Thalidomide therapy for patients with refractory Crohn's disease: An open-label trial

Background & Aims: Inhibition of tumor necrosis factor is a proposed mechan ism for the anti-inflammatory properties of thalidomide. We performed an op en-label trial of thalidomide in refractory Crohn's disease. Methods: Twent y-two patients with refractory Crohn's disease (Crohn's Disease Activity In dex [CDAI] > 200 and/or draining perianal disease) initiated therapy with t halidomide, 200 mg at bedtime (18 patients), or 300 mg at bedtime (4 patien ts). CDAI and goal interval scores (GIS) were assessed at weeks 0, 4, and 1 2, Clinical response for patients with luminal disease was defined as reduc tion in CDAI score of > 150 points and for fistula patients was 2 scores of greater than or equal to 1+ in 3 parameters of the GIS, Clinical remission was defined as a total CDAI < 150 (luminal patients) or greater than or eq ual to 2+ for all parameters of the GIS (fistula patients). Results: Nine p atients with luminal disease and 13 with fistulas (16 male, 6 female) were enrolled. The median CDAI score at entry was 371 (95-468), Sixteen patients completed 4 weeks of treatment (12 clinical responses, 4 clinical remissio ns). All 14 patients completing 12 weeks met criteria for clinical response . Nine achieved clinical remission (3 luminal, 6 fistula patients), The med ian CDAI score was 175 (30-468; P < 0.001 vs. baseline). Conclusions: Thali domide is efficacious in some patients with refractory Crohn's disease.