title: Low-dose continuous oral melphalan for the treatment of primary systemic (AL) amyloidosis.
abstract: Median survival of patients with AL amyloidosis with clinically significant cardiac involvement is 5 months when treated with cyclic melphalan and prednisone. We investigated a regimen of continuous oral melphalan as a single agent for patients with cardiac amyloidosis who were unable to tolerate prednisone or more aggressive chemotherapy. Thirty patients with amyloid cardiomyopathy were treated with continuous oral melphalan. Seven of 13 patients, evaluable after 3-4 months of treatment, achieved a partial haematological response and three achieved a complete haematological response; six patients have survived for > 1 year. This regimen appeared to be effective in inducing haematological responses in patients who received total doses of melphalan > 300 mg.
authors.affiliation: Department of Medicine, Section of Hematology/Oncology, Boston Medical Center, Boston University School of Medicine, 88 East Newton Street, Boston, 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: David C
authors.initials: DC
authors.lastname: Seldin
authors.forename: Rodney H
authors.initials: RH
authors.lastname: Falk
authors.forename: John L
authors.initials: JL
authors.lastname: Berk
authors.forename: Laura M
authors.initials: LM
authors.lastname: Dember
authors.forename: Kathleen T
authors.initials: KT
authors.lastname: Finn
authors.forename: Martha
authors.initials: M
authors.lastname: Skinner
mesh_terms: D000368:Aged; D000369:Aged, 80 and over; D000686:Amyloidosis / Q000188:drug therapy* / Q000401:mortality; D018906:Antineoplastic Agents, Alkylating / Q000008:administration & dosage* / Q000627:therapeutic use; D004334:Drug Administration Schedule; D005260:Female; D006331:Heart Diseases / Q000188:drug therapy* / Q000401:mortality; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000627:therapeutic use; D008875:Middle Aged; D016019:Survival Analysis
chemical_list: D018906:Antineoplastic Agents, Alkylating; D008558:Melphalan