THALIDOMIDE IN THE TREATMENT OF THE MUCOCUTANEOUS LESIONS OF THE BEHCET-SYNDROME - A RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL

Background: Recurrent oral and genital ulcers are the most frequent pr oblem in the management of the Behcet syndrome. Uncontrolled experienc e suggests that thalidomide may help prevent recurrences of these ulce rs. Objective: To determine the efficacy of two thalidomide dosages in the treatment of mucocutaneous lesions of the Behcet syndrome. Design : Randomized, double-blind, placebo-controlled trial. Setting: Special ist outpatient clinic for the Behcet syndrome in Turkey. Patients: 96 male patients with the Behcet syndrome who primarily had mucocutaneous lesions without major organ involvement. Intervention: Thalidomide, 1 00 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 addit ional evaluation was done 4 weeks after treatment ended. Results: A co mplete 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 t he 32 patients (0% [CI, 0% to 10.9%]) receiving placebo (P = 0.031). T he 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 discontin ued. Both thalidomide dosages led to significant increases in the numb er 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 g roup and 3 in the 300-mg/d group); in 3 of these patients, the conditi on was diagnosed after the trial had ended. Conclusions: Thalidomide i s effective for treating the oral and genital ulcers and follicular le sions of the Behcet syndrome. A dosage of 100 mg/d is as effective as a dosage of 300 mg/day.