title: A trial of three regimens for primary amyloidosis: colchicine alone, melphalan and prednisone, and melphalan, prednisone, and colchicine.
abstract: BACKGROUND
  Primary systemic amyloidosis is an uncommon disease characterized by the accumulation in vital organs of a fibrillar protein consisting of monoclonal light chains.
  
  
  METHODS
  We treated 220 patients with biopsy-proved amyloidosis. The patients were randomly assigned to receive colchicine (72 patients), melphalan and prednisone (77), or melphalan, prednisone, and colchicine (71). They were stratified according to their chief clinical manifestations: renal disease (105 patients), cardiac involvement (46), peripheral neuropathy (19), or other (50).
  
  
  RESULTS
  The median duration of survival after randomization was 8.5 months in the colchicine group, 18 months in the group assigned to melphalan and prednisone, and 17 months in the group assigned to melphalan, prednisone, and colchicine (P<0.001). Among patients who had a reduction in serum or urine monoclonal protein at 12 months, the overall length of survival was 50 months, whereas among those without a reduction at 12 months, the overall length of survival was 36 months (P=0.03). Thirty-four patients (15 percent) survived for five years or longer.
  
  
  CONCLUSIONS
  Therapy with melphalan and prednisone results in objective responses and prolonged survival as compared with colchicine in patients with primary amyloidosis.
authors.affiliation: Division of Hematology and Internal Medicine, Mayo Clinic and Mayo Foundation, Rochester, MN 55905, USA.
authors.forename: R A
authors.initials: RA
authors.lastname: Kyle
authors.forename: M A
authors.initials: MA
authors.lastname: Gertz
authors.forename: P R
authors.initials: PR
authors.lastname: Greipp
authors.forename: T E
authors.initials: TE
authors.lastname: Witzig
authors.forename: J A
authors.initials: JA
authors.lastname: Lust
authors.forename: M Q
authors.initials: MQ
authors.lastname: Lacy
authors.forename: T M
authors.initials: TM
authors.lastname: Therneau
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality; D000970:Antineoplastic Agents / Q000627:therapeutic use; D009202:Cardiomyopathies / Q000188:drug therapy / Q000401:mortality; D003078:Colchicine / Q000627:therapeutic use*; D004359:Drug Therapy, Combination; D006801:Humans; D007674:Kidney Diseases / Q000188:drug therapy / Q000401:mortality; D008558:Melphalan / Q000627:therapeutic use*; D008875:Middle Aged; D011241:Prednisone / Q000627:therapeutic use*; D011446:Prospective Studies; D016019:Survival Analysis; D016896:Treatment Outcome
chemical_list: D000970:Antineoplastic Agents; D008558:Melphalan; D003078:Colchicine; D011241:Prednisone