Journal of Pediatrics: X最新文献

Objective

To assess the safety and efficacy of the endothelin receptor antagonist ambrisentan in pediatric pulmonary arterial hypertension (PAH).

Study design

In this open-label, phase IIb study, patients with PAH aged 8 to <18 years were randomized to low- or high-dose ambrisentan for 24 weeks. Most patients were receiving other PAH medication(s) that could not be changed during the trial. The primary outcome was safety (treatment-emergent adverse events [TEAEs]); secondary outcome was efficacy (including change from baseline to week 24 in 6-minute walking distance and World Health Organization functional class). Study staff were blinded to treatment. No statistical testing was performed.

Results

Most of the 41 patients randomized (80%) experienced ≥1 TEAE; most were mild (22%) or moderate (49%) in severity (no difference between dose groups). Most common TEAEs were headache (24%), nausea (17%), abdominal pain (12%), and nasopharyngitis (12%). Eight patients had serious TEAEs; 2 were fatal (unrelated to study treatment). Improved 6-minute walking distance was observed from baseline to week 24: total mean (SD) change, +40.69 (84.58) meters; World Health Organization functional class was maintained or improved in 70% and 27% patients, respectively.

Conclusions

Ambrisentan was well tolerated; TEAEs were consistent with the adult safety profile. Efficacy was similar to previous findings in adult PAH; however, interpretation is limited by small sample size. Findings support a potentially similar benefit:risk profile in pediatric (8 to <18 years) and adult patients with PAH.

Trial registration

ClinicalTrials.gov: NCT01332331

Objectives

To measure case detection and response time of severe pediatric dyslipidemia, defined as non-high-density lipoprotein cholesterol (HDL-C) ≥190 mg/dL on the initial screening panel. Although low adherence to guidelines recommending universal pediatric lipid screening is well-documented, it is unknown how clinicians respond to pediatric lipid screening results suggestive of severe dyslipidemia.

Study design

This study is a single-institution, retrospective review of patients 0-18 years of age with initial lipid panels completed from January 1, 2010, to June 30, 2018. A chart review was conducted on all patients with non-HDL-C ≥190 mg/dL to determine indication(s) for the initial lipid panel, specialty of ordering clinician, type of action taken to an abnormal result (repeat laboratory tests, treatment, and/or referral), time from result to clinician action, and diagnosis.

Results

There were 16 860 initial lipid panels that met the inclusion criteria; 178 (1.1%) had non-HDL-C ≥190 mg/dL, indicating severe dyslipidemia. The most common indication for screening was universal screening (52%). For all lipid panels with non-HDL ≥190 mg/dL, a clinician action was documented for 47% within 7 days and 69% within 30 days. No follow-up action was documented in 18 (9%). A clinical diagnosis of familial hypercholesterolemia was the most common diagnosis, in 24% of patients.

Conclusions

The majority of lipid panels with non-HDL-C ≥190 mg/dL had some action documented, although the actions varied. Universal screening was the most common indication for testing, clarifying its significance in identifying severe dyslipidemia. Further education and improved management protocols may help responses to severe dyslipidemia in children at high risk for premature cardiovascular disease.

Objective

To assess food insecurity during pediatric visits to federally qualified health centers (FQHCs) during the coronavirus disease-19 pandemic.

Study design

Interviews using the validated American Academy of Pediatrics 2-question food insecurity screen were performed with 200 consecutive families presenting for pediatric care to 2 FQHC in Central Texas from April 14 to May 20, 2020, during the initial phase of the pandemic in Texas. Brief qualitative interviews were conducted to determine whether families found a worsening of food insecurity during the pandemic.

Results

Overall, 47% of families had a positive food insecurity screen. More than 90% of these were worrying about food running out and about 60% were positive for the question related to food not lasting. Among families with food insecurity, 94% indicated this had begun or worsened during the pandemic. Of the 115 families volunteering information about employment, 46% reported job loss during this time period. Both ethnicity (P < .001) and Special Supplementation Nutrition Program for Women, Infants and Children (WIC) participation (P = .03) were associated with greater levels of food insecurity. Among primarily Spanish-speaking families participating in the WIC program, 64% reported food insecurity.

Conclusions

Approximately one-half of families receiving routine pediatric care at a FQHC during the coronavirus disease-19 pandemic reported food insecurity and this was associated with loss of jobs during the pandemic. Participation in the WIC program was not protective against food insecurity. Increased frequency of food insecurity was detected in Hispanic and Spanish-speaking families. Screening of families at an FQHC should be strongly considered as a part of routine pediatric care. Knowledge of community resources is important for providers to share with patients. (J Pediatr: X 2020;4:100044).

Trial Registration

ClinicalTrials.gov: NCT04378595

There is variation in cardiorespiratory monitoring practices for neonatal abstinence syndrome. We examined the incidence of cardiorespiratory adverse events in infants with neonatal abstinence syndrome who were treated or nontreated pharmacologically. Eight (10%) in the nontreated and 23 (19%) in the treated group experienced adverse events. This warrants further investigation in a larger cohort.

Objective

To develop a simplified clinical prediction tool for identifying children with clinically important traumatic brain injuries (ciTBIs) after minor blunt head trauma by applying machine learning to the previously reported Pediatric Emergency Care Applied Research Network dataset.

Study design

The deidentified dataset consisted of 43 399 patients <18 years old who presented with blunt head trauma to 1 of 25 pediatric emergency departments between June 2004 and September 2006. We divided the dataset into derivation (training) and validation (testing) subsets; 4 machine learning algorithms were optimized using the training set. Fitted models used the test set to predict ciTBI and these predictions were compared statistically with the a priori (no information) rate.

Results

None of the 4 machine learning models was superior to the no information rate. Children without clinical evidence of a skull fracture and with Glasgow Coma Scale scores of 15 were at the lowest risk for ciTBIs (0.48%; 95% CI 0.42%-0.55%).

Conclusions

Machine learning algorithms were unable to produce a more accurate prediction tool for ciTBI among children with minor blunt head trauma beyond the absence of clinical evidence of skull fractures and having Glasgow Coma Scale scores of 15.