@article {7708, title = {Effect of Thalidomide on Clinical Remission in Children and Adolescents with Ulcerative Colitis Refractory to Other Immunosuppressives: Pilot Randomized Clinical Trial.}, journal = {Inflamm Bowel Dis}, volume = {21}, year = {2015}, month = {2015 Aug}, pages = {1739-49}, abstract = {

BACKGROUND: In a randomized controlled trial, thalidomide has shown to be effective in refractory Crohn{\textquoteright}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.

}, issn = {1536-4844}, doi = {10.1097/MIB.0000000000000437}, author = {Lazzerini, Marzia and Martelossi, Stefano and Magazz{\`u}, Giuseppe and Pellegrino, Salvatore and Lucanto, Maria Cristina and Barabino, Arrigo and Calvi, Angela and Arrigo, Serena and Lionetti, Paolo and Lorusso, Monica and Mangiantini, Francesca and Fontana, Massimo and Zuin, Giovanna and Palla, Gabriella and Maggiore, Giuseppe and Bramuzzo, Matteo and Pellegrin, Maria Chiara and Maschio, Massimo and Villanacci, Vincenzo and Manenti, Stefania and Decorti, Giuliana and De Iudicibus, Sara and Paparazzo, Rossella and Montico, Marcella and Ventura, Alessandro} } @article {1796, title = {Amenorrhea in women treated with thalidomide: report of two cases and literature review.}, journal = {Inflamm Bowel Dis}, volume = {19}, year = {2013}, month = {2013 Jan}, pages = {E10-1}, keywords = {Adolescent, Amenorrhea, Colitis, Ulcerative, Crohn Disease, Female, Humans, Review Literature as Topic, Thalidomide}, issn = {1536-4844}, doi = {10.1002/ibd.22845}, author = {Lazzerini, Marzia and Bramuzzo, Matteo and Martelossi, Stefano and Magazz{\`u}, Giuseppe and Pellegrino, Salvatore and Ventura, Alessandro} }