Primary Systemic Amyloidosis (AL): Comparison of Melphalan-Prednisone vs. Colchicine Treatment in 101 Cases
One hundred one patients with primary amyloidosis (AL) were stratified in four groups on the basis of their major clinical features: 1) Nephrotic syndrome or renal insufficiency; 2) congestive heart failure; 3) peripheral neuropathy; and 4) other. The patients were randomized to 1) melphalan (0.15 mg/kg daily) and prednisone (0.8 mg/kg daily) for 7 days every 6 weeks, or 2) colchicine (0.6 mg twice a day). Forty-nine patients were randomized to melphalan-prednisone (M-P) therapy, while 52 were to receive colchicine (C). No important differences were found between the two groups in the history or the initial physical examination and laboratory findings. If there was progressive disease or lack of benefit from therapy, either M-P or C was added to the original therapeutic regimen. Twenty-one of the 40 patients with the nephrotic syndrome who received M-P had >50% reduction in urinary protein. Four patients died of acute nonlymphocytic leukemia. The median survival was 25 months for the M-P group and 18 months for the C group (P = 0.23). When survival was calculated from the onset of randomization to death or progression of disease, the median survival was 16 months for the M-P group and 6 months for the C group (P = 0.0001).
KeywordsCongestive Heart Failure Nephrotic Syndrome Peripheral Neuropathy Familial Mediterranean Fever Pernicious Anemia
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