Hereditary Haemorrhagic Telangiectasia (HHT)
Conditions
Keywords
Hereditary Haemorrhagic Telangiectasia, Nose bleeding, Iron supplements
Brief summary
Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people. The purpose of this study is to provide data regarding the diet and nosebleed frequency using a questionnaire. This will be filled in by people with HHT. The questionnaire has been designed in paper format.
Detailed description
Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people, usually causing nosebleeds, skin blood spots, and/or anaemia 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 whether the diet influences HHT or its complications in any way. In this study, people will fill in two questionnaires, one giving details of their diet, and another details of their nosebleeds. They will also be asked to consider participating in an accessory study arm which includes weighing food for one week and providing a food diary, in addition to having a single set of blood test.
Interventions
OTHERQuestionnaire on dietary history
OTHERQuestionnaire on nosebleed severity
OTHEROne week food diary generated by weighing foods
PROCEDUREBlood tests for full blood count, albumin, and indices of nutritional status
Sponsors
Imperial College London
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
SUPPORTIVE_CARE
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* A diagnosis of hereditary Haemorrhagic Telangiectasia (HHT)
Exclusion criteria
* Unable to provide informed consent * Presence of another major organ disorder that may affect nutritional status, such as inflammatory bowel disease, or celiac disease.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants That Achieving Personal Recommended Intake of Iron | 1 year | Dietary food item iron content assessed by the European Prospective Investigation into Cancer and Nutrition (EPIC) food frequency questionnaire. This method has been validated against the gold standard for dietary assessment, a 7-day weighed food diary. Questions are asked about consumption of 130 major food items over the previous year, requiring participants to indicate the frequency of consumption, and to provide details about the methods of cooking, type of produce, and use of dietary supplements. The EPIC FFQ has been widely validated in a number of studies and is deemed an adequate assessment tool to assess dietary intake. |
| Nosebleed Severity | 3 months | Nosebleeds were quantified using the validated Epistaxis Severity Score (ESS). The six questions provide an objective measure of nosebleeds: three relate to different characteristics of typical nosebleeds within the previous three months (frequency, duration and intensity (gushing/pouring or not)), three to medical attention, anemia and transfusion requirements. The final ESS score ranges from 0-10, where a higher score equates to greater blood losses. |
| Number of Participants That Achieving the Hemorrhage Adjusted Iron Requirement (HAIR) | 1 year | The hemorrhage-adjusted iron requirement (HAIR) was calculated as the sum of the normal recommended dietary iron intake, and requirements to compensate for non-menstrual blood losses. |
Other
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants That Ingested Chocolate | 3 months | Chocolate can precipitate nosebleeds Food items intake assessed by 7-day weighed food diary |
| Number of Participants That Ingested Bread | 3 months | Bread not precipitate nosebleeds Food items intake assessed by 7-day weighed food diary |
Countries
United Kingdom
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) Patients with hereditary hemorrhagic telangiectasia (HHT) | 50 |
| Total | 50 |
Baseline characteristics
| Characteristic | Hereditary Hemorrhagic Telangiectasia (HHT) | — |
|---|---|---|
| Age, Continuous | 55 years | — |
| Race and Ethnicity Not Collected | — | — Participants |
| Region of Enrollment United Kingdom | 50 participants | — |
| Sex: Female, Male Female | 22 Participants | — |
| Sex: Female, Male Male | 28 Participants | — |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 50 |
| other Total, other adverse events | 0 / 50 |
| serious Total, serious adverse events | 0 / 50 |
Outcome results
Primary
Nosebleed Severity
Nosebleeds were quantified using the validated Epistaxis Severity Score (ESS). The six questions provide an objective measure of nosebleeds: three relate to different characteristics of typical nosebleeds within the previous three months (frequency, duration and intensity (gushing/pouring or not)), three to medical attention, anemia and transfusion requirements. The final ESS score ranges from 0-10, where a higher score equates to greater blood losses.
Time frame: 3 months
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) | Nosebleed Severity | 4.4 units |
Primary
Number of Participants That Achieving Personal Recommended Intake of Iron
Dietary food item iron content assessed by the European Prospective Investigation into Cancer and Nutrition (EPIC) food frequency questionnaire. This method has been validated against the gold standard for dietary assessment, a 7-day weighed food diary. Questions are asked about consumption of 130 major food items over the previous year, requiring participants to indicate the frequency of consumption, and to provide details about the methods of cooking, type of produce, and use of dietary supplements. The EPIC FFQ has been widely validated in a number of studies and is deemed an adequate assessment tool to assess dietary intake.
Time frame: 1 year
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) | Number of Participants That Achieving Personal Recommended Intake of Iron | 43 Participants |
Primary
Number of Participants That Achieving the Hemorrhage Adjusted Iron Requirement (HAIR)
The hemorrhage-adjusted iron requirement (HAIR) was calculated as the sum of the normal recommended dietary iron intake, and requirements to compensate for non-menstrual blood losses.
Time frame: 1 year
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) | Number of Participants That Achieving the Hemorrhage Adjusted Iron Requirement (HAIR) | 10 Participants |
Other Pre-specified
Number of Participants That Ingested Bread
Bread not precipitate nosebleeds Food items intake assessed by 7-day weighed food diary
Time frame: 3 months
Population: 25 participants were filled up the 7-day weighed food diary
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) | Number of Participants That Ingested Bread | 25 Participants |
Other Pre-specified
Number of Participants That Ingested Chocolate
Chocolate can precipitate nosebleeds Food items intake assessed by 7-day weighed food diary
Time frame: 3 months
Population: 25 participants were filled up the 7-day weighed food diary
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Hereditary Hemorrhagic Telangiectasia (HHT) | Number of Participants That Ingested Chocolate | 14 Participants |