Frequency of Common Medical Conditions in People With and Without HHT

NCT ID: NCT02464644

Last Updated: 2023-09-28

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

NA

Total Enrollment

2174 participants

Study Classification

INTERVENTIONAL

Study Start Date

2012-04-30

Study Completion Date

2015-05-31

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.

Conditions

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Hereditary Hemorrhagic Telangiectasia (HHT)

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

PREVENTION

Blinding Strategy

NONE

Study Groups

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People with HHT

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.

Group Type EXPERIMENTAL

Questionnaire

Intervention Type OTHER

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.

Controls without HHT

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.

Group Type OTHER

Questionnaire

Intervention Type OTHER

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.

Interventions

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Questionnaire

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.

Intervention Type OTHER

Other Intervention Names

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Survey Confidential SurveyMonkey SSL encryption feature Web-link collector does not collect IP addresses

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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

Yes

Sponsors

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Imperial College London

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Claire L Shovlin, PhD MB BChir FRCP

Role: PRINCIPAL_INVESTIGATOR

Imperial College London

Locations

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NHLI Cardiovascular Sciences, Imperial College London

London, , United Kingdom

Site Status

Countries

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United Kingdom

References

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Silva BM, Hosman AE, Devlin HL, Shovlin CL. Lifestyle and dietary influences on nosebleed severity in hereditary hemorrhagic telangiectasia. Laryngoscope. 2013 May;123(5):1092-9. doi: 10.1002/lary.23893. Epub 2013 Feb 12.

Reference Type RESULT
PMID: 23404156 (View on PubMed)

Devlin HL, Hosman AE, Shovlin CL. Antiplatelet and anticoagulant agents in hereditary hemorrhagic telangiectasia. N Engl J Med. 2013 Feb 28;368(9):876-8. doi: 10.1056/NEJMc1213554. No abstract available.

Reference Type RESULT
PMID: 23445111 (View on PubMed)

Finnamore H, Le Couteur J, Hickson M, Busbridge M, Whelan K, Shovlin CL. Hemorrhage-adjusted iron requirements, hematinics and hepcidin define hereditary hemorrhagic telangiectasia as a model of hemorrhagic iron deficiency. PLoS One. 2013 Oct 16;8(10):e76516. doi: 10.1371/journal.pone.0076516. eCollection 2013.

Reference Type RESULT
PMID: 24146883 (View on PubMed)

Hosman AE, Devlin HL, Silva BM, Shovlin CL. Specific cancer rates may differ in patients with hereditary haemorrhagic telangiectasia compared to controls. Orphanet J Rare Dis. 2013 Dec 20;8:195. doi: 10.1186/1750-1172-8-195.

Reference Type RESULT
PMID: 24354965 (View on PubMed)

Elphick A, Shovlin CL. Relationships between epistaxis, migraines, and triggers in hereditary hemorrhagic telangiectasia. Laryngoscope. 2014 Jul;124(7):1521-8. doi: 10.1002/lary.24526. Epub 2014 Jan 23.

Reference Type RESULT
PMID: 24458873 (View on PubMed)

Shovlin CL, Awan I, Cahilog Z, Abdulla FN, Guttmacher AE. Reported cardiac phenotypes in hereditary hemorrhagic telangiectasia emphasize burdens from arrhythmias, anemia and its treatments, but suggest reduced rates of myocardial infarction. Int J Cardiol. 2016 Jul 15;215:179-85. doi: 10.1016/j.ijcard.2016.04.006. Epub 2016 Apr 7.

Reference Type RESULT
PMID: 27116331 (View on PubMed)

Patel T, Elphick A, Jackson JE, Shovlin CL. Injections of Intravenous Contrast for Computerized Tomography Scans Precipitate Migraines in Hereditary Hemorrhagic Telangiectasia Subjects at Risk of Paradoxical Emboli: Implications for Right-to-Left Shunt Risks. Headache. 2016 Nov;56(10):1659-1663. doi: 10.1111/head.12963. Epub 2016 Oct 11.

Reference Type RESULT
PMID: 27727478 (View on PubMed)

Shovlin CL, Patel T, Jackson JE. Embolisation of PAVMs reported to improve nosebleeds by a subgroup of patients with hereditary haemorrhagic telangiectasia. ERJ Open Res. 2016 Apr 29;2(2):00035-2016. doi: 10.1183/23120541.00035-2016. eCollection 2016 Apr.

Reference Type RESULT
PMID: 27730189 (View on PubMed)

Shovlin CL, Gilson C, Busbridge M, Patel D, Shi C, Dina R, Abdulla FN, Awan I. Can Iron Treatments Aggravate Epistaxis in Some Patients With Hereditary Hemorrhagic Telangiectasia? Laryngoscope. 2016 Nov;126(11):2468-2474. doi: 10.1002/lary.25959. Epub 2016 Apr 23.

Reference Type RESULT
PMID: 27107394 (View on PubMed)

Hosman AE, Shovlin CL. Cancer and hereditary haemorrhagic telangiectasia. J Cancer Res Clin Oncol. 2017 Feb;143(2):369-370. doi: 10.1007/s00432-016-2298-x. Epub 2016 Nov 11.

Reference Type RESULT
PMID: 27837281 (View on PubMed)

Other Identifiers

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CLS/2012/2

Identifier Type: -

Identifier Source: org_study_id

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