Objective:
The purpose of our study was to understand the healthcare burden and incidence of Krabbe disease (Krabbe).
Methods:
Retrospective analysis of Krabbe patients identified October 1, 2015 ...through December 31, 2020, ages birth through age 3, evaluated in two national databases. We estimated point prevalence and incidence from year 2016 data.
Results:
We identified 98 unique Krabbe patients with 736 visits including 260 were inpatient admissions. Total healthcare charges were $51.5 million dollars. We determined a point prevalence of 34 68 Krabbe patients in 2016 ages 0 3 years. This estimates a birth incidence of ~1 in 310,000 live births. Significance: Krabbe disease patients had over $51 million in health care charges and hundreds of hospitalizations. Estimated prevalence and birth incidence is similar to rates observed from newborn screening. Our findings show the tremendous health impacts of Krabbe disease, and provide guidance for efforts in screening and treatment planning.
Invasive plant species should be evaluated and prioritized for management according to their impacts, which include reduction in native diversity, changes to nutrient pools, and alteration of fire ...regimes. However, the impacts of most invasive species have not been quantified and, when measured, those impacts are based on a limited number of response metrics. As a result, invasion ecology has been overwhelmed by speculation and bias regarding the ecological consequences of invasive plants. We propose a quantitative mathematical framework that integrates any number of impact metrics as a function of groundcover and geographic extent. By making relative comparisons between invaded and uninvaded landscapes at the population scale, which results in a percent change for each metric, we overcome previous limitations that confounded the integration of metrics based on different units. Our model offers a quantitative approach to ecological impact that may allow identification of the transition from benign introduction to impactful invader, while also allowing empirical comparisons at the species and population levels that will be useful for management prioritization.
•Bisexual youth reported greatest severity of depression, victimization, and trauma.•Parent-family connectedness among bisexual youth was lower than all SMY subgroups.•Suicide ideation and attempts ...did not differ for bisexual and gay/lesbian youth.•Associations between risk factors and suicide ideation differed among SMY subgroups.
Background: Differences in risk and protective factors (e.g., victimization, abuse, social support) have been used to explain elevated rates of suicidal ideation and suicide attempts in sexual minority youth (SMY) relative to heterosexual peers. However, little is known regarding how risk and protective factors may explain suicide risk differences among subgroups of SMY. The aims of this study were to 1) examine differences in prevalence and severity for suicide risk and protective factors among SMY, and 2) explore whether risk and protective factors are differentially associated with suicidal ideation and suicide attempts for SMY subgroups. Methods: Participants were 6,423 adolescents (ages 12–17) recruited from 14 Emergency Departments across the United States who completed an assessment of suicide risk and protective factors. SMY were 20% of the sample (n = 1,275) and categorized as bisexual (8%), gay/lesbian (2%), mostly straight (5%), or other sexual minority (5%). Results: Bisexual youth had elevated rates of suicidal ideation and attempts, more risk factors (e.g., bullying victimization, depression), and fewer protective factors (e.g., parent-family connectedness, positive affect) relative to mostly straight and other sexual minority youth. Bisexual and gay/lesbian youth only differed in parent-family connectedness (lower among bisexual youth). Depression and parent-family connectedness had weaker associations with suicidal ideation for bisexual youth. Limitations: Emergency departments were not nationally representative. Study design was cross-sectional, preventing causal inferences. Conclusions: Interventions seeking to mitigate risk factors and promote protective factors are greatly needed for SMY and may benefit from tailoring to address unique stressors for sexual minority subgroups.
Despite evidence of the importance of interpersonal connectedness to our understanding of suicide risk, relatively little research has examined the protective and buffering effects of connectedness ...among adolescents. The aims of this study were to determine: (a) whether overall connectedness (composite of family, peer, and school) and specific domains of connectedness were related to a lower likelihood of suicide attempts, and (b) whether these factors buffer the prospective risk of suicide attempt for high-risk subgroups (i.e., recent suicidal ideation and/or lifetime history of suicide attempt, peer victimization, or sexual and gender minority status).
Participants were 2,897 adolescents (64.7% biological female), ages 12 to 17 (M = 14.6, SD = 1.6), recruited in collaboration with the Pediatric Emergency Care Applied Research Network (PECARN) from 14 emergency departments for the Emergency Department Screen for Teens at Risk for Suicide Study (ED-STARS). Suicide risk and protective factors were assessed at baseline; 3- and 6-month follow-ups were completed (79.5% retention). Multivariable logistic regressions were conducted, adjusting for established suicide risk factors.
Higher overall connectedness and, specifically, school connectedness were associated with decreased likelihood of a suicide attempt across 6 months. Overall connectedness and connectedness domains did not function as buffers for future suicide attempts among certain high-risk subgroups. The protective effect of overall connectedness was lower for youth with recent suicidal ideation or a suicide attempt history than for those without this history. Similarly, overall connectedness was protective for youth without peer victimization but not those with this history. Regarding specific domains, family connectedness was protective for youth without recent suicidal ideation or a suicide attempt history and peer connectedness was protective for youth without peer victimization but not youth with these histories.
In this large and geographically diverse sample, overall and school connectedness were related prospectively to lower likelihood of suicide attempts, and connectedness was more protective for youth not in certain high-risk subgroups. Results inform preventive efforts aimed at improving youth connectedness and reducing suicide risk.
The objective of this study was to determine the association of the use of extracorporeal cardiopulmonary resuscitation (ECPR) with survival to hospital discharge in pediatric patients with a ...noncardiac illness category. A secondary objective was to report on trends in ECPR usage in this population for 20 years.
Retrospective multicenter cohort study.
Hospitals contributing data to the American Heart Association's Get With The Guidelines-Resuscitation registry between 2000 and 2021.
Children (<18 yr) with noncardiac illness category who received greater than or equal to 30 minutes of cardiopulmonary resuscitation (CPR) for in-hospital cardiac arrest.
None.
Propensity score weighting balanced ECPR and conventional CPR (CCPR) groups on hospital and patient characteristics. Multivariable logistic regression incorporating these scores tested the association of ECPR with survival to discharge. A Bayesian logistic regression model estimated the probability of a positive effect from ECPR. A secondary analysis explored temporal trends in ECPR utilization. Of 875 patients, 159 received ECPR and 716 received CCPR. The median age was 1.0 interquartile range: 0.2-7.0 year. Most patients (597/875; 68%) had a primary diagnosis of respiratory insufficiency. Median CPR duration was 45 35-63 minutes. ECPR use increased over time ( p < 0.001). We did not identify differences in survival to discharge between the ECPR group (21.4%) and the CCPR group (16.2%) in univariable analysis ( p = 0.13) or propensity-weighted multivariable logistic regression (adjusted odds ratio 1.42 95% CI, 0.84-2.40; p = 0.19). The Bayesian model estimated an 85.1% posterior probability of a positive effect of ECPR on survival to discharge.
ECPR usage increased substantially for the last 20 years. We failed to identify a significant association between ECPR and survival to hospital discharge, although a post hoc Bayesian analysis suggested a survival benefit (85% posterior probability).
Children managed for asthma in an emergency department (ED) may be less likely to be hospitalized if they receive intravenous magnesium sulfate (IVMg). Asthma guidelines recommend IVMg for severely ...sick children but note a lack of evidence to support this recommendation. All previous trials of IVMg in children with asthma have been too small to answer whether IVMg is effective and safe. A few major questions remain about IVMg. First, it has not been tested early in the course of ED treatment, when the impact on hospitalization would be greatest. Second, the clinical impact of hypotension, a known adverse effect of IVMg, has not been well characterized in previous research. Third, no trials have compared different IVMg doses or serial serum magnesium (total and ionized) concentrations to optimize dosing, so the most effective dose is unknown. A large, conclusive, randomized, placebo-controlled clinical trial of IVMg might be challenging due to the need to enroll and complete study procedures quickly, a lack of understanding of blood pressure changes after IVMg, and a lack of pharmacologic information to guide the optimal doses of IVMg to be tested. Therefore, a pilot study to inform the above gaps is warranted before conducting a definitive trial.
The objectives of this study are to (1) demonstrate the feasibility of enrolling children with severe acute asthma in the ED in a multicenter, randomized controlled trial of a placebo, low-dose IVMg, or high-dose IVMg; (2) demonstrate the feasibility of timely delivery of study medication, assessment of blood pressure, and evaluation of adverse events in a standardized protocol; and (3) externally validate a previously constructed pharmacokinetic model and develop a combined pharmacokinetic/pharmacodynamic model for IVMg using magnesium (total and ionized) serum concentrations and their correlation with measures of efficacy and safety.
This pilot trial tests procedures and gathers information to plan a definitive trial. The pilot trial will enroll as many as 90 children across 3 sites, randomize each child to 1 of 3 study arms, measure blood pressure frequently, and collect 3 blood samples from each participant with corresponding clinical asthma scores.
The project was funded by the National Heart, Lung, and Blood Institute (1 R34HL152047-2) in March 2022. Enrollment began in September 2022, and 43 children have been enrolled as of April 2023. We will submit the results for publication in late 2023.
The results of this study will guide the planning of a large, definitive, multicenter trial powered to evaluate if IVMg reduces hospitalization. Blood pressure measurements will inform a monitoring plan for the larger trial, and blood samples and asthma scores will be used to validate pharmacologic models to select the optimal dose of IVMg to be evaluated in the definitive trial.
ClinicalTrials.gov NCT05166811; https://clinicaltrials.gov/ct2/show/NCT05166811.
DERR1-10.2196/48302.
We propose new summary measures of biomarker accuracy which can be used as companions to existing diagnostic accuracy measures. Conceptually, our summary measures are tantamount to the so-called ...Hellinger affinity and we show that they can be regarded as measures of agreement constructed from similar geometrical principles as Pearson correlation. We develop a covariate-specific version of our summary index, which practitioners can use to assess the discrimination performance of a biomarker, conditionally on the value of a predictor. We devise nonparametric Bayes estimators for the proposed indexes, derive theoretical properties of the corresponding priors, and assess the performance of our methods through a simulation study. The proposed methods are illustrated using data from a prostate cancer diagnosis study.
Longitudinal growth curves, based on repeated measurements from the same group of infants, exist for preterm infant weight and length but not for BMI. Our existing BMI (weight divided by length ...squared) curves are based on cross-sectional birth data obtained from a different group of infants at each gestational age (GA).
We calculated BMI over time for 68 693 preterm infants between 24 and 36 weeks GA. Stratifying infants by sex, GA at birth, and quintiles based on birth BMI, we created longitudinal median curves using R and validated the resulting curves for empirical fit, proper classification, and normality of
scores.
We created 2 sets of BMI growth charts. The first set displays fitted median curves for all 5 percentile groups in each GA group by sex. The second set displays fitted median curves with their corresponding third and 97th percentiles by percentile group, GA, and sex. In the validation analysis, percentage of daily observations below the median curve approximated the expected 50th percentile after the initial 3 days. Unlike the cross-sectional curves, the longitudinal curves reveal the pattern of change corresponding to nadir; postnadir, these curves remained consistently below the cross-sectional curves and varied by GA and sex as expected. The percentage of observations falling below the 50th percentile for cross-sectional curves (revealing optimal growth) was generally much higher than for longitudinal curves (revealing actual growth).
These new longitudinal curves provide clinicians data on how premature infants' body proportionality changes over time.
The present study explored the Immigrant Paradox (IP), generational differences in problematic alcohol use (alcohol consumption and alcohol-related consequences), among immigrants and US born groups ...from a number of ethnic minority backgrounds. Our approach separates group differences in problematic alcohol consumption in a counterfactual manner for immigrants and the US born to answer the following counterfactual question: "What would problematic alcohol use levels be for the US born had they been exposed to the alcohol use generation (or protective) processes of immigrants and vice versa?" Multidimensional measures of enculturation (involvement with heritage culture), acculturation (involvement with US culture), acculturative stress, and demographic covariates were used to statistically explain these differences. The sample consisted of Asian American (n = 1,153), Black American (n = 833), and Latinx (n = 1,376) college students from 30 universities. Results indicated significant generational differences in mean levels of alcohol consumption but not alcohol-related consequences. Differences in measured characteristics (endowments) marginally explained differences between immigrants and the US born. On the other hand, endowments significantly explained generational differences and represented an increase in alcohol consumption among immigrants if they had the endowments of the U.S. born. Results are discussed in light of cultural and social factors that contribute to the IP.
Celotno besedilo
Dostopno za:
DOBA, IJS, IZUM, KILJ, NUK, OILJ, PILJ, PNG, SAZU, SIK, UILJ, UKNU, UL, UM, UPUK, VSZLJ
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