Long-term safety and effectiveness of evinacumab in people with homozygous familial hypercholesterolemia: a plain language summary

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Plain Language SummaryWhat is this summary about?This summary explains the results of a study looking at the long-term effects of a treatment called evinacumab. This study included people aged over 12 years with homozygous familial hypercholesterolemia, also known as HoFH. HoFH is a rare genetic disorder that makes the liver less able to remove excess ‘bad’ cholesterol (called low-density lipoprotein cholesterol, or LDL-C) from the body. LDL-C is considered ‘bad’ cholesterol because it increases the risk of heart disease and stroke. High levels of LDL-C increase the risk of developing serious heart problems early in life. Treatment options for lowering LDL-C often do not work very well for people with HoFH. Evinacumab is a medicine that is approved for the treatment of HoFH as an add-on to other therapies that lower LDL-C. The effect of evinacumab on heart problems such as heart attacks, stroke, or death is not known. In the UK, evinacumab is approved for people with HoFH aged 6 months and above. In Canada and the USA, evinacumab is approved for people with HoFH aged 5 years and above. In the European Union/European Economic Area, evinacumab is approved for people with HoFH aged 6 months and above. In Japan, evinacumab is approved for all ages of people with HoFH. More recently, evinacumab has also been approved in Brazil and Israel.What were the results?People with HoFH who took part in the study were treated with evinacumab every 4 weeks for up to 44 months.After 6 months of treatment, LDL-C levels were almost 44% lower than when people started the study.Treatment-emergent adverse events, also called TEAEs, were reported by 80% of people in the study but most of these were mild (23.3%) or moderate (41.4%) in severity. The most commonly reported side effects which happened during the study were common cold, COVID-19, headache, flu-like illness, general pain, back pain, nausea, and cough. The study researchers determined that most of these reported side effects (91%) were not related to the use of evinacumab.No serious TEAEs were considered related to evinacumab treatment. Nearly all of the people (97%) could continue with the study treatment and, based on this, the researchers concluded that evinacumab was generally well tolerated.What do the results mean?This study showed that evinacumab lowered ‘bad’ cholesterol in people with HoFH, and that this result lasted for a long period of time.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

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.