Recurrent bleeding due to small-intestinal angiodysplasia (SIA) is a challenging condition that lacks effective treatment options. However, a recent randomized trial has shed light on a potential therapy using thalidomide. This article critically examines the trial’s findings and discusses the implications of this novel approach.
The randomized trial, conducted by Huimin Chen, MD, and colleagues from Shanghai Jiao-Tong University, involved 150 patients with recurrent bleeding. The patients were divided into three groups: one group received 100-mg thalidomide daily for 4 months, another received 50-mg thalidomide, and the third received a placebo. The primary endpoint was a reduction of at least 50% in bleeding episodes in the year following treatment.
According to the results published in the New England Journal of Medicine, 68.6% of patients in the 100-mg thalidomide group experienced a reduction of 50% or more in bleeding episodes, compared to 51% in the 50-mg group and a mere 16% in the placebo group. These findings indicate a significant improvement in bleeding reduction with the use of thalidomide.
The trial not only demonstrated the effectiveness of thalidomide in reducing bleeding episodes but also showed positive results in various secondary endpoints. Patients in the 100-mg thalidomide group had a lower rate of hospitalization for re-bleeding compared to those in the other groups. Additionally, other measures such as the number and duration of bleeding episodes, hemoglobin level, and cessation of bleeding without re-bleeding were all consistent with the positive effects of thalidomide.
The study highlighted the unmet medical need for a safe and effective oral medication for recurrent bleeding due to SIA. Current treatments, such as endoscopic procedures and somatostatin analogues, have limited evidence supporting their efficacy. The high rates of re-bleeding after endoscopic treatment further emphasize the need for alternative therapeutic options.
One intriguing aspect of the trial is the suggestion that thalidomide may be disease-modifying, with its efficacy persisting even after discontinuation. This finding opens up new possibilities for long-term management of recurrent bleeding due to SIA. However, further research is necessary to confirm this potential and explore the underlying mechanisms of action.
While thalidomide showed promising results in the trial, the appropriate dosage remains uncertain. Experts suggest starting with a lower dose and increasing it based on clinical response and side effects. However, caution is necessary due to the increased occurrence of adverse events associated with thalidomide, including constipation, somnolence, limb numbness, peripheral edema, dizziness, and elevated liver enzyme levels.
The trial’s findings provide higher-quality evidence for thalidomide compared to any other therapy available for recurrent bleeding due to SIA. However, the potential for better adherence and safety with somatostatin analogues may still make them preferable as a first-line treatment. Thalidomide can be reserved for patients who experience continued bleeding or side effects with somatostatin analogues.
Although the trial provides valuable insights, it also has some limitations. The sample sizes were relatively small for adequately comparing different doses of thalidomide. Additionally, the lack of racial and ethnic diversity in the trial participants raises questions about the generalizability of the findings. Further research is needed to confirm these results in non-Han Chinese populations.
The use of thalidomide as a treatment for recurrent bleeding due to SIA holds promise based on the results of the recent randomized trial. The findings suggest that thalidomide can effectively reduce bleeding episodes and improve patient outcomes. However, optimal dosing and long-term effects require further investigation. This study opens up new avenues for the management of this challenging condition and highlights the need for larger, more diverse trials to validate these findings.