Phase II trial of α‐tocopherol (vitamin E) in the treatment of primary systemic amyloidosis

Morie A. Gertz, Robert A. Kyle

Research output: Contribution to journalArticlepeer-review

17 Scopus citations


Primary systemic amyloidosis (AL) is a rare disorder characterized by deposition of a monoclonal immunoglobulin light chain or fragment thereof, resulting in dysfunction of the heart, kidney, liver, or nerves. Despite the use of melphalan, prednisone, colchicine, and dimethyl sulfoxide (DMSO), no improvement in median survival has been reported in prospective randomized studies. We undertook a study of α‐tocopherol acetate (vitamin E) in the treatment of 16 patients with AL because of its reported benefits in animal models of senile and secondary amyloidosis as well as reported benefit in the treatment of secondary amyloidosis in humans. None of the patients showed any objective regression of their disease. The median survival of the entire group was 19.4 months. This survival is not superior to that reported with other agents used in this disease. We conclude that α‐tocopherol is not a valuable agent in the treatment of AL.

Original languageEnglish (US)
Pages (from-to)55-58
Number of pages4
JournalAmerican journal of hematology
Issue number1
StatePublished - May 1990


  • cardiomyopathy
  • nephrotic syndrome
  • primary amyloidosis

ASJC Scopus subject areas

  • Hematology


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