TOPLINE:
In a computer simulation of children with familial hypercholesterolemia, starting lipid-lowering treatment in childhood and continuing it for life produced the lowest projected lifetime cardiovascular risk. Delaying, interrupting, or stopping treatment was projected to increase the estimated risk, according to a brief report published in JACC.
METHODOLOGY:
- Researchers used a computer simulation model to estimate how the timing and continuity of lipid-lowering therapy would affect lifetime cardiovascular risk in children with heterozygous familial hypercholesterolemia.
- The model simulated health outcomes in 100,000 10-year-olds with familial hypercholesterolemia in the United States, scaled to represent approximately 15,900 real children over a lifetime.
- The analysis estimated lifetime exposure to LDL cholesterol from 2 to 100 years of age.
- Researchers compared six strategies: (1) no treatment; (2) usual care; (3) lipid-lowering therapy started at age 10 years and continued for life; (4) therapy started at age 18 and continued for life; (5) therapy started at age 10 then interrupted at age 18 and transitioned to usual care; and (6) therapy started at age 10 then permanently discontinued at age 18.
- Outcomes included cardiovascular disease (CVD) events, life years, quality-adjusted life years, and therapy-related adverse events. The models were run 100 times to report 95% uncertainty ranges.
TAKEAWAY:
- Untreated familial hypercholesterolemia produced a mean cumulative LDL-cholesterol exposure of 14,921 mg/dL-years, whereas lifelong lipid-lowering therapy starting at age 10 years significantly reduced the exposure.
- Lipid-lowering therapy started in childhood and continued for life prevented approximately 10,754 CVD events (95% uncertainty interval [UI], 10,188-11,343) and added 54,136 life-years (95% UI, 48,597-58,218) compared with no treatment.
- Therapy started in childhood and continued for life cut lifetime CVD event rates by nearly half compared with no lipid-lowering treatment (11.4 vs 22.4 per 1000 person-years) and showed rates comparable to the general population of US 10-year-olds (10.7 events per 1000 person-years).
- Starting therapy later or stopping it was associated with an increase in LDL-cholesterol exposure, more lifetime CVD events, and fewer life-years than continuous therapy starting in childhood. Starting therapy at age 18 prevented fewer events and added fewer life-years than starting in childhood.
IN PRACTICE:
“The best approach to prevent premature CVD [cardiovascular disease] in people with FH [familial hypercholesterolemia] is likely LLT [lipid-lowering therapy] initiated during childhood,” the researchers of the study write.
“These findings underline how essential it is to support patients with FH as they navigate the transition to adult medical care and ensure uninterrupted LLT into adulthood,” they add.
SOURCE:
The study was led by Yun-Lin Huang, PharmD, MPH, of Columbia University Irving Medical Center in New York City. It was published online January 21 as a brief report in JACC.
LIMITATIONS:
The study lacked population-level data on people with familial hypercholesterolemia in the US; thus, the researchers used a published regression model to assign disease status to survey participants. The findings depended on assumptions about patient and clinician behavior built into the model.
DISCLOSURES:
This study received funding from the National Institutes of Health. One author reported receiving royalties from an UpToDate online textbook. Another author reported being an employee of and holding equity in Verve Therapeutics, a subsidiary of Eli Lilly and Company, and receiving personal fees from three other organizations. One author reported being a Vice Chair of the US Preventive Services Task Force, and one author reported being an employee of a real-world evidence analytics company.
This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.
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