TREATMENT OF 100 PATIENTS WITH PRIMARY AMYLOIDOSIS - A RANDOMIZED TRIAL OF MELPHALAN, PREDNISONE AND COLCHICINE VERSUS COLCHICINE ONLY

PURPOSE: A clinical trial designed to test whether treatment with melp halan, prednisone, and colchicine (MPC) is superior to colchicine (C) alone was performed in patients with primary amyloidosis (AL), a nonma lignant plasma cell dyscrasia. PATIENTS AND METHODS: Patients were ran domized to MPC or C with stratification according to sex, time from di agnosis to study entry (ie, less than 3 months or 3 to 12 months), and dominant organ system involvement (ie, cardiac, renal, neurologic, or other). Data were gathered monthly from patients, quarterly from phys icians, and annually in the Clinical Research Center. One hundred cons ecutive patients with AL amyloidosis admitted between 1987 and 1992 wh o met eligibility requirements were treated and followed for a minimum of 18 months. Fifty patients (group A) received daily oral colchicine and 50 patients (group B) received cycles of oral melphalan and predn isone every 6 weeks for 1 year as well as colchicine. RESULTS: The pri ncipal outcome measure was median survival, which was compared in the two treatment groups and in the subgroups. The overall survival of all patients from study entry was 8.4 months. Comparing group A (C) to gr oup B (MPC), the survival was 6.7 months versus 12.2 months (P = 0.087 ). Both treatment groups had poor survival for patients in the cardiac subgroup, longest survival in the renal group, and significant differ ences favoring MPC treatment only in patients whose major system manif estations were neurologic (P = 0.037) or other (P = 0.007). Multivaria te analysis showed a strongly significant treatment effect (P = 0.003) and improved survival associated with not having cardiac or gastroint estinal involvement. CONCLUSIONS: MPC was advantageous for patients wh ose major manifestations of amyloid disease were other than cardiac or renal. Better survival regardless of treatment was noted in patients for whom a satisfactory supportive treatment such as transplant or dia lysis exists for their organ failure.