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Blood, 15 April 2004, Vol. 103, No. 8, pp. 2936-2938. Prepublished online as a Blood First Edition Paper on December 18, 2003; DOI 10.1182/blood-2003-08-2788. This Article Full Text (PDF) All Versions of this Article: 2003-08-2788v1 103/8/2936    most recent Alert me when this article is cited Alert me if a correction is posted Services Email this article to a friend Similar articles in this journal Similar articles in PubMed Alert me to new issues of the journal Download to citation manager Rights and Permissions Citing Articles Citing Articles via HighWire Citing Articles via CrossRef Citing Articles via Google Scholar Google Scholar Articles by Palladini, G. Articles by Merlini, G. Search for Related Content PubMed PubMed Citation Articles by Palladini, G. Articles by Merlini, G. Social Bookmarking                   What's this? Submitted August 13, 2003 Accepted December 4, 2003 The association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis ineligible for stem cell transplantation Giovanni Palladini, Vittorio Perfetti, Laura Obici, Riccardo Caccialanza, Alessandra Semino, Fausto Adami, Giobatta Cavallero, Roberto Rustichelli, Giovambattista Virga, and Giampaolo Merlini* Amyloid Center, Biotechnology Research Laboratories, Department of Biochemistry, IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy Department of Internal Medicine and Medical Oncology, IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy Department of Applied Health Sciences, Human Nutrition Unit, IRCCS Policlinico San Matteo and University of Pavia, Pavia, Italy Department of Clinical and Experimental Medicine, Padua University School of Medicine, Padova, Italy Internal Medicine Unit, Hospital 'Santa Croce e Carle', Cuneo, Italy Nephrology and Dialysis Unit, 'Arcispedale Santa Maria Nuova', Reggio Emilia, Italy Nephrology and Dialysis Unit, Provincial Hospital, Camposampiero, Padova, Italy * Corresponding author; email: gmerlini{at}smatteo.pv.it. The most efficient therapeutic approach for immunoglobulin light chain amyloidosis (AL) is autologous stem cell transplantation (ASCT), however, the toxicity of ASCT limits its feasibility to a minority of patients. Patients ineligible for ASCT are usually treated with standard oral melphalan and prednisone, but the response rate to this regimen is unsatisfactory and time to response is long. High-dose dexamethasone provides a rapid response time in AL patients. We evaluated the combination of oral melphalan and high-dose dexamethasone (M-Dex) in 46 AL patients ineligible for ASCT. Thirty-one (67%) achieved a hematologic response and 15 (33%) a complete remission. In 22 of the responsive patients (48%) functional improvement of the organs involved was observed. Five patients (11%) experienced severe adverse events, 3 required hospitalization, no treatment-related deaths were observed. M-Dex represents a feasible and effective therapeutic option for patients with advanced AL who are ineligible for ASCT. CiteULike    Connotea    Del.icio.us    Digg    Reddit    Technorati    What's this? This article has been cited by other articles: R. L. Comenzo How I treat amyloidosis Blood, October 8, 2009; 114(15): 3147 - 3157. [Abstract] [Full Text] [PDF] P. Ronco and P. Aucouturier Renal involvement in AL amyloidosis: the facts, the promise and the hope Nephrol. Dial. Transplant., October 1, 2009; 24(10): 2967 - 2969. [Full Text] [PDF] A. V. Kristen, F.-U. Sack, S. O. Schonland, U. Hegenbart, B. M. Helmke, A. Koch, P. A. Schnabel, C. Rocken, S. Hardt, A. Remppis, et al. Staged heart transplantation and chemotherapy as a treatment option in patients with severe cardiac light-chain amyloidosis Eur J Heart Fail, October 1, 2009; 11(10): 1014 - 1020. [Abstract] [Full Text] [PDF] D. E. Reece, V. Sanchorawala, U. Hegenbart, G. Merlini, G. Palladini, J.-P. Fermand, R. A. Vescio, X. 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