Retrospective Chart Review Study Evaluating Clinical Effectiveness and Impact on Quality of Life Among Patients Who Initiated Long-term Prophylaxis With Takhzyro® in a Real-World Setting in the Kingdom of Saudi Arabia - the REFLEQT-KSA Study (Retrospective Evaluation Focusing on Lanadelumab's Effectiveness and Impact on Quality of Life in The Kingdom of Saudi Arabia)
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 发起方
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Change in HAE- Activity Score (AS) From Pre to Post Index Period
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 but does not cause itching or hives. HAE is classified based on the amount of a protein in the blood called C1 inhibitor (C1INH): HAE with normal C1INH levels (HAE-nC1INH) and HAE with limited or insufficient C1INH levels (HAE-C1INH); HAE-C1INH can be divided into Type 1, with low levels of C1INH, and Type 2, in which the protein is there, but does not work properly. This study will concentrate on people with HAE-C1INH Type 1 or 2 who have received Takhzyro® (lanadelumab) as prophylactic treatment for at least half a year (6 months). Prophylactic means that treatment is given to prevent the happening of HAE attacks.
The main goal of the study is to see how well Takhzyro® works in everyday life to reduce the condition's activity after 6 months of treatment, or 12 months (if data is available). This will be measured by checking the change of the HAE activity from before treatment to after 6 months of treatment.
The study design will permit a study follow-up of up to 12 months following the index event (i.e. date of first dose administration of Takhzyro®) unless the patient discontinues the index treatment, dies or is lost to follow-up within this timeframe. Chart abstractions will only occur once patients have at least 6 months' duration between the index event date and the date of chart abstraction initiation. Other goals are to find out how a person's quality of life changes after using Takhzyro® for 6 months, how often they had attacks before and after treatment and to learn which factors may have an impact on the treatment.
The study will only look at data already existing in the participants' medical records. No treatment will be given as part of the study.
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 is diagnosed with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 and initiated on long-term prophylaxis (LTP) with Takhzyro® (lanadelumab).
- •Participant is aged greater than or equal to (>=) 12 years at the time of Takhzyro® initiation.
- •Participant has received at least 6 months of continuous treatment with Takhzyro® before data abstraction.
排除标准
- •Participants who have normal C1INH function or HAE-nC1INH (formerly type III HAE).
- •Participants who discontinued Takhzyro® before completing 6 months of treatment.
- •Participants with insufficient or incomplete medical records which prevent the assessment of baseline HAE-AS at the time of Takhzyro® initiation, as well as 6 months HAE-AS after treatment initiation.
- •Participants who are participating in an interventional clinical trial involving other HAE-C1INH treatments during the observation period.
研究组 & 干预措施
Participants with HAE-C1INH-Type 1 or HAE-C1INH-Type 2
All participants diagnosed with HAE-C1INH type 1 or type 2 will be included, and retrospective clinical data will be collected from patient medical records between 01 September 2025 and 28 February 2026. Participants who received Takhzyro® during the eligibility period beginning in 2021 will be observed across two periods: a pre-index period, defined as the time before the first dose of Takhzyro® (index date), used to establish baseline characteristics and disease activity, and a post-index period, defined as at least 6 months following the index date, used to assess treatment outcomes. The index date is defined as the date of the first Takhzyro® administration during the eligibility period.
干预措施: No intervention (Other)
结局指标
主要结局
Change in HAE- Activity Score (AS) From Pre to Post Index Period
时间窗: Up to 6 Months
HAE-AS is a patient-reported outcome (PRO) instrument used to measure the activity of HAE. It comprises 12 items assessing factors like attack frequency, emergency visits, and impact on daily life over a recall period. A higher HAE-AS score indicates greater disease activity. A score of 13 or higher suggests severe HAE activity, while a score below 12 indicates mild or low activity.
次要结局
- Change in Quality of Life (QoL) Scores From Pre to Post Index Period(Up to 6 months)
- Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period(Up to 6 months)
- Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response(From Baseline up to end of study (up to 6 months))