lastname: Sloan
authors.forename: Anthony C
authors.initials: AC
authors.lastname: Shelton
authors.forename: Jerome B
authors.initials: JB
authors.lastname: Zeldis
authors.forename: David C
authors.initials: DC
authors.lastname: Seldin
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality; D000971:Antineoplastic Combined Chemotherapy Protocols / Q000009:adverse effects / Q000627:therapeutic use*; D003907:Dexamethasone / Q000008:administration & dosage; D018450:Disease Progression; D005260:Female; D006801:Humans; D007147:Immunoglobulin Light Chains; D000077269:Lenalidomide; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D013792:Thalidomide / Q000008:administration & dosage / Q000031:analogs & derivatives; D016896:Treatment Outcome
chemical_list: D007147:Immunoglobulin Light Chains; D013792:Thalidomide; D003907:Dexamethasone; D000077269:Lenalidomide; D008558:Melphalan