Bevacizimab, HHT
Conditions
Keywords
HHT, bevacizimab
Brief summary
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.
Interventions
DRUGBevacizumab
Bevacizumab is administered intravenously at 5 mg/kg body weight, 1 to 2 times annually, starting one month after nasal electrocoagulation.
PROCEDUREnasal electrocoagulation.
nasal electrocoagulation.
Oral thalidomide is initiated at 50 mg twice daily (100 mg per day) one month after nasal electrocoagulation.
Sponsors
Second Affiliated Hospital, Zhejiang University, School of Medicine
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
SINGLE (Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Definite HHT diagnosis is established by either clinical assessment according to the Curaçao criteria or genetic confirmation. Clinical diagnosis (Curaçao criteria) Patients who meet at least 3 of the following 4 items are diagnosed with definite HHT: Spontaneous, recurrent epistaxis Multiple mucocutaneous telangiectasias at typical sites Visceral arteriovenous malformations (lung, liver, brain, gastrointestinal tract, etc.) Positive family history of HHT in a first-degree relative Genetic diagnosis Identification of a pathogenic germline mutation in HHT-associated genes (ENG, ACVRL1) confirms the diagnosis of HHT.
Exclusion criteria
* Minors, patients with mild epistaxis that can be well controlled by other treatments, and patients with any contraindication to bevacizumab treatment.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| ESS | 1,3.6.12 months after surgery | Epistaxis Severity Score |
| Hb | 1,3,6,12months after surgery | Hemoglobin (Hb) |
Countries
China
Contacts
CONTACTHong-gang Duan, doctor
Outcome results
None listed