Thalidomide for CGD-related inflammatory bowel disease: A randomized, double-blind trial.

Chronic granulomatous disease-related inflammatory bowel disease (CGD-IBD) requires effective and safe therapies, as conventional immunosuppressants increase infection risk. Thalidomide, with anti-inflammatory and immunomodulatory effects, may represent a therapeutic option. This study was conducted to explore the preliminary efficacy and safety of thalidomide for CGD-IBD in a multicenter, randomized, double-blind, placebo-controlled, parallel-group phase II trial. Patients were randomized to thalidomide or placebo for a 12-wk blinded phase, followed by a 12-wk extension thalidomide phase. The primary endpoint was achievement of remission or a ≥20-point reduction in the Pediatric Ulcerative Colitis Activity Index. Eight male patients were randomized and analyzed. The primary endpoint was achieved by 1/3 of thalidomide patients versus 0/5 in the placebo group during the blinded phase, and by 5/8 in the extension phase. Secondary endpoints and exploratory endoscopy showed improvements. CGD-related infection rates were comparable before and after treatment. Thalidomide met the prespecified efficacy criterion with an acceptable safety profile in CGD-IBD, suggesting a promising therapeutic option that warrants further clinical studies.