Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

A Questionnaire Based Study on Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia (HHT)


Lead Sponsor: University Hospital, Essen

Source University Hospital, Essen
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.

Detailed Description

Specific aspects include potential consequences from iron deficiency, efficacy and safety of self-packing, effect of female sex hormones, mortality, effects on the immune system.

Overall Status Unknown status
Start Date February 2016
Completion Date December 2019
Primary Completion Date August 14, 2017
Study Type Observational
Primary Outcome
Measure Time Frame
Effect of nasal self-packing to treat epistaxis on the quality of life in patients with HHT measured mainly by Glasgow Benefit Inventory (GBI) 34 months
Secondary Outcome
Measure Time Frame
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 34 months
Enrollment 915

Intervention Type: Other

Intervention Name: questionnaire

Description: a questionnaire based study

Arm Group Label: patients with HHT


Sampling Method: Non-Probability Sample


Inclusion Criteria:

- a diagnosis of Hereditary Haemorrhagic Telangiectasia

Exclusion Criteria:

- unable to provide informed consent

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: Accepts Healthy Volunteers

Overall Official
Last Name Role Affiliation
Urban Geisthoff, Prof. Dr. Study Director University Hospital, Essen
Facility: University Hospital Essen
Location Countries


Verification Date

May 2018

Responsible Party

Type: Principal Investigator

Investigator Affiliation: University Hospital, Essen

Investigator Full Name: Urban Geisthoff

Investigator Title: Prof. Dr. med.

Has Expanded Access No
Condition Browse
Arm Group

Label: patients with HHT

Description: patients with Hereditary Haemorrhagic Telangiectasia

Label: control cohort

Description: indirectly only as a control cohort in questionnaire of affected persons

Patient Data Undecided
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective