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
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.
No biological mechanisms were identified in this study. This may be an epidemiological, observational, or survey-based study that reports associations rather than proposing causal biological pathways.
Systematic Reviews & Meta-Analyses
Max 100Randomized Controlled Trials
Max 90Cohort Studies
Max 72Case-Control Studies
Max 58Cross-Sectional Studies
Max 44Case Reports & Case Series
Max 30Expert Opinion & Narrative Reviews
Max 533 / 72
Evidence 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.
Not medical advice. For informational purposes only. Always consult a healthcare professional. Terms
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.
No biological mechanisms were identified in this study. This may be an epidemiological, observational, or survey-based study that reports associations rather than proposing causal biological pathways.
Systematic Reviews & Meta-Analyses
Max 100Randomized Controlled Trials
Max 90Cohort Studies
Max 72Case-Control Studies
Max 58Cross-Sectional Studies
Max 44Case Reports & Case Series
Max 30Expert Opinion & Narrative Reviews
Max 533 / 72
Evidence 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.
Publication
Authors
Alshahrani A, Kholaif N, Al-Khnifsawi M, Zarif H, Mahzari M
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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.