Effect of Thalidomide on Clinical Remission in Children and Adolescents with Ulcerative Colitis Refractory to Other Immunosuppressives

Abstract

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.

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“…Except for 2 prospective placebo-controlled randomized trials, all studies were uncontrolled before and after studies (case series). Risk of bias was ranked as low for the primary analysis of the 2 RCTs (8 weeks) [10,15] and moderate to high in all the other studies.…”

“…Seven studies [12–15,19,23–25] enrolled patients with UC, but only 2 focused specifically on UC. In a pilot RCT on children with UC 18/23 (78.3%) children treated with thalidomide achieved clinical remission, compared to 2/11 (18.2%) in placebo.…”

“…In a pilot RCT on children with UC 18/23 (78.3%) children treated with thalidomide achieved clinical remission, compared to 2/11 (18.2%) in placebo. [15] In 4 other studies [13,23,24,29] outcomes of patients with UC were as follows: of the 16 patients identified, 9 (56.2%) responded to thalidomide, and 7 (43.7%) achieved remission.…”

“…[10,13–15,17,22,27,33–36,38–41,43] The number of patients evaluated at each time-point is specified in Table 4. The remission rate over time was as follows: 128/1160 (80.0%) at 6 months (15 studies); 96/133 patients (72.2%) at 12 months (13 studies); and 61/112 patients (54.5%) at 24 months (11 studies).…”

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“…Thalidomide is a small molecule with anti-tumor necrosis factor α, immunomodulatory and antiangiogenic properties. Randomized controlled trials demonstrated that the use of thalidomide, compared with placebo, resulted in improved clinical remission among children and adolescents with refractory CD and UC [43,44] . Recently, some case series showed that thalidomide could induce MH [45] .…”