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Cerebral Hemorrhage Risk in Hereditary Hemorrhagic Telangiectasia

Hereditary Hemorrhagic Telangiectasia

This study is one of the three projects of an NIH Rare Disease Clinical Research Consortium. A "consortium" is a group of centres sharing information and resources to perform research. The consortium research focuses on brain blood vessel malformations in three different rare diseases.

The focus of this specific study is on Hemorrhagic Telangiectasia (HHT).

HHT is a condition characterized by blood vessel malformations, called telangiectasia and arteriovenous malformations (AVMs), occurring in the brain, nose, lungs, stomach, bowels and liver. Brain AVMs (BAVMs) in HHT are difficult to study because they are rare, affecting approximately 10% of people with HHT. While other types of BAVMs have been studied in depth, studies in the HHT population have been very small. Here, we propose the first large-scale collaboration by joining with 12 HHT Centers of Excellence in North America to perform a large study of risk factors for bleeding from BAVMs, called intracranial hemorrhage (ICH) in HHT patients.

The current standard of clinical practice across North America, is to screen all HHT patients for BAVMs with magnetic resonance imaging (MRI). If BAVMs are detected, patients are referred to a multidisciplinary neurovascular team for consideration for treatment. Treatment decisions are made on a case by case basis, balancing risks of complications from the BAVM with risks of therapy, but are limited by the few studies available in HHT. We hope that the knowledge we obtain about the risk factors for intracranial bleeding in these patients from this larger study will help us to improve the care of HHT patients.

We plan to study risk factors for rupture of BAVMs, including primarily genetics and imaging characteristics of the BAVMs. Knowledge about risk factors will help in the care and management of HHT patients. This will be achieved through the collection of health information to construct a HHT database, blood sampling and banking (through the National Institute of Neurological Disorders and Stroke \[NINDS\]), and through genetic analysis at the University of California San Francisco.

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Participation Requirements

  • Sex:

    ALL
  • Eligible Ages:

    0 and up

Participation Criteria

Inclusion criteria:

* Definite clinical HHT diagnosis (at least 3 Curacao criteria)or genetic diagnosis of HHT or
* Definite clinical HHT diagnosis (at least 3 Curacao criteria) or genetic diagnosis of HHT and Presence of Brain Arteriovenous Malformation
* Able to provide informed consent

Curacao criteria:

1. spontaneous recurrent nosebleeds;
2. mucocutaneous telangiectasia at characteristic sites (lips, oral cavity or the nose);
3. visceral involvement such as pulmonary, hepatic or CNS BAVM; and (d) an affected first degree relative by same criteria.

Willingness

* Willingness to participate in the study and ability to give informed consent

Exclusion Criteria:

* Patients not complying with Inclusion criteria

Study Location

St. Paul's Hospital, University of British Columbia
St. Paul's Hospital, University of British Columbia
Vancouver, British Columbia
Canada

Contact Study Team

University of Alberta
University of Alberta
Edmonton, Alberta
Canada

Contact Study Team

St. Michael's Hospital
St. Michael's Hospital
Toronto, Ontario
Canada

Contact Study Team

Primary Contact

Marie E Faughnan, MD MSc FRCPC

[email protected]
416 864 6060
Backup Contact

Dewi Clark

[email protected]
416-864-6060
Hospital for Sick Children
Hospital for Sick Children
Toronto, Ontario
Canada

Contact Study Team

Primary Contact

Stephanie J Manning

[email protected]
(416) 813 - 4903
Study Sponsored By
Unity Health Toronto
Participants Required
More Information
Study ID: NCT01158807