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Previous Volume 336:1202-1207 April 24, 1997 Number 17 Next

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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.

Source Information

From the Division of Hematology and Internal Medicine (R.A.K., M.A.G., P.R.G., T.E.W., J.A.L., M.Q.L.) and the Section of Biostatistics (T.M.T.), Mayo Clinic and Mayo Foundation, Rochester, Minn.

Address reprint requests to Dr. Kyle at the Mayo Clinic, 200 First St. SW, Rochester, MN 55905.

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• Palladini, G., Campana, C., Klersy, C., Balduini, A., Vadacca, G., Perfetti, V., Perlini, S., Obici, L., Ascari, E., d'Eril, G. M., Moratti, R., Merlini, G. (2003). Serum N-Terminal Pro-Brain Natriuretic Peptide Is a Sensitive Marker of Myocardial Dysfunction in AL Amyloidosis. Circulation 107: 2440-2445 [Abstract] [Full Text]  
• Abraham, R. S., Geyer, S. M., Price-Troska, T. L., Allmer, C., Kyle, R. A., Gertz, M. A., Fonseca, R. (2003). Immunoglobulin light chain variable (V) region genes influence clinical presentation and outcome in light chain-associated amyloidosis (AL). Blood 101: 3801-3807 [Abstract] [Full Text]  
• Ohno, S., Yoshimoto, M., Honda, S., Miyachi, S., Ishida, T., Itoh, F., Endo, T., Chiba, S., Imai, K. (2002). The Antisense Approach in Amyloid Light Chain Amyloidosis: Identification of Monoclonal Ig and Inhibition of Its Production by Antisense Oligonucleotides in In Vitro and In Vivo Models. J. Immunol. 169: 4039-4045 [Abstract] [Full Text]  
• Perfetti, V., Casarini, S., Palladini, G., Vignarelli, M. C., Klersy, C., Diegoli, M., Ascari, E., Merlini, G. (2002). Analysis of Vlambda -Jlambda expression in plasma cells from primary (AL) amyloidosis and normal bone marrow identifies 3r (lambda III) as a new amyloid-associated germline gene segment. Blood 100: 948-953 [Abstract] [Full Text]  
• Lachmann, H. J., Booth, D. R., Booth, S. E., Bybee, A., Gilbertson, J. A., Gillmore, J. D., Pepys, M. B., Hawkins, P. N. (2002). Misdiagnosis of Hereditary Amyloidosis as AL (Primary) Amyloidosis. NEJM 346: 1786-1791 [Abstract] [Full Text]  
• Saraiva, M. J. M. (2002). Sporadic Cases of Hereditary Systemic Amyloidosis. NEJM 346: 1818-1819 [Full Text]  
• Comenzo, R. L., Gertz, M. A. (2002). Autologous stem cell transplantation for primary systemic amyloidosis. Blood 99: 4276-4282 [Abstract] [Full Text]  
• Reyners, A.K.L., Hazenberg, B.P.C., Reitsma, W.D., Smit, A.J. (2002). Heart rate variability as a predictor of mortality in patients with AA and AL amyloidosis. Eur Heart J 23: 157-161 [Abstract] [Full Text]  
• Khan, M F, Falk, R H (2001). Amyloidosis. Postgrad. Med. J. 77: 686-693 [Abstract] [Full Text]  
• Comenzo, R. L., Zhang, Y., Martinez, C., Osman, K., Herrera, G. A. (2001). The tropism of organ involvement in primary systemic amyloidosis: contributions of Ig VL germ line gene use and clonal plasma cell burden. Blood 98: 714-720 [Abstract] [Full Text]  
• Dispenzieri, A., Lacy, M. Q., Kyle, R. A., Therneau, T. M., Larson, D. R., Rajkumar, S. V., Fonseca, R., Greipp, P. R., Witzig, T. E., Lust, J. A., Gertz, M. A. (2001). Eligibility for Hematopoietic Stem-Cell Transplantation for Primary Systemic Amyloidosis Is a Favorable Prognostic Factor for Survival. JCO 19: 3350-3356 [Abstract] [Full Text]  
• RONCO, P. M., ALYANAKIAN, M.-A., MOUGENOT, B., AUCOUTURIER, P. (2001). Light Chain Deposition Disease: A Model of Glomerulosclerosis Defined at the Molecular Level. J. Am. Soc. Nephrol. 12: 1558-1565 [Full Text]  
• Hrncic, R., Wall, J., Wolfenbarger, D. A., Murphy, C. L., Schell, M., Weiss, D. T., Solomon, A. (2000). Antibody-Mediated Resolution of Light Chain-Associated Amyloid Deposits. Am. J. Pathol. 157: 1239-1246 [Abstract] [Full Text]  
• van Woerkom, J. M., van Toorn, D. W. (2000). A domestic fight or something else?. Nephrol Dial Transplant 15: 1253-1254 [Full Text]  
• Iggo, N., Littlewood, T., Winearls, C.G. (2000). Prospects for effective treatment of AL amyloidosis?. QJM 93: 257-260 [Full Text]  
• Gillmore, J. D, Hawkins, P. N (1999). Amyloidosis and the respiratory tract. Thorax 54: 444-451 [Full Text]  
• Kyle, R. A., Gertz, M. A., Greipp, P. R., Witzig, T. E., Lust, J. A., Lacy, M. Q., Therneau, T. M. (1999). Long-Term Survival (10 Years or More) in 30 Patients With Primary Amyloidosis. Blood 93: 1062-1066 [Abstract] [Full Text]  
• Gertz, M. A., Lacy, M. Q., Lust, J. A., Greipp, P. R., Witzig, T. E., Kyle, R. A. (1999). Prospective Randomized Trial of Melphalan and Prednisone Versus Vincristine, Carmustine, Melphalan, Cyclophosphamide, and Prednisone in the Treatment of Primary Systemic Amyloidosis. JCO 17: 262-262 [Abstract] [Full Text]  
• Ruiz-Laiglesia, F. J., Perez-Calvo, J. I., Torrubia-Perez, C. B., Castiella-Muruzabal, T., Morandeira-Garcia, M. J. (1998). Liver Failure and Peripheral Facial Paralysis in a Case of Primary Amyloidosis. Arch Intern Med 158: 2066-2067 [Full Text]  
• Ropper, A. H., Gorson, K. C. (1998). Neuropathies Associated with Paraproteinemia. NEJM 338: 1601-1607 [Full Text]  
• Comenzo, R. L., Vosburgh, E., Falk, R. H., Sanchorawala, V., Reisinger, J., Dubrey, S., Dember, L. M., Berk, J. L., Akpek, G., LaValley, M., O'Hara, C., Arkin, C. F., Wright, D. G., Skinner, M. (1998). Dose-Intensive Melphalan With Blood Stem-Cell Support for the Treatment of AL (Amyloid Light-Chain) Amyloidosis: Survival and Responses in 25 Patients. Blood 91: 3662-3670 [Abstract] [Full Text]  
• Falk, R. H., Comenzo, R. L., Skinner, M. (1997). The Systemic Amyloidoses. NEJM 337: 898-909 [Full Text]  
• (1997). TREATMENT OF PRIMARY AMYLOIDOSIS. JWatch General 1997: 8-8 [Full Text]