An Assessment of Public Preferences for Newborn Screening Using Best-Worst Scaling.

OBJECTIVE: To identify and quantify public preferences for attributes of newborn screening conditions. STUDY DESIGN: We conducted an online national survey of the public (n = 502) to evaluate preferences for attributes of candidate newborn screening conditions. Respondents were presented with hypothetical condition profiles that were defined using 10 attributes with 2-6 levels per attribute. Participants indicated whether they would recommend screening for a condition and which condition attributes were most and least important when making this decision (best-worst scaling). Difference scores were calculated and stratified by condition recommendation (recommend or not recommend for screening). Regression analyses were used to evaluate the effect of attributes on choice to screen or not screen. RESULTS: The number of babies diagnosed was important to those who would recommend newborn screening for a profile, and age at which the treatment would start was important to those who would not recommend newborn screening. Cost was considered to be a key attribute, and treatment effectiveness and impact of making the diagnosis through newborn screening were of low importance for both groups. CONCLUSION: Public preferences identified through survey methods that provide an adequate baseline understanding of newborn screening can be used to inform newborn screening decisions.

Association between enhanced access services in pediatric primary care and utilization of emergency departments: a national parent survey.

OBJECTIVES: To measure the prevalence of enhanced access services in pediatric primary care and to assess whether enhanced access services are associated with lower emergency department (ED) utilization. STUDY DESIGN: Internet-based survey of a national sample of parents (n = 820, response rate 41%). We estimated the prevalence of reported enhanced access services and ED use in the prior 12 months. We then used multivariable negative binomial regression to assess associations between enhanced access services and ED use. RESULTS: The majority of parents reported access to advice by telephone during office hours (80%), same-day sick visits (79%), and advice by telephone outside office hours (54%). Less than one-half of parents reported access to their child's primary care office on weekends (47%), after 5:00 p.m. on any night (23%), or by email (13%). Substantial proportions of parents reported that they did not know if these services were available (7%-56%, depending on service). Office hours after 5:00 p.m. on ≥ 5 nights a week was the only service significantly associated with ED utilization in multivariable analysis (adjusted incidence rate ratio: 0.51 [95% CI 0.28-0.92]). CONCLUSIONS: The majority of parents report enhanced access to their child's primary care office during office hours, but many parents do not have access or do not know if they have access outside of regular office hours. Extended office hours may be the most effective practice change to reduce ED use. Primary care practices should prioritize the most effective enhanced access services and communicate existing services to families.

Health-related quality of life in adolescents with or at risk for type 2 diabetes mellitus.

OBJECTIVE: To evaluate how adolescents with or at risk for type 2 diabetes mellitus (T2DM) and their parent/guardians (parents) perceive adolescents' health-related quality of life. STUDY DESIGN: We interviewed overweight/obese, 12- to 18-year-old youth with T2DM, prediabetes, or insulin resistance and one parent from 5 US sites. Assessments included Pediatric Quality of Life Inventory (PedsQL), Health Utilities Index, family conflict, and diabetes burden. RESULTS: In 108 adolescents, diagnoses included 40.7% with T2DM, 25.0% with prediabetes, and 34.3% with insulin resistance. PedsQL summary score (SS) was higher in adolescents than parents (P=.02). Parents rated physical functioning lower than adolescents (P<.0001), but there were no differences in psychosocial health. Adolescent PedsQL SS did not differ with diagnosis, but was inversely associated with adolescent body mass index z-score (P=.0004) and family conflict (P<.0001) and associated with race/ethnicity (P<.0001). Number of adolescent co-morbidities (P=.007) and burden of diabetes care (P<.05) were inversely associated with parent PedsQL SS. There were no differences in the Health Utilities Index-Mark 3 multi-attribute utility score. CONCLUSIONS: Parents perceive their adolescents' physical functioning as more impaired than adolescents themselves. Contextual factors including severity of obesity, race/ethnicity, family conflict, and burden of diabetes care influence health-related quality of life. Family-based approaches to treatment and prevention of T2DM may benefit from increased attention to the biopsychosocial context.

Cost-effectiveness of oseltamivir treatment for children with uncomplicated seasonal influenza.

OBJECTIVE: To evaluate the cost-effectiveness of oseltamivir treatment for seasonal influenza in children and consider the impact of oseltamivir resistance on these findings. STUDY DESIGN: We developed a model to evaluate 1-year clinical and economic outcomes associated with 3 outpatient management strategies for unvaccinated children with influenza-like-illness: no antiviral treatment; diagnostic testing and oseltamivir treatment when positive; and empiric oseltamivir treatment. The model depicted a hypothetical non-pandemic influenza season with a 29% level of oseltamivir resistance in circulating viruses, and 14% to 54% probability of seasonal influenza with influenza-like-illness. Strategies were compared with incremental cost-effectiveness ratios. RESULTS: In our primary analysis, empiric oseltamivir treatment consistently produced the greatest benefit. The incremental cost-effectiveness of this alternative, compared with testing and treating, was <$100,000 per quality-adjusted life year gained in all age groups except the oldest. The testing strategy was consistently more effective compared with no treatment and cost between $25,900 and $71,200 per quality-adjusted life year gained, depending on age. Results were sensitive to the prevalence of oseltamivir resistance in circulating viruses. CONCLUSION: Empiric oseltamivir treatment of seasonal influenza is associated with favorable cost-effectiveness ratios, particularly in children aged 1 to <12 years, but ratios are highly dependent on the prevalence of oseltamivir resistance among circulating influenza viruses.

Effectiveness and cost-effectiveness of blood pressure screening in adolescents in the United States.

OBJECTIVE: To compare the long-term effectiveness and cost-effectiveness of 3 approaches to managing elevated blood pressure (BP) in adolescents in the United States: no intervention, "screen-and-treat," and population-wide strategies to lower the entire BP distribution. STUDY DESIGN: We used a simulation model to combine several data sources to project the lifetime costs and cardiovascular outcomes for a cohort of 15-year-old U.S. adolescents under different BP approaches and conducted cost-effectiveness analysis. We obtained BP distributions from the National Health and Nutrition Examination Survey 1999-2004 and used childhood-to-adult longitudinal correlation analyses to simulate the tracking of BP. We then used the coronary heart disease policy model to estimate lifetime coronary heart disease events, costs, and quality-adjusted life years (QALY). RESULTS: Among screen-and-treat strategies, finding and treating the adolescents at highest risk (eg, left ventricular hypertrophy) was most cost-effective ($18000/QALY [boys] and $47000/QALY [girls]). However, all screen-and-treat strategies were dominated by population-wide strategies such as salt reduction (cost-saving [boys] and $650/QALY [girls]) and increasing physical education ($11000/QALY [boys] and $35000/QALY [girls]). CONCLUSIONS: Routine adolescents BP screening is moderately effective, but population-based BP interventions with broader reach could potentially be less costly and more effective for early cardiovascular disease prevention and should be implemented in parallel.