Thalidomide in the treatment of the mucocutaneous lesions of the Behçet syndrome. A randomized, double-blind, placebo-controlled trial.

ABSTRACT

BACKGROUND: Recurrent oral and genital ulcers are the most frequent problem in the management of the Behçet syndrome. Uncontrolled experience suggests that thalidomide may help prevent recurrences of these ulcers. OBJECTIVE: To determine the efficacy of two thalidomide dosages in the treatment of mucocutaneous lesions of the Behçet syndrome. DESIGN: Randomized, double-blind, placebo-controlled trial. SETTING: Specialist outpatient clinic for the Behçet syndrome in Turkey. PATIENTS 96 male patients with the Behçet syndrome who primarily had mucocutaneous lesions without major organ involvement. INTERVENTION Thalidomide, 100 mg/d or 300 mg/d, or placebo for 24 weeks. MEASUREMENTS Sustained absence of any oral and genital ulceration during treatment (complete response) and changes in the number of mucocutaneous lesions. An additional evaluation was done 4 weeks after treatment ended. RESULTS: A complete response occurred in 2 of the 32 patients (6% [95% CI, 0.8% to 20.8%]) receiving thalidomide, 100 mg/d; in 5 of the 31 patients (16% [CI, 5.5% to 33.7%]) receiving thalidomide, 300 mg/d; and in none of the 32 patients (0% [CI, 0% to 10.9%]) receiving placebo (P = 0.031). The suppressive effect of thalidomide with either dosage was evident at 4 weeks for oral ulcers (P < 0.001) and at 8 weeks for genital ulcers (P < 0.001) and follicular lesions (P = 0.008). This effect persisted during treatment but diminished rapidly after treatment was discontinued. Both thalidomide dosages led to significant increases in the number of erythema nodosum lesions during the first 8 weeks of treatment (P = 0.03). Polyneuropathy developed in 4 patients (1 in the 100-mg/d group and 3 in the 300-mg/d group); in 3 of these patients, the condition was diagnosed after the trial had ended. CONCLUSIONS: Thalidomide is effective for treating the oral and genital ulcers and follicular lesions of the Behçet syndrome. A dosage of 100 mg/d is as effective as a dosage of 300 mg/day.