Bernadette J. Prentice , Kathryn J. Potter , Adèle Coriati , Valérie Boudreau , Leah Rusnell , Tamizan Kherani , Peter A. Senior , Shihab Hameed , Rémi Rabasa-Lhoret
{"title":"Cystic Fibrosis-Related Diabetes: Clinical approach and knowledge gaps","authors":"Bernadette J. Prentice , Kathryn J. Potter , Adèle Coriati , Valérie Boudreau , Leah Rusnell , Tamizan Kherani , Peter A. Senior , Shihab Hameed , Rémi Rabasa-Lhoret","doi":"10.1016/j.prrv.2022.10.001","DOIUrl":"10.1016/j.prrv.2022.10.001","url":null,"abstract":"<div><p><span>Cystic Fibrosis-Related Diabetes (CFRD) is a unique type of diabetes mellitus that shares some features with both type 1 and type 2 diabetes. Yet, its distinguishing feature of acute pulmonary complications associated with hyperglycemia<span> and the catabolic metabolism associated with a relative insulin deficiency<span> poses challenges to the application of traditional definitions and treatments for diabetes mellitus. People with CF (pwCF) undergo rigorous annual screening starting at age 10, a process that is challenging for patients and limited by sensitivity, specificity, and reproducibility. As pwCF continue to live longer, over 50% are expected to develop CFRD over their lifetime, including up to 20% of adolescents. Increasing numbers of people with CFRD will make this disease increasingly relevant to diabetes practitioners. Evidence-guided practice in CFRD care is limited by small and short studies. Our current understanding of CFRD may change significantly with the recent introduction of CF Transmembrane Regulator (CFTR) modulator medications. This review will explore current challenges in the diagnosis and </span></span></span>management of CFRD<span>, specifically highlighting knowledge gaps in the pathophysiology of CFRD, optimal screening methods, priorities for research and provide guidance with regards to screening, diagnosis, and treatment.</span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 3-11"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10037532","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}
Charlotte Wells , Ioannis Makariou , Nicki Barker , Ravi Thevasagayam , Samatha Sonnappa
{"title":"Exercise induced laryngeal obstruction (EILO) in children and young people: Approaches to assessment and management","authors":"Charlotte Wells , Ioannis Makariou , Nicki Barker , Ravi Thevasagayam , Samatha Sonnappa","doi":"10.1016/j.prrv.2023.04.003","DOIUrl":"10.1016/j.prrv.2023.04.003","url":null,"abstract":"<div><p>Exercise Induced Laryngeal Obstruction (EILO) is characterised by breathlessness, cough and/or noisy breathing particularly during high intensity exercise. EILO is a subcategory of inducible laryngeal obstruction where exercise is the trigger that provokes inappropriate transient glottic or supraglottic narrowing. It is a common condition affecting 5.7–7.5% of the general population and is a key differential diagnosis for young athletes presenting with exercise related dyspnoea where prevalence rates go as high as 34%. Although the condition has been recognised for a long time, little attention, and awareness of the condition results in many young people dropping out of sporting participation due to troublesome symptoms. With evolving understanding of the condition, diagnostic tests and interventions, this review looks to present the current available evidence and best practice when managing young people with EILO.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 37-48"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10057971","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":"Psychosocial needs and interventions for young children with cystic fibrosis and their families","authors":"Stella Li , Tonia Douglas , Dominic A. Fitzgerald","doi":"10.1016/j.prrv.2023.04.002","DOIUrl":"10.1016/j.prrv.2023.04.002","url":null,"abstract":"<div><p><span>This review summarises the experiences of young children and their families living with CF during the first five years of life following </span>NBS<span> diagnosis, as well as the options of psychosocial support available to them. We present strategies embedded within routine CF care that focus on prevention, screening, and intervention for psychosocial health and wellbeing that constitute essential components of multidisciplinary care in infancy and early childhood.</span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 30-36"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9675555","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}
Nicholas Bolden , Joshua Chang Mell , Jennifer Bouso Logan , Paul J. Planet
{"title":"Phylogenomics of nontuberculous mycobacteria respiratory infections in people with cystic fibrosis","authors":"Nicholas Bolden , Joshua Chang Mell , Jennifer Bouso Logan , Paul J. Planet","doi":"10.1016/j.prrv.2023.02.001","DOIUrl":"10.1016/j.prrv.2023.02.001","url":null,"abstract":"<div><p>Nontuberculous mycobacteria (NTM) can cause severe pulmonary disease in people with cystic fibrosis (pwCF). These infections present unique challenges for diagnosis and treatment, prompting a recent interest in understanding NTM transmission and pathogenesis during chronic infection. Major gaps remain in our knowledge regarding basic pathogenesis, immune evasion strategies, population dynamics, recombination potential, and the evolutionary implications of host and antibiotic pressures of long-term NTM infections in pwCF. Phylogenomic techniques have emerged as an important tool for tracking global patterns of transmission and are beginning to be used to ask fundamental biological questions about adaptation to the host during pathogenesis. In this review, we discuss the burden of NTM lung disease (NTM-LD), highlight the use of phylogenomics in NTM research, and address the clinical implications associated with these studies.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 63-70"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9679768","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}
Bernadette Prentice , Michael Nicholson , Grace Y. Lam
{"title":"Cystic fibrosis related diabetes (CFRD) in the era of modulators: A scoping review","authors":"Bernadette Prentice , Michael Nicholson , Grace Y. Lam","doi":"10.1016/j.prrv.2022.11.005","DOIUrl":"10.1016/j.prrv.2022.11.005","url":null,"abstract":"<div><p>Cystic fibrosis-related diabetes (CFRD) is a common complication of CF that increases in incidence as patients age. Poor glycemic control has been shown to negatively impact lung function and weight, resulting in higher risk of recurrent pulmonary exacerbations. With the advent of highly effective modulator therapies (HEMT), patients with CF are living longer and healthier lives. Consequently, CFRD and its microvascular complications are rising in prominence, becoming one of the most urgent clinical concerns. As HEMT were developed with the primary focus of improving pulmonary outcomes, it is not clear from the original phase III studies what the short- or long-term benefits of modulators might be on CFRD development and trajectory. In this review, we will examine the pathophysiology of CFRD, summarize and synthesize the available evidence of HEMT impact on CFRD and describe the emerging research needs in this field.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 23-29"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9672830","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}
Megan Sheppard , Hiran Selvadurai , Paul D. Robinson , Chetan Pandit , S. Murthy Chennapragada , Dominic A. Fitzgerald
{"title":"Approaches to the management of haemoptysis in young people with cystic fibrosis","authors":"Megan Sheppard , Hiran Selvadurai , Paul D. Robinson , Chetan Pandit , S. Murthy Chennapragada , Dominic A. Fitzgerald","doi":"10.1016/j.prrv.2022.10.002","DOIUrl":"10.1016/j.prrv.2022.10.002","url":null,"abstract":"<div><p><span><span>Haemoptysis occurs in up to 25 % of young people with </span>Cystic fibrosis<span> (CF) [1]. We undertook a literature review and described the management approach to haemoptysis in CF between 2010 and 2020 at an Australian tertiary paediatric centre, The Children’s Hospital Westmead, Sydney, New South Wales, using a retrospective review of the </span></span>medical records<span> which identified 67 episodes. Sixty episodes met inclusion criteria, including 31 patients. Using the US CF Foundation guidelines, episodes were classified as scant (53.3 %), moderate (38.3 %) or massive (8.3 %). Fifty-two percent of patients were female, mean age at presentation was 15.4 years (SD+/- 2.4) and 58 % were homozygous for the Fdel508 genotype. Twelve episodes (9 patients) required bronchial artery embolization (BAE). BAE was used in all cases of massive haemoptysis 5/5 (100 %), 6/23 (22 %) episodes of moderate and 1/32 (3 %) episode of scant haemoptysis as an elective procedure for recurrent haemoptysis. Our literature review and institutional experience highlights the need for up-to-date management guidelines in the management of haemoptysis in Cystic Fibrosis. Based on our experience, we provide a proposed algorithm to help guide the management of haemoptysis in CF.</span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 17-22"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9679227","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}
Brigitte Fauroux , Karen Waters , Joanna E. MacLean
{"title":"Sleep in children and young adults with cystic fibrosis","authors":"Brigitte Fauroux , Karen Waters , Joanna E. MacLean","doi":"10.1016/j.prrv.2021.09.006","DOIUrl":"10.1016/j.prrv.2021.09.006","url":null,"abstract":"<div><p><span><span><span><span>Large gains have been made in the management of respiratory diseases associated with cystic fibrosis (CF). Initial studies evaluating sleep issues in CF focused on respiratory problems of nocturnal </span>hypoxia<span>, alveolar hypoventilation and risk of airway obstruction from </span></span>nasal polyps<span> with treatment<span> evaluations including long term oxygen therapy or noninvasive ventilation in case of nocturnal </span></span></span>hypercapnia. More recent studies include patients whose lung function is better preserved, and have permitted more focus on sleep patterns and sleep quality. This literature identified that reduced sleep duration and poor sleep quality are common and may be explained by chronic pain and cough, frequent stools, gastro-oesophageal reflux, nasal obstruction or sinusitis, and </span>drugs<span><span> such as corticosteroids or beta-agonists. In the teenage years, poor sleep hygiene<span><span>, sleep debt and poor sleep quality are associated with depression, poor academic performance, less physical activity, and a decrease in quality of life. </span>Restless leg syndrome also seems to be common in adult patients with CF. These sleep problems seem more important </span></span>in patients<span> with a low lung function but may also be observed in patients with preserved lung function. The consequences of poor sleep may potentially exaggerate the multi-organ morbidity of CF, such as pain, inflammation, susceptibility to infection, and glucose intolerance, but these aspects are largely under-evaluated. Sleep should be evaluated on a routine basis in CF and prospective studies assessing the benefits of interventions aiming at improving sleep duration and sleep quality urgently needed.</span></span></p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 12-16"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"10054562","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":"Surgical treatment of non-cystic fibrosis bronchiectasis in children and adolescents: A review","authors":"Helena Teresinha Mocelin , Gilberto Bueno Fischer , Júlia Danezi Piccini , Renata Baú , Cristiano Feijó Andrade , Janice Luisa Lukrafka","doi":"10.1016/j.prrv.2022.11.003","DOIUrl":"10.1016/j.prrv.2022.11.003","url":null,"abstract":"<div><h3>Objective</h3><p>To discuss surgery for non-cystic fibrosis [CF] bronchiectasis in children and adolescents.</p></div><div><h3>Sources</h3><p>Non-systematic review including articles in English, mainly from the last 5 years.</p></div><div><h3>Summary of findings</h3><p>In this review, we present that in low- and middle-income countries [LMIC] clinical treatment fails in around 20% of cases due to low socioeconomic status and poor adherence. This causes the disease to progress and require surgery. We emphasize that the indications for surgery are not well defined and must be considered on an individual basis. The surgical treatment of bronchiectasis in children may be indicated in selected cases; especially in localized disease with frequent exacerbations despite an optimized clinical approach. Surgery can improve quality of life [QoL] and reduce exacerbations. It has few postoperative complications and low morbidity and mortality. Finally, we propose an algorithm for managing bronchiectasis, which takes into account LMIC settings with limited resources.</p></div><div><h3>Conclusion</h3><p>We conclude that in LMICs, surgery is a treatment strategy for selected children/adolescents with bronchiectasis.</p></div>","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 57-62"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9680731","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":"Editorial: Optimism grows after 10 years of modulator therapies in Cystic Fibrosis","authors":"Dominic A. Fitzgerald","doi":"10.1016/j.prrv.2023.05.004","DOIUrl":"10.1016/j.prrv.2023.05.004","url":null,"abstract":"","PeriodicalId":19658,"journal":{"name":"Paediatric Respiratory Reviews","volume":"46 ","pages":"Pages 1-2"},"PeriodicalIF":5.8,"publicationDate":"2023-06-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"9687744","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}