For treating recurrent bleeding caused by small-intestinal angiodysplasia (SIA), thalidomide significantly reduced bleeding compared with placebo, a Chinese study shows. “The results of the current trial need to be further confirmed in non–Han Chinese populations,” the authors write. In addition, the study required participants to take “thalidomide and placebo tablets … four times a day, a requirement that may have decreased adherence and may limit generalizability to a more typical group of patients who would be expected to have lower adherence.”
At 10 sites, eligible patients with recurrent bleeding (at least 4 episodes of bleeding during the previous year) due to SIA were randomly assigned to receive thalidomide at an oral daily dose of 100 mg or 50 mg or placebo for 4 months. Based on a primary endpoint of effective response ( a reduction of at least 50% in the number of bleeding episodes that occurred during the year after the end of thalidomide treatment), the study showed the following: “Overall, 150 patients underwent randomization: 51 to the 100-mg thalidomide group, 49 to the 50-mg thalidomide group, and 50 to the placebo group. The percentages of patients with an effective response in the 100-mg thalidomide group, 50-mg thalidomide group, and placebo group were 68.6%, 51.0%, and 16.0%, respectively (P <0.001 for simultaneous comparison across the three groups). The results of the analyses of the secondary end points supported those of the primary end point. Adverse events were more common in the thalidomide groups than in the placebo group overall; specific events included constipation, somnolence, limb numbness, peripheral edema, dizziness, and elevated liver-enzyme levels.”
Editorial: “The fact that thalidomide was effective during the year after therapy was stopped suggests that thalidomide alters angiodysplasias,” an editorialist writes. “Nevertheless, among 42 patients in the thalidomide groups without rebleeding during the year after the end of treatment, 20 had rebleeding during the subsequent 3 to 27 months, a finding that suggests that retreatment will be needed. However, the duration of time before treatment with thalidomide would need to be repeated and the appropriate duration of a course of thalidomide treatment are unclear. In addition, given the possible dose response with respect to efficacy and adverse events, the appropriate dose is uncertain: clinicians may administer 50 mg, 100 mg, or 50 mg initially, with an increase to 100 mg on the basis of clinical response and side effects.”