Lanadelumab treatment helps prevent swelling attacks in children aged 2 to 11 years with hereditary angioedema (HAE): a plain language summary of the SPRING Study

Summary What is this summary about? This is a summary of a clinical trial called the SPRING Study. The study looked at a treatment for children aged 2 to 11 years with a disease called hereditary angioedema, or HAE for short. The treatment is called lanadelumab. It is used to prevent swelling attacks of the skin or other tissues in people with HAE. What happened in the SPRING Study? The SPRING Study included 21 children who had HAE. Before starting lanadelumab treatment, the children’s symptoms were tracked for 4 to 12 weeks. This was to help understand how HAE was affecting each child. The researchers used data from completed studies of lanadelumab in adults to help select the doses for children. The study had 2 parts (Part A and Part B) that lasted for 26 weeks each. In Part A, children aged 2 to 5 years had lanadelumab injections every 4 weeks and children aged 6 to 11 years had lanadelumab injections every 2 weeks. In Part B, some children aged 6 to 11 years who did not have any attacks in the first part of the study changed to receiving lanadelumab injections every 4 weeks instead of every 2 weeks. This helped researchers understand if these children could still be protected if they had lanadelumab treatment less often. What are the results of the SPRING Study? Lanadelumab prevented HAE attacks in the children during the study. Before starting lanadelumab treatment, all 21 children had at least 1 HAE attack in the previous 3 months, and each child had an average of 24 attacks each year. While they were receiving treatment, 16 of 21 children did not have any HAE attacks. After 1 year of lanadelumab treatment, each child had on average about 1 attack each year. The average duration of an HAE attack in the children receiving lanadelumab was also half as long as before they started lanadelumab treatment. The most common side effect was mild pain at the site of the lanadelumab injection. This happened in 6 of the 21 children. Most of the children had a better quality of life after starting lanadelumab treatment. What do the results of the SPRING Study mean? These results suggest that lanadelumab treatment is safe and works well to help prevent HAE attacks in children aged 2 to 11 years. Children in this age group are likely to benefit from taking lanadelumab treatment when their doctor thinks it is the most suitable medicine for them. After seeing the results of the SPRING Study, many medical agencies around the world agreed that lanadelumab could be used in children as young as 2 years of age. Where can I find the original article on which this summary is based? The original article is called “Lanadelumab in patients 2 to less than 12 years old with hereditary angioedema: results from the phase 3 SPRING Study.” You can read the article that was published in the Journal of Allergy and Clinical Immunology: In Practice in 2024 for free here: https://www.jaci-inpractice.org/article/S2213-2198(23)01019-X/fulltext Who is this article for? The aim of this plain language summary is to help people without specialist knowledge of HAE to understand the findings of the SPRING Study. Who sponsored the study? The SPRING Study was sponsored by Takeda Pharmaceuticals.