Thalidomide appears to be effective in some cases in about 30% of patients but only phase I and II studies have been performed and much remains unknown. Such data show that the combination of dexamethasone and thalidomide at full dosage (400 mg/d) in AL patients whose disease is refractory to or has relapsed after previous treatment provides good response rates (around 70% hematologic response, 23-30% complete remission, 30% organ improvement). Only 35% of patients could reach and tolerate the target dose because of the high incidence of adverse reactions to thalidomide. Organ improvement was more frequent in patients with renal involvement, whereas cardiac amyloidosis did not generally improve. Guidelines suggest that some patients, if they can tolerate thalidomide, can obtain some benefit from it.
Giovanni Palladini, Vittorio Perfetti, Stefano Perlini, Laura Obici, Francesca Lavatelli, Riccardo Caccialanza, Rosangela Invernizzi, Benedetto Comotti, and Giampaolo Merlini
The combination of thalidomide and intermediate-dose dexamethasone is an effective but toxic treatment for patients with primary amyloidosis (AL)
Blood 105: 2949-2951
Guidelines on the diagnosis and management of AL amyloidosis. Br J Haematol 2004; 125:681–700.