A Study to Review the Treatment and Outcomes of Teenagers and Adults With Non-histaminergic Angioedema With Normal C1 Inhibitor in Canada

Study Overview
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Brief Summary
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The main aim of this study is to describe the characteristics of participants, check for number of hereditary angioedema (HAE) cases, their treatment and outcomes. Another aim is to check how the healthcare facilities were utilized for treatment. Participants' data will be taken from their medical records (charts), which were already collected as a part of their routine care between January 1, 2012, and January 1, 2022.

Detailed Description
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This is a non-interventional, retrospective, observational study of participants with Hereditary Angioedema with normal C1 esterase inhibitor (HAE) and Non-histaminergic Angioedema (NHAE) with normal C1-inhibitor function (nC1-INH) receiving various treatments between January 1, 2012, and January 1, 2022. The study will assess case numbers, treatments, outcomes, and healthcare resource utilization in the real-world setting. This study will enroll approximately 90 to 150 participants. Participants will be enrolled in the following two cohorts: Participants with HAE nC1-INH Participants with NHAE nC1-INH This study will have a retrospective data collection from January 1, 2012 to January 1, 2022 by using data from the participant medical charts that were already collected as part of routine care. This multi-center trial will be conducted in Canada. The overall time for data collection in this study will be approximately 10 years.

Official Title
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Treatment and Outcomes of Patients Identified With Non-Histaminergic Angioedema With Normal C1 Inhibitor in Canada

Conditions
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Hereditary Angioedema (HAE), Angioedema

Intervention / Treatment
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* Other: No Intervention


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: 1. Participants ≥12 years of age with HAE nC1-INH or NHAE nC1-INH diagnosed by an HAE treating specialist based on some or all of the following: Recurrent angioedema as documented by a healthcare professional within specialist charts Normal C4 Normal C1 level and function Condition worsened with estrogen if estrogen is/was being received Lack of response to corticosteroid and high-dose regular and/or prophylactic antihistamine(s) treatment(s) Family history of non-histaminergic angioedema for patients with HAE nC1-INH Exclusion Criteria: Other types of angioedema (type-1 HAE, type-2 HAE, acquired angioedema, etc.) Not meeting the above diagnostic criteria for inclusion Response to treatments used for histamine-related angioedema

Ages Eligible for Study
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Minimum Age: 12 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 nC1-INH<br>Participants with HAE nC1-INH will be observed retrospectively for the treatments they receive and their outcomes in the real-world setting from January 1, 2012, to January 1, 2022. | Other: No Intervention<br>* As this is an observational study, no intervention will be administered.<br>|
| NHAE nC1-INH<br>Participants with NHAE nC1-INH will be observed retrospectively for the treatments they receive and their outcomes in the real-world setting from January 1, 2012, to January 1, 2022. | Other: No Intervention<br>* As this is an observational study, no intervention will be administered.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants Diagnosed With HAE nC1-INH at Each Site |  | Up to approximately 10 years | 
| Number of Participants Diagnosed With NHAE nC1-INH at Each Site |  | Up to approximately 10 years | 
| Number of Participants Categorized by Type of Treatments Received by Participants |  | Up to approximately 10 years | 
| Average Dose for Each Type of Treatment Received by Participants |  | Up to approximately 10 years | 
| Average Duration of Treatment for Each Type of Treatment Received by Participants |  | Up to approximately 10 years | 
| Number of Participants Categorized by Type of Settings Where Treatment was Received | Categories of treatment settings include: home, hospital, physician office, emergency room visits (ER), and other. | Up to approximately 10 years | 
| Number of Participants Receiving Multiple Long-Term Prophylaxis (LTP) Therapies at Once | Number of participants will be reported by number of LTP therapies received at once. | Up to approximately 10 years | 
| Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received |  | Up to approximately 10 years | 
| Number of Attacks Occurring per Three-Months |  | Before and after initiation of treatment up to end of the study (up to approximately 10 years) | 
| Number of Participants Categorized by Severity of Attack per Three Months | Categories of severity are planned to include ER, use of rescue medication(s), (%) intubation, hospitalization, and laryngeal involvement. | Before and after initiation of treatment up to end of the study (up to approximately 10 years) | 
| Number of Participants by Body Sites Affected by Attacks per Three Months | Body sites will include face, lip, tongue, gastrointestinal (GI) system, larynx, extremities, and other. | Before and after initiation of treatment up to end of the study (up to approximately 10 years) | 
| Number of Participants with Symptoms of the Attack |  | Up to approximately 10 years | 
| Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution |  | Up to approximately 10 years | 
| Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 
| Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 
| Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 
| Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 
| Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 
| Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH |  | Up to approximately 10 years | 

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