title: Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide.
abstract: Patients with primary (AL) amyloidosis and heart failure have a very poor prognosis and cannot tolerate aggressive therapy, such as autologous stem cell transplantation and high-dose dexamethasone-based regimens. We prospectively treated 22 patients with advanced cardiac amyloidosis combining oral melphalan, thalidomide, and reduced intensity dexamethasone (MTD). Six patients died due to cardiac amyloidosis before completing cycle 3. Early death was associated with reduced ejection fraction. Eight patients achieved a hematological response and four achieved a durable improvement of cardiac dysfunction. Treatment with MTD is feasible in patients with advanced cardiac AL amyloidosis and effective in subjects with preserved systolic function.
authors.affiliation: Biotechnology Research Laboratories, Center for Amyloidosis, Department of Biochemistry, Fondazione Istituto di Ricovero e Cura a Carattere Scientifico San Matteo and University of Pavia, Piazzale Golgi, 19, 27100 Pavia, Italy. g.palladini@smatteo.pv.it
authors.forename: Giovanni
authors.initials: G
authors.lastname: Palladini
authors.forename: Paola
authors.initials: P
authors.lastname: Russo
authors.forename: Francesca
authors.initials: F
authors.lastname: Lavatelli
authors.forename: Mario
authors.initials: M
authors.lastname: Nuvolone
authors.forename: Riccardo
authors.initials: R
authors.lastname: Albertini
authors.forename: Tiziana
authors.initials: T
authors.lastname: Bosoni
authors.forename: Vittorio
authors.initials: V
authors.lastname: Perfetti
authors.forename: Laura
authors.initials: L
authors.lastname: Obici
authors.forename: Stefano
authors.initials: S
authors.lastname: Perlini
authors.forename: Remigio
authors.initials: R
authors.lastname: Moratti
authors.forename: Giampaolo
authors.initials: G
authors.lastname: Merlini
mesh_terms: D000368:Aged; D000686:Amyloidosis / Q000188:drug therapy*; D003907:Dexamethasone / Q000008:administration & dosage; D004359:Drug Therapy, Combination; D005260:Female; D006331:Heart Diseases / Q000188:drug therapy*; D006333:Heart Failure / Q000188:drug therapy; D006801:Humans; D008297:Male; D008558:Melphalan / Q000008:administration & dosage; D008875:Middle Aged; D013318:Stroke Volume; D015996:Survival Rate; D013792:Thalidomide; D016896:Treatment Outcome
chemical_list: D013792:Thalidomide; D003907:Dexamethasone; D008558:Melphalan