Clinical Trial: Symptoms and Treatment Results in Hereditary Hemorrhagic Telangiectasia

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Observational

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

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 Summary: 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.
Sponsor: University Hospital, Essen

Current Primary Outcome: Effect of nasal self-packing to treat epistaxis on the quality of life in patients with HHT measured mainly by Glasgow Benefit Inventory (GBI) [ Time Frame: 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.


Original Primary Outcome: Same as current

Current Secondary Outcome: Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: 34 months ]

Original Secondary Outcome: Same as current

Information By: University Hospital, Essen

Dates:
Date Received: February 9, 2016
Date Started: February 2016
Date Completion: November 2017
Last Updated: December 2, 2016
Last Verified: December 2016