Retrospective and Prospective Real-World Evidence (RWE) Study Investigating Diagnosis and Therapeutic Management of Patients With HAE Type I or II in the Current Medical Practice in Egypt: the EXPLORHAE Study (EXPLORing Prospective and Retrospective Health Assessments of HAE in Egypt)
试验速览
- 阶段
- 不适用
- 状态
- 尚未招募
- 发起方
- 入组人数
- 100
- 试验地点
- 7
- 主要终点
- Age of Participants at Diagnosis (in years) of HAE-C1INH Type1 or Type2
研究概览
简要总结
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1-INH): HAE with normal C1-INH levels and HAE with limited or insufficient C1-INH levels (C1-INH deficiency); HAE with C1-INH deficiency can be divided into Type1, with low levels of C1-INH, and Type 2, in which the protein is there, but does not work properly. This study will look at people with HAE Type1 or Type2.
The main aim of this study is to describe the diagnosis and management of people with HAE Type1 or HAE Type2 in Egypt. Other aims are to learn more about people with HAE Type1 or Type2, including, but not limited to, other conditions they may have, family history, impact of HAE on daily life, if treatment is stopped and the reasons.
During the study, information from already existing data in a participant's medical record will be reviewed and new data will be collected during routine visits of a participant to the study clinic.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Retrospective Phase:
- •The participant has a confirmed HAE-C1INH type1 or type2 diagnosis in medical records (based on clinical history and/or laboratory diagnosis in medical records).
- •The participant was diagnosed and/or treated from January 2015 to August
- •Inclusion Criteria: Prospective Phase:
- •The participant has a physician-confirmed HAE-C1INH type1 or type2 diagnosis (based on clinical history and/or laboratory diagnosis).
- •The participant signed an informed consent or assent.
- •The participant should have had at least one visit to the treating physician/investigator during enrollment and the follow-up period.
- •The participant was not enrolled in the study's retrospective phase.
排除标准
- •(For both retrospective and prospective phases):
- •Participant with AAE-C1INH (acquired angioedema), drug-induced angioedema (AE-DI) (example angiotensin-converting enzyme inhibitors-I [ACE-I] angioedema), allergic mediated angioedema, inflammatory angioedema, or idiopathic angioedema.
- •HAE participants with normal C1-INH (HAE-nC1INH).
- •Participant deemed unsuitable for participation for any reason, based on the investigator's clinical judgment.
研究组 & 干预措施
Retrospective Phase
All participants who were diagnosed with HAE-C1INH type1 or type2 from January 2015 to August 2025; retrospective clinical data will be extracted for 5 months exclusively from patients' medical charts, and no direct patient interaction will occur.
干预措施: No intervention (Other)
Prospective Phase
All participants with any newly diagnosed or admitted or follow-up HAE-C1INH type1 or type2; prospective clinical data will be actively collected over 12 months through routine visits and electronic data capture (EDC) entries.
干预措施: No intervention (Other)
结局指标
主要结局
Age of Participants at Diagnosis (in years) of HAE-C1INH Type1 or Type2
时间窗: At Day 1
Percentage of Participants With HAE-C1INH Type1 or Type2
时间窗: At Day 1
Number and Type of First Symptom at Initial Manifestation
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants With First Symptom at Initial Manifestation
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number and Type of Confirmed Diagnosis of HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Routes of Administration Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Duration of First Symptom at Initial Manifestation
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants who Received Prescriptions for Therapeutic Management With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Treatment Options Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Dose Regimens Used for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants Switching Treatment for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants With Confirmed Diagnosis of HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants with Recorded Diagnostic Test Results for C1-INH Antigenic Level, Serum Complement Component 4 (C4) Level, and C1-INH Functional Level
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Duration of Confirmed Diagnosis of HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants Receiving On-demand (OD), Short-term prophylaxis (STP), and Multiple Long-Term Prophylaxis (LTP) Therapies
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants With Reason for Switching Treatment for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
Number of Participants With Frequency of Treatment Changes for the Therapeutic Management of Participants With HAE-C1INH Type1 or Type2
时间窗: Up to 5 months retrospectively, up to 12 months prospectively
次要结局
- Number of Participants With Clinical Symptom Types of HAE-C1INH Type1 or Type2 at Last Clinical Appointment(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With History of Upper Airway Edema(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With History of Abdominal Surgery due to HAE Misdiagnosis(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With HAE-related Death(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Prodromal Symptom(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Clinical Symptoms of HAE-C1INH Type1 or Type2 at Last Clinical Appointment(Up to 5 months retrospectively, up to 12 months prospectively)
- Average Number of Attacks Requiring Emergency Care or Hospitalization per Month(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Severity of HAE Attack per Each Attack(Up to 5 months retrospectively, up to 12 months prospectively)
- Duration of Symptoms for Each HAE Attack Onset During Subsequent Follow-Up Visits(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of HAE Attacks per Month(Up to 5 months retrospectively, up to 12 months prospectively)
- Number and Type of Hospitalizations due to HAE Attacks(Up to 5 months retrospectively, up to 12 months prospectively)
- Duration of Clinical Symptoms of HAE-C1INH Type1 or Type2 at Last Clinical Appointment(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Known Triggers at Diagnosis(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Presence of Comorbidities(Up to 5 months retrospectively, up to 12 months prospectively)
- Time of HAE Attack(s) at Each Onset During Follow-Up Visits or Date of Last Recorded HAE Attack(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Rate of Testing and of Diagnosis Among Tested Family Members(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Response to Standard Allergy Medications(Up to 5 months retrospectively, up to 12 months prospectively)
- Average Number of HAE Attacks per Month(Up to 5 months retrospectively, up to 12 months prospectively)
- Average Number of Attacks Requiring Acute Treatment per Month(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants Categorized by HAE Attack Characteristics(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With any Known Triggers Identified by the Participant as Related to the Onset of the Attack per Each Attack Onset for Each Subsequent Follow-up Visits(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With On-demand Treatment Medication for Each HAE Attack(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Life-threatening Attacks(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants (Family Members) Diagnosed With HAE-C1INH Type1 or Type2(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Degree of Relation (Parent, Sibling, Child) to Diagnosed Family Members(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Presence of Laryngeal or Life-threatening Attacks in Relatives(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Clinical Assessment for HAE Diagnosis With Symptoms Description for the Patient, their Descendants, or Relatives(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Laboratory Testing for the Participant, Their Descendants, or Relatives(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With History of HAE-related Deaths in the Family(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participant's Relatives who Underwent Clinical and/or Lab Assessment for HAE diagnosis(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Follow-up Visits Routinely Performed (Average per Year) During the Retrospective and Prospective Phase(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants Who Missed Days at Work or School and Social Life due to HAE Attack(Up to 5 months retrospectively, up to 12 months prospectively)
- Number of Participants With Treatment Discontinuation(Up to 5 months retrospectively, up to 12 months prospectively)