Association of melphalan and high-dose dexamethasone is effective and well tolerated in patients with AL (primary) amyloidosis who are ineligible for stem cell transplantation

Giovanni Palladini, Vittorio Perfetti, Laura Obici, Riccardo Caccialanza, Alessandra Semino, Fausto Adami, Giobatta Cavallero, Roberto Rustichelli, Giovambattista Virga, Giampaolo Merlini

Research output: Contribution to journalArticle

Abstract

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 patients with AL. We evaluated the combination of oral melphalan and high-dose dexamethasone (M-Dex) in 46 patients with AL ineligible for ASCT. Thirty-one (67%) achieved a hematologic response and 15 (33%) a complete remission. In 22 (48%) of the responsive patients functional improvement of the organs involved was observed. Five patients (11%) experienced severe adverse events, 3 required hospitalization, and 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.

Original languageEnglish
Pages (from-to)2936-2938
Number of pages3
JournalBlood
Volume103
Issue number8
DOIs
Publication statusPublished - Apr 15 2004

ASJC Scopus subject areas

  • Hematology

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