Amyloidosis: prognosis and treatment

Semin Arthritis Rheum. 1994 Oct;24(2):124-38. doi: 10.1016/s0049-0172(05)80006-x.

Abstract

The objective of this study was to review (1) the factors that have been linked to prediction of clinical outcome and survival in amyloidosis and (2) the available studies on the therapy for localized and systemic forms of amyloidosis. We made a retrospective review of the relevant literature on treatment and prognosis in localized and systemic amyloidosis dating back to 1975. The most important prognostic factors in amyloidosis are the presence of congestive heart failure, beta 2-microglobulin, and whether peripheral neuropathy dominates the presentation. The presence of a monoclonal light chain in serum or urine, multiple myeloma, and hepatic involvement are also important adverse factors. Colchicine is beneficial in treating familial Mediterranean fever and may play a role in managing secondary amyloidosis in inflammatory bowel disease. Chlorambucil is particularly useful in juvenile rheumatoid arthritis with amyloidosis. Dimethyl sulfoxide provides benefit in bladder and lichen amyloidosis. A trial of alkylating agent-based chemotherapy is reasonable in symptomatic primary systemic amyloidosis. Advances have been made in the treatment of amyloidosis and include chemotherapy, dialysis, transplantation, and improved supportive care. Definite disease regressions with long-term survival (> 10 years) are seen. Unfortunately, alternatives still need to be developed: Of 859 patients with primary systemic amyloidosis seen at the Mayo Clinic from 1982 to 1992, the median survival was 2.1 years.

Publication types

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

MeSH terms

  • Amyloidosis* / mortality
  • Amyloidosis* / physiopathology
  • Amyloidosis* / therapy
  • Cardiomyopathy, Dilated / etiology
  • Cardiomyopathy, Dilated / therapy
  • Humans
  • Nephrotic Syndrome / etiology
  • Nephrotic Syndrome / therapy
  • Prognosis
  • Survival Rate