Comparing the predicted benefits and harms of using adult versus child guidelines for lipid testing and treatment in 17- to 21-year-olds
For aim 2, we performed a literature review and evidence synthesis and analyzed national data to estimate FH prevalence as key inputs for simulation and statistical models comparing the effectiveness of pediatric versus adult cholesterol guidelines for (1) 1-year screening and treatment outcomes in...
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| Format: | eBook |
| Language: | English |
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[Washington, D.C.]
Patient-Centered Outcomes Research Institute (PCORI)
2018, [2018]
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| Series: | Final research report
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| Online Access: | |
| Collection: | National Center for Biotechnology Information - Collection details see MPG.ReNa |
| Summary: | For aim 2, we performed a literature review and evidence synthesis and analyzed national data to estimate FH prevalence as key inputs for simulation and statistical models comparing the effectiveness of pediatric versus adult cholesterol guidelines for (1) 1-year screening and treatment outcomes in AYAs (including patient-centered quality of life estimates [VAS] from aim 1); (2) 30-year cardiovascular disease incidence; and (3) life expectancy for AYAs, including FH patients. RESULTS: In aim 1, AYAs (n = 41) and parents (n = 35) from all groups (FH, OB, HA) perceived that all cholesterol screening scenarios would result in comparatively less than perfect health, with the high-risk result being furthest from perfect health (median = 0.50; IQR = 0.31-0.60). Notably, responses varied substantially, with minimum and maximum VAS ranging from 0.25 to 1.0 for lifestyle changes and 0.20 to 0.99 for statin. For the 20.4 million 20-year-olds in the United States, the Pencina model predicted the pediatric guidelines would produce lower average individual 30-year CVD event likelihood compared with adult guidelines (3.13% [95% CI, 2.97%-3.30%] versus 3.18% [95% CI, 3.00%-3.35%]), resulting in 10 200 fewer CVD events over 30 years. The primary benefit of screening from either approach accrued to AYA with FH, who experienced a 2.1-year increased survival. The pediatric guidelines provided additional benefit over the adult guidelines to the 530 000 AYA with LDL of 130 mg/dL to 189 mg/dL, increasing average life expectancy by 63 days. CONCLUSIONS: We found cholesterol screening and treatment impacts AYAs' perceptions of their current health, with broad variability in responses. The pediatric versus adult lipid guidelines would lead to more diagnoses and treatment, thereby lowering LDL and increasing survival. Long-term follow-up of AYAs undergoing cholesterol screening and treatment is needed to understand the real-world impact of cholesterol guidelines on patient-centered experiences and CVD events In aim 2, over 1 year the pediatric versus adult guidelines in the US population resulted in 240 000 additional AYAs receiving new statin prescriptions, 2 million AYAs being referred for intensive lifestyle interventions, and 600 000 fewer AYAs receiving standard-of-care lifestyle advice. Both guidelines yielded similar 1-year cholesterol levels for AYAs with high low-density lipoprotein cholesterol (LDL-C) levels, but pediatric guidelines yielded larger declines for those with moderately high LDL-C. When applying VAS scores, adult guidelines resulted in higher 1-year VAS-adjusted outcomes for diagnosis, treatment, and diagnosis plus treatment because the pediatric guidelines led to more diagnoses of high cholesterol and more statin and lifestyle interventions, which negatively affected perceived health. BACKGROUND: Early cholesterol screening and treatment in adolescents and young adults (AYAs) may improve cardiovascular disease (CVD) outcomes from lifelong high cholesterol; however, it remains controversial, and pediatric and adult lipid guidelines conflict. OBJECTIVES: (Aim 1) To assess variation in patient-centered preferences about cholesterol screening and treatment from 17- to 21-year-old AYAs with familial hypercholesterolemia (FH), with obesity (OB), and also healthy (HA) and parents. (Aim 2) To compare cholesterol guideline strategies for AYAs. METHODS: A diverse 20-member stakeholder panel informed our approach. For aim 1, we conducted an observational study of preferences using semistructured interviews incorporating vignettes, visual analogue scales (VAS), and open-ended questions about cholesterol screening and treatment scenarios. |
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| Physical Description: | 1 PDF file (121 pages) illustrations |