A Multi-national Real-world Outcomes and Treatment Patterns Study of Lanadelumab (Takhzyro) in Pediatric Patients with Hereditary Angioedema

What this Trial Is About

Hereditary Angioedema (HAE) is a rare condition causing sudden swelling under the skin and inside the body, such as in the belly, throat, or genitals. This swelling results from a temporary leak in blood vessels and does not cause itching or hives. HAE is classified by the level of a blood protein called C1 inhibitor (C1-INH), with some patients having normal levels and others having limited or insufficient levels. This research focuses on children with HAE who have C1-INH deficiency and have been treated with lanadelumab as a preventive therapy. The study aims to understand how well lanadelumab works in real-life settings by measuring how long children remain free of HAE attacks. The study is observational and collects existing data from medical records of pediatric patients aged 2 to under 12 years who started long-term prophylaxis treatment with lanadelumab during routine care. No new treatments are given as part of the study. Researchers will analyze various outcomes over a period of up to 13 months, including attack rates, attack severity and location, treatment patterns, healthcare usage, and any side effects or reactions to the injections. Participants' involvement includes reviewing their medical history and treatment records to assess the effectiveness and safety of lanadelumab. The main outcome measured is the percentage of children who remain free of HAE attacks during the study period. Other important data include healthcare visits, hospital stays, and adverse events. The study will provide insights based on up to 13 months of follow-up information per participant without requiring additional visits or interventions.

A Study of Lanadelumab in Children With Hereditary Angioedema (HAE) in Multiple Countries