PRIMARY SYSTEMIC AMYLOIDOSIS - COMPARISON OF MELPHALAN AND PREDNISONE VERSUS PLACEBO

  • 1 January 1978
    • journal article
    • research article
    • Vol. 52  (4) , 818-827
Abstract
Satisfactory treatment for primary amyloidosis does not exist. Because the amyloid fibrils consist of a portion of a monoclonal L chain, it appeared reasonable to treat amyloidosis with alkylating agents that are effective against the plasma cells that synthesize monoclonal L chains. Patients (55) with primary systemic amyloidosis were randomized (double blind) to melphalan-prednisone or placebo. In comparison with the placebo group, patients given melphalan-prednisone continued on treatment for a longer time and to receive larger doses before the code was broken. Among this group, the nephrotic syndrome disappeared in 2 patients and urinary excretion of protein was reduced by more than 50% in 8 others. Of 13 patients who received melphalan-prednisone for more than 12 mo., 6 improved, 3 were stable, and 4 had progression of disease. Survival did not differ significantly between the groups.

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