Is Thalidomide Effective In Treating Recurrent Small-Intestinal Bleeding, Study Insights

A recent multicenter, double-blind, randomized, placebo-controlled trial investigated the efficacy and safety of thalidomide as a potential solution for recurrent bleeding due to small-intestinal angiodysplasia (SIA). Thalidomide treatment significantly reduced recurrent bleeding in patients with small-intestinal angiodysplasia, with an effective response observed in 68.6% of those receiving 100 mg thalidomide and 51.0% of those receiving 50 mg, compared to 16.0% in the placebo group.

In the study, eligible patients with a history of recurrent bleeding, experiencing at least four episodes within the previous year due to SIA, were randomly assigned to receive thalidomide at a daily oral dose of either 100 mg or 50 mg or a placebo for four months. Following the treatment period, patients were monitored for a minimum of one year.

The results were derived from 150 patients who underwent randomization: 51 to the 100-mg thalidomide group, 49 to the 50-mg thalidomide group, and 50 to the placebo group. Notably, the percentages of patients with an effective response in these respective groups were 68.6%, 51.0%, and 16.0%, indicating a highly significant outcome (P<0.001). Moreover, the analysis of secondary endpoints consistently supported the primary results.

However, it is important to acknowledge that adverse events were more frequent in the thalidomide groups compared to the placebo group. These adverse events included constipation, somnolence, limb numbness, peripheral edema, dizziness, and elevated liver enzyme levels.

Ref: N Engl J Med 2023; 389:1649-1659 DOI: 10.1056/NEJMoa2303706