The Efficacy of Thalidomide in Treating Recurrent Bleeding due to Small-Intestinal Angiodysplasia

Recurrent bleeding due to small-intestinal angiodysplasia (SIA) poses a significant medical challenge, as current treatment options are limited. However, a recent randomized trial conducted by Huimin Chen, MD, and colleagues at Shanghai Jiao-Tong University in China has shown promising results with the use of thalidomide. Thalidomide treatment led to a significant reduction in bleeding episodes among patients with SIA, providing potential hope for those suffering from this condition.

The study included 150 patients who experienced at least four bleeding episodes in the year prior to treatment. These patients were randomized into three groups: the first group received a daily dose of 100 mg thalidomide, the second group received 50 mg thalidomide, and the third group received a placebo. Treatment lasted for 4 months, and patients were followed for 1 year before and after treatment to assess outcomes.

Results

The study’s primary endpoint was a reduction of at least 50% in bleeding episodes in the year after treatment. The results showed that 68.6% of patients in the 100-mg thalidomide group experienced a 50% or more reduction in bleeding episodes, compared to 51% in the 50-mg thalidomide group and 16% in the placebo group. These findings indicate a statistically significant reduction in bleeding episodes among patients treated with thalidomide compared to placebo.

The efficacy of thalidomide was further supported by secondary endpoints. The study found that the 100-mg thalidomide group had a lower rate of hospitalization for re-bleeding in the year after treatment (28%) compared to the 50-mg thalidomide group (35%) and the placebo group (74%). Moreover, other secondary endpoints such as the number and duration of bleeding episodes, hemoglobin level, and the percentage of patients who had cessation of bleeding without re-bleeding were consistent with the primary findings.

An accompanying editorial by Loren Laine, MD, of Yale School of Medicine, highlighted that thalidomide may have disease-modifying properties. The study results suggest that the efficacy of thalidomide persists even after discontinuation of treatment. This finding is particularly encouraging as it indicates a potential long-term benefit for patients with SIA.

Optimal Dose and Treatment Approach

While the study provides valuable evidence for the efficacy of thalidomide in treating recurrent bleeding due to SIA, the optimal dose of thalidomide remains unclear. Laine suggests that clinicians may consider administering 50 mg or 100 mg initially based on individual patient response and side effects. However, he also emphasizes that somatostatin analogues, which have better adherence and safety profiles, may still be preferred as a first-line treatment option.

Adverse Events

The study reported a higher incidence of adverse events in the thalidomide groups compared to the placebo group. These events included constipation, somnolence, limb numbness, peripheral edema, dizziness, and elevated liver enzyme levels. While these side effects are worth considering, further research is needed to fully understand the safety profile of thalidomide in treating SIA.

Limitations

The study had some limitations that should be acknowledged. The sample sizes for each thalidomide group were relatively small, limiting a comprehensive comparison of the two doses. Additionally, the trial lacked racial and ethnic diversity, as all participants were Han Chinese. Therefore, further research is needed to replicate these findings in non-Han Chinese populations.

The study conducted by Huimin Chen et al. provides compelling evidence for the efficacy of thalidomide in reducing bleeding episodes among patients with recurrent bleeding due to SIA. Thalidomide may offer a promising treatment option for those who have not responded to other therapies. However, more research is warranted to determine the optimal dosage, evaluate the long-term effects, and expand the study population to include diverse racial and ethnic groups. Nevertheless, these findings bring hope to patients with SIA and pave the way for further investigations into the potential role of thalidomide in managing this challenging condition.