Frequency of Common Medical Conditions in People With and Without HHT

Study Overview
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Brief Summary
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Hereditary Hemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people. The purpose of this study is to provide data regarding the frequency of common health conditions and the tolerability of therapies in HHT by using a questionnaire. This will be filled in by both people with HHT, and controls who will be members of the general population without HHT. The questionnaire has been designed primarily for web based entry, but can also be circulated in paper format on request

Detailed Description
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Hereditary Hemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people, usually causing nosebleeds, skin blood spots, and/or anemia as a result of bleeding from the nose or gut. The majority of people with HHT also have abnormal blood vessels (arteriovenous malformations) in internal organs such as the lungs, liver and brain. Management of this multisystem disorder is highly challenging. The Lead Applicant has spent 20 years working on this rare disease, and identified multiple areas where more evidence is required to assist clinicians and patients with this lifelong condition. A particular issue is what happens when people with HHT have other common medical conditions such as asthma, cancer, diabetes, or heart disease. Do they have the same pattern of problems as the general population? Can they use the same drugs? Are further safeguards needed? For these important questions, current advice can only be based on theoretical considerations and anecdotal data. The ultimate goal of this study is to use information derived from a questionnaire to provide evidence to assist clinicians treating people with HHT. Data will be entered from April 2012. Data will be analysed on average 6-8 months after entry.

Official Title
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A Questionnaire Study on Hereditary Hemorrhagic Telangiectasia (HHT) and Other Medical Conditions, Compared to the General Population

Conditions
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Hereditary Hemorrhagic Telangiectasia (HHT)

Intervention / Treatment
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* Other: Questionnaire


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Aged 18 or over. Capacity to fill in a questionnaire. Exclusion Criteria: Age under 18 Unable to fill in a questionnaire

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Prevention
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: People with HHT<br>People with HHT will identify themselves within the questionnaire, first by statement of what they think is their diagnosis, and second by provision of the HHT diagnostic criteria within specific questions. They will be directed to appropriate questions, according to answers to the previous questions. | Other: Questionnaire<br>* To capture large sufficiently large numbers of individuals for appropriate statistical power (see below), a web based questionnaire design was considered the most appropriate tool. SurveyMonkey was identified as the most suitable means to generate the questionnaire, facilitate confidential answers by the target populations, and for analysis of questionnaire data. The designed survey can also be presented in paper format, and used in our clinical service.<br>* Other names: Web-link collector does not collect IP addresses;|
| Other: Controls without HHT<br>People without HHT will identify themselves within the questionnaire, first by statement of what they think is their diagnosis, and second by provision of the HHT diagnostic criteria within specific questions. They will be directed to appropriate questions, according to answers to the previous questions. | Other: Questionnaire<br>* To capture large sufficiently large numbers of individuals for appropriate statistical power (see below), a web based questionnaire design was considered the most appropriate tool. SurveyMonkey was identified as the most suitable means to generate the questionnaire, facilitate confidential answers by the target populations, and for analysis of questionnaire data. The designed survey can also be presented in paper format, and used in our clinical service.<br>* Other names: Web-link collector does not collect IP addresses;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Prevalence of nosebleeds. | The data outcome will be captured at the time of reporting by study participants, indicating the % of respondents affected by nosebleeds at that time. Subsequent calculations will standardise for age and other participant variables. | Day 1 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Efficacy of nosebleed treatments | The data outcome will be captured at the time of reporting by study participants, indicating the % of respondents reporting beneficial, null or detrimental effects from nosebleed treatments. Subsequent calculations will standardise for age and other participant variables. | Day 1 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Health, On-line Questionnaire, No travel, Medicines, Drugs, Lifestyle, Family, Help for others, Rare condition, General population