Low-dose continuous oral melphalan for the treatment of primary systemic (AL) amyloidosis

Br J Haematol. 2002 Jun;117(4):886-9. doi: 10.1046/j.1365-2141.2002.03541.x.

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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Aged
  • Aged, 80 and over
  • Amyloidosis / drug therapy*
  • Amyloidosis / mortality
  • Antineoplastic Agents, Alkylating / administration & dosage*
  • Antineoplastic Agents, Alkylating / therapeutic use
  • Drug Administration Schedule
  • Female
  • Heart Diseases / drug therapy*
  • Heart Diseases / mortality
  • Humans
  • Male
  • Melphalan / administration & dosage*
  • Melphalan / therapeutic use
  • Middle Aged
  • Survival Analysis

Substances

  • Antineoplastic Agents, Alkylating
  • Melphalan