Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide.
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.
Palladini
Giovanni
G
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
Russo
Paola
P
Lavatelli
Francesca
F
Nuvolone
Mario
M
Albertini
Riccardo
R
Bosoni
Tiziana
T
Perfetti
Vittorio
V
Obici
Laura
L
Perlini
Stefano
S
Moratti
Remigio
R
Merlini
Giampaolo
G
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
D013792:Thalidomide; D003907:Dexamethasone; D008558:Melphalan