The following is a summary of “How I Treat Bleeding in Hereditary Hemorrhagic Telangiectasia,” published in the June 2024 issue of Hematology by Al-Samkari et al.
Hereditary hemorrhagic telangiectasia (HHT), or Osler-Weber-Rendu disease, is a rare genetic disorder affecting approximately 1 in 5,000 people globally, characterized by vascular malformations that lead to recurrent and often debilitating bleeding episodes. The hallmark manifestations include frequent nosebleeds (epistaxis) and chronic gastrointestinal bleeding, both of which can result in profound iron deficiency anemia and significant psychosocial burden for affected individuals.
Despite the lack of therapies approved by regulatory agencies specifically for HHT, recent clinical studies, including rigorous randomized controlled trials, have demonstrated the efficacy and safety of specific treatments. Antifibrinolytic medications have shown promise in managing mild to moderate bleeding by preventing the breakdown of blood clots, thereby reducing bleeding episodes. For more severe cases, systemic antiangiogenic drugs like pomalidomide and bevacizumab have emerged as effective options, targeting the abnormal blood vessel growth characteristic of HHT.
This shift from predominantly procedural interventions, often temporizing measures, to targeted medical therapies, represents a significant advancement in managing HHT-associated bleeding. This approach not only aims to alleviate acute bleeding episodes but also seeks to improve patients’ long-term outcomes and quality of life. This article presents four clinical cases to illustrate typical scenarios encountered in the daily practice of hematologists managing HHT-related bleeding complications.
These cases are informed by a robust framework of published evidence and extensive clinical experience. They offer practical guidance on evidence-based strategies for the use of antifibrinolytics, antiangiogenic therapies, and the management of iron deficiency anemia across the spectrum of disease severity in HHT. This comprehensive review underscores the importance of integrating modern therapeutic approaches into clinical practice to optimize outcomes and address the diverse challenges posed by HHT-associated bleeding disorders.
Source: sciencedirect.com/science/article/abs/pii/S0006497124016008
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