P441 Thalidomide induces clinical remission and mucosal healing in adults with active Crohn's disease: a prospective open-label study

Abstract

Background: Thalidomide is effective in inducing clinical remission and longer-term maintenance of remission in children and adolescents with refractory Crohn's disease (CD). However, little is known about efficacy and safety of thalidomide therapy for adult CD patients—particularly with respect to mucosal healing. Methods: We conducted a prospective observational open-label study (ClinicalTrials.gov, NCT02501291) between January 2013 and April 2015. Forty-seven adult patients with active CD who were resistant or intolerant to corticosteroids and/or immunomodulators or biologics received 50–100mg of thalidomide daily. Primary outcome was clinical remission evaluated at week 8. Endoscopic assessment was performed at week 24 and defined as endoscopic response (decrease in CDEIS score >5 points from baseline CDEIS of 6 or more), endoscopic remission (CDEIS score <3) and mucosal healing (no ulceration). Results: The clinical remission rate was 14.9% and 23.4% at week 4 and 8, but increased to 46.8% at week 12 and 53.2% at week 24 out of all 47 included patients (intention-to-treat analysis). Out of the 47 patients, 32 consented and underwent ileocolonoscopy at week 24. The rate of endoscopic response and endoscopic remission were 68.4% and 43.8%. Mucosal healing (no ulceration) was achieved in 28.1% of patients. Adverse events occurred in 27 (57.4%, 27/47) patients but necessitated therapy discontinuation in only 5/47 (10.7%) of patients. Conclusions: Low-dose thalidomide was effective and tolerated for inducing and maintaining clinical remission in adult patients with active CD, but the optimal time frame for thalidomide to induce clinical remission may be longer than previously appreciated and is probably optimal at 12 weeks. Mucosal healing could be reasonably achievable with thalidomide.