Pediatrics
There are nearly 75 million children and adolescents in the United States, representing almost one-quarter, or 25%, of the American population. Health issues can have a significant impact on a child’s learning and development; nearly 4% of children have missed 11 or more days of school because of injury or illness in the past 12 months. From infancy and childhood through adolescence and emerging adulthood, children and their caregivers have changing needs and preferences for care.
To address this, PCORI funds patient-centered comparative clinical effectiveness research and related projects that focus on pediatric health. PCORI funds research on pediatric health that addresses questions important to patients, caregivers and the broader healthcare community, generating meaningful evidence that enables better-informed health and healthcare decisions.
PCORI is actively involving patients affected by pediatric health and health decision-makers as collaborators in PCORI’s work, ensuring research findings are easily accessible and usable by those who need them.
Study Results that Support Better-Informed Decisions
The COMBINE TRIAL: Improving Treatment for Pediatric Crohn’s Disease
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Pediatric patients receiving the tumor necrosis factor (TNF) inhibitor adalimumab, combined with a low dose of methotrexate, a second immunosuppressant, to treat Crohn’s disease did better than those treated with adalimumab alone, according to findings of a PCORI-funded study published in Gastroenterology. However, patients taking infliximab, another anti-TNF medication, had similar outcomes with or without methotrexate.
The results of The COMBINE Trial — led by Michael Kappelman, MD, MPH, from the School of Medicine at the University of North Carolina at Chapel Hill — provide important evidence to help answer questions about which medicines and combinations work best to treat Crohn’s disease in children. The lifelong inflammatory bowel disease is harder to treat in pediatric patients, but left untreated can impair growth, psychosocial development and puberty.
Telehealth-Based Referrals Improve Access to Hearing Care for Children in Rural Areas
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Children in school-based hearing screening programs who received telehealth-based specialist referrals experienced follow-up care up to 17.6 times faster, compared with children who received standard primary care referrals, according to results from the PCORI-funded Hearing Norton Sound Study published in Lancet Global Health. The randomized controlled trial with about 1,500 children in Alaska was led by Susan D. Emmett, MD, MPH, and Samantha Kleindienst Robler, PhD, AuD, from the Center for Hearing Health Equity at the University of Arkansas for Medical Sciences.
The study, which was conducted in 15 rural Alaskan communities between 2017 and 2020, is considered to be the first to demonstrate that telemedicine can reduce a key rural health disparity in access to care. The researchers also suggest that the benefits of telemedicine could translate to other preventive school-based services to improve specialty health care for children in rural areas.
2024 PCORI Annual Meeting Breakout Session Spotlights
Partnerships in Research: Expanding Mental Health Care for Children and Youth
Make Way for Mental Health: Pathways to Promoting Mental Wellness for Children and Youth
Evidence for Decisions from PCORI-Funded Studies
Treating Resistant Kawasaki Disease
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Kawasaki disease (KD) is a rare disease that most commonly affects children. KD can be treated, but some children have resistant KD and do not respond to initial treatment. A PCORI-funded study compared the safety and effectiveness of two treatments for resistant KD. This study is the largest clinical trial to date on treatment for resistant KD.
A pair of Evidence Updates provides information about second treatment options for children with KD whose fever doesn’t go away after their first treatment, and to help inform clinicians and parents as they make treatment decisions.
Choosing the Best Option for Managing Mild Persistent Asthma in Children
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Asthma is a common chronic illness among children in the US, with disproportionate impact among racial and ethnic minorities. A PCORI-funded study, which focused exclusively on African-American children, who have traditionally been underserved in research and have had worse asthma-related health outcomes than other children, looked at two different approaches to managing mild persistent asthma.
A pair of Evidence Updates is now available to help support conversations between parents and clinicians about choosing the best option for managing mild persistent asthma in children.
Systematic Reviews
Report Evaluates Treatments for Infantile Epilepsies
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Uncontrolled seizures in children up to 36 months old have serious short-term health risks and may be associated with substantial developmental, behavioral, and psychological impairments.
A systematic review, Management of Infantile Epilepsies, supported by PCORI through a research partnership with the Agency for Healthcare Research and Quality, evaluates the effectiveness, comparative effectiveness, and harms of several treatments for infantile epilepsies.
The main findings of the review include the following: Levetiracetam may be effective in increasing seizure freedom. Ketogenic and modified Atkins diets may reduce seizure frequency. Surgical procedures (Hemispherectomy/Hemispherotomy and Non-Hemispheric procedures) may lead to seizure freedom in some infants.
Pediatrics Study Spotlights
Increasing Community Participation for People of All Abilities
People living with intellectual and developmental disabilities (IDD) and their families often face barriers to participating in their own communities. These challenges can include a building’s design that makes entry difficult to misperceptions about a person’s behavior. An occupational therapist is leading a PCORI-funded project to make community participation easier for people with IDD and their families.
Narrow- versus Broad-Spectrum Antibiotics for Common Infections in Children
When bacteria cause an ear, nose, or throat infection in children, a clinician may prescribe narrow- or broad-spectrum antibiotics. This study found that narrow-spectrum antibiotics worked just as well as broad-spectrum antibiotics. Also, children taking narrow-spectrum antibiotics had fewer side effects than children taking broad-spectrum antibiotics.
Using Computer Alert Systems in the Emergency Room to Screen for Child Abuse
Hospital staff look for signs of abuse in children cared for in emergency rooms but don’t always order standard tests that help detect child abuse. This study found that clinicians were more likely to check for and report suspected abuse when prompted by an alert system built into the hospital’s electronic health record. PCORI is now funding a project to implement the system in other children’s hospitals.
PCORI Stories
Enhancing the Delivery of Mental Health Services to Children in Underserved Areas
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While many individuals have come to rely on telehealth services as a way to receive mental health care during the COVID-19 pandemic, children in lower-income families have experienced barriers to mental health care access that existed prior to the pandemic, and in many ways were exacerbated by it.
This PCORI Story highlights findings from a PCORI-funded study in Seattle that was designed to address these parents’ concerns using telehealth platforms and the community partnerships that came out of it.
More Study Results that Support Better-Informed Decisions
Informing Treatment of Juvenile Arthritis
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Clinicians and patients have been uncertain about the optimal time to start biologics for polyarticular juvenile idiopathic arthritis (pJIA). However, results from a PCORI-funded study, published in Arthritis & Rheumatology, provide useful new evidence about first-line treatment options for this disease. Researchers compared the effectiveness of three treatment approaches among 400 children with pJIA.
The approaches included starting patients on a non-biologic disease-modifying antirheumatic drug (DMARD) only, starting patients with a DMARD and a biologic medication, and starting patients with a biologic only. After one year, the approaches did not differ significantly in achieving clinically inactive disease without the use of glucorticoids, and patients reported decreased pain interference levels and increased mobility with all three approaches.
The Effect of Treatment Duration for Newborn Infants Who Have Seizures
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Every year, about 16,000 newborns in the United States have seizures. Medications can treat seizures but can also have serious side effects and may harm children’s brain development. A PCORI-funded study at University of Michigan compared functional development outcomes of 270 newborns who developed seizures due to acute brain injury in the first days to weeks after birth and were treated with antiseizure medication (ASM) prior to hospital discharge and those who continued ASM after hospital discharge.
Reporting in JAMA Neurology, the study found that after two years, children in both groups did not differ significantly in their functional development and risk of epilepsy. These results support discontinuation of ASM prior to hospital discharge for most newborns with acute symptomatic neonatal seizures.