Etiology, diagnosis, and treatment in childhood atelectasis

Introduction: Atelectasis is the loss of lung volume secondary to collapse. Narrow and collapsible airways and underlying chronic diseases facilitate the development of atelectasis in children. Since atelectasis is an important cause of morbidity and mortality in children, early diagnosis and treatment are of great importance. Methods: Thirty-six patients who were followed up in the pediatric service and pediatric intensive care unit of our clinic be - tween December 1, 2018, and June 1, 2019, and were diagnosed radiologically with atelectasis were evaluated retrospectively. Results: The median age was 1.85 years (1.0–7.37). The most common cause for hospitalization was pneumonia (n=30, 83%). Except for two patients, all patients had an underlying disease that increased the risk of atelectasis. Neurological diseases were the most common diseases among the underlying diseases (n=12, 36%). For the treatment, 4 (11.1%) patients received chest physiotherapy, 19 (52.7%) patients received nebulized medications, and chest physiotherapy, and 13 (36.1%) patients received positive end-expiratory pressure support in addition to these treatments. The frequency of atelectasis in more than one localization was higher in children with the neurological disease than in other patients (n=7, [54%] vs. n=3, [13%]; p=0.018). In patients with atelectasis in more than one localization, the duration of hospitalization was longer (median 12.5 days [9.5–16.75] vs. 19 days [13–22.75]; p=0.034). Discussion and Conclusion: Atelectasis is common in hospitalized children with an underlying disease. In the presence of pathological respiratory symptoms and signs, atelectasis should be kept in mind, and treatment should be started early

The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN

Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by impaired mucociliary clearance leading to irreversible lung damage. In contrast to other rare lung diseases like cystic fibrosis (CF), there are only few clinical trials and limited evidence-based treatments. Management is mainly based on expert opinions and treatment is challenging due to a wide range of clinical manifestations and disease severity. To improve clinical and translational research and facilitate development of new treatments, the clinical trial network for PCD (PCD-CTN) was founded in 2020 under the framework of the European Reference Network (ERN)-LUNG PCD Core. Applications from European PCD sites interested in participating in the PCD-CTN were requested. Inclusion criteria consisted of patient numbers, membership of ERN-LUNG PCD Core, use of associated standards of care, experience in PCD and/or CF clinical research, resources to run clinical trials, good clinical practice (GCP) certifications and institutional support. So far, applications from 22 trial sites in 18 European countries have been approved, including >1400 adult and >1600 paediatric individuals with PCD. The PCD-CTN is headed by a coordinating centre and consists of a steering and executive committee, a data safety monitoring board and committees for protocol review, training and standardisation. A strong association with patient organisations and industrial companies are further cornerstones. All participating trial sites agreed on a code of conduct. As CTNs from other diseases have demonstrated successfully, this newly formed PCD-CTN operates to establish evidence-based treatments for this orphan disease and to bring new personalised treatment approaches to patients

Demographic and microbial characteristics and drug resistance of childhood tuberculosis in Istanbul: analysis of 1,541 cases

Introduction: Although tuberculosis (TB) is frequently seen in Turkey, there are limited studies on childhood TB. We aimed to describe clinical and laboratory findings, including drug resistance, of children with TB in Istanbul, Turkey. Methodology: The study included all children aged 0-14 years who were registered in public dispensaries between 2006 and 2010. Results: The study included 1,541 cases. Forty-four percent (n = 763) of the patients were male with a mean age of 8.86 +/- 4.19 years. Fifty-five percent of the patients had pulmonary TB, 39% had extrapulmonary TB, and 6% had both pulmonary and extrapulmonary TB. The most common extrapulmonary involvement sites were the pleura (n = 193), lymph nodes (n = 247), and central nervous system (n = 41). Forty-one percent of the patients were evaluated microbiologically and 35% of them were positive. For the total study group, 14% of them were positive. A drug susceptibility test was performed on 108 patients. Drug resistance to at least one drug was detected in 16% (n = 17), to isoniazid in 15% (n = 16), streptomycin in 12% (n = 12), rifampicin in 9% (n = 10), ethambutol in 7% (n = 8), and multi-drug resistance in 8% (n=9) of patients. Conclusions: This is the largest analysis on demographic features and drug resistance of childhood TB in Turkey. In Turkey, the rate of microbiological diagnosis is low, similar to rates worldwide. More microbiological studies and drug resistance tests should be done and annual changes should be followed with multi-center studies

Inhaled corticosteroids in treatment of cystic fibrosis

Reduction of lung inflammation is one of the goals in the treatment of cystic fibrosis (CF). As a result, anti-inflammatory therapies are often used to decrease the excessive and persistent inflammatory response. Although effective, the use of systemic corticosteroids has been limited due to unacceptable adverse effects. Inhaled corticosteroids (ICS) are often used empirically to treat children and adults with CF despite the lack of evidence of their benefit. Concern about ef-fects on growth and adrenal suppression have been reduced, but not eliminated with the use of ICS. Herein, mechanisms of action of corticosteroids, the effectiveness and safety of ICS usage in CF are reviewed. © 2012 Bentham Science Publishers

Neurologic Adverse Events Associated with Voriconazole Therapy: Report of Two Pediatric Cases

Although voriconazole, a triazole antifungal, is a safe drug, treatment with this agent is associated with certain adverse events such as hepatic, neurologic, and visual disturbances. The current report presents two cases, one a 9-year-old boy and the other a 17-year-old girl, who experienced neurologic side effects associated with voriconazole therapy. Our aim is to remind readers of the side effects of voriconazole therapy in order to prevent unnecessary investigations especially for psychological and ophthalmologic problems. The first case was a 9-year-old boy with cystic fibrosis and invasive aspergillosis that developed photophobia, altered color sensation, and fearful visual hallucination. The second case was a 17-year-old girl with cystic fibrosis and allergic bronchopulmonary aspergillosis, and she experienced photophobia, fatigue, impaired concentration, and insomnia, when the dose of voriconazole therapy was increased from 12mg/kg/day to 16mg/kg/day. The complaints of the two patients disappeared after discontinuation of voriconazole therapy. Our experience in these patients reminded us of the importance of being aware of the neurologic adverse events associated with voriconazole therapy in establishing early diagnosis and initiating prompt treatment. In addition, although serum voriconazole concentration was not measured in the present cases, therapeutic drug monitoring for voriconazole seems to be critically important in preventing neurologic side effects in pediatric patients

Study protocol: the ear-nose-throat (ENT) prospective international cohort of patients with primary ciliary dyskinesia (EPIC-PCD)

Introduction Primary ciliary dyskinesia (PCD) is a rare, genetic, multiorgan disease with an estimated prevalence of 1 in 10 000. It affects mainly the upper and lower airways due to impaired mucociliary clearance. Almost all patients have sinonasal or otologic (ear-nose-throat, ENT) problems, although the ENT clinical phenotype may present great variability. Despite that, data on PCD ENT manifestations are scarce and based on small single-centre studies. To date, we know little about the spectrum and severity of PCD ENT disease, its association with lung disease, its course over life and its determinants of prognosis. This study protocol describes the aims and methods of the first prospective, observational, multinational cohort study focusing on ENT disease in patients with PCD. Methods and analysis The ENT prospective international cohort of patients with PCD (EPIC-PCD) is a prospective standardised observational clinical cohort set up as a multinational multicentre study, embedded into routine patient care. It aims to longitudinally characterise ENT disease in patients with PCD and its association with lung disease, and to identify determinants of its prognosis. Patients of all ages, diagnosed with PCD who undergo an ENT clinical assessment at least once a year at one of the participating centres will be invited to participate. Collected data include diagnostic test results, results of ENT examinations, lung function measurements, information on management of ENT disease and patient-reported data on clinical symptoms and health-related quality of life (QoL). Data are collected using the standardised PCD-specific FOLLOW-PCD form and the validated QoL-PCD questionnaire. Ethics and dissemination The study has been reviewed and approved by the Human Research Ethics Committees at all participating centres, based on local legislation. The results of the study will be published in scientific journals, presented at scientific conferences and disseminated to participants and national patient organisations

The disease-specific clinical trial network for primary ciliary dyskinesia: PCD-CTN

Primary ciliary dyskinesia (PCD) is a rare genetic disorder characterised by impaired mucociliary clearance leading to irreversible lung damage. In contrast to other ram lung diseases like cystic fibrosis (CF), there are only few clinical trials and limited evidence-based treatments. Management is mainly based on expert opinions and treatment is challenging due to a wide range of clinical manifestations and disease severity. To improve clinical and translational research and facilitate development of new treatments, the clinical trial network for PCD (PCD-CTN) was founded in 2020 under the framework of the European Reference Network (ERN)-LUNG PCD Core. Applications from European PCD sites interested in participating in the PCD-CTN were requested. Inclusion criteria consisted of patient numbers, membership of ERN-LUNG PCD Core, use of associated standards of care, experience in PCD and/or CF clinical research, resources to run clinical trials, good clinical practice (GCP) certifications and institutional support. So far, applications from 22 trial sites in 18 European countries have been approved, including >1400 adult and >1600 paediatric individuals with PCD. The PCD-CTN is headed by a coordinating centre and consists of a steering and executive committee, a data safety monitoring board and committees for protocol review, training and standardisation. A strong association with patient organisations and industrial companies are further cornerstones. All participating trial sites agreed on a code of conduct. As CTNs from other diseases have demonstrated successfully, this newly formed PCD-CTN operates to establish evidence-based treatments for this orphan disease and to bring new personalised treatment approaches to patients

Comparison of intravenous and non-intravenous antibiotic regimens in eradication of P. aeruginosa and MRSA in cystic fibrosis

Background Chronic pulmonary infection is the leading cause of mortality and morbidity in patients with cystic fibrosis (CF). The most common pathogens isolated in CF are Staphylococcus aureus (SA) and Pseudomonas aeruginosa (PA). Chronic infection of PA and methicillin-resistant S. aureus (MRSA) are associated with worse survival and antibiotic eradication treatment is recommended for both. This study compared the outcomes between intravenous (IV) and non-IV antibiotics in eradication of PA and MRSA. Methods This was a single-center retrospective study. All respiratory specimen cultures of 309 CF patients and eradication regimens between 2015 and 2019 were reviewed. Patients received eradication treatment in case of first ever isolation or new isolation after being infection-free >= 1 year. The primary analysis was the comparison of the percentage of successful eradication after receiving IV and non-IV eradication regimens. Demographic and clinical risk factors for eradication failure were also analyzed. Results One hundred and two patients with PA isolations and 48 patients with MRSA were analyzed. At 1 year, 21.6% in PA group and 35.4% in MRSA group were successfully eradicated. There was not any statistically significant difference between IV versus non-IV antibiotic regimens on eradication in either group. Additionally, none of the clinical risk factors was significantly associated with eradication failure in PA and MRSA groups. Conclusion In the eradication of PA and MRSA, IV and non-IV treatment regimens did not show any superiority to one another. Non-parenteral eradication could be a better option considering the cost-effectiveness and the treatment burden of IV treatments due to hospitalization and the need for IV access

Comparison of conventional chest physiotherapy and oscillatory positive expiratory pressure therapy in primary ciliary dyskinesia

Background Chest physiotherapy (CP) is a recommended treatment modality in primary ciliary dyskinesia (PCD). Objective Primary aim was to compare the efficacy and safety of the conventional chest physiotherapy (CCP) and oscillatory positive expiratory pressure therapy (OPEPT). Secondary aims were to compare the exacerbation rate, time until the first exacerbation, patient compliance and comfort between the two CP methods. Methods This is a 6 month randomized, controlled crossover trial. Patients >6 years of age with PCD were randomized into two groups, first group was assigned to OPEPT (Acapella (R)) for 3 months while second group was assigned to CCP. Groups were crossed over to the other modality after a 15-day washout period. Pulmonary function tests (PFTs) and compliance were monitored by monthly clinic visits. Results There was a significant increase in FEV1, FEF25-75, and PEF values (p = .018,p = .020, andp = .016, respectively) in the OPEPT group and in FVC values (p = .007) in CCP group compared to baseline. However PFT increase at 3rd month was not superior to each other with both physiotherapy methods. Median acute pulmonary exacerbation rate and time period until the first exacerbation were similar in both groups (p = .821,p = .092, respectively). Comfort and effectiveness of OPEPT was higher than CCP according to patients (p = .029 andp = .042, respectively). There were no adverse effects with either therapy. Conclusions OPEPT was as effective as CCP in PCD patients. OPEPT was more comfortable and effective than CCP according to patients. OPEPT might be an efficient alternative method for airway cleareance in PCD patients