Question: What is the most current and efficacious treatment of amyloidosis?

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Unique Identifier: 97244527

Authors: Kyle RA. Gertz MA. Greipp PR. Witzig TE. Lust JA. Lacy MQ. Therneau TM.

Institution: Division of Hematology and Internal Medicine, Mayo Clinic and Mayo Foundation, Rochester, MN 55905, USA.

Title: A trial of three regimens for primary amyloidosis: colchicine alone, melphalan and prednisone, and melphalan, prednisone, and colchicine

Source: New England Journal of Medicine. 336(17):1202-7, 1997 Apr 24. [see comments]

Abstract: BACKGROUND: Primary systemic amyloidosis is an uncommon disease characterized by the accumulation in vital organs of a fibrillar protein consisting of monoclonal light chains. METHODS: We treated 220 patients with biopsy-proved amyloidosis. The patients were randomly assigned to receive colchicine (72 patients), melphalan and prednisone (77), or melphalan, prednisone, and colchicine (71). They were stratified according to their chief clinical manifestations: renal disease (105 patients), cardiac involvement (46), peripheral neuropathy (19), or other (50). RESULTS: The median duration of survival after randomization was 8.5 months in the colchicine group, 18 months in the group assigned to melphalan and prednisone, and 17 months in the group assigned to melphalan, prednisone, and colchicine (P<0.001). Among patients who had a reduction in serum or urine monoclonal protein at 12 months, the overall length of survival was 50 months, whereas among those without a reduction at 12 months, the overall length of survival was 36 months (P=0.03). Thirty-four patients (15 percent) survived for five years or longer. CONCLUSIONS: Therapy with melphalan and prednisone results in objective responses and prolonged survival as compared with colchicine in patients with primary amyloidosis.Sent Tue Jun 16 17:51:06 1998 -0400

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Unique Identifier: 96202846

Authors: Skinner M. Anderson J. Simms R. Falk R. Wang M. Libbey C. Jones LA. Cohen AS.

Institution: Arthritis Center, Thorndike Memorial Laboratories, Boston City Hospital, Massachusetts, USA.

Title: Treatment of 100 patients with primary amyloidosis: a randomized trial of melphalan, prednisone, and colchicine versus colchicine only.

Source: American Journal of Medicine. 100(3):290-8, 1996 Mar.

Abstract: PURPOSE: A clinical trial designed to test whether treatment with melphalan, prednisone, and colchicine (MPC) is superior to colchicine (C) alone was performed in patients with primary amyloidosis (AL), a nonmalignant plasma cell dyscrasia. PATIENTS AND METHODS: Patients were randomized to MPC or C with stratification according to sex, time from diagnosis to study entry (ie, less than 3 months or 3 to 12 months), and dominant organ system involvement (ie, cardiac, renal, neurologic, or others). Data were gathered monthly from patients, quarterly from physicians, and annually in the Clinical Research Center. One hundred consecutive patients with AL amyloidosis admitted between 1987 and 1992 who 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 prednisone every 6 weeks for 1 year as well as colchicine. RESULTS: The principal 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 group 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 differences favoring MPC treatment only in patients whose major system manifestations were neurologic (P = 0.037) or other (P = 0.007). Multivariate analysis showed a strongly significant treatment effect (P = 0.003) and improved survival associated with not having cardiac or gastrointestinal involvement. CONCLUSIONS: MPC was advantageous for patients whose 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 dialysis exists for their organ failure.

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Unique Identifier: 95184021

Authors: Merlini G.

Institution: Institute of Clinical Medicine II, University Hospital S. Matteo, University of Pavia, Italy.

Title: Treatment of primary amyloidosis. [Review] [262 refs]

Source: Seminars in Hematology. 32(1):60-79, 1995 Jan.

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