Hereditary Angioedema (HAE)
Conditions
Keywords
Drug Therapy
Brief summary
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.
Interventions
OTHERNo intervention
This is a non-interventional study.
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
RETROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Participant is diagnosed with HAE-C1INH-Type 1 or HAE-C1INH-Type 2 and initiated on long-term prophylaxis (LTP) with Takhzyro® (lanadelumab). 2. Participant is aged greater than or equal to (\>=) 12 years at the time of Takhzyro® initiation. 3. Participant has received at least 6 months of continuous treatment with Takhzyro® before data abstraction.
Exclusion criteria
1. Participants who have normal C1INH function or HAE-nC1INH (formerly type III HAE). 2. Participants who discontinued Takhzyro® before completing 6 months of treatment. 3. 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. 4. Participants who are participating in an interventional clinical trial involving other HAE-C1INH treatments during the observation period.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in HAE- Activity Score (AS) From Pre to Post Index Period | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Quality of Life (QoL) Scores From Pre to Post Index Period | Up to 6 months | A study-specific quality-of-life (QoL) score will be developed. This custom score will be derived from select parameters conceptually aligned with the domains of the validated Angioedema (AE)-QoL (functioning, fatigue/mood, fear/shame, and nutrition), as captured in the available participant records. The goal is to approximate core aspects of health-related quality of life relevant to participants with recurrent angioedema, while ensuring feasibility and compliance within the constraints of retrospective data collection. |
| Change in HAE-C1INH Attack Frequency (number/month) From Pre to Post Index Period | Up to 6 months | HAE attack is defined as a discrete episode during which the participant progresses from no angioedema to symptoms of angioedema. A "change in HAE-C1INH attack frequency" refers to the reduction in the number of HAE attacks (number per month) after the implementation of treatments. |
| Number of Participants Categorized by Demographic and Clinical Predictors of Treatment Response | From Baseline up to end of study (up to 6 months) | Number of participants will be reported by baseline demographic (age, sex) and clinical characteristics (disease duration, baseline attack frequency). The relationship between these characteristics and change from baseline in HAE-AS score and attack frequency will be further explored using regression analysis, provided sufficient sample size and model stability. |
Countries
Saudi Arabia
Contacts
CONTACTTakeda Contact
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed