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Evaluating Therapeutic Interventions in the SHIP-deficient Mouse Model of Crohn Disease-like Ileitis and Fibrosis
Published on: October 14, 2025
Thalidomide for CGD-related inflammatory bowel disease: A randomized, double-blind trial
Toshinao Kawai1, Mayumi Sako2, Katsuhiro Arai3
1Division of Immunology, National Center for Child Health and Development, Tokyo, Japan.
Journal of Human Immunity
|June 25, 2026
Summary
Thalidomide shows promise for treating chronic granulomatous disease-related inflammatory bowel disease (CGD-IBD). This therapy demonstrated efficacy and an acceptable safety profile, suggesting it as a potential treatment option for CGD-IBD patients.
Area of Science:
- Immunology
- Gastroenterology
- Pharmacology
Background:
- Chronic granulomatous disease-related inflammatory bowel disease (CGD-IBD) presents a therapeutic challenge due to the risks associated with conventional immunosuppressants.
- Thalidomide possesses anti-inflammatory and immunomodulatory properties, indicating its potential utility in managing CGD-IBD.
Purpose of the Study:
- To evaluate the preliminary efficacy and safety of thalidomide in patients with CGD-IBD.
- To assess thalidomide's impact on disease activity and infection rates in this patient population.
Main Methods:
- A multicenter, randomized, double-blind, placebo-controlled, parallel-group phase II trial was conducted.
- Patients received either thalidomide or placebo for 12 weeks, followed by a 12-week open-label thalidomide extension phase.
- The primary endpoint was remission or a significant reduction in the Pediatric Ulcerative Colitis Activity Index (PUCAI).
Main Results:
- The primary efficacy endpoint was met by 33.3% of thalidomide recipients versus 0% in the placebo group during the blinded phase.
- In the extension phase, 62.5% of patients achieved the primary endpoint.
- Improvements were observed in secondary endpoints and endoscopic findings, with comparable CGD-related infection rates before and after treatment.
Conclusions:
- Thalidomide demonstrated preliminary efficacy and an acceptable safety profile for treating CGD-IBD.
- The findings support thalidomide as a promising therapeutic option for CGD-IBD, warranting further investigation in larger clinical studies.
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