To evaluate the efficacy and safety of bevacizumab for treating severe epistaxis in patients with Hereditary Hemorrhagic Telangiectasia (HHT) and to explore how clinical and genetic factors affect treatment outcomes.
Conducted a systematic evaluation involving patient data on HHT severity, clinical response to bevacizumab treatment, and associated side effects. The study included both clinical assessments and genetic analysis to identify predictors of treatment response.
Preliminary findings suggest that bevacizumab significantly reduces the frequency and severity of epistaxis in severe HHT patients, with some patients showing remarkable long-term benefits. However, variability in treatment response was noted among individuals.
The study is limited by its potential for interindividual variability in drug response and the incidence of adverse events. Additionally, the underlying mechanisms contributing to treatment heterogeneity remain inadequately understood.
This research is crucial as it addresses significant gaps in the treatment of HHT, particularly for patients experiencing severe bleeding episodes. It provides insights into personalized medicine approaches in HHT management, which may enhance patient outcomes and quality of life.
Clinical Study Abstract (Ethics Application) Hereditary hemorrhagic telangiectasia (HHT) is a rare autosomal dominant vascular disorder characterized by recurrent intractable epistaxis and multi-organ arteriovenous malformations. Moderate-to-severe patients often develop refractory anemia with severely impaired quality of life. Current stepwise therapeutic strategies have substantial limitations, including frequent adverse reactions of thalidomide, high recurrence rates after electrocoagulation, and severe surgical trauma, resulting in a lack of safe and individualized treatment options. Our preliminary clinical practice has verified the promising efficacy and long-term benefits of bevacizumab in severe HHT-related epistaxis. However, obvious interindividual variability and occasional adverse events exist, and the underlying mechanism remains unclear. This study aims to systematically evaluate the efficacy and safety of bevacizumab for severe HHT-associated epistaxis, explore the influences of baseline clinical and genetic factors on prognosis, and investigate the mechanism of treatment heterogeneity, so as to optimize individualized therapeutic strategies and provide clinical evidence for precise management of HHT in China.