Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide

Giovanni Palladini (1) , Paola Russo (1, 2) , Francesca Lavatelli (1, 2) , Mario Nuvolone (1, 2) , Riccardo Albertini (3) , Tiziana Bosoni (3) , Vittorio Perfetti (1, 2) , Laura Obici (1) , Stefano Perlini (2) , Remigio Moratti (3) , Giampaolo Merlini (1)

1 Biotechnology Research Laboratories, Center for Amyloidosis, Department of Biochemistry
2 Department of Internal Medicine
3 Clinical Chemistry Laboratory

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.

Mots clés: Amyloidosis; Melphalan; Dexamethasone; Thalidomide; Response

Giovanni Palladini, Paola Russo, Francesca Lavatelli, Mario Nuvolone, Riccardo Albertini, et al.. Treatment of patients with advanced cardiac AL amyloidosis with oral melphalan, dexamethasone, and thalidomide. Annals of Hematology, 2008, 88 (4), pp.347-350. ⟨10.1007/s00277-008-0600-y⟩. ⟨hal-00478494⟩