A National Process to Enhance the Validity of Entrustment Decisions for Dutch Pediatric Residents.

BACKGROUND: Postgraduate medical education (PGME) has become increasingly individualized, and entrustable professional activities (EPAs) have been adopted to operationalize this. At the same time, the process and content to determine residents' progress using high-stakes summative entrustment decisions by clinical competency committees (CCCs) is not yet well established. OBJECTIVE: We evaluated the experiences with a structured process for assessment of EPAs to attain uniform summative entrustment decisions for a national sample of pediatric residents. METHODS: An EPA-based national PGME program for pediatric residents was introduced in the Netherlands, including a process of uniform summative entrustment decisions, termed the Evaluation and Assessment of Residents by Supervisors (EARS) procedure. To evaluate the program, we assessed survey data and information from invitational conferences. RESULTS: Beginning in January 2017, 125 pediatric residents in all 8 Dutch residency regions started training in the EARS program. The program enabled robust summative entrustment decisions. Preliminary data suggested that faculty, despite increased preparation time, appreciated the comprehensive appraisal of resident qualifications. The EPA-based program was well accepted by residents. Fifty-one percent (57 of 112) had at least 2 EARS procedures per year, and for 75% (84 of 112) the level of supervision was often or always adjusted to their level of training. CONCLUSIONS: A national EPA-based program provided a structured process for summative entrustment decisions by CCCs and enabled individualized stepwise progression of residents toward unsupervised practice. Broader application of these concepts may require adaptations to accommodate different health care systems and specialties.

Evaluation of interrupter resistance in methacholine challenge testing in children.

Bronchial hyperresponsiveness (BHR) is a key feature of asthma and is assessed using bronchial provocation tests. The primary outcome in such tests (a 20% decrease in forced expiratory volume in 1 sec (FEV1)) is difficult to measure in young patients. This study evaluated the sensitivity and specificity of the interrupter resistance (Rint ) technique, which does not require active patient participation, by comparing it to the primary outcome measure. Methacholine challenge tests were performed in children with a history of moderate asthma and BHR. Mean and individual changes in Rint and FEV1 were studied. A receiver operating characteristic (ROC) curve was used to describe sensitivity and specificity of Rint . Seventy-three children (median age: 9.2 years; range: 6.3-13.4 years) participated. There was a significant (P < 0.01) increase in mean Rint with increasing methacholine doses. However, individual changes of Rint showed large fluctuations. There was great overlap in change of Rint between children who did and did not reach the FEV1 endpoint. A ROC curve showed an area under the curve of 0.65. Because of low sensitivity and specificity, the use of Rint to diagnose BHR in individual patients seems limited.

Combination therapy salmeterol/fluticasone versus doubling dose of fluticasone in children with asthma.

RATIONALE: For children with symptomatic asthma despite low to moderate doses of inhaled corticosteroids, evidence is still lacking whether to add a long-acting bronchodilator or to increase the dose of inhaled corticosteroids. OBJECTIVE: To evaluate whether salmeterol/fluticasone propionate (SFP), 50/100 µg twice a day, is noninferior regarding symptom control compared with fluticasone propionate (FP), 200 µg twice a day Diskus in children with symptomatic asthma. METHODS: A multicenter, randomized, parallel-group, double-blind study was performed comparing SFP and FP treatment during 26 weeks on asthma control and lung function. MEASUREMENTS AND MAIN RESULTS: A total of 158 children, 6-16 years old, still symptomatic on FP, 100 µg twice a day, during a 4-week run-in period, were included. Percentage of symptom-free days during the last 10 weeks of the treatment period did not differ between treatment groups (per protocol analysis: adjusted mean difference [FP minus SFP] 2.6%; 95% confidence interval, -8.1 to 13.4). Both groups showed substantial improvements of about 25 percent points in symptom-free days (both P < 0.001 from baseline). Lung function measurements (FEV(1), FVC, PEF rate, and maximal expiratory flow) did not differ between groups except for a slight advantage in maximal expiratory flow in the SFP group at 1 week. No differences were found between FP and SFP regarding exacerbation rates, adverse events, or growth. CONCLUSIONS: In our study the efficacy on symptom control and lung function of the combination of a long-acting bronchodilator with inhaled corticosteroid is equal to doubling the dose of the inhaled corticosteroid in children still symptomatic on a moderate dose of inhaled corticosteroid.

Biomarkers in exhaled breath condensate indicate presence and severity of cystic fibrosis in children.

Chronic airway inflammation is present in cystic fibrosis (CF). Non-invasive inflammometry may be useful in disease management. The aim of the present cross-sectional study was to investigate: (i) the ability of fractional exhaled nitric oxide and inflammatory markers (IM) [exhaled breath condensate (EBC) acidity, nitrite, nitrate, hydrogen peroxide (H(2)O(2)), 8-isoprostane, Th1/Th2 cytokines] to indicate (exacerbations of) CF; and (ii) the ability of these non-invasive IM to indicate CF disease severity. In 98 children (48 CF/50 controls), exhaled nitric oxide was measured using the NIOX, and condensate was collected using a glass condenser. In CF interferon (IFN-gamma) and nitrite concentrations were significantly higher, whereas exhaled nitric oxide levels were significantly lower compared with controls (3.3 +/- 0.3 pg/ml, 2.2 +/- 0.2 microM, 10.0 +/- 1.2 p.p.b. vs. 2.6 +/- 0.2 pg/ml, 1.4 +/- 0.1 microM, 15.4 +/- 1.4 p.p.b. respectively). Using multivariate logistic regression models, the presence of CF was best indicated by 8-isoprostane, nitrite and IFN-gamma [sensitivity 78%, specificity 83%; area under receiver operating characteristic curve (AUC) 0.906, p < 0.001]. An exacerbation of CF was best indicated by 8-isoprostane and nitrite (sensitivity 40%, specificity 97%, AUC curve 0.838, p = 0.009). Most indicative biomarkers of CF severity were exhaled nitric oxide, and condensate acidity (sensitivity 96%, specificity 67%; AUC curve 0.751, p = 0.008). In this cross-sectional study, the combination of different exhaled IM could indicate (exacerbations of) CF, and severity of the disease in children. Longitudinal data are necessary to further confirm the role of these markers for the management of CF in children.

A novel microdeletion in 1(p34.2p34.3), involving the SLC2A1 (GLUT1) gene, and severe delayed development.

A de novo 4.1-megabase microdeletion of chromosome 1p34.2p34.3 has been identified by array-based comparative genomic hybridization in a young male with severely delayed development, microcephaly, pronounced hypotonia, and facial dysmorphism. The deleted region encompasses 48 genes, among them the glucose transporter 1 (SLC2A1 or GLUT1) gene. The deletion of the GLUT1 gene was in line with the abnormal ratio of cerebrospinal fluid (CSF) glucose to blood glucose, indicative of GLUT1 deficiency syndrome (MIM #606777). GLUT1 deficiency syndrome is characterized by therapy-resistant infantile seizures, developmental delay, acquired microcephaly, spasticity, ataxia, and a low concentration of glucose in the CSF. It is known that a ketogenic diet can lead to better control of seizures. This case study shows that identifying a microdeletion as the cause of learning disability is not only important for genetic counselling but might also lead to therapeutic intervention.

Poor adherence to self-medication instructions in children with asthma and their parents.

This study describes a self-treatment program for parents of children with asthma. The aim was to prevent asthma exacerbations by learning to recognise prodromal signs and acting upon them by increasing inhaled corticosteroids (ICS). The study questions were: (1) can we teach parents and children to recognise prodromal signs? (2) are instructions to increase inhalation medication followed? (3) will frequency and severity of asthma attacks diminish subsequently? Due to physicians' changed attitude towards prescription of ICS, fewer children could be recruited who were "ICS-naive" than expected. Twenty-nine children of the age of 4-11 years with moderate asthma, participated in a one year prospective randomised study. Structured information was given to all patients on asthma, symptoms and medication. The experimental group received additional information on recognising prodromal signs and doubling ICS during one week. Only in 25% of the patients who recognised prodromal signs the dose of ICS was doubled (as prescribed), in 75% inadequately or not at all. Recognition of prodromal signs was poor as well as compliance to increase as-needed medication. No significant decrease of asthma symptoms occurred in the experimental group. Clinical implications are important for self-treatment instructions: an individually tailored and multi-component program should be offered by health care providers in order to help the patient to recognise early alarm symptoms, comply to self-treatment instructions and to make adaptations for continuous self-regulation.