Physician, PhD-student
Pernille Darre Haahr
Department of ORL - Head and neck surgery, OUH
| Projekt styring | ||
| Projekt status | Open | |
| Data indsamlingsdatoer | ||
| Start | 01.01.2023 | |
| Slut | 31.12.2015 | |
Bevacizumab usage in HHT patients
Short summary
This database aims to determine the usage of bevacizumab treatment in HHT. This is done by quantifying the total number of HHT patients, using the treatment, their demographics, effects and side effects of the treatment.
Rationale
Hereditary Haemorrhagic Telangiectasia (HHT) also known as Osler-Weber-Rendu syndrome affects one in 5000 to 8000 individuals (around 85,000 European citizens). The disease is diagnosed using the Curaçao criteria, which include; 1; Recurrent epistaxis, 2; telangiectasia located on mucus membranes of the nose, mouth and skin, 3; visceral arteriovenous malformations and 4; family history of HHT. The diseases cause multiple arteriovenous malformations (AVMs), which are abnormal connections between arteries and veins. Small AVMs are known as telangiectasias. In most cases, the patients experience recurrent epistaxis, and muco-cutaneous telangiectasias which often appear from child age. Further, in some of the patients, arteriovenous malformations (AVMs) in the lungs, liver, nervous system and digestive tract are part of the clinical picture. Epistaxis affects more than 90% of patients after the age of 21. Further around 30% of HHT patients above the age of 50 experience gastrointestinal bleeding. Both GI-bleeding and epistaxis may cause severe transfusion dependent anemia, despite local treatment modalities, treatment of iron depletion, and medical treatment with tranexamic acid. During the last 15 years, awareness regarding the presence of hepatic arteriovenous malformations (HAVMs) have increased. It is known that severe HAVM can cause early morbidity and mortality in patients. The symptoms of HAVMs, are dependent on the shunt type (arteriovenous, arterioportal and venous-venous shunt). The arteriovenous shunt can cause high output heart failure in HHT. Arterioportal shunting can result in portal hypertension and venous-venous shunting can give rise to hepatic encephalopathy. All of these conditions represent a challenging clinical entity where liver transplantation is the last resort. In the above patients (both bleeding and high output cardiac failure) effective treatment modalities are warranted. Since the first case report published in 2006, four prospective studies (Dupuis-Girod S 2012 and 2023, Thompson 2014, Chavan A 2017), many retrospective studies and case reports have been published and it has become clear that bevacizumab helps HHT patients with bleeding anemia as well as patients with high output cardiac failure. In 2022 we published VASCERN guidelines on the use of Bevacizumab in HHT. In 2019 we published our first experience with the safety of Bevacizumab. Aim The aim of this study is to exhibit the VASCERN experience with the use of Bevacizumab for treatment of HHT patients.
Description of the cohort
HHT patients, treated with Bevacizumab.
Data and biological material
Categories of data (demographic), data from the patient journal.
Collaborating researchers and departments
Department of gastroenterology, Odense Universitetshospital