An open-label pilot study of low-dose thalidomide in chronically active, steroid-dependent Crohn's disease

Background & Aims: Thalidomide decreases production of tumor necrosis factor alpha, a proinflammatory cytokine associated with Crohn's disease (CD). In this study the safety, tolerance, and efficacy of low-dose thalidomide were evaluated for treatment of moderate-to-severe, steroid-dependent CD. Methods: Twelve adult male patients with Crohn's Disease Activity Index (CDAI) scores of greater than or equal to 250 and less than or equal to 500 despite greater than or equal to 20 mg prednisone/day were enrolled. The first 6 patients received 50 mg thalidomide every night, the next 6 received 100 mg every night. Steroid doses were stable during the first 4 weeks of treatment, then tapered during weeks 5-12, CDAI was used to assess response. Results: (1) Disease activity improved consistently in all patients during weeks 1-4: 58% response, 17% remission, (2) Clinical improvement was generally maintained despite steroid taper during weeks 5-12, All patients were able to reduce steroids by greater than or equal to 50%, Forty-four percent discontinued steroids entirely. In weeks 5-12, 70% of patients responded and 20% achieved remission, (3) Side effects were mild and mostly transient, with the most common being drowsiness, peripheral neuropathy, edema, and dermatitis, Conclusions: Low-dose thalidomide appears to be well tolerated and effective over a 12-week period. Results of this pilot study support the need for controlled multicenter trials of thalidomide for treatment of CD.