Thalidomide is active in patients with refractory myeloma. Seventeen patien
ts (nine men/eight women, median age 73 years) with multiple myeloma (MM) w
ere treated with thalidomide. Fifteen patients had refractory disease and t
wo untested relapse. The median dose of thalidomide was 500 mg (range, 200-
800 mg). Nine of the 17 patients (53%) responded. The response rate was sig
nificantly higher in patients with no extramedullary disease than in those
with soft tissue masses (75% CI: 43-95% versus 0%: P = 001)). Of note, no d
ecrease in the size of soft tissue plasmacytomas was observed in all the fi
ve patients who had extramedullary involvement. This data suggests that the
mechanism of action and effectiveness of thalidomide might depend on the s
ite of the tumour cells.