Studio randomizzato controllato vs placebo su efficacia e sicurezza della Talidomide nelle mici in pediatria: disegno, conduzione, risultati preliminari

Abstract

Thalidomide is a small molecule with anti-TNF properties, recently emerged for the treatment of inflammatory bowel diseases (IBD) in adults and children. This is a report of the preliminary results of a multicenter double-blind vs placebo controlled trial to evaluate thalidomide efficacy and safety in inducing remission in untreatable patient with IBD aged 2-20 years. Patients non-responder to placebo are further treated with thalidomide open-label, and followed up for 52 weeks. Overall, thalidomide induced remission in 15/27 (55%) patients with CD and 7/10 (66%) patients with UC, compared to only one children in the placebo group (p=0.01, p=0.05). All 15 patients with CD and 6/7 pts with UC reaching remission with thalidomide are maintaining remission, with a mean follow up respectively of 58 weeks (range 12 to 104 weeks) and 52 weeks (range 26-78 weeks). Clinical and electromiographical sign of neuropathy occurred in 5/36 (13%), while persistent isolated electromiographical signs occurred in 3 more patient. There was one case of arterial cerebral thrombosis in a child with severe disease, for which thalidomide role is under discussion.