Paediatric Respiratory Reviews最新文献

{"title":"Cystic fibrosis liver disease in the new era of cystic fibrosis transmembrane conductance regulator (CFTR) modulators","authors":"Jessica A. Eldredge ,&nbsp;Mark R. Oliver ,&nbsp;Chee Y. Ooi","doi":"10.1016/j.prrv.2023.12.005","DOIUrl":"10.1016/j.prrv.2023.12.005","url":null,"abstract":"<div><h3>Summary</h3><p>Cystic fibrosis liver disease (CFLD) is characterised by a wide heterogenity of manifestations and severity. It represents a major cause of morbidity in people with cystic fibrosis (PwCF), which will be of increasing relevance as survival increases in the new era of cystic fibrosis care. No medical therapy currently available has evidence to treat or prevent progression of liver disease. Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators may be transformative on pulmonary, nutritional and quality of life, but direct effect on long term liver disease outcomes is not yet established. Drug-associated hepatic adverse effects may be common, and clinician familiarity with drug-monitoring recommendations is essential. Longitudinal studies are required to understand the effect of CFTR modulators on the incidence and natural history of CFLD, including with early treatment initiation, in established advanced liver disease, and post liver transplantation.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 54-61"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054223000878/pdfft?md5=a103ef97f990bc20ce0578c92a341733&pid=1-s2.0-S1526054223000878-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139071226","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neurodevelopmental outcomes of extremely preterm infants with bronchopulmonary dysplasia (BPD) – A retrospective cohort study","authors":"Khoa L. Nguyen ,&nbsp;Dominic A. Fitzgerald ,&nbsp;Annabel Webb ,&nbsp;Barbara Bajuk ,&nbsp;Himanshu Popat","doi":"10.1016/j.prrv.2024.02.004","DOIUrl":"10.1016/j.prrv.2024.02.004","url":null,"abstract":"<div><h3>Objective</h3><p>To investigate the neurodevelopmental outcomes for preterm infants born &lt; 29 weeks gestation with/without bronchopulmonary dysplasia (BPD).</p></div><div><h3>Study design</h3><p>Preterm infants &lt; 29 weeks’ gestation born 2007–2018 in New South Wales and the Australian Capital Territory, Australia, were included. Infants who died &lt; 36 weeks’ postmenstrual age and those with major congenital anomalies were excluded. Subjects were assessed at 18–42 months corrected age using the Bayley Scales of Infant Development, 3rd edition.</p></div><div><h3>Results</h3><p>1436 infants without BPD (non-BPD) and 1189 infants with BPD were followed. The BPD group, 69 % infants were discharged without respiratory support (BPD1), 29 % on oxygen (BPD2) and 2 % on pressure support/tracheostomy (BPD3). Moderate neurodevelopmental impairment (NDI) was evident in 5.7 % of non-BPD infants, 11 % BPD1, 15 % BPD2, 15 % BPD3 infants. Severe NDI was seen in 1.7 % non-BPD infants, 3.4 % BPD1, 7.3 % BPD2, 35 % BPD3 infants. After adjusting for confounders, infants with BPD2 (OR 2.24, 99.9 % CI 1.25 to 5.77) or BPD3 (OR 5.99, 99.9 % CI 1.27 to 46.77) were more likely to have moderate-severe NDI compared to non-BPD infants.</p></div><div><h3>Conclusion</h3><p>The majority of infants with BPD were discharged home without respiratory support and had better neurocognitive outcomes in early childhood compared to those that required home-based oxygen or respiratory support.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 23-30"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140047067","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Learning from cystic fibrosis: How can we start to personalise treatment of Children’s Interstitial Lung Disease (chILD)?","authors":"Andrew Bush","doi":"10.1016/j.prrv.2023.11.001","DOIUrl":"10.1016/j.prrv.2023.11.001","url":null,"abstract":"<div><p>Cystic fibrosis (CF) is a monogenic disorder cause by mutations in the CF Transmembrane Regulator (<em>CFTR</em>) gene. The prognosis of cystic fibrosis has been transformed by the discovery of highly effective modulator therapies (HEMT). Treatment has changed from reactive therapy dealing with complications of the disease to pro-active correction of the underlying molecular functional abnormality. This has come about by discovering the detailed biology of the different CF molecular sub-endotypes; the development of biomarkers to assess response even in mild disease or young children; the performance of definitive large randomised controlled trials in patients with a common mutation and the development of <em>in vitro</em> testing systems to test efficacy in those patients with rare CFTR mutations. As a result, CF is now an umbrella term, rather than a specific diagnostic label; we have moved from clinical phenotypes to molecular subendotypes. Children’s Interstitial Lung Diseases (chILDs) comprise more than 200 entities, and are a diverse group of diseases, for an increasing number of which an underlying gene mutation has been discovered. Many of these entities are umbrella terms, such as pulmonary alveolar proteinosis or hypersensitivity pneumonitis, for each of which there are multiple and very different endotypes. Even those chILDs for which a specific gene mutation has been discovered comprise, as with CF, different molecular subendotypes likely mandating different therapies. For most chILDs, current treatment is non-specific (corticosteroids, azithromycin, hydroxychloroquine). The variability of the different entities means that there is little evidence for the efficacy of any treatment. This review considers how some of the lessons of the success story of CF are being applied to chILD, thus opening the opportunities for truly personalised medicine in these conditions. Advances in knowledge in the molecular biology of surfactant protein C and Adenosine triphosphate binding cassette subfamily A member 3 (ABCA3), and the possibilities of discovering novel therapies by <em>in vitro</em> studies will especially be highlighted.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 46-53"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054223000787/pdfft?md5=45bbcf973d6d171153110b14f0d05b42&pid=1-s2.0-S1526054223000787-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138299796","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Expiratory airflow limitation in adults born extremely preterm: A systematic review and meta-analysis","authors":"Henriette Lahn-Johannessen Lillebøe ,&nbsp;Merete Salveson Engeset ,&nbsp;Hege H Clemm ,&nbsp;Thomas Halvorsen ,&nbsp;Ola Drange Røksund ,&nbsp;Thomas Potrebny ,&nbsp;Maria Vollsæter","doi":"10.1016/j.prrv.2024.02.002","DOIUrl":"10.1016/j.prrv.2024.02.002","url":null,"abstract":"<div><p>Extreme preterm (EP) birth, denoting delivery before the onset of the third trimester, interrupts intrauterine development and causes significant early-life pulmonary trauma, thereby posing a lifelong risk to respiratory health. We conducted a systematic review and <em>meta</em>-analysis to investigate adult lung function following EP birth (gestational age &lt;28 weeks); comparing forced expiratory volume in first second (FEV₁), forced vital capacity (FVC), and FEV₁/FVC to reference values. Subgroup differences were explored based on timing of birth relative to surfactant use (1991) and bronchopulmonary dysplasia (BPD) status. Systematic searches were performed in Medline, EMBASE, Web of Science and Cochrane Central. Quality assessments were carried out using a modified Newcastle-Ottawa Scale for cohort studies. Sixteen studies encompassing 1036 EP-born adults were included, with 14 studies (n = 787) reporting data as %predicted, and 11 (n = 879) as z-score (not mutually exclusive). Overall mean [95 % confidence interval (CI)] %FEV₁ was 85.30 (82.51; 88.09), %FVC was 94.33 (91.74; 96.91), and FEV₁/FVC was 79.54 (77.71 to 81.38), all three with high heterogeneity. Overall mean (95 %CI) zFEV₁ was −1.05 (-1.21; −0.90) and zFVC was.</p><p>−0.45 (-0.59; −0.31), both with moderate heterogeneity. Subgroup analyses revealed no difference in FEV₁ before versus after widespread use of surfactant, but more impairments after neonatal BPD. This <em>meta</em>-analysis revealed significant airflow limitation in EP-born adults, mostly explained by those with neonatal BPD. FEV₁ was more reduced than FVC, and FEV₁/FVC was at the lower limit of normal. Although at a group level, most adult EP-born individuals do not meet COPD criteria, these findings are concerning.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 2-22"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054224000198/pdfft?md5=1cbd340fc004995d5289d5a0dc978981&pid=1-s2.0-S1526054224000198-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139927099","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Characterising the lifelong consequences of bronchopulmonary dysplasia","authors":"Dominic A. Fitzgerald","doi":"10.1016/j.prrv.2024.03.001","DOIUrl":"10.1016/j.prrv.2024.03.001","url":null,"abstract":"","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Page 1"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140403352","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adherence in paediatric respiratory medicine: A review of the literature","authors":"Ella A. Kotecha ,&nbsp;Dominic A. Fitzgerald ,&nbsp;Sailesh Kotecha","doi":"10.1016/j.prrv.2023.09.004","DOIUrl":"10.1016/j.prrv.2023.09.004","url":null,"abstract":"<div><p>Poor adherence is an important factor in unstable disease control and treatment failure. There are multiple ways to monitor a patient’s adherence, each with their own advantages and disadvantages. The reasons for poor adherence are multi-factorial, inter-related and often difficult to target for improvement. Although practitioners can implement different methods of adherence, the ultimate aim is to improve health outcomes for the individual and the health care system. Asthma is a common airway disease, particularly diagnosed in children, often treated with inhaled corticosteroids and long-acting bronchodilators. Due to the disease’s tendency for exacerbations and consequently, when severe will require unscheduled health care utilisation including hospital admissions, considerable research has been done into the effects of medication adherence on asthma control. This review discusses the difficulties in defining adherence, the reasons for and consequences of poor adherence, and the methods of recording and improving adherence in asthma patients, including an in-depth analysis of the uses of smart inhalers.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 41-45"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054223000660/pdfft?md5=633e8afb7aa539e37c80492304a0f737&pid=1-s2.0-S1526054223000660-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41208098","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Should an inhaled corticosteroid accompany each dose of fast-acting beta2-agonist for relief of asthma symptoms?","authors":"Leslie Hendeles ,&nbsp;Miles Weinberger","doi":"10.1016/j.prrv.2023.05.005","DOIUrl":"10.1016/j.prrv.2023.05.005","url":null,"abstract":"","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"50 ","pages":"Pages 38-40"},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140402126","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Assessment of obstructive sleep apnoea in children: What are the challenges we face?","authors":"Dominic A. Fitzgerald, Joanna MacLean, Brigitte Fauroux","doi":"10.1016/j.prrv.2024.04.002","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.04.002","url":null,"abstract":"There is an increasing demand for the assessment of sleep-disordered breathing in children of all ages to prevent the deleterious neurocognitive and behaviour consequences of the under-diagnosis and under-treatment of obstructive sleep apnoea [OSA]. OSA can be considered in three broad categories based on predominating contributory features: OSA type 1 [enlarged tonsils and adenoids], type II [Obesity] and type III [craniofacial abnormalities, syndromal, storage diseases and neuromuscular conditions]. The reality is that sleep questionnaires or calculations of body mass index in isolation are poorly predictive of OSA in individuals. Globally, the access to testing in tertiary referral centres is comprehensively overwhelmed by the demand and financial cost. This has prompted the need for better awareness and focussed history taking, matched with simpler tools with acceptable accuracy used in the setting of likely OSA. Consequently, we present key indications for polysomnography and present scalable, existing alternatives for assessment of OSA in the hospital or home setting, using polygraphy, oximetry or contactless sleep monitoring.","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"30 1","pages":""},"PeriodicalIF":5.8,"publicationDate":"2024-04-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140577448","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Positive end-expiratory pressure in chronic care of children with obstructive sleep apnoea","authors":"Brigitte Fauroux ,&nbsp;Meryl Vedrenne-Cloquet","doi":"10.1016/j.prrv.2023.01.001","DOIUrl":"10.1016/j.prrv.2023.01.001","url":null,"abstract":"<div><p>Positive end-expiratory pressure (PEEP) consists of the delivery of a constant positive pressure in the airways by means of a noninvasive interface aiming to maintain airway patency throughout the entire respiratory cycle. PEEP is increasingly used in the chronic care of children with anatomical or functional abnormalities of the upper airways to correct severe persistent obstructive sleep apnea despite optimal management which commonly includes adenotonsillectomy<span> in young children<span>. PEEP may be used at any age, due to improvements in equipment and interfaces. Criteria for CPAP/NIV initiation, optimal setting, follow-up and monitoring, as well as weaning criteria have been established by international experts, but validated criteria are lacking. As chronic PEEP is a highly specialised treatment<span>, patients should be managed by an expert pediatric multidisciplinary team.</span></span></span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"49 ","pages":"Pages 2-4"},"PeriodicalIF":5.8,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9179042","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Diagnostic challenges in CFTR-related metabolic syndrome: Where the guidelines fall short","authors":"Erin F. Kallam ,&nbsp;Ajay S. Kasi ,&nbsp;Eileen Barr ,&nbsp;Rachel W. Linnemann ,&nbsp;Lokesh Guglani","doi":"10.1016/j.prrv.2023.08.004","DOIUrl":"10.1016/j.prrv.2023.08.004","url":null,"abstract":"<div><p><span>Newborn screening<span> (NBS) for cystic fibrosis (CF) has enabled earlier diagnosis and has improved nutritional and growth-related outcomes in children with CF. For those with a positive NBS for CF that do not meet the diagnostic criteria for CF, the clinical entity called CFTR-Related </span></span>Metabolic Syndrome (CRMS) or CF Screen- Positive, Inconclusive Diagnosis (CFSPID) is used. Although most children with CRMS remain relatively asymptomatic, studies have shown that between 11% and 48% of these patients may eventually progress to a diagnosis of CF over time. Although the CF Foundation guidelines for CRMS management and European CF Society guidelines for CFSPID have some similarities, there are also some differences. Here, we review challenging case scenarios that highlight remaining gaps in CRMS guidelines, thus supporting the need to update and unify existing guidelines.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"49 ","pages":"Pages 28-33"},"PeriodicalIF":5.8,"publicationDate":"2024-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10137990","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}