Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

NCT02690246 | Completed

• 1 other identifier: University Clinic Essen
• Study Type: observational
• Target: 915
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary haemorrhagic telangiectasia (HHT), also known as Rendu-Osler-Weber syndrome, is an inherited multisystemic disorder with recurrent epistaxis, mucocutaneous telangiectasia and visceral arteriovenous malformations. The purpose of this study is to provide data about multiple clinical aspects of HHT and responses to treatment. For comparison of some aspects also data of non-affected relatives is collected (second cohort). the questionnaire has been designed primarily for web based entry, but can also be circulated in paper format on request.

Conditions

Hereditary Haemorrhagic Telangiectasia (HHT)

Outcome Measures

Primary Outcomes (1)

• Effect of nasal self-packing to treat epistaxis on the quality of life in patients with HHT measured mainly by Glasgow Benefit Inventory (GBI)

  Hereditary Haemorrhagic Telangiectasia is characterised by recurrent epistaxis which can lead to a feeling to lose control. The investigators examine whether the use of high volume low pressure nasal packing is a secure and practical method to improve patients' quality of life.

  34 months

Secondary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  34 months

Other Outcomes (3)

• Number of patients with restless legs syndrome according to a questionnaire including the criteria by Allen et al., Sleep Medicine 4(2003) 101-119

  34 months

• Number of infectious diseases and complications in comparison to healthy spouses

  34 months

• Hormonal changes: Change of number of visible telangiectases during menstrual cycle and pregnancy

  34 months

Study Arms (2)

patients with HHT

patients with Hereditary Haemorrhagic Telangiectasia

Other: questionnaire

control cohort

indirectly only as a control cohort in questionnaire of affected persons

Interventions

questionnaire OTHER

a questionnaire based study

patients with HHT

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with HHT; Indirectly we accept healthy volunteers only as control cohort in questionnaires of affected persons.

You may qualify if:

• a diagnosis of Hereditary Haemorrhagic Telangiectasia

You may not qualify if:

• unable to provide informed consent

Sponsors & Collaborators

• University Hospital, Essen: lead

Study Sites (1)

University Hospital Essen

Essen, 45147, Germany

Location

MeSH Terms

Conditions

Telangiectasia, Hereditary Hemorrhagic

Interventions

Surveys and Questionnaires

Condition Hierarchy (Ancestors)

Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Telangiectasis; Hemorrhagic Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Vascular Malformations; Cardiovascular Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Urban Geisthoff, Prof. Dr.

  University Hospital, Essen

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prof. Dr. med.

Study Record Dates

• First Submitted: February 9, 2016
• First Posted: February 24, 2016
• Study Start: April 1, 2014
• Primary Completion: August 1, 2019
• Study Completion: March 1, 2021
• Last Updated: March 22, 2022

Record last verified: 2022-03

Locations

DE (1)