Chen H, et al. NEJM 2023; 389: 1649-1659. Thalidomide for Recurrent Bleeding Due to Small-Intestinal Angiodysplasia

In this multicenter, double-blind, randomized, placebo-controlled trial, 150 adult patients underwent randomization: 51 to the 100-mg thalidomide group, 49 to the 50-mg thalidomide group, and 50 to the placebo group. Thalidomide has antiangiogenic activity, with inhibition of VEGF.  It also has many adverse effects of thalidomide including peripheral neuropathy, fatigue, and teratogenic effects.

Key finding:

• 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

Discussion points -(from the associated editorial by Loren Laine): The data for thalidomide for small-intestinal angiodysplasia is “of higher quality than evidence for any other therapy for this indication. In addition,….thalidomide may be disease-modifying, with efficacy persisting after discontinuation. However, many clinicians will still use somatostatin analogues first” due to convenience and safety.

My take: I am glad this is a rare problem in pediatrics. I am not at all excited about using thalidomide.

Related blog posts:

• The Original Anti-TNF Therapy: Thalidomide
• JAMA Thalidomide Study for Crohn’s Disease
• Telomere Biology Disorders and GI Bleeding
• Desperate Measures in Refractory IBD
• When nothing else is working