When Jack Howaniec was six years old, he had his first test of his cholesterol levels. Testing children for cholesterol levels is not routine, but Jack’s circumstances were special. His father has a condition called familial hypercholesterolemia (FH), a genetic disorder causing high levels of blood lipids, including cholesterol. The condition can lead to early heart attacks and strokes.

“Jack’s cholesterol came back sky-high,” says his mother, Barbara Howaniec, a psychiatric nurse practitioner in Portland, Maine. At eight, Jack started taking one of the cholesterol-lowering drugs known as statins; he’s now a healthy and upbeat twelve-year-old.

High cholesterol, or hyperlipidemia, is one of the most common medical problems faced by adults in the United States. Some have FH, but in others—generally those with milder abnormalities—the condition is related to obesity, diabetes, or other factors. It is typically treated first with diet and exercise, and if that doesn’t do the job, with statins.

Adolescents found to have high cholesterol first receive advice on getting more exercise and on improving their diets. (iStock)

Who Should Be Tested

Increasingly, children and adolescents, as well as adults, are being diagnosed with high cholesterol. Until recently, doctors would check children and teens only if they, like Jack, were known to have a risk factor. But some experts recommend that all young people be screened at certain ages. Those found to have high cholesterol could receive advice on diet and lifestyle changes, and if that doesn’t work, they could be offered statins.

This recommendation is controversial. Although there’s no question that high cholesterol can have serious consequences and that family history isn’t always enough to lead to a diagnosis, there’s little evidence so far that screening of children and adolescents at set times (ages 9–11 and 17–21) leads to better long-term health outcomes, says Laurel K. Leslie, MD, MPH, of Tufts University School of Medicine.

Available information suggests important benefits of statins for those with familial hypercholesterolemia, and short-term studies and some limited longer-term data haven’t shown worrisome side effects. However, the long-term safety and effectiveness of statins have not been comprehensively studied in children and adolescents, and research studies in adults have linked long-term use of the drugs with diabetes, she says. Also as with many other health conditions, screening can lead to some false positives, causing unnecessary treatment and anxiety for patients and their families.

Risks and Benefits

A two-year PCORI-funded study that will be completed this year takes up these issues, and more. It addresses critical questions that young people and their parents have about how best to control cholesterol.

Leslie and Sarah D. de Ferranti, MD, MPH, of Boston Children’s Hospital wanted to understand the risks and benefits of cholesterol screening and treatment strategies for young patients. The researchers also wanted to learn how a patient’s clinical and personal characteristics, and preferences, might affect which approach is best.

“This project is about better understanding what would be a good way to screen,” says de Ferranti.

Leslie, who specializes in comparative effectiveness research and stakeholder engagement, and de Ferranti, a pediatric cardiologist who cares for children with cholesterol disorders, worked with adolescents and parents to design an ambitious project. The researchers convened young adults and parents representing three groups: healthy adolescents, adolescents with FH, and obese adolescents.

Throughout the study, these stakeholders helped the research team refine its approach, for example, advising when to conduct focus groups versus individual interviews with patients and families. The group’s input also made interview and survey questions easier to understand. “They helped us with the wording, the flow, the understandability,” says de Ferranti.

The team also brought together a panel of 11 researchers, clinicians, and policymakers to provide advice on the project and help disseminate its eventual results.

During in-depth interviews, the study team asked 72 participants, both adolescents and parents, what health outcomes are most important to them and what their preferences are about screening and treatment. The results pointed the team to consider an area they had not previously recognized: the needs of young people as they move away from the family home. 

“From a provider point of view, even though I had felt pretty engaged with my patients, I learned quite a lot about the adolescent perspective,” says de Ferranti. “Adolescents go in and out of being grown-up in a very interesting and fluid way.”

The researchers next compiled data from existing studies of screening, hyperlipidemia, FH, and obesity. Using these data, they are employing a technique called decision analysis to model hypothetical patients’ likely long-term health outcomes in different screening and treatment scenarios. In the analysis, they are considering whether variations in a patient’s clinical characteristics (e.g., good health, FH, obesity) and sociodemographic profile (e.g., gender, race, ethnicity) influence which approach is most effective. The analysis is incorporating insights about patients’ preferences that the team gleaned earlier.

As teens become young adults, they must take on more responsibility for their own health care. (iStock)

Involving Teens in Their Own Care

The researchers hope the results will shed light on whether universal screening for adolescents is effective for identifying those who need treatment. The study is also examining how adolescents with chronic health conditions participate in their own care. Leslie and de Ferranti have been surprised by how often teens with chronic conditions have a poor understanding of their own health problems or the reasons for treatment.

“When you have a chronic illness that was diagnosed in childhood, your parents have made so many decisions for you,” says Leslie. She says doctors need to ensure that adolescents become informed, active participants in care decisions before they leave home and become responsible for their own care.

“On a number of fronts, we now understand better what it’s like to be an adolescent or young adult with a chronic illness,” Leslie says.

At stakeholder meetings, patients and parents revealed a variety of attitudes toward screening and treatment. Some focused on how angry they’d be if screening gave them a false positive result. Others worried about how little is known about the safety of statins in young people. Still others, including Jack’s mother, Barbara Howaniec, embraced universal screening. She says, “Anything that advances our ability to screen and prevent illness is extremely important.”

Patients and parents also discussed whether they’d prefer lifestyle management or drug treatment.

“It was powerful to hear the perspectives of the teens and parents who were on the panel,” says Barbara Howaniec. “Everybody had a different viewpoint. As a layperson, I found it amazing and inspiring to be a part of that group. The researchers always emphasize that what we have given to the study is just as important as the scientific piece.”

On a number of fronts, we now understand better what it’s like to be an adolescent or young adult with a chronic illness.

Laurel K. Leslie, MD, MPH Tufts University School of Medicine, PCORI-funded Study Principal Investigator

Use of the Study Results

The research team plans to submit its results for publication soon. It has already published one paper examining how using pediatric versus adult criteria among people aged 17 to 21 years might affect the number that would qualify for drug treatment of high cholesterol. A second paper estimates the prevalence of FH in the United States, a key factor for modeling the effect of screening at the population level. The research team reported a prevalence of 1 in 250, double what had been commonly estimated.

The team is planning to use the results of its study of teens and parents to develop a decision aid to help adolescents, parents, and providers make choices about screening and act on the results. The team is also working to get the word out on their work. Many members of the expert panel that advised the study are engaged in efforts to improve patient care, serving on work groups affiliated with the American Heart Association, the American Academy of Pediatrics, and the US Preventive Services Task Force. Those connections increase the project’s likelihood of influencing clinical practice and pediatric health policy, Leslie says. 

The team will also make efforts to reach out directly to young people. “The issue of transitions got our stakeholders excited,” says Leslie. So de Ferranti approached the Familial Hypercholesterolemia Foundation, which is considering adding to its website some advice, based on the study results, for young people navigating transitions to adulthood and adult health care.

Leslie and de Ferranti hope that in addition to providing insight into screening, their work will serve as a model for stakeholder decision-making for other chronic disorders of childhood and adolescence. “Clinicians may not be able to assume that adolescents and young adults understand their disorder,” Leslie says. Instead, clinicians may need to help young patients own their treatment plans.

“These clinical implications align with PCORI’s vision of activated, engaged patients who share in the decision-making process for their care,” Leslie says.

At a Glance

Comparing the Predicted Benefits and Harms of Using Adult versus Child Guidelines for Lipid Testing and Treatment in 17- to 21-Year-Olds

Principal Investigators: Laurel K. Leslie, MD, MPH & Sarah D. de Ferranti, MD, MPH

Goal: To compare the effectiveness of different lipid screening and treatment strategies for adolescents, taking into account sociodemographic and clinical differences among patient groups, as well as variation in adolescents’ and their families’ preferences about screening, treatment, and outcomes.

View Project Details | View Related Materials

Posted: March 29, 2016


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