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Introduction: Cystic fibrosis (CF) is an inherited disorder that leads to the production of thick, sticky mucus in the lungs and digestive system. CFTR protein modulators, such as Kaftrio and Kalydeco, represent a significant advancement in CF treatment by targeting the underlying molecular defect. Aim: to evaluate the clinical efficacy and safety of CFTR protein modulators in pediatric CF patients at the Clinical Center University of Sarajevo. Materials and methods: eight pediatric patients aged 6 to 18 years with a confirmed CF diagnosis were included. Clinical outcomes such as weight, height, lung function (FEV1), and liver enzyme levels were monitored. Results: all patients showed significant improvements in weight and lung function, with an average FEV1 increase of 13%. Liver enzyme levels normalized in all cases, and no serious adverse events were reported. Conclusion: CFTR protein modulators, particularly Kaftrio and Kalydeco, offer significant clinical benefits for pediatric CF patients, highlighting their potential as a first-line therapy. [ABSTRACT FROM AUTHOR]

The aim of our study was to assess the nutritional status of hospitalized children with cystic fibrosis. We extracted data from the ePINUT surveys. Undernutrition was defined as a body mass index (BMI) of <18.5 according to the International Obesity Task Force cut-off, and the nutritional status goal was defined as a BMI z-score ≥0 SD for children older than 2 years and a weight-for-height z-score ≥0 SD for those younger than 2 years. Undernutrition frequency in the 114 patients with cystic fibrosis was 46% and was higher than in children with other chronic diseases (n = 5863; 30.5%; p = 0.001); 81% of children were below the nutritional status goal. Undernutrition frequency in cystic fibrosis is higher than in other chronic diseases. [ABSTRACT FROM AUTHOR]

Background: Different respiratory sampling methods exist to identify lower airway pathogens in patients with cystic fibrosis (CF). Bronchoalveolar lavage (BAL) is considered the "gold standard.". Because BAL is invasive and cannot be repeated frequently, identification of lower respiratory tract pathogens in CF patients is challenging. Other sampling techniques are sputum, cough, and nasal swabs. The aim of the work: To differentiate between the microbiological results of bronchoalveolar lavage (BAL), sputum and cough swabs in CF children and to evaluate their accuracy in detecting lower respiratory tract organisms. Patients and Methods: Microbiological results detected by these different sampling methods were compared in all enrolled CF patients (n=31). Specificity, sensitivity, positive (PPV), and negative (NPV) predictive values were calculated. Results: Regarding microbiological results, Concordance was found between BAL and sputum as regards Pseudomonas spp., Klebsiella spp. and Enterobacter spp., except for Staph aureus (P 0.039). overall low sensitivity (7.1%) and low accuracy (16.1%) of cough swabs to detect lower airway pathogens [Pseudomonas spp., Staphylococcus aureus spp., Klebsiella spp. and Enterobacter spp.] in children with CF followed by sputum analysis as it showed sensitivity (85.7%) and accuracy (80.6%) while BAL showed highest sensitivity (86.2%) and highest accuracy (82.9%). Conclusion: Our findings suggested that sputum samples showed a good bacteriologic concordance with BAL in detecting lower airway pathogens, While Cough swabs showed the lowest accuracy in detecting the presence of CF pathogens in clinically stable CF children. However, BAL should be performed in symptomatic patients who could not expectorate and had a negative cough swab and sputum sample. [ABSTRACT FROM AUTHOR]

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The study aimed to assess the regional peculiarities of the respiratory profile of children with cystic fibrosis (CF) in the Dnipro region (Ukraine). Methods. Children living in the Dnipro region and aged younger than 18 years old with molecular-genetic confirmation of CF were enrolled in the study. Lung colonization was evaluated using a culture-dependent method. Sputum, mucus from the posterior pharyngeal wall and bronchoalveolar lavage fluid (BALF) were utilized. Results. The Firmicutes phylum was the most common and occupied 54.00 % of the general proportion. On the other hand, the Proteobacteria phylum demonstrated overexpression in CF airways and kept the second rank with 28.87 %. Sorensen's species similarity coefficient showed an allied affinity between the microbial burden of oropharyngeal samples with nasopharyngeal and sputum, QS = 0.61 and 0.91, respectively. However, the species composition within the nasal cavity was distinct from sputum and BALF (QS = 0.47). The primary pathogens in childhood were S. aureus, H. influenza, P. aeruginosa and A. fumigatus. In contrast to gram-negative non-fermenters (GNNF), the prevalence of S. aureus isolates by age had a non-linear character. The commensal microbiota changed negatively with age. Among children under 12 years, the Streptococcus genus was identified in 23.08 % of the samples, but among the age category older than 15 - only in 9.22 %. 11.06 % of S. aureus had small colony variants (SCVs) morphotypes. Isolates of P. aeruginosa with the properties of SCVs were also found in children who underwent prolonged antimicrobial treatment. However, the most prominent was the mucoid phenotype - 34.31 % of isolates. Conclusions. Along with conventional microbiological properties, obligate pathobionts in children with CF exhibited changes, resulting in difficulties in identification. These included auxotrophic modification into SCVs and mucoid transformation. The culture-dependent technique gives crucial data about the profile of pathogens usually associated with CF, although it is sufficiently limited. [ABSTRACT FROM AUTHOR]

Although many Clinical Practice Guidelines (CPGs) have been published for the care of patients with Cystic Fibrosis (CF), including a variety of nutrition recommendations, the quality of these CPGs has never been evaluated. The aim of this study was to compare, review, and critically appraise CPGs for the nutritional management of CF, throughout the lifespan. We searched PubMed, Guidelines International Network (GIN), ECRI Institute, and Guidelines Central for CPGs, with information on the nutritional management of CF. Retrieved CPGs were appraised by three independent reviewers, using the Appraisal of Guidelines, Research and Evaluation II (AGREE II) instrument and checklist. A total of 22 CPGs (seven solely nutrition oriented), by 14 different publishers, were retrieved. The Thoracic Society of Australia and New Zealand CPGs scored the highest overall quality (94.4%), while the Paediatric Gastroenterology Society/Dietitians Association of Australia CPGs had the lowest score (27.8%). Great variation in AGREE II domain-specific scores was observed in all CPGs, suggesting the existence of different strengths and weaknesses. Despite the availability of several CPGs, many appear outdated, lacking rigor, transparency, applicability, and efficiency, while incorporating bias. Considering that CPGs adherence is associated with better outcomes and the need for improving life expectancy in patients with CF, the development of CPGs of better quality is deemed necessary. [ABSTRACT FROM AUTHOR]

The article reports on the use of magnetic resonance imaging (MRI) and newborn screening to detect lung disease progression in children with cystic fibrosis. Topics include microbiological pathogen detection from throat swabs, morphofunctional chest MRI in sedated children and use of analysis of variance to analyze quantitative data.

Methods to identify children with cystic fibrosis (CF) at risk for development of advanced liver disease are lacking. We aim to determine the association between liver stiffness measurement (LSM) by vibration‐controlled transient elastography (VCTE) with research ultrasound (US) patterns and conventional hepatic markers as a potential means to follow liver disease progression in children with CF. ELASTIC (Longitudinal Assessment of Transient Elastography in CF) is a nested cohort of 141 patients, ages 7‐21, enrolled in the Prediction by US of Risk of Hepatic Cirrhosis in CF (PUSH) Study. We studied the association between LSM with research‐grade US patterns (normal [NL], heterogeneous [HTG], homogeneous [HMG], or nodular [NOD]) and conventional hepatic markers. In a subgroup (n = 79), the association between controlled attenuation parameter (CAP) and US pattern was explored. Among 133 subjects undergoing VCTE, NOD participants (n = 26) had a significantly higher median (interquartile range) LSM of 9.1 kPa (6.3, 15.8) versus NL (n = 72, 5.1 kPa [4.2, 7.0]; P < 0.0001), HMG (n = 17, 5.9 kPa [5.2, 7.8]; P = 0.0013), and HTG (n = 18, 6.1 kPa [4.7, 7.0]; P = 0.0008) participants. HMG participants (n = 14) had a significantly higher mean CAP (SD) (270.5 dB/m [61.1]) compared with NL (n = 40, 218.8 dB/m [46.5]; P = 0.0027), HTG (n = 10, 218.1 dB/m [60.7]; P = 0.044), and NOD (n = 15, 222.7 dB/m [56.4]; P = 0.041) participants. LSM had a negative correlation with platelet count (rs = ‐0.28, P = 0.0071) and positive correlation with aspartate aminotransferase–to‐platelet ratio index (rs = 0.38, P = 0.0002), Fibrosis‐4 index (rs = 0.36, P = 0.0007), gamma‐glutamyltransferase (GGT; rs = 0.35, P = 0.0017), GGT‐to‐platelet ratio (rs = 0.35, P = 0.003), and US spleen size z‐score (rs = 0.27, P = 0.0073). Conclusion: VCTE is associated with US patterns and conventional markers in patients with liver disease with CF. [ABSTRACT FROM AUTHOR]

OBJECTIVE Cystic fibrosis (CF) patients and caregivers are impacted by the number of pharmacological agents and unique administration needs; however, no data currently assesses how medication regimen complexity impacts clinical outcomes in this population. The objective of this study is to evaluate if an association exists between increased medication regimen complexity and clinical endpoints in pediatric patients with CF. METHODS This retrospective analysis included all pediatric patients with CF (ages 5--20 years) with at least 2 pharmacist encounters and acceptable pulmonary function tests at our pediatric pulmonary clinic during 2017. Each patient's medication regimen was scored using the validated Medication Regimen Complexity Index (MRCI) tool. The primary outcome was the correlation between MRCI score and lung function. Secondary endpoints included growth, number of infections requiring antibiotics, and hospitalizations. RESULTS MRCI scores of the 113 included patients ranged from 2 to 101 points. A negative correlation was found between initial and final MRCI score and initial and final forced expiratory volume in 1 second (FEV1; r = -0.323, p = 0.0005 and r = -0.287, p = 0.0021, respectively). MRCI scores were negatively correlated with BMI percentile for both encounters (r = -0.162 and r = -0.125) but were not significant. Higher MRCI scores were associated with increased use of oral and intravenous antibiotics and hospital admissions. CONCLUSIONS Higher MRCI scores are correlated with a significant decrease in FEV1, increased need for antibiotic therapy, and more hospital admissions in pediatric patients with CF. Larger studies are needed to determine if a correlation exists between MRCI score and growth. ABBREVIATIONS BMI, body mass index; CF, cystic fibrosis; CFTR, Cystic fibrosis transmembrane conductance regulator; FEV1, forced expiratory volume in 1 second; HIV, human immunodeficiency virus; IV, intravenous; MRCI, Medication Regimen Complexity Index; UNC, University of North Carolina [ABSTRACT FROM AUTHOR]

Rationale: We previously reported that ivacaftor was safe and well tolerated in cohorts aged 12 to <24 months with cystic fibrosis and gating mutations in the ARRIVAL study; here, we report results for cohorts aged 4 to <12 months.Objectives: To evaluate the safety, pharmacokinetics, and pharmacodynamics of ivacaftor in infants aged 4 to <12 months with one or more gating mutations.Methods: ARRIVAL is a single-arm phase 3 study. Infants received 25 mg or 50 mg ivacaftor every 12 hours on the basis of age and weight for 4 days in part A and 24 weeks in part B.Measurements and Main Results: Primary endpoints were safety (parts A and B) and pharmacokinetics (part A). Secondary/tertiary endpoints (part B) included pharmacokinetics and changes in sweat chloride levels, growth, and markers of pancreatic function. Twenty-five infants received ivacaftor, 12 in part A and 17 in part B (four infants participated in both parts). Pharmacokinetics was consistent with that in older groups. Most adverse events were mild or moderate. In part B, cough was the most common adverse event (n = 10 [58.8%]). Five infants (part A, n = 1 [8.3%]; part B, n = 4 [23.5%]) had serious adverse events, all of which were considered to be not or unlikely related to ivacaftor. No deaths or treatment discontinuations occurred. One infant (5.9%) experienced an alanine transaminase elevation >3 to ≤5× the upper limit of normal at Week 24. No other adverse trends in laboratory tests, vital signs, or ECG parameters were reported. Sweat chloride concentrations and measures of pancreatic obstruction improved.Conclusions: This study of ivacaftor in the first year of life supports treating the underlying cause of cystic fibrosis in children aged ≥4 months with one or more gating mutations.Clinical trial registered with clinicaltrials.gov (NCT02725567). [ABSTRACT FROM AUTHOR]

Background: Medication use is important to collect accurately in medically complex patients in both clinical and research settings. Aim: We assessed patient‐level agreement for medication use between self‐reported survey and electronic health record (EHR) for children with cystic fibrosis (CF). Methods: Our retrospective cross‐sectional study focused on children with CF ages 6‐20 years from Seattle Children's Hospital in Washington state, USA (N = 85). A self‐ or parent‐reported survey included questions on current use of specific medications and antibiotic use in the past 2 months. We compared survey data with data abstracted from the individual's EHR and derived Cohen's Kappa statistics to estimate the level of agreement between the two methods. Results: Self‐reported medication use was generally higher in the survey than in the EHR. The level of agreement ranged from slight for probiotics (74.1% agreement; 95% confidence interval [CI]: 64.6%‐83.6%; kappa: 0.07), pancreatic enzymes (80% agreement; 95% CI: 71.3%‐88.7%; kappa: 0.12), and vitamin D (55.3% agreement; 95% CI: 44.5%‐66.1%; kappa: 0.20) to moderate for chronic azithromycin (80% agreement; 95% CI: 7.13%‐88.7%; kappa: 0.50), proton pump inhibitors (76.5% agreement; 95% CI: 67.3%‐85.7%; kappa: 0.46), and oral antibiotics (70.6% agreement; 95% CI: 60.7%‐80.5%; kappa: 0.42). Conclusion: There is considerable heterogeneity in level of agreement in medication use between self‐reported survey and EHR data for children with CF. Standardized approaches are needed to improve the accuracy of medication data collected in clinical practice and research. [ABSTRACT FROM AUTHOR]

Nebulizers are used by the great majority of cystic fibrosis patients for delivery of cornerstone treatments. Inhalation technique and adequate disinfection and maintenance are important for optimizing medication delivery. In this study, inhalation technique and nebulizer disinfection/maintenance were assessed in cystic fibrosis patients by direct observation in clinic and completion of a scoring sheet. A total of 108 patients were recruited. The maximum inhalation technique score was attained by 30.5% and adequate inhalation technique score by 74.08% of patients. The inhalation technique score was best with the vibrating mesh nebulizer (p = 0.038), while patient age and number of nebulized medications did not affect ITS significantly (p > 0.05). Nebulizer disinfection/maintenance score was excellent in only 31.48%. Most families kept the nebulizer clean and used appropriate disinfection method, but only half of them replaced the nebulizer and nebulizer cup at the recommended time intervals. Nebulizer disinfection/maintenance score was positively affected by a number of nebulized medications and negatively by years of equipment use (p = 0.009 and p = 0.001, respectively). Even though inhalation technique and disinfection/maintenance practices were found to be adequate in a large proportion of cases, there is still a need for regular review and education. The type of nebulizer was associated with improved inhalation technique, but more data are required before making specific recommendations. [ABSTRACT FROM AUTHOR]

Symptomatic congenital pulmonary malformations (CPM) in child are rare. The diagnosis is based on clinical, radiological and histopathological confrontation. The aim of this study is to derterminate the diagnostic, therapeutic and evolutionary profile of CPM in a poor ressources country. This was a retrospective study over a period of 8 years (from January 2012 to March 2020),including 13 cases treated for CPM in the thoracic surgery department of a teaching hospital in Mali. Among patients, we found 5 cases of congenital lobar emphysema (38.8%), 4 cases of pulmoary cyst (30.8%), 3 cases of cystic adenomatoid malformation of the lung (23%) and 1 case of pulmonary sequestration(7,7%). The sex ratio was 2,2. All patients were symptomatic with an ave-rage age of 8,5 months. The symptoms were dominated by respiratory infection (38,4%), dyspnea (30,8%), dyspnea with cyanosis(7,7), dyspnea with thoracic pain (7,7%), respiratory distress (7,7%) and hemoptysis (7,7%). The chest X-ray has al-lowed to orient the diagnosis in most cases and thethoracic scan set it in 100% of the cases. All the patients underwent surgery by thoracotomy. Lobectomy was done for 53,8 % followed by cystectomy in 30,8%, segmentectomy (7,7%) and pneumonectomy (7,7%) of patients. Histopathological examination confirmed the diagnosis ofmalformation in all cases. The postoperative outcome was uneventful for all the patients. Clinical manifestations evoks the diagnosis of symptomatic CPM, the chest X-ray allowed to orient it and the thoracic scan set it in all cases. The treatment is mainly surgical. [ABSTRACT FROM AUTHOR]