title: 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.
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.
authors.affiliation: Amyloid Center Biotechnology Research Laboratories, Department of Biochemistry, University Hospital, Istituto di Ricovero e Cura a Carattere Scientifico-Policlinico San Matteo, Pavia, Italy.
authors.forename: Giovanni
authors.initials: G
authors.lastname: Palladini
authors.forename: Vittorio
authors.initials: V
authors.lastname: Perfetti
authors.forename: Laura
authors.initials: L
authors.lastname: Obici
authors.forename: Riccardo
authors.initials: R
authors.lastname: Caccialanza
authors.forename: Alessandra
authors.initials: A
authors.lastname: Semino
authors.forename: Fausto
authors.initials: F
authors.lastname: Adami
authors.forename: Giobatta
authors.initials: G
authors.lastname: Cavallero
authors.forename: Roberto
authors.initials: R
authors.lastname: Rustichelli
authors.forename: Giovambattista
authors.initials: G
authors.lastname: Virga
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Merlini
mesh_terms: D000328:Adult; D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy* / Q000628:therapy; D000075202:Contraindications; D003907:Dexamethasone / Q000008:administration & dosage* / Q000009:adverse effects; D004359:Drug Therapy, Combination; D004361:Drug Tolerance; D005260:Female; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage* / Q000009:adverse effects; D008875:Middle Aged; D033581:Stem Cell Transplantation
chemical_list: D003907:Dexamethasone; D008558:Melphalan