A Study in Adults With Hereditary Angioedema (HAE) Who Currently Receive Icatibant at Home

Study Overview
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Brief Summary
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The main aim of this study is to describe the treatment patterns, characteristics and outcomes of people with HAE who are currently receiving icatibant in the homecare setting in the United Kingdom (UK). Participants will be treated with icatibant according to their routine practice via homecare service for icatibant within the UK. Data will be directly collected from participants via study diaries and questionnaires. Participants will be contacted approximately every 90 days during study duration (this can occur via phone or as a face-to-face visit).

Official Title
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A Real-World Study to Determine Patient Characteristics, Treatment Patterns, Clinical and Patient-Reported Outcomes of Patients With Hereditary Angioedema That Self-Administered Icatibant Using Homecare Services in the UK

Conditions
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Hereditary Angioedema (HAE)

Intervention / Treatment
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* Other: No Intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria Participants self-administering or receiving care assisted administration of icatibant treatment for patient confirmed diagnosis of HAE Type I or II in the homecare setting Participants who are able and willing to complete the study questionnaires Participants who are willing to be visited or contacted by a member of the homecare or research team at pre-arranged intervals in order to complete questionnaires Exclusion Criteria - Participants who have received icatibant as an investigational medicine as part of a clinical trial

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| HAE Participants<br>Adult participants with a diagnosis of HAE who are receiving treatment according to routine clinical practice and prescribed all treatments in accordance with the approved marketing authorization will be enrolled in this study. Data will be directly collected from participants via patient reported diaries and paper- based and/or electronic homecare records as appropriate for UK participants using homecare services for icatibant. No study specific intervention will be administered in this study. | Other: No Intervention<br>* No Intervention will be administered in this study.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Treated with Icatibant in the Homecare Setting who Were Receiving Prophylaxis Treatment at Index |  | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Number of Participants Treated with Icatibant in the Homecare Setting in Each Prophylactic Treatment Type |  | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Categorized by Their Demographic Characteristics | Demographic characteristics will include age at index (categories: 18-29, 30-39, 40-49, 50-59, 60+ years), sex (male, female, non-binary), and ethnic group. | Baseline Period [3 months prior to Index (day of the first homecare telephonic consultation/visit)] | 
| Number of Participants Categorized by Their Clinical Characteristics | Clinical characteristics will include patient reported HAE type (Type I or Type II), categories of duration (participant's self-administration of icatibant at home and time since HAE diagnosis) at index, comorbidities, prior HAE related concomitant medication details, categories of details of deaths during the study observation period. | At Index (day of the first homecare telephonic consultation/visit) | 
| Rate of New HAE Attacks | Rate of new HAE attacks and rate of new HAE attacks that required treatment in homecare setting will be recorded as number of HAE attacks per participant per month. Data would be reported for pre-defined time-period categories as follows: between index to month 3, between month 3 to 6, month 6 to 9 and month 9 to 12. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Frequency of Visits to Accident and Emergency Services Related to an HAE Attack | Frequency of visits to Accident and Emergency Services related to an HAE attack will be defined as number of visits to Accidents and Emergency Services during pre-defined time period. Data would be reported for pre-defined time-period categories as follows: between index to month 3, between month 3 to 6, month 6 to 9 and month 9 to 12. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Time from the Start of Each HAE Attack to Administration of Icatibant in the Homecare Setting | The summary data for time (in hours) from the start of each HAE attack to administration of icatibant in the homecare setting will be reported. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Frequency of Icatibant Administration Following Start of Each HAE Attack | The summary data for number of icatibant administration following start of each HAE attack during the predefined time period will be reported. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Number of Participants by Treatment Patterns | Treatment patterns will include assessment of number of participants by treatment dose received for HAE attack and average dose per participant received post-index. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Number of Participants by Treatment Management Patterns | Treatment management patterns will include assessment of number of participants by discontinuations of icatibant treatment and reasons for discontinuation (this would be assessed for participants who received prophylaxis treatment during the study), number of face-to-face and telephone-based homecare consultations. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| EQ-5D-5L Score | The EQ-5D-5L questionnaire will be used to record impact of HAE on participant's quality-of-life (QoL). The EQ-5D-5L is a descriptive system of Health-Related Quality-of-Life (HRQoL) states consisting of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression), each of which can take 1 of 5 responses. The responses record 5 levels of severity [no problems, slight problems, moderate problems, severe problems, and extreme problems] within a particular dimension. The scores will be the sum of scores from each dimension and can range from 5 (no problems) to 25 (extreme problems). | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| AE-QoL Total Score | The Angioedema Quality of Life (AE-QoL) includes 17 items and 4 domains: functioning, fatigue/mood, fears/shame, and food. Raw scores from each domain will be linearly transformed to a total score of 0 to 100, with higher scores indicating a stronger impairment. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 
| Angioedema Control Test (AECT) Total Score | The AECT is used to assess participants with recurrent angioedema. The test uses a questionnaire with 4 items, each of which has 5 options and is used to measure the participant's current disease control. HAE control score will be recorded with 5 levels of answers [1=very often, 2=often, 3=sometimes, 4=seldom, 5=not at all] to the following questions: 1) In the last 3 months, how often have you had angioedema? 2) In the last 3 months, how much has your QoL been affected by angioedema? 3) In the last 3 months, how much has the unpredictability of your angioedema bothered you? 4) In the last 3 months, how well has your angioedema been controlled by your therapy? The total scores are the sum of individual scores from 4 items and will range from 4 (Very often) to 20 (not at all). Higher scores will indicate no recurrence. | Up to 12 months from Index (day of the first homecare telephonic consultation/visit) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Drug Therapy