The Study
The Effect of PCSK9 Inhibitors on LDL-C Target Achievement in Patients with Homozygous Familial Hypercholesterolemia: A Retrospective Cohort Analysis
This study is like looking back at medical records to see what happened to 28 patients with a rare cholesterol condition after they started a new drug. It can tell us what was observed, but it can't prove the drug caused the results because other treatments and factors might have played a role.
Analysis score
Maximum 72 for a cohort study.
Where the score came from
Doctors gave special cholesterol drugs (PCSK9 inhibitors) to kids and young adults with a rare, severe form of high cholesterol to see if it helped lower their levels enough.
Where does this study sit?
Reviews of RCTs (Meta-analyses)
Max 100Randomized Trials
Max 90Reviews of Cohort Studies
Max 85Cohort Studies
Max 72Reviews of Case-Control Studies
Max 63Case-Control Studies
Max 58Cross-Sectional & Case Series
Max 50Expert Opinion
Max 533 / 100
Quality score
Groups of people are followed over time to see who develops an outcome. Strong for identifying risk factors and associations, but cannot prove causation as firmly as RCTs.
Key takeaways
Summary
Based on the study abstract and findings.
- 1The drug doesn’t help most of these patients much, especially those with the worst genetic form.
- 2Staying on it for over a year without benefit may waste time and money.
- 3On average, cholesterol went down by less than 9%.
- 47 out of 10 didn’t even get the minimum benefit.
- 5None reached the safe cholesterol level.
- 6If their body couldn’t make working cholesterol receptors, the drug barely worked (-5%).
- 7If they had partly working receptors, it worked better (-20%).
Score breakdown, methodology, conflicts of interest, evidence analysis & raw study data
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.
People with a rare genetic cholesterol disorder who still have a little bit of working cholesterol-cleaning system in their body tend to respond better to a certain type of cholesterol drug, lowering their bad cholesterol by about 20% on average.
Not medical advice. For informational purposes only. Always consult a qualified healthcare professional before making health decisions.