1. In this randomized controlled trial, thalidomide resulted in a dose-dependent reduction in bleeding among patients with recurrent bleeding from small-intestinal angiodysplasia (SIA).
2. Thalidomide was associated with a higher rate of adverse events than the placebo.
Evidence Rating Level: 1 (Excellent)
Study Rundown: SIA is a vascular abnormality within the walls of the small intestine that results in vessels prone to occult bleeding and resultant iron-deficiency anemia, especially in older patients. Current procedural interventions are associated with complications and high recurrent rates due to the multi-focal nature of angiodysplasia. Thalidomide has shown benefits in observational and small studies in treating SIA-related bleeding. This study was a multi-center randomized trial to assess the efficacy and safety of thalidomide for the treatment of SIA-related recurrent bleeding. In the one year following a four-month regimen, compared to placebo, thalidomide was associated higher response rate, defined as at least a 50% reduction of bleeding episodes from baseline, and other bleeding-related complications. The thalidomide groups were associated with more adverse events, including constipation, somnolence, and dizziness. The study was limited by a small sample size precluding the differentiation between the two thalidomide doses and its lack of patients with aortic stenosis and hereditary hemorrhagic telangiectasia. Nonetheless, these results demonstrated the efficacy of thalidomide in treating recurrent bleeding related to SIA.
In-Depth [randomized controlled trial]: The current study was a randomized, double-blind, placebo-controlled trial investigating the impact of thalidomide in treating recurrent bleeding in patients with SIA. Adult patients with SIA, as confirmed by endoscopy, and at least four episodes of recurrent bleeding within the preceding year were eligible for inclusion. Exclusion criteria included severe liver, kidney, respiratory, and heart diseases as well as severe gastrointestinal bleeding, and hypersensitivity to thalidomide. In total, 150 patients were randomized 1:1:1 to receive thalidomide 100mg or 50mg or placebo once daily for four months. The primary outcome was effective response, defined as a reduction of ≥50% from baseline in bleeding episodes in the year following the completion of the four-month treatment. Secondary outcomes included blood transfusion, hospitalization from bleeding, and hemoglobin levels. The primary outcome occurred in 68.6% of patients in the 100mg thalidomide group, 51.0% in the 50mg thalidomide group, and 16.0% in the placebo group (p<0.001). Correspondingly, both thalidomide groups had lower rates of blood transfusion and hospitalization due to bleeding than the placebo group. The mean (+/-standard deviation) change of hemoglobin level from baseline was 30.2+/-22.4 g/L in the 100mg thalidomide group, 22.6+/-21.7 g/L in the 50mg thalidomide group, and -3.8+/-9.5 g/L in the placebo group. The thalidomide groups were also associated with significantly higher incidence of adverse events, including constipation, somnolence, limb numbness and edema, dizziness, and elevated liver enzymes. In summary, these results showed that thalidomide was efficacious in treating recurrent bleeding from SIA.
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