Do these fancy cholesterol drugs work for kids with super high cholesterol?
The Effect of PCSK9 Inhibitors on LDL-C Target Achievement in Patients with Homozygous Familial Hypercholesterolemia: A Retrospective Cohort Analysis
Not medical advice. For informational purposes only. Always consult a healthcare professional. Terms
Surprising Findings
PCSK9 inhibitors — known to slash LDL by up to 60% in regular patients — barely moved the needle in HoFH, with only an 8.6% average drop.
These drugs are hailed as breakthroughs for high cholesterol, so seeing such weak results in HoFH contradicts their reputation and marketing.
Practical Takeaways
If you or a loved one has HoFH, ask for genetic testing to determine LDLR mutation type before starting PCSK9 inhibitors.
Not medical advice. For informational purposes only. Always consult a healthcare professional. Terms
Surprising Findings
PCSK9 inhibitors — known to slash LDL by up to 60% in regular patients — barely moved the needle in HoFH, with only an 8.6% average drop.
These drugs are hailed as breakthroughs for high cholesterol, so seeing such weak results in HoFH contradicts their reputation and marketing.
Practical Takeaways
If you or a loved one has HoFH, ask for genetic testing to determine LDLR mutation type before starting PCSK9 inhibitors.
Publication
Journal
Advances in Therapy
Year
2024
Authors
Awad Alshahrani, Naji Kholaif, Mutaz Al-khnifsawi, Hawazen A. Zarif, Moeber M. Mahzari
Related Content
Claims (6)
Adding PCSK9 drugs to cholesterol-lowering statins can slash bad cholesterol by as much as 81% in people with high cholesterol.
For people with a rare cholesterol condition called HoFH who are already on cholesterol meds, adding a newer drug (PCSK9 inhibitor) barely lowers bad cholesterol for most — and 7 out of 10 don’t get enough benefit to keep taking it.
People with a rare genetic form of high cholesterol caused by two broken copies of the LDLR gene don’t get much benefit from PCSK9 inhibitor drugs — most see almost no drop in their bad cholesterol.
People with a rare cholesterol condition stayed on a pricey cholesterol drug for about a year on average, even though it didn’t help much — doctors might just keep prescribing it because they’re hesitant to stop treatments that aren’t working.
Even when people with a rare genetic cholesterol disorder take powerful new drugs called PCSK9 inhibitors along with their usual treatments, none of them got their bad cholesterol down to the recommended healthy levels.