TY - JOUR
T1 - Evinacumab for children with homozygous familial hypercholesterolemia
T2 - a plain language summary
AU - Wiegman, Albert
AU - Pordy, Robert
N1 - Publisher Copyright:
© 2025 The Author(s). Published by Informa UK Limited, trading as Taylor & Francis Group.
PY - 2025
Y1 - 2025
N2 - Plain Language Summary: What is this summary about? This is a plain language summary explaining the results of a study assessing a treatment, called evinacumab, for children with a condition called homozygous familial hypercholesterolemia, also known as HoFH. HoFH is a rare genetic disorder that makes the liver less able to remove ‘bad’cholesterol (called low-density lipoprotein cholesterol, or LDL-C) from the body. High levels of LDL-C increase the risk of early development of serious heart problems. Only a small number of options are available to treat children with HoFH, and these do not always work well. Evinacumab is approved in the European Union, United Kingdom, Canada, and the USA as the first medicine approved for children with HoFH aged 5 to 11 years as an add-on to other therapies that lower LDL-C. In Japan, evinacumab is also approved for treatment of people with HoFH when given with other LDL-C-lowering therapies. Evinacumab was recently approved to treat HoFH in Brazil and Israel. What were the results of the study? Children with HoFH aged 5 to 11 years treated with evinacumab every 4 weeks for 24 weeks had LDL-C levels in their blood that were almost half what they were before treatment was started.The reduction in LDL-C was seen as early as 1 week after treatment was started and was maintained through 24 weeks of treatment.Overall, evinacumab was generally well accepted by most of the children in the study. Side effects were similar to those seen in adults and adolescents with HoFH. The most common side effects in children were throat pain, stomach pain, diarrhea, nausea,vomiting, headache, and symptoms like those seen in a common cold. What do the results mean? This study showed that evinacumab lowered ‘bad’ cholesterol in children of 5 to 11 years with HoFH. This is an abstract of the Plain Language Summary of Publication article. View the full Plain Language Summary PDF of this article to read the full-text.
AB - Plain Language Summary: What is this summary about? This is a plain language summary explaining the results of a study assessing a treatment, called evinacumab, for children with a condition called homozygous familial hypercholesterolemia, also known as HoFH. HoFH is a rare genetic disorder that makes the liver less able to remove ‘bad’cholesterol (called low-density lipoprotein cholesterol, or LDL-C) from the body. High levels of LDL-C increase the risk of early development of serious heart problems. Only a small number of options are available to treat children with HoFH, and these do not always work well. Evinacumab is approved in the European Union, United Kingdom, Canada, and the USA as the first medicine approved for children with HoFH aged 5 to 11 years as an add-on to other therapies that lower LDL-C. In Japan, evinacumab is also approved for treatment of people with HoFH when given with other LDL-C-lowering therapies. Evinacumab was recently approved to treat HoFH in Brazil and Israel. What were the results of the study? Children with HoFH aged 5 to 11 years treated with evinacumab every 4 weeks for 24 weeks had LDL-C levels in their blood that were almost half what they were before treatment was started.The reduction in LDL-C was seen as early as 1 week after treatment was started and was maintained through 24 weeks of treatment.Overall, evinacumab was generally well accepted by most of the children in the study. Side effects were similar to those seen in adults and adolescents with HoFH. The most common side effects in children were throat pain, stomach pain, diarrhea, nausea,vomiting, headache, and symptoms like those seen in a common cold. What do the results mean? This study showed that evinacumab lowered ‘bad’ cholesterol in children of 5 to 11 years with HoFH. This is an abstract of the Plain Language Summary of Publication article. View the full Plain Language Summary PDF of this article to read the full-text.
UR - http://www.scopus.com/inward/record.url?scp=85218252685&partnerID=8YFLogxK
U2 - 10.1080/14796678.2025.2461425
DO - 10.1080/14796678.2025.2461425
M3 - Article
C2 - 39931838
SN - 1479-6678
VL - 21
SP - 139
EP - 147
JO - Future cardiology
JF - Future cardiology
IS - 3
ER -