%0 Journal Article %J Inflamm Bowel Dis %D 2015 %T Effect of Thalidomide on Clinical Remission in Children and Adolescents with Ulcerative Colitis Refractory to Other Immunosuppressives: Pilot Randomized Clinical Trial. %A Lazzerini, Marzia %A Martelossi, Stefano %A Magazzù, Giuseppe %A Pellegrino, Salvatore %A Lucanto, Maria Cristina %A Barabino, Arrigo %A Calvi, Angela %A Arrigo, Serena %A Lionetti, Paolo %A Lorusso, Monica %A Mangiantini, Francesca %A Fontana, Massimo %A Zuin, Giovanna %A Palla, Gabriella %A Maggiore, Giuseppe %A Bramuzzo, Matteo %A Pellegrin, Maria Chiara %A Maschio, Massimo %A Villanacci, Vincenzo %A Manenti, Stefania %A Decorti, Giuliana %A De Iudicibus, Sara %A Paparazzo, Rossella %A Montico, Marcella %A Ventura, Alessandro %X

BACKGROUND: In a randomized controlled trial, thalidomide has shown to be effective in refractory Crohn's disease in children. This pilot study aimed at evaluating thalidomide in refractory pediatric ulcerative colitis (UC).

METHODS: Double-blind, placebo-controlled randomized clinical trial on thalidomide 1.5 to 2.5 mg/kg/day in children with active UC despite multiple immunosuppressive treatments. In an open-label extension, nonresponders to placebo received thalidomide for an additional 8 weeks; all responders were followed up for a minimum of 52 weeks.

RESULTS: Twenty-six children with refractory UC were randomized to thalidomide or placebo. Clinical remission at week 8 was achieved by significantly more children treated with thalidomide {10/12 (83.3%) versus 2/11 (18.8%); risk ratio, 4.5 (95% confidence interval [CI], 1.2-16.4); P = 0.005; number needed to treat, 1.5}. Of the nonresponders to placebo who were switched to thalidomide, 8 of 11 (72.7%) subsequently reached remission at week 8 (risk ratio, 4.0 [95% CI, 1.1-14.7]; number needed to treat, 2.45; P = 0.01). Clinical remission in the thalidomide group was 135.0 weeks (95% CI, 32-238), compared with 8.0 weeks (95% CI, 2.4-13.6) in the placebo group (P < 0.0001). Cumulative incidence of severe adverse events was 3.1 per 1000 patient-weeks. Peripheral neuropathy and amenorrhea were the most frequent adverse events.

CONCLUSIONS: In this pilot randomized controlled trial on cases of UC refractory to immunosuppressive therapy, thalidomide compared with placebo resulted in improved clinical remission at 8 weeks of treatment and in longer term maintenance of remission. These findings require replication in larger clinical studies evaluating both thalidomide efficacy and safety.

%B Inflamm Bowel Dis %V 21 %P 1739-49 %8 2015 Aug %G eng %N 8 %1 http://www.ncbi.nlm.nih.gov/pubmed/26185909?dopt=Abstract %R 10.1097/MIB.0000000000000437