Treatment and Outcomes of Patients Identified With Non-Histaminergic Angioedema With Normal C1 Inhibitor in Canada
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 60
- 试验地点
- 5
- 主要终点
- Number of Participants Categorized by Type of Treatments Received by Participants
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Retrospective
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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 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
排除标准
- •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
结局指标
主要结局
Number of Participants Categorized by Type of Treatments Received by Participants
时间窗: Up to approximately 10 years
Number of Walk-in Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
Number of Participants Receiving Multiple Long-Term Prophylaxis (LTP) Therapies at Once
时间窗: Up to approximately 10 years
Number of participants will be reported by number of LTP therapies received at once.
Number of Participants Receiving Multiple LTP Therapies Categorized by Combination and Order of Treatments Received
时间窗: Up to approximately 10 years
Number of Attacks Occurring per Three-Months
时间窗: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
Number of Participants Diagnosed With HAE nC1-INH at Each Site
时间窗: Up to approximately 10 years
Number of Participants by Body Sites Affected by Attacks per Three Months
时间窗: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
Body sites will include face, lip, tongue, gastrointestinal (GI) system, larynx, extremities, and other.
Number of Participants with Symptoms of the Attack
时间窗: Up to approximately 10 years
Time from Treatment (On-demand Therapy) to Symptom Improvement/Resolution
时间窗: Up to approximately 10 years
Number of Emergency Room Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
Number of Participants Diagnosed With NHAE nC1-INH at Each Site
时间窗: Up to approximately 10 years
Average Duration of Treatment for Each Type of Treatment Received by Participants
时间窗: Up to approximately 10 years
Number of Participants Categorized by Type of Settings Where Treatment was Received
时间窗: Up to approximately 10 years
Categories of treatment settings include: home, hospital, physician office, emergency room visits (ER), and other.
Average Dose for Each Type of Treatment Received by Participants
时间窗: Up to approximately 10 years
Number of Participants Categorized by Severity of Attack per Three Months
时间窗: Before and after initiation of treatment up to end of the study (up to approximately 10 years)
Categories of severity are planned to include ER, use of rescue medication(s), (%) intubation, hospitalization, and laryngeal involvement.
Number of Primary Care Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
Number of Unscheduled Physician Visits per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
Number of Hospitalizations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
Number of Intubations per Year Associated With Management of HAE nC1-INH or NHAE nC1-INH
时间窗: Up to approximately 10 years
次要结局
未报告次要终点