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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.

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.

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