Principles for Primary Care Screening in the Context of Population Health.

A key component of primary care pediatrics is health promotion through screening: applying a test or procedure to detect a previously unrecognized disease or disease risk. How do we decide whether to screen? In 1965, Wilson and Jungner published an influential set of screening principles focused on the health problem's importance, the screening tool's performance, and the evidence for treatment efficacy. However, if we want realistic estimates of the population effects of routine screening, we must also account for the health care system's real-world functioning and disparities in care. We offer revised principles to guide discussions about routine screening in the primary care setting. We add to Wilson and Jungner's principles: 1. A focus on life course epidemiology and its consequences for population health, 2. A need to screen for the early stages of chronic health problems, 3. A concern for screening's acceptability to providers and the community, 4. A recommendation for estimating the uncertainty in benefits and harms in evaluating screening, 5. Inclusion of systematic plans for population data collection and monitoring, and 6. Recognition that achieving population health improvement requires a high-performing system with sufficient throughput and monitoring to deliver accessible, affordable, and effective care, especially for the groups experiencing the greatest inequities in access. Above all, instead of assuming best practices in treatment delivery and monitoring after screening, we argue for realism about the health care system functioning in routine practice.

Celebrating 75 Years of Innovation in Pediatrics.

The inaugural issue of Pediatrics was published in 1948. Although the journal has remained steadfast in its mission of helping pediatricians and other child health care clinicians improve outcomes for children and families, the approach it uses to achieve its mission continues to evolve. This special article provides a broad historical overview of changes to the journal, focusing on the last 25 years, including the move to online publication and use of social media, the adoption of new article types, the commitment to transparency, the expansion of the editorial board, and the commitment to diversity, equity, inclusion, and justice. These changes ensure that Pediatrics remains timely and relevant for everyone invested in improving child health outcomes.

Trends and Economic Implications of Disparities in Postoperative Pneumonia.

BACKGROUND: Postoperative pneumonia is the third most common surgical complication and can seriously impair surgical rehabilitation and lead to related morbidity and mortality. We evaluated the temporal trends in racial and ethnic disparities in postoperative pneumonia and quantified the economic burden resulting from these inequalities in the United States. METHODS: This population-based study includes 195 028 children (weighted to 964 679) admitted for elective surgery across 5340 US hospitals reporting to the Nationwide Inpatient Sample between 2010 and 2018. We estimated the risk-adjusted incidence of postoperative pneumonia, comparing racial and ethnic groups. We also quantified the inflation-adjusted hospital costs attributable to racial and ethnic disparities in postoperative pneumonia. RESULTS: The risk-adjusted rates of pneumonia declined across all racial and ethnic categories, with Black children having the lowest annual rate of decline (Black: 0.03 percentage points, Hispanic: 0.05 percentage points, white: 0.05 percentage points). The risk-adjusted rates of pneumonia trended consistently higher for Black and Hispanic children, relative to white children, throughout the study period (Black versus white: relative risk, 1.31 (95% confidence interval, 1.14-1.51), P < .01; Hispanic versus white: relative risk, 1.16 (95% confidence interval, 1.02-1.32), P = .02). These disparities did not narrow significantly over time. During the study period, the excess hospitalization cost attributable to racial and ethnic disparities in postoperative pneumonia was $24 533 458 for Black children and $26 200 783 for Hispanic children (total, $50 734 241). CONCLUSIONS: Against the backdrop of decreasing postoperative pneumonia, Black and Hispanic children continue to experience higher rates compared with white children. These persistent disparities in postoperative pneumonia were associated with considerable excess cost of surgical care.

Improving Patient-Provider Continuity in a Large Urban Academic Primary Care Network.

OBJECTIVE: Although patient-provider continuity improves care delivery and satisfaction, poor continuity with primary care providers (PCP) often exists in academic centers. We aimed to increase patient empanelment from 0% to 90% and then increase the percent of well-child care (WCC) visits scheduled with the PCP from 25.6% to 50%, without decreasing timely access that might result if patients waited for PCP availability. METHODS: Nationwide Children's Hospital Primary Care Network cares for >120,000 mostly Medicaid-enrolled patients across 13 offices. Before 2017, patients were empaneled to an office, not individual PCPs. We empaneled patients to PCPs, reduced provider floating, implemented continuity-promoting scheduling guidelines, scheduled future WCC visits for patients ≤15 months during check-in for their current one, and encouraged online scheduling. We tracked the percentage of all WCC visits that were scheduled with the patient's PCP and the percentage of subsequent WCC visits for patients ≤15 months that were scheduled during the current visit, and provided feedback to schedulers. We followed emergency department (ED) utilization and visit show rates. WCC visit completion rates were tracked using HEDIS metrics. RESULTS: Patient empanelment increased from 0% to >90% (P < .001). Patient-provider WCC continuity increased from 25.6% to 54.7% (P < .001). A 20.5% decrease in ED utilization rate was associated with continuity project initiation. Empaneled patients demonstrated higher show rates (76.9%) versus unempaneled patients (71.4%; P < .001). WCC completion rates increased from 52.6% to 60.7%. CONCLUSIONS: WCC continuity more than doubled after interventions and was associated with decreased ED utilization, higher show rates, and increased timely WCC completion.

Anti-Poverty Medicine Through Medical-Financial Partnerships: A New Approach to Child Poverty.

Poverty threatens child health. In the United States, financial strain, which encompasses income and asset poverty, is common with many complex etiologies. Even relatively successful antipoverty programs and policies fall short of serving all families in need, endangering health. We describe a new approach to address this pervasive health problem: antipoverty medicine. Historically, medicine has viewed poverty as a social problem outside of its scope. Increasingly, health care has addressed poverty's downstream effects, such as food and housing insecurity. However, strong evidence now shows that poverty affects biology, and thus, merits treatment as a medical problem. A new approach uses Medical-Financial Partnerships (MFPs), in which healthcare systems and financial service organizations collaborate to improve health by reducing family financial strain. MFPs help families grow assets by increasing savings, decreasing debt, and improving credit and economic opportunity while building a solid foundation for lifelong financial, physical, and mental health. We review evidence-based approaches to poverty alleviation, including conditional and unconditional cash transfers, savings vehicles, debt relief, credit repair, financial coaching, and employment assistance. We describe current national MFPs and highlight different applications of these evidence-based clinical financial interventions. Current MFP models reveal implementation opportunities and challenges, including time and space constraints, time-sensitive processes, lack of familiarity among patients and communities served, and sustainability in traditional medical settings. We conclude that pediatric health care practices can intervene upon poverty and should consider embracing antipoverty medicine as an essential part of the future of pediatric care.

Economic Trends of Racial Disparities in Pediatric Postappendectomy Complications.

BACKGROUND: Despite unparalleled advances in perioperative medicine, surgical outcomes remain poor for racial minority patients relative to their white counterparts. Little is known about the excess costs to the health care system related to these disparities. METHODS: We performed a retrospective analysis of data from the Nationwide Inpatient Sample between 2001 and 2018. We included children younger than 18 years admitted with appendicitis who underwent an appendectomy during their hospital stay. We examined the inflation-adjusted hospital costs attributable to the racial disparities in surgical complications and perforation status, focusing on differences between non-Hispanic white patients and non-Hispanic Black patients. RESULTS: We included 100 639 children who underwent appendectomy, of whom 89.9% were non-Hispanic white and 10.1% were non-Hispanic Black. Irrespective of perforation status at presentation, surgical complications were consistently higher for Black compared with white children, with no evidence of narrowing of the racial disparity gap over time. Black children consistently incurred higher hospital costs (median difference: $629 [95% confidence interval: $500-$758; P < .01). The total inflation-adjusted hospital costs for Black children were $518 658 984, and $59 372 044 (11.41%) represented the excess because of the racial disparities in perforation rates. CONCLUSIONS: Although all patients had a progressive decline in post appendectomy complications, Black children consistently had higher rates of complications and perforation, imposing a significant economic burden. We provide an empirical economic argument for sustained efforts to reduce racial disparities in pediatric surgical outcomes, notwithstanding that eliminating these disparities is simply the right thing to do.

Incorporation of Social Risk in US Preventive Services Task Force Recommendations and Identification of Key Challenges for Primary Care.

IMPORTANCE: In its mission to improve health, the US Preventive Services Task Force (USPSTF) recognizes the strong relationship between a person's health and social and economic circumstances as well as persistent inequities in health care delivery. OBJECTIVE: To assess how social risks have been considered in USPSTF recommendation statements and identify current gaps in evidence needed to expand the systematic inclusion of social risks in future recommendations. EVIDENCE: The USPSTF commissioned a technical brief that reviewed existing literature on screening and interventions for social risk factors and also audited the 85 USPSTF recommendation statements active as of December 2019 to determine how social risks were addressed in clinical preventive services recommendations. FINDINGS: Among the 85 USPSTF recommendation statements reviewed, 14 were focused on preventive services that considered health-related social risks. Social risks were commonly referenced in parts of USPSTF recommendations, with 57 of 85 recommendations including some comment on social risks within the recommendation statement, although many comments were not separate prevention services. Social risks were commented on in USPSTF recommendations as part of risk assessment, as a marker of worse health outcomes from the condition of focus, as a consideration for clinicians when implementing the preventive service, and as a research need or gap on the topic. CONCLUSIONS AND RELEVANCE: This report identified how social risks have been considered in the USPSTF recommendation statements. It serves as a benchmark and foundation for ongoing work to advance the goal of ensuring that health equity and social risks are incorporated in USPSTF methods and recommendations.

Health and economic outcomes of newborn screening for infantile-onset Pompe disease.

PURPOSE: To estimate health and economic outcomes associated with newborn screening (NBS) for infantile-onset Pompe disease in the United States. METHODS: A decision analytic microsimulation model simulated health and economic outcomes of a birth cohort of 4 million children in the United States. Universal NBS and treatment was compared with clinical identification and treatment of infantile-onset Pompe disease. Main outcomes were projected cases identified, costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) over the life course. RESULTS: Universal NBS for Pompe disease and confirmatory testing was estimated to cost an additional $26 million annually. Additional medication costs associated with earlier treatment initiation were $181 million; however, $8 million in medical care costs for other services were averted due to delayed disease progression. Infants with screened and treated infantile-onset Pompe disease experienced an average lifetime increase of 11.66 QALYs compared with clinical detection. The ICER was $379,000/QALY from a societal perspective and $408,000/QALY from the health-care perspective. Results were sensitive to the cost of enzyme replacement therapy. CONCLUSION: Newborn screening for Pompe disease results in substantial health gains for individuals with infantile-onset Pompe disease, but with additional costs.

Changes in Provider Type and Prescription Refills Among Privately Insured Children and Youth With ADHD.

OBJECTIVE: The aim of this paper is to understand associations between age and health care provider type in medication continuation among transition-aged youth with ADHD. METHOD: Using an employer-sponsored insurance claims database, we identified patients with likely ADHD and receipt of ADHD medications. Among patients who had an outpatient physician visit at baseline and maintained enrollment at follow-up 3 years later, we evaluated which ones continued to fill prescriptions for ADHD medications. RESULTS: Patients who were younger at follow-up more frequently continued medication (77% of 11-12 year-olds vs. 52% of 19-20 year-olds). Those who saw a pediatric provider at baseline and follow-up more frequently continued to fill ADHD medication prescriptions than those who saw a pediatric provider at baseline and non-pediatric providers at follow-up (71% vs. 53% among those ages 15-16 years at follow-up). CONCLUSION: Adolescents and young adults with ADHD who changed from pediatric to exclusively non-pediatric providers less frequently continued to receive ADHD medications.

Developing Primary Care-Based Recommendations for Social Determinants of Health: Methods of the U.S. Preventive Services Task Force.

The purpose of the U.S. Preventive Services Task Force (USPSTF) is to provide evidence-based recommendations on primary care screening, behavioral counseling, and preventive medications. A person's health is strongly influenced by social determinants of health, such as economic and social conditions; therefore, preventive recommendations that address these determinants would be ideal. However, differing social determinants have been proposed by a wide range of agencies and organizations, little prevention evidence is available, and responsible parties are in competition, all of which make the creation of evidence-based prevention recommendations for social determinants of health challenging. This article highlights social determinants already included in USPSTF recommendations and proposes a process by which others may be considered for primary care preventive recommendations. In many ways, incorporating social determinants of health into evidence-based recommendations is an evolving area. By reviewing the evidence on the effects of screening and interventions on social determinants relevant to primary care, the USPSTF will continue to provide recommendations on clinical preventive services to improve the health of all Americans.

Early Check: translational science at the intersection of public health and newborn screening.

BACKGROUND: Newborn screening (NBS) occupies a unique space at the intersection of translational science and public health. As the only truly population-based public health program in the United States, NBS offers the promise of making the successes of translational medicine available to every infant with a rare disorder that is difficult to diagnose clinically, but for which strong evidence indicates that presymptomatic treatment will substantially improve outcomes. Realistic NBS policy requires data, but rare disorders face a special challenge: Screening cannot be done without supportive data, but adequate data cannot be collected in the absence of large-scale screening. The magnitude and scale of research to provide this expanse of data require working with public health programs, but most do not have the resources or mandate to conduct research. METHODS: To address this gap, we have established Early Check, a research program in partnership with a state NBS program. Early Check provides the infrastructure needed to identify conditions for which there have been significant advances in treatment potential, but require a large-scale, population-based study to test benefits and risks, demonstrate feasibility, and inform NBS policy. DISCUSSION: Our goal is to prove the benefits of a program that can, when compared with current models, accelerate understanding of diseases and treatments, reduce the time needed to consider inclusion of appropriate conditions in the standard NBS panel, and accelerate future research on new NBS conditions, including clinical trials for investigational interventions. TRIAL REGISTRATION: Clinicaltrials.gov registration # NCT03655223 . Registered on August 31, 2018.

Using Decision Analysis to Support Newborn Screening Policy Decisions: A Case Study for Pompe Disease.

Background: Newborn screening is a public health program to identify conditions associated with significant morbidity or mortality that benefit from early intervention. Policy decisions about which conditions to include in newborn screening are complex because data regarding epidemiology and outcomes of early identification are often incomplete. Objectives: To describe expected outcomes of Pompe disease newborn screening and how a decision analysis informed recommendations by a federal advisory committee. Methods: We developed a decision tree to compare Pompe disease newborn screening with clinical identification of Pompe disease in the absence of screening. Cases of Pompe disease were classified into three types: classic infantile-onset disease with cardiomyopathy, nonclassic infantile-onset disease, and late-onset disease. Screening results and 36-month health outcomes were projected for classic and nonclassic infantile-onset cases. Input parameters were based on published and unpublished data supplemented by expert opinion. Results: We estimated that screening 4 million babies born each year in the United States would detect 40 cases (range: 13-56) of infantile-onset Pompe disease compared with 36 cases (range: 13-56) detected clinically without screening. Newborn screening would also identify 94 cases of late-onset Pompe disease that might not become symptomatic for decades. By 36 months, newborn screening would avert 13 deaths (range: 8-19) and decrease the number of individuals requiring mechanical ventilation by 26 (range: 20-28). Conclusions: Pompe disease is a rare condition, but early identification can improve health outcomes. Decision analytic modeling provided a quantitative data synthesis that informed the recommendation of Pompe disease newborn screening.

Risk Assessment for Cardiovascular Disease With Nontraditional Risk Factors: US Preventive Services Task Force Recommendation Statement.

Importance: Cardiovascular disease (CVD) is the most common cause of death among adults in the United States. Treatment to prevent CVD events by modifying risk factors is currently informed by the Framingham Risk Score, the Pooled Cohort Equations, or similar CVD risk assessment models. If current CVD risk assessment models could be improved by adding more risk factors, treatment might be better targeted, thereby maximizing the benefits and minimizing the harms. Objective: To update the 2009 US Preventive Services Task Force (USPSTF) recommendation on using nontraditional risk factors in coronary heart disease risk assessment. Evidence Review: The USPSTF reviewed the evidence on using nontraditional risk factors in CVD risk assessment, focusing on the ankle-brachial index (ABI), high-sensitivity C-reactive protein (hsCRP) level, and coronary artery calcium (CAC) score; the health benefits and harms of CVD risk assessment and treatment guided by nontraditional risk factors combined with the Framingham Risk Score or Pooled Cohort Equations compared with using either risk assessment model alone; and whether adding nontraditional risk factors to existing CVD risk assessment models improves measures of calibration, discrimination, and risk reclassification. Findings: The USPSTF found adequate evidence that adding the ABI, hsCRP level, and CAC score to existing CVD risk assessment models results in small improvements in discrimination and risk reclassification; however, the clinical meaning of these changes is largely unknown. Evidence on adding the ABI, hsCRP level, and CAC score to the Pooled Cohort Equations is limited. The USPSTF found inadequate evidence to assess whether treatment decisions guided by the ABI, hsCRP level, or CAC score, in addition to risk factors in existing CVD risk assessment models, leads to reduced incidence of CVD events or mortality. The USPSTF found adequate evidence to conceptually bound the harms of early detection and interventions as small. The USPSTF concludes that the current evidence is insufficient to assess the balance of benefits and harms of using the ABI, hsCRP level, or CAC score in risk assessment for CVD in asymptomatic adults to prevent CVD events. Conclusions and Recommendation: The USPSTF concludes that the current evidence is insufficient to assess the balance of benefits and harms of adding the ABI, hsCRP level, or CAC score to traditional risk assessment for CVD in asymptomatic adults to prevent CVD events. (I statement).

Screening for Peripheral Artery Disease and Cardiovascular Disease Risk Assessment With the Ankle-Brachial Index: US Preventive Services Task Force Recommendation Statement.

Importance: Peripheral artery disease (PAD) is a manifestation of atherosclerosis in the lower limbs. It can impair walking and, in severe cases, can lead to tissue loss, infection, and amputation. In addition to morbidity directly caused by PAD, patients with PAD are at increased risk for cardiovascular disease (CVD) events, because atherosclerosis is a systemic disease that also causes coronary and cerebrovascular events. Objective: To update the 2013 US Preventive Services Task Force (USPSTF) recommendation on screening for PAD and CVD risk with the ankle-brachial index (ABI). Evidence Review: The USPSTF reviewed the evidence on whether screening for PAD with the ABI in generally asymptomatic adults reduces morbidity or mortality from PAD or CVD. The current review expanded on the previous review to include individuals with diabetes and interventions that include supervised exercise and physical therapy intended to improve outcomes in the lower limbs. Findings: The USPSTF found few data on the accuracy of the ABI for identifying asymptomatic persons who can benefit from treatment of PAD or CVD. There are few studies addressing the benefits of treating screen-detected patients with PAD; 2 good-quality studies showed no benefit of using the ABI to manage daily aspirin therapy in unselected populations, and 2 studies showed no benefit from exercise therapy. No studies addressed the harms of screening, although the potential exists for overdiagnosis, labeling, and opportunity costs. Studies that addressed the harms of treatment showed nonsignificant results. Therefore, the USPSTF concludes that the current evidence is insufficient and that the balance of benefits and harms of screening for PAD with the ABI in asymptomatic adults cannot be determined. Conclusions and Recommendation: The USPSTF concludes that the current evidence is insufficient to assess the balance of benefits and harms of screening for PAD and CVD risk with the ABI in asymptomatic adults. (I statement).

Use of Decision Models in the Development of Evidence-Based Clinical Preventive Services Recommendations: Methods of the U.S. Preventive Services Task Force.

The U.S. Preventive Services Task Force (USPSTF) develops evidence-based recommendations about preventive care based on comprehensive systematic reviews of the best available evidence. Decision models provide a complementary, quantitative approach to support the USPSTF as it deliberates about the evidence and develops recommendations for clinical and policy use. This article describes the rationale for using modeling, an approach to selecting topics for modeling, and how modeling may inform recommendations about clinical preventive services. Decision modeling is useful when clinical questions remain about how to target an empirically established clinical preventive service at the individual or program level or when complex determinations of magnitude of net benefit, overall or among important subpopulations, are required. Before deciding whether to use decision modeling, the USPSTF assesses whether the benefits and harms of the preventive service have been established empirically, assesses whether there are key issues about applicability or implementation that modeling could address, and then defines the decision problem and key questions to address through modeling. Decision analyses conducted for the USPSTF are expected to follow best practices for modeling. For chosen topics, the USPSTF assesses the strengths and limitations of the systematically reviewed evidence and the modeling analyses and integrates the results of each to make preventive service recommendations.

Lessons Learned From Newborn Screening for Critical Congenital Heart Defects.

Newborn screening for critical congenital heart defects (CCHD) was added to the US Recommended Uniform Screening Panel in 2011. Within 4 years, 46 states and the District of Columbia had adopted it into their newborn screening program, leading to CCHD screening being nearly universal in the United States. This rapid adoption occurred while there were still questions about the effectiveness of the recommended screening protocol and barriers to follow-up for infants with a positive screen. In response, the Centers for Disease Control and Prevention partnered with the American Academy of Pediatrics to convene an expert panel between January and September 2015 representing a broad array of primary care, neonatology, pediatric cardiology, nursing, midwifery, public health, and advocacy communities. The panel's goal was to review current practices in newborn screening for CCHD and to identify opportunities for improvement. In this article, we describe the experience of CCHD screening in the United States with regard to: (1) identifying the target lesions for CCHD screening; (2) optimizing the algorithm for screening; (3) determining state-level challenges to implementation and surveillance of CCHD; (4) educating all stakeholders; (5) performing screening using the proper equipment and in a cost-effective manner; and (6) implementing screening in special settings such as the NICU, out-of-hospital settings, and areas of high altitude.

Evaluating Harms in the Assessment of Net Benefit: A Framework for Newborn Screening Condition Review.

BACKGROUND: The Department of Health and Human Services (HHS) Advisory Committee on Heritable Disorders in Newborns and Children ("Advisory Committee") makes recommendations to the HHS Secretary regarding addition of new conditions to the national Recommended Uniform Screening Panel for newborns. The Advisory Committee's decision-making process includes assessing the net benefit of screening for nominated conditions, informed by systematic evidence reviews generated by an independent Condition Review Workgroup. The evidence base regarding harms associated with screening for specific conditions is often more limited than that for benefits. PROCEDURES: The process for defining potential harms from newborn screening reviewed the frameworks from other public health evidence-based review processes, adapted to newborn screening by experts in systematic review, newborn screening programs and bioethics, with input from and approval by the Advisory Committee. MAIN FINDINGS: To support the Advisory Committee's review of nominated conditions, the Workgroup has developed a standardized approach to evaluation of harms and relevant gaps in the evidence. Types of harms include the physical burden to infants; psychosocial and logistic burdens to families from screening or diagnostic evaluation; increased risk of medical treatment for infants diagnosed earlier than children with clinical presentation; delayed diagnosis from false negative results; psychosocial harm from false positive results; uncertainty of clinical diagnosis, age of onset or clinical spectrum; and disparities in access to diagnosis or therapy. CONCLUSIONS: Estimating the numbers of children at risk, the magnitude, timing and likelihood of harms will be integrated into Workgroup reports to the Advisory Committee.