Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

Completed

• Conditions: Hereditary Haemorrhagic Telangiectasia (HHT)
• Interventions: Other: questionnaire

• Registration Number: NCT02690246
• Lead Sponsor: 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.

Study Timeline

• Study Start: 2014-04
• Study First Submitted: 2016-02-09
• Study First Submitted QC: 2016-02-19
• Study First Posted: 2016-02-24
• Primary Completion: 2019-08
• Study Completion: 2021-03
• Status Verified: 2022-03
• Last Update Submitted: 2022-03-21
• Last Update Posted: 2022-03-22

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 915

Inclusion Criteria

• a diagnosis of Hereditary Haemorrhagic Telangiectasia

Exclusion Criteria

• unable to provide informed consent

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
patients with HHT	questionnaire	patients with Hereditary Haemorrhagic Telangiectasia

Primary Outcome Measures

Name	Time	Method
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	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.

Secondary Outcome Measures

Name	Time	Method
Number of participants with treatment-related adverse events as assessed by CTCAE v4.0	34 months

Trial Locations

Locations (1)

University Hospital Essen; 🇩🇪 Essen, Germany