A Retrospective Observational Chart Review Study Evaluating the Burden of Illness and Treatment Patterns in Hereditary Angioedema Type I and II
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 发起方
- 入组人数
- 221
- 试验地点
- 36
- 主要终点
- Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment
研究概览
简要总结
This study is about teenagers and adults with hereditary angioedema (HAE) type I and type II. In this study, the burden of illness means the impact of HAE in terms of long-term health outcomes and the financial cost.
The main aims of this study are as follows:
- to learn how often, how severe and where on the body HAE attacks occur.
- to describe how HAE prophylaxis and on-demand medicines are prescribed and used. (Prophylaxis medicines prevent a bleed from happening and on-demand medicines treat a bleed when it occurs.)
This study is about collecting data only; participants will not receive treatment as part of this study. Existing data available in the participant's medical records will be collected. Participants will be asked to complete an electronic questionnaire either on a website or by using an app on their mobile phone; data from this questionnaire will also be collected.
Participants do not need to visit their doctor in addition to their normal visits.
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
- •Participant aged >= 12 years at the time of the last documented HAE attack in the eligibility period.
- •Physician-confirmed diagnosis (or confirmation in medical records) of HAE type I or type II.
- •Participant had at least one documented HAE attack during the eligibility period.
- •Participant is not adequately or sub-optimally controlled according to treating physician.
- •Participant (or parent/legal guardian) is willing and able to comply with the study requirements and provides informed consent or assent.
排除标准
- •Participant enrolled in a therapeutic investigational drug or device trial during the observation period.
- •Participant initiated long-term prophylaxis with Takhzyro® at any time since diagnosis.
结局指标
主要结局
Number of Participants Based on Long-term or Short-term HAE Prophylactic Treatment
时间窗: Throughout the study period (up to 6 months)
Number of participants based on long-term or short-term HAE prophylactic treatment will be reported.
Number of Participants Associated Between Prescriptions Patterns and HAE Attacks
时间窗: Throughout the study period (up to 6 months)
Number of participants with association between prescriptions patterns and HAE attacks related to frequency or severity. Generalized linear models (GLM) will be used to evaluate the association of outcomes of interest with relevant covariates between prescription pattern, HAE attacks, frequency and severity of HAE attacks will be reported.
Duration of Each HAE Attacks During Study Period
时间窗: Throughout the study period (up to 6 months)
Duration of each HAE attacks during study period will be reported.
Number of Participants Based on Type of HAE Prophylactic Treatments
时间窗: Throughout the study period (up to 6 months)
Number of participants based on type of HAE prophylactic treatment will be reported.
Number of Participants With HAE Attacks Occurrence During Study Period
时间窗: Throughout the study period (up to 6 months)
HAE attack is defined as the symptoms or signs consistent with an attack in at least 1 of the following locations: peripheral angioedema (cutaneous swelling involving an extremity, the face, neck, torso, and/or genitourinary region), abdominal angioedema (abdominal pain, with or without abdominal distention, nausea, vomiting, or diarrhea), laryngeal angioedema (stridor, dyspnea, difficulty speaking, difficulty swallowing, throat tightening, or swelling of the tongue, palate, uvula, or larynx). Number of participants with HAE attacks occurrence (start and end date of each HAE attack) during study period will be reported.
Duration of HAE Prophylactic Treatment
时间窗: Throughout the study period (up to 6 months)
Duration of HAE prophylactic treatment includes treatment start and end dates will be reported.
Number of Participants Categorized Based on Characteristics of HAE Attacks During Study Period
时间窗: Throughout the study period (up to 6 months)
HAE attack characteristics will be based on prodromal symptoms, severity and location, whether attack was life-threatening will be reported.
Time Interval Between HAE Attacks During Study Period
时间窗: Throughout the study period (up to 6 months)
Time interval between HAE attacks during the study period will be reported.
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Initiation
时间窗: Throughout the study period (up to 6 months)
Number of participants categorized based on reasons for HAE prophylactic treatment initiation will be reported.
Number of Participants Categorized Based on Dose and Route of Administration for HAE Prophylactic Treatment
时间窗: Throughout the study period (up to 6 months)
Number of participants categorized based on dose and route of administration for HAE prophylactic treatment will be reported.
Number of Participants Categorized Based on Reasons for HAE Prophylactic Treatment Discontinuation
时间窗: Throughout the study period (up to 6 months)
Number of participants categorized based on reasons for HAE prophylactic treatment discontinuation will be reported.
Duration of HAE On-demand Treatment
时间窗: Throughout the study period (up to 6 months)
Duration of HAE on-demand treatment includes treatment start and end dates will be reported.
Number of Participants Based on Type of HAE On-demand Treatment
时间窗: Throughout the study period (up to 6 months)
Number of participants based on type of HAE on-demand treatment will be reported.
Number of Participants Categorized Based on Dose and Route of Administration for HAE On-demand Treatment
时间窗: Throughout the study period (up to 6 months)
Number of participants categorized based on dose, route of administration and setting of administration for HAE on-demand treatment will be reported.
次要结局
- Healthcare Resource Utilization (HRU): Number of Visits to Healthcare Professionals (HCP)(Throughout the study period (up to 6 months))
- Health-related Quality of Life (HRQoL): Angioedema Quality of Life (AE-QoL) Questionnaire(Throughout the study period (up to 6 months))
- Time Interval Between HAE Attacks in Relevant Subgroups(Throughout the study period (up to 6 months))
- Number of Participants With HAE Attacks Occurrence in Relevant Subgroups(Throughout the study period (up to 6 months))
- HRU: Number of Participants With Hospitalizations due to HAE Attacks(Throughout the study period (up to 6 months))
- HRQoL: EuroQoL Group 5-Dimension 5-Level (EQ-5D-5L) Questionnaire(Throughout the study period (up to 6 months))
- Number of Participants Categorized Based on Characteristics of HAE Attacks in Relevant Subgroups(Throughout the study period (up to 6 months))
- Duration of Each HAE Attacks in Relevant Subgroups(Throughout the study period (up to 6 months))
- HAE Attacks Prescription Pattern in Relevant Subgroups(Throughout the study period (up to 6 months))
- HRU: Number of Participants With Emergency Room/Emergency Departments (ER/ED) Visits due to HAE Attacks(Throughout the study period (up to 6 months))
- HRQoL: EuroQoL-five Dimensions, Youth Version (EQ-5D-Y)(Throughout the study period (up to 6 months))