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

Background & Aims: Inhibition of tumor necrosis factor is a proposed mechanism for the anti-inflammatory properties of thalidomide. We performed an open-label trial of thalidomide in refractory Crohn's disease. Methods: Twenty-two patients with refractory Crohn's disease (Crohn's Disease Activity Index [CDAI] > 200 and/or draining perianal disease) initiated therapy with thalidomide, 200 mg at bedtime (18 patients), or 300 mg at bedtime (4 patients). CDAI and goal interval scores (GIS) were assessed at weeks 0, 4, and 12, Clinical response for patients with luminal disease was defined as reduction 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 equal to 2+ for all parameters of the GIS (fistula patients). Results: Nine patients 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 remissions). All 14 patients completing 12 weeks met criteria for clinical response. Nine achieved clinical remission (3 luminal, 6 fistula patients), The median CDAI score was 175 (30-468; P < 0.001 vs. baseline). Conclusions: Thalidomide is efficacious in some patients with refractory Crohn's disease.