Pub Date : 2024-09-01DOI: 10.1016/j.prrv.2023.12.004
Maternal asthma affects up to 17% of pregnancies and is associated with adverse infant, childhood, and adult respiratory outcomes, including increased risks of neonatal respiratory distress syndrome, childhood wheeze and asthma. In addition to genetics, these poor outcomes are likely due to the mediating influence of maternal asthma on the in-utero environment, altering fetal lung and immune development and predisposing the offspring to later lung disease. Maternal asthma may impair glucocorticoid signalling in the fetus, a process critical for lung maturation, and increase fetal exposure to proinflammatory cytokines. Therefore, interventions to control maternal asthma, increase glucocorticoid signalling in the fetal lung, or Vitamin A, C, and D supplementation to improve alveologenesis and surfactant production may be beneficial for later lung function. This review highlights potential mechanisms underlying maternal asthma and offspring respiratory morbidities and describes how pregnancy interventions can promote optimal fetal lung development in babies of asthmatic mothers.
产妇哮喘影响到高达 17% 的孕妇,并与婴儿、儿童和成人呼吸系统的不良后果有关,包括新生儿呼吸窘迫综合征、儿童喘息和哮喘的风险增加。除遗传因素外,这些不良后果很可能是由于母体哮喘对子宫内环境的介导影响,改变了胎儿肺部和免疫系统的发育,使后代日后易患肺部疾病。母体哮喘可能会损害胎儿体内糖皮质激素信号的传递(这是肺成熟的关键过程),并增加胎儿暴露于促炎细胞因子的机会。因此,采取干预措施控制母体哮喘、增加胎儿肺中的糖皮质激素信号,或补充维生素 A、C 和 D 以改善肺泡生成和表面活性物质的产生,可能对日后的肺功能有益。本综述强调了母体哮喘和后代呼吸系统疾病的潜在机制,并介绍了妊娠干预措施如何促进哮喘母亲的胎儿肺部发育达到最佳状态。
{"title":"The impact of maternal asthma on the fetal lung: Outcomes, mechanisms and interventions","authors":"","doi":"10.1016/j.prrv.2023.12.004","DOIUrl":"10.1016/j.prrv.2023.12.004","url":null,"abstract":"<div><p>Maternal asthma affects up to 17% of pregnancies and is associated with adverse infant, childhood, and adult respiratory outcomes, including increased risks of neonatal respiratory distress syndrome, childhood wheeze and asthma. In addition to genetics, these poor outcomes are likely due to the mediating influence of maternal asthma on the <em>in-utero</em> environment, altering fetal lung and immune development and predisposing the offspring to later lung disease. Maternal asthma may impair glucocorticoid signalling in the fetus, a process critical for lung maturation, and increase fetal exposure to proinflammatory cytokines. Therefore, interventions to control maternal asthma, increase glucocorticoid signalling in the fetal lung, or Vitamin A, C, and D supplementation to improve alveologenesis and surfactant production may be beneficial for later lung function. This review highlights potential mechanisms underlying maternal asthma and offspring respiratory morbidities and describes how pregnancy interventions can promote optimal fetal lung development in babies of asthmatic mothers.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054223000866/pdfft?md5=1add1cb172faa202e6b911055c66cf87&pid=1-s2.0-S1526054223000866-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139020502","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}
Pub Date : 2024-09-01DOI: 10.1016/j.prrv.2024.01.001
Objectives
Aim of this study was to identify risk factors for a progression to cystic fibrosis (CF) in individuals detected as CF Screening Positive, Inconclusive Diagnosis (CFSPID).
Methods
This is a systematic review through literature databases (2015–2023). Blood immunoreactive trypsinogen (b-IRT) values, CFTR genotype, sweat chloride (SC) values, isolation of Pseudomonas aeruginosa (Pa) from respiratory samples, Lung Clearance Index (LCI) values in CFSPIDs who converted to CF (CFSPID > CF) and age at CF transition were assessed.
Results
Percentage of CFSPID > CF varies from 5.3 % to 44 %. Presence of one CF-causing CFTR variant in trans with a variant with variable clinical consequences (VVCC), an initial SC ≥ 40 mmol/L, an increase of SC > 2.5 mmol/L/year and recurrent isolation of pseudomonas aeruginosa (Pa) from airway samples could allow identification of subjects at risk of progression to CF.
Conclusions
CFSPIDs with CF causing variant/VVCC genotype and first SC in the higher borderline range may require more frequent and prolonged clinical follow-up.
{"title":"Biochemical and genetic tools to predict the progression to Cystic Fibrosis in CRMS/CFSPID subjects: A systematic review","authors":"","doi":"10.1016/j.prrv.2024.01.001","DOIUrl":"10.1016/j.prrv.2024.01.001","url":null,"abstract":"<div><h3>Objectives</h3><p>Aim of this study was to identify risk factors for a progression to cystic fibrosis (CF) in individuals detected as CF Screening Positive, Inconclusive Diagnosis (CFSPID).</p></div><div><h3>Methods</h3><p>This is a systematic review through literature databases (2015–2023). Blood immunoreactive trypsinogen (b-IRT) values, <em>CFTR</em> genotype, sweat chloride (SC) values, isolation of <em>Pseudomonas aeruginosa</em> (Pa) from respiratory samples, Lung Clearance Index (LCI) values in CFSPIDs who converted to CF (CFSPID > CF) and age at CF transition were assessed.</p></div><div><h3>Results</h3><p>Percentage of CFSPID > CF varies from 5.3 % to 44 %. Presence of one CF-causing <em>CFTR</em> variant in trans with a variant with variable clinical consequences (VVCC), an initial SC ≥ 40 mmol/L, an increase of SC > 2.5 mmol/L/year and recurrent isolation of pseudomonas aeruginosa (Pa) from airway samples could allow identification of subjects at risk of progression to CF.</p></div><div><h3>Conclusions</h3><p>CFSPIDs with CF causing variant/VVCC genotype and first SC in the higher borderline range may require more frequent and prolonged clinical follow-up.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.sciencedirect.com/science/article/pii/S1526054224000022/pdfft?md5=3444d1ed965aa205ceb61fe210d668de&pid=1-s2.0-S1526054224000022-main.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139498422","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}
Pub Date : 2024-08-16DOI: 10.1016/j.prrv.2024.08.001
Rebecca Dobra, Sarah Carroll, Jane C. Davies, Fiona Dowdall, Alistair Duff, Anna Elderton, Anna M. Georgiopoulos, Rachel Massey-Chase, Paul McNally, Michèle Puckey, Susan Madge
Cystic fibrosis (CF) is traditionally associated with considerable and progressive multisystem pathology, onerous treatment burden, complex psychosocial challenges, and reduced life-expectancy .This decade has seen transformative change in management for many, but not all, people with CF. The most notable change comes from Cystic Fibrosis Transmembrane Receptor (CFTR) modulators, which bring significant benefits for people who are eligible for, and able to access, them . However alongside, or perhaps because of, this exciting progress, the past few years have also brought important novel challenges to the psychosocial wellbeing of people with CF.
{"title":"Exploring the complexity of cystic fibrosis (CF) and psychosocial wellbeing in the 2020s: Current and future challenges","authors":"Rebecca Dobra, Sarah Carroll, Jane C. Davies, Fiona Dowdall, Alistair Duff, Anna Elderton, Anna M. Georgiopoulos, Rachel Massey-Chase, Paul McNally, Michèle Puckey, Susan Madge","doi":"10.1016/j.prrv.2024.08.001","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.08.001","url":null,"abstract":"Cystic fibrosis (CF) is traditionally associated with considerable and progressive multisystem pathology, onerous treatment burden, complex psychosocial challenges, and reduced life-expectancy .This decade has seen transformative change in management for many, but not all, people with CF. The most notable change comes from Cystic Fibrosis Transmembrane Receptor (CFTR) modulators, which bring significant benefits for people who are eligible for, and able to access, them . However alongside, or perhaps because of, this exciting progress, the past few years have also brought important novel challenges to the psychosocial wellbeing of people with CF.","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-08-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142224056","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}
Pub Date : 2024-08-02DOI: 10.1016/j.prrv.2024.07.003
Yoshua Selvadurai, Emily R Le Fevre, Jonathan Mervis, Dominic A Fitzgerald
Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant condition characterised by small telangiectasias and larger multisystem arteriovenous malformations (AVMs). Common sites of AVMs include in the nose, lungs, brain and liver. These lesions are prone to rupture, leading to complications including recurrent epistaxis and significant haemorrhage. Pulmonary hypertension (PH) can also occur. This review presents an update on the genetics, clinical manifestations, management options, and screening recommendations for children with HHT.
{"title":"Hereditary haemorrhagic telangiectasia: A primer for the paediatrician.","authors":"Yoshua Selvadurai, Emily R Le Fevre, Jonathan Mervis, Dominic A Fitzgerald","doi":"10.1016/j.prrv.2024.07.003","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.07.003","url":null,"abstract":"<p><p>Hereditary haemorrhagic telangiectasia (HHT) is an autosomal dominant condition characterised by small telangiectasias and larger multisystem arteriovenous malformations (AVMs). Common sites of AVMs include in the nose, lungs, brain and liver. These lesions are prone to rupture, leading to complications including recurrent epistaxis and significant haemorrhage. Pulmonary hypertension (PH) can also occur. This review presents an update on the genetics, clinical manifestations, management options, and screening recommendations for children with HHT.</p>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-08-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142110526","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}
Climate change has significant consequences for children's respiratory health. Rising temperatures and extreme weather events increase children's exposure to allergens, mould, and air pollutants. Children are particularly vulnerable to these airborne particles due to their higher ventilation per unit of body weight, more frequent mouth breathing, and outdoor activities. Children with asthma and cystic fibrosis are at particularly high risk, with increased risks of exacerbation, but the effects of climate change could also be observed in the general population, with a risk of impaired lung development and growth. Mitigation measures, including reducing greenhouse gas emissions by healthcare professionals and healthcare systems, and adaptation measures, such as limiting outdoor activities during pollution peaks, are essential to preserve children's respiratory health. The mobilisation of society as a whole, including paediatricians, is crucial to limit the impact of climate change on children's respiratory health.
{"title":"Climate change and children's respiratory health.","authors":"Camille Bignier, Lucile Havet, Margot Brisoux, Céline Omeiche, Swati Misra, Apolline Gonsard, David Drummond","doi":"10.1016/j.prrv.2024.07.002","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.07.002","url":null,"abstract":"<p><p>Climate change has significant consequences for children's respiratory health. Rising temperatures and extreme weather events increase children's exposure to allergens, mould, and air pollutants. Children are particularly vulnerable to these airborne particles due to their higher ventilation per unit of body weight, more frequent mouth breathing, and outdoor activities. Children with asthma and cystic fibrosis are at particularly high risk, with increased risks of exacerbation, but the effects of climate change could also be observed in the general population, with a risk of impaired lung development and growth. Mitigation measures, including reducing greenhouse gas emissions by healthcare professionals and healthcare systems, and adaptation measures, such as limiting outdoor activities during pollution peaks, are essential to preserve children's respiratory health. The mobilisation of society as a whole, including paediatricians, is crucial to limit the impact of climate change on children's respiratory health.</p>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-07-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141897945","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":"Evaluation of vitamin D in the diagnosis of infants with respiratory distress, the clinical value: A systematic review and meta-analysis","authors":"Hassan Boskabadi, Maryam Zakarihamidi, Hassan Mehrad-Majd, Sahar Ghoflchi","doi":"10.1016/j.prrv.2024.06.005","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.06.005","url":null,"abstract":"","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141710982","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}
Pub Date : 2024-07-01DOI: 10.1016/j.prrv.2024.07.001
E. Nkereuwem, Sheila Ageiwaa Owusu, Victory Fabian Edem, Beate Kampmann, T. Togun
{"title":"Post-tuberculosis lung disease in children and adolescents: A scoping review of definitions, measuring tools, and research gaps","authors":"E. Nkereuwem, Sheila Ageiwaa Owusu, Victory Fabian Edem, Beate Kampmann, T. Togun","doi":"10.1016/j.prrv.2024.07.001","DOIUrl":"https://doi.org/10.1016/j.prrv.2024.07.001","url":null,"abstract":"","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":4.7,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141843666","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}
Pub Date : 2024-06-01DOI: 10.1016/j.prrv.2023.11.002
Olivia Jarrett , Soputhirith Seng , Dominic A. Fitzgerald
Melioidosis is a tropical infectious disease caused by the saprophytic gram-negative bacterium Burkholderia pseudomallei. Despite the infection being endemic in southeast Asia and northern Australia, the broad clinical presentations and diagnostic difficulties limit its early detection, particularly in children. Melioidosis more commonly affects the immunocompromised and adults. Melioidosis is increasingly being diagnosed around the world and whole-genome sequencing indicates that these cases are not linked with travel to endemic areas. Research has concentrated on the adult population with limited experience reported in the care of this uncommon, but potentially fatal condition in children presenting with bacteraemia and pneumonia.
{"title":"Paediatric melioidosis","authors":"Olivia Jarrett , Soputhirith Seng , Dominic A. Fitzgerald","doi":"10.1016/j.prrv.2023.11.002","DOIUrl":"10.1016/j.prrv.2023.11.002","url":null,"abstract":"<div><p>Melioidosis is a tropical infectious disease caused by the saprophytic gram-negative bacterium <span><em>Burkholderia pseudomallei</em></span><span>. Despite the infection being endemic in southeast Asia and northern Australia, the broad clinical presentations and diagnostic difficulties limit its early detection, particularly in children. Melioidosis more commonly affects the immunocompromised and adults. Melioidosis is increasingly being diagnosed around the world and whole-genome sequencing indicates that these cases are not linked with travel to endemic areas. Research has concentrated on the adult population with limited experience reported in the care of this uncommon, but potentially fatal condition in children presenting with bacteraemia and pneumonia.</span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"138516921","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}
Pub Date : 2024-06-01DOI: 10.1016/j.prrv.2023.12.006
Megha Sharma , Andrew W Brown , Nicholas M. Powell , Narasimhan Rajaram , Lauren Tong , Peter M. Mourani , Mario Schootman
Race-based and skin pigmentation-related inaccuracies in pulse oximetry have recently been highlighted in several large electronic health record-based retrospective cohort studies across diverse patient populations and healthcare settings. Overestimation of oxygen saturation by pulse oximeters, particularly in hypoxic states, is disparately higher in Black compared to other racial groups. Compared to adult literature, pediatric studies are relatively few and mostly reliant on birth certificates or maternal race-based classification of comparison groups. Neonates, infants, and young children are particularly susceptible to the adverse life-long consequences of hypoxia and hyperoxia. Successful neonatal resuscitation, precise monitoring of preterm and term neonates with predominantly lung pathology, screening for congenital heart defects, and critical decisions on home oxygen, ventilator support and medication therapies, are only a few examples of situations that are highly reliant on the accuracy of pulse oximetry. Undetected hypoxia, especially if systematically different in certain racial groups may delay appropriate therapies and may further perpetuate health care disparities. The role of biological factors that may differ between racial groups, particularly skin pigmentation that may contribute to biased pulse oximeter readings needs further evaluation. Developmental and maturational changes in skin physiology and pigmentation, and its interaction with the operating principles of pulse oximetry need further study. Importantly, clinicians should recognize the limitations of pulse oximetry and use additional objective measures of oxygenation (like co-oximetry measured arterial oxygen saturation) where hypoxia is a concern.
{"title":"Racial and skin color mediated disparities in pulse oximetry in infants and young children","authors":"Megha Sharma , Andrew W Brown , Nicholas M. Powell , Narasimhan Rajaram , Lauren Tong , Peter M. Mourani , Mario Schootman","doi":"10.1016/j.prrv.2023.12.006","DOIUrl":"10.1016/j.prrv.2023.12.006","url":null,"abstract":"<div><p><span><span><span>Race-based and skin pigmentation-related inaccuracies in pulse oximetry have recently been highlighted in several large electronic health record-based </span>retrospective cohort studies across diverse patient populations and healthcare settings. Overestimation of oxygen saturation by pulse oximeters, particularly in hypoxic states, is disparately higher in Black compared to other racial groups. Compared to adult literature, pediatric studies are relatively few and mostly reliant on birth certificates or maternal race-based classification of comparison groups. Neonates, infants, and </span>young children<span><span> are particularly susceptible to the adverse life-long consequences of hypoxia and </span>hyperoxia<span>. Successful neonatal resuscitation, precise monitoring of preterm and term neonates with predominantly lung pathology, screening for </span></span></span>congenital heart defects<span><span>, and critical decisions on home oxygen, ventilator support and medication therapies, are only a few examples of situations that are highly reliant on the accuracy of pulse oximetry. Undetected hypoxia, especially if systematically different in certain racial groups may delay appropriate therapies and may further perpetuate </span>health care disparities<span>. The role of biological factors that may differ between racial groups, particularly skin pigmentation that may contribute to biased pulse oximeter readings needs further evaluation. Developmental and maturational changes in skin physiology and pigmentation, and its interaction with the operating principles of pulse oximetry need further study. Importantly, clinicians should recognize the limitations of pulse oximetry and use additional objective measures of oxygenation (like co-oximetry measured arterial oxygen saturation) where hypoxia is a concern.</span></span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":null,"pages":null},"PeriodicalIF":5.8,"publicationDate":"2024-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"139376187","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}
Pub Date : 2024-06-01DOI: 10.1016/j.prrv.2023.12.005
Jessica A. Eldredge , Mark R. Oliver , Chee Y. Ooi
Summary
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.
{"title":"Cystic fibrosis liver disease in the new era of cystic fibrosis transmembrane conductance regulator (CFTR) modulators","authors":"Jessica A. Eldredge , Mark R. Oliver , 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":null,"pages":null},"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}