Medicolegal issues for the respiratory paediatrician.

The legal profession depends on expert witnesses, and indeed the first time an English Court relied on an expert medical witness was in the 14th century. Asking a specialist to comment on the standard of professional practice expected in their own specialty was first introduced in a 1767 case [1]. This article draws on 20 years of experience in medicolegal work relating to paediatric respiratory medicine. It highlights some of the legal principles that lie behind an expert opinion and what constitutes clinical negligence. It aims to set out lessons for medicolegal experts and clinicians, but also offers some advice to lawyers and parents. Finally, it illustrates some issues that arise more commonly in paediatric respiratory practice.

Isolated bulbar palsy and dysphagia in children with respiratory symptoms.

Oropharyngeal dysphagia can cause chronic aspiration leading to significant respiratory symptoms. When dysphagia is diagnosed, an underlying cause is sought. We present a case series of 15 children diagnosed aged 6 months to 5 years (mean 2y 5mo; 11 males, four females) over a 6-year period, who were found to have an isolated bulbar palsy on genioglossus electromyography, with no accompanying neurological or neurodevelopmental disorder. Eight children had dysphagia but a normal EMG. In those with isolated bulbar palsy, management included thickened fluids (n=13), cooled boiled water (n=1), and nasogastric tube feeding (n=1). Follow-up over 1 to 8 years (mean 5y) showed complete resolution in six children, improvement in four children, and no improvement in five children (including two requiring fluids via a gastrostomy). Eight children no longer had any respiratory symptoms. Isolated bulbar palsy is under-recognized and has not been reported previously as a cause of significant dysphagia in children.

Home Oxygen Therapy for Children. An Official American Thoracic Society Clinical Practice Guideline.

BACKGROUND: Home oxygen therapy is often required in children with chronic respiratory conditions. This document provides an evidence-based clinical practice guideline on the implementation, monitoring, and discontinuation of home oxygen therapy for the pediatric population. METHODS: A multidisciplinary panel identified pertinent questions regarding home oxygen therapy in children, conducted systematic reviews of the relevant literature, and applied the Grading of Recommendations, Assessment, Development, and Evaluation approach to rate the quality of evidence and strength of clinical recommendations. RESULTS: After considering the panel's confidence in the estimated effects, the balance of desirable (benefits) and undesirable (harms and burdens) consequences of treatment, patient values and preferences, cost, and feasibility, recommendations were developed for or against home oxygen therapy specific to pediatric lung and pulmonary vascular diseases. CONCLUSIONS: Although home oxygen therapy is commonly required in the care of children, there is a striking lack of empirical evidence regarding implementation, monitoring, and discontinuation of supplemental oxygen therapy. The panel formulated and provided the rationale for clinical recommendations for home oxygen therapy based on scant empirical evidence, expert opinion, and clinical experience to aid clinicians in the management of these complex pediatric patients and identified important areas for future research.

Inhaled corticosteroids for cystic fibrosis.

BACKGROUND: The reduction of lung inflammation is one of the goals of cystic fibrosis therapy. Inhaled corticosteroids are often used in this respect to treat children and adults with cystic fibrosis. The rationale for this is their potential to reduce lung damage arising from inflammation, as well as their effect on symptomatic wheezing. It is important to establish the current level of evidence for the risks and benefits of inhaled corticosteroids, especially in the light of their known adverse effects on growth. This is an update of a previously published review; however, due to the lack of research in this area, we do not envisage undertaking any further updates. OBJECTIVES: To assess the effectiveness of taking regular inhaled corticosteroids compared to not taking them in children and adults with cystic fibrosis. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We requested information from pharmaceutical companies manufacturing inhaled corticosteroids and authors of identified trials.Date of most recent search of the Group's Trials Register: 19 November 2018. SELECTION CRITERIA: Randomised or quasi-randomised trials, published and unpublished, comparing inhaled corticosteroids to placebo or standard treatment in individuals with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two independent authors assessed methodological quality and risk of bias in trials using established criteria and extracted data using standard pro formas. The quality of the evidence was assessed using the GRADE criteria. MAIN RESULTS: The searches identified 35 citations, of which 27 (representing 13 trials) were eligible for inclusion. These 13 trials reported the use of inhaled corticosteroids in 525 people with cystic fibrosis aged between 6 and 55 years. One was a withdrawal trial in 171 individuals who were already taking inhaled corticosteroids. Methodological quality and risk of bias were difficult to assess from published information.Objective measures of airway function were reported in most trials but were often incomplete and reported at different time points. We found no difference in forced expiratory volume in one second (FEV1) or forced vital capacity (FVC) % predicted in any of the trials, although the quality of the evidence was low due to risks of bias within the included trials and low participant numbers. We are uncertain whether inhaled corticosteroids result in an improvement in exercise tolerance, bronchial hyperreactivity or exacerbations as the quality of the evidence was very low. Data from one trial suggested that inhaled corticosteroids may make little or no difference to quality of life (low-quality evidence).Three trials reported adverse effects, but the quality of the evidence is low and so we are uncertain whether inhaled corticosteroids increase the risk of adverse effects. However, one study did show that growth was adversely affected by high doses of inhaled corticosteroids. AUTHORS' CONCLUSIONS: Evidence from these trials is of low to very low quality and insufficient to establish whether inhaled corticosteroids are beneficial in cystic fibrosis, but withdrawal in those already taking them has been shown to be safe. There is some evidence they may cause harm in terms of growth. It has not been established whether long-term use is beneficial in reducing lung inflammation, which should improve survival, but it is unlikely this will be proven conclusively in a randomised controlled trial.

Pulmonary function deficits in newborn screened infants with cystic fibrosis managed with standard UK care are mild and transient.

With the advent of novel designer molecules for cystic fibrosis (CF) treatment, there is huge need for early-life clinical trial outcomes, such as infant lung function (ILF). We investigated the degree and tracking of ILF abnormality during the first 2 years of life in CF newborn screened infants.Forced expiratory volume in 0.5 s (FEV0.5), lung clearance index (LCI) and plethysmographic functional residual capacity were measured at ∼3 months, 1 year and 2 years in 62 infants with CF and 34 controls.By 2 years there was no significant difference in FEV0.5 z-score between CF and controls, whereas mean LCI z-score was 0.81 (95% CI 0.45-1.17) higher in CF. However, there was no significant association between LCI z-score at 2 years with either 3-month or 1-year results. Despite minimal average group changes in any ILF outcome during the second year of life, marked within-subject changes occurred. No child had abnormal LCI or FEV0.5 on all test occasions, precluding the ability to identify "high-risk" infants in early life.In conclusion, changes in lung function are mild and transient during the first 2 years of life in newborn screened infants with CF when managed according to a standardised UK treatment protocol. Their potential role in tracking disease to later childhood will be ascertained by ongoing follow-up.

Hypoxic Challenge Test for airflight in children with respiratory disease.

During airflight, cabins are pressurised to 8000ft (2438m) leading to an effective FiO2 of 0.15. This leads to a fall in oxygen saturation in all passengers, and especially those with underlying lung disease. The hypoxic challenge test using a body plethysmograph can predict a need for supplemental oxygen during airflight, and the process is described.

Inhaled corticosteroids for cystic fibrosis.

BACKGROUND: Reduction of lung inflammation is one of the goals of cystic fibrosis therapy. Inhaled corticosteroids are often used to treat children and adults with cystic fibrosis. The rationale for this is their potential to reduce lung damage arising from inflammation, as well as their effect on symptomatic wheezing. It is important to establish the current level of evidence for the risks and benefits of inhaled corticosteroids, especially in the light of their known adverse effects on growth. This is an update of a previously published review. OBJECTIVES: To assess the effectiveness of taking regular inhaled corticosteroids, compared to not taking them, in children and adults with cystic fibrosis. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We requested information from pharmaceutical companies manufacturing inhaled corticosteroids and authors of identified trials.Date of most recent search of the Group's Trials Register: 15 August 2016. SELECTION CRITERIA: Randomised or quasi-randomised trials, published and unpublished, comparing inhaled corticosteroids to placebo or standard treatment in individuals with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two independent authors assessed methodological quality and risk of bias in trials using established criteria and extracted data using standard pro formas. MAIN RESULTS: The searches identified 34 citations, of which 26 (representing 13 trials) were eligible for inclusion. These 13 trials reported the use of inhaled corticosteroids in 506 people with cystic fibrosis aged between six and 55 years. One was a withdrawal trial in individuals who were already taking inhaled corticosteroids. Methodological quality and risk of bias were difficult to assess from published information. Many of the risk of bias judgements were unclear due to a lack of available information. Only two trials specified how participants were randomised and less than half of the included trials gave details on how allocation was concealed. Trials were generally judged to have a low risk of bias from blinding, except for two which were open label or did not use a placebo. There were some concerns that a number of trials had not been published in peer-reviewed journals, but the risk of bias from this was unclear. Inclusion criteria varied between trials, as did type and duration of treatment and timing of outcome assessments. Objective measures of airway function were reported in most trials but were often incomplete. Significant benefit has not been conclusively demonstrated. Four trials systematically documented adverse effects and growth was significantly affected in one study using high doses. AUTHORS' CONCLUSIONS: Evidence from these trials is insufficient to establish whether inhaled corticosteroids are beneficial in cystic fibrosis, but withdrawal in those already taking them has been shown to be safe. There is some evidence they may cause harm in terms of growth. It has not been established whether long-term use is beneficial in reducing lung inflammation, which should improve survival, but it is unlikely this will be proven conclusively in a randomised controlled trial.

Critical timing of gastrostomy insertion in a child with cystic fibrosis.

Pulmonary exacerbations and malabsorption in children with cystic fibrosis (CF) can lead to faltering growth and poor weight gain. Children with a higher BMI (body mass index) show a slower decline in lung function. Our specialist CF centre has experienced a death following gastrostomy insertion in a young CF child, despite maximal medical intervention, which has made us reflect on our practice and the urgency with which we discuss the option for a gastrostomy to improve nutrition.

Personalised medicine in cystic fibrosis is unaffordable.

Personalised medicine refers to a tailored approach to treatment of an individual based on molecular analysis of genes, proteins or metabolites, and commonly involves a companion diagnostic test. It usually applies to small subsets of patients, often with rare diseases. In cystic fibrosis (CF), the best example is the CFTR (CF transmembrane conductance regulator) potentiator, ivacaftor, relevant to the 5% of cystic fibrosis patients with the p.Gly551Asp gene mutation. However the cost of personalised medicine is too high, making it unaffordable in the long term for many healthcare systems. Society needs to find a way to make personalised medicine affordable in order to not deny life-changing treatments from patients.

Inhaled corticosteroids for cystic fibrosis.

BACKGROUND: Reduction of lung inflammation is one of the goals of cystic fibrosis therapy. Inhaled corticosteroids are often used to treat children and adults with cystic fibrosis. The rationale for this is their potential to reduce lung damage arising from inflammation, as well as their effect on symptomatic wheezing. It is important to establish the current level of evidence for the risks and benefits of inhaled corticosteroids, especially in the light of their known adverse effects on growth. OBJECTIVES: To assess the effectiveness of taking regular inhaled corticosteroids, compared to not taking them, in children and adults with cystic fibrosis. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We requested information from pharmaceutical companies manufacturing inhaled corticosteroids and authors of identified trials.Date of most recent search of the Group's Trials Register: 17 July 2014. SELECTION CRITERIA: Randomised or quasi-randomised trials, published and unpublished, comparing inhaled corticosteroids to placebo or standard treatment in individuals with cystic fibrosis. DATA COLLECTION AND ANALYSIS: Two independent authors assessed methodological quality and risk of bias in trials using established criteria and extracted data using standard pro formas. MAIN RESULTS: The searches identified 34 citations, of which 26 (representing 13 trials) were eligible for inclusion. These 13 trials reported the use of inhaled corticosteroids in 506 people with cystic fibrosis aged between six and 55 years. One was a withdrawal trial in individuals who were already taking inhaled corticosteroids. Methodological quality and risk of bias were difficult to assess from published information. Many of the risk of bias judgements were unclear due to a lack of available information. Only two trials specified how participants were randomised and less than half of the included trials gave details on how allocation was concealed. Trials were generally judged to have a low risk of bias from blinding, except for two which were open label or did not use a placebo. There were some concerns that a number of trials had not been published in peer-reviewed journals, but the risk of bias from this was unclear. Inclusion criteria varied between trials, as did type and duration of treatment and timing of outcome assessments. Objective measures of airway function were reported in most trials but were often incomplete. Significant benefit has not been conclusively demonstrated. Four trials systematically documented adverse effects and growth was significantly affected in one study using high doses. AUTHORS' CONCLUSIONS: Evidence from these trials is insufficient to establish whether inhaled corticosteroids are beneficial in cystic fibrosis, but withdrawal in those already taking them has been shown to be safe. There is some evidence they may cause harm in terms of growth. It has not been established whether long-term use is beneficial in reducing lung inflammation, which should improve survival, but it is unlikely this will be proven conclusively in a randomised controlled trial.

Haemoptysis: is it really from the lungs? The well child who spits out blood.

Blood appearing in a previously well child's mouth may have many sources, and it should not be assumed to be haemoptysis, that is, coming from the respiratory tract below the larynx. In addition to the lungs and lower airways, consider also the upper airways, the mouth, gastrointestinal tract and cardiovascular conditions. This article discusses the differential diagnosis and appropriate investigations.

Inhaled corticosteroids for cystic fibrosis.

BACKGROUND: Reduction of lung inflammation is one of the goals of cystic fibrosis (CF) therapy. Inhaled corticosteroids (ICS) are often used to treat children and adults with CF. The rationale for this is their potential to reduce lung damage arising from inflammation, as well as their effect on symptomatic wheezing. It is important to establish the current level of evidence for the risks and benefits of ICS, especially in the light of their known adverse effects on growth. OBJECTIVES: To assess the effectiveness of taking regular ICS, compared to not taking them, in children and adults with CF. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We requested information from pharmaceutical companies manufacturing inhaled corticosteroids and authors of identified trials.Date of most recent search of the Group's Trials Register: 03 September 2012. SELECTION CRITERIA: Randomised or quasi-randomised trials, published and unpublished, comparing ICS to placebo or standard treatment in individuals with CF. DATA COLLECTION AND ANALYSIS: Two independent authors assessed methodological quality of trials using established criteria and extracted data using standard pro formas. MAIN RESULTS: The searches identified 34 citations, of which 26 (representing 13 trials) were eligible for inclusion. These 13 trials reported the use of ICS in 506 people with CF aged between 6 and 55 years. One trial was a withdrawal study in individuals who were already taking ICS. Methodological quality was difficult to assess from published information. Inclusion criteria varied between trials, as did type and duration of treatment and timing of outcome assessments. Objective measures of airway function were reported in most trials but were often incomplete. Significant benefit has not been conclusively demonstrated. Four trials systematically documented adverse effects and growth was significantly affected in one study using high doses. AUTHORS' CONCLUSIONS: Evidence from these trials is insufficient to establish whether ICS are beneficial in CF, but withdrawal in those already taking them has been shown to be safe. There is some evidence they may cause harm in terms of growth. It has not been established whether long-term use is beneficial in reducing lung inflammation, which should improve survival, but it is unlikely this will be proven conclusively in a randomised controlled trial.

Resolution of lobe collapse in a child with cystic fibrosis with Mycobacterium abscessus using serial intrabronchial rhDNase.

An eight-year-old girl with cysticfibrosis (CF) developed a left upper lobe collapse failing to resolve withinitial conventional antibiotic treatment, mucolytics and intensifiedphysiotherapy. Mycobacterium abscessus was isolated from her sputum. Bronchoscopy revealed thick viscousmucus plugging of the left upper lobe bronchus with complete obliteration.Three bronchoscopies with saline lavage and Dornase alfa, a rhDNase, at the endof each procedure resulted in removal of this mucus plug and the re-inflationof the affected lobe, with clinical and radiological resolution. The use of flexible bronchoscopy as a 'secondary' treatment with 0.9% saline lavage and instillation of rhDNase isdescribed sparsely in the literature. This is the first reported successfultherapeutic resolution of a lung collapse in a CF patient with Mycobacteriumabscessus, with sequential therapeutic bronchoscopies with instillation ofDornase alfa. This should be considered for lobar collapse in CF not respondingto the standard therapeutic regime.

Initiating Self-Administration of Medicines for inpatients with cystic fibrosis.

INTRODUCTION: Children with cystic fibrosis (CF) take a multitude of therapies at home. Self-Administration of Medicines (SAM) is a scheme whereby the parent/carer and/or older child keep control of their own medicines in hospital. We initiated a scheme and assessed drug errors, cost implications, and parent and nurse satisfaction. METHODS: Following a pilot stage, the SAM protocol was initiated and amended as necessary. Drug errors were analysed from the Datix hospital electronic reporting system. Cost analysis of use of the patents own drugs was carried out. Questionnaires were given to parents and nursing staff. RESULTS: In the initial 10 months, 97 children had 159 admissions, and 60% were deemed suitable for SAM. Drug errors still occurred-33 in 5 years. Cost savings for the hospital over 1 year were £20 022 for 123 admissions. Patient/parent satisfaction was high, and all wished to partake in SAM for further admissions. CONCLUSIONS: The scheme was a success although it took 3 years to bring to fruition. Drug errors still occurred but we were able to amend the protocol appropriately to react to these. Cost savings are an incidental benefit from use of patient's own medication. The SAM scheme is applicable to all children with chronic disease on long term medications when they are in hospital.

Combination antifungal therapy for Scedosporium species in cystic fibrosis.

OBJECTIVE: To evaluate safety and efficacy of oral posaconazole and terbinafine for Lomentospora prolificans and Scedosporium apiospermum in children with cystic fibrosis. METHODS: Retrospective case review. RESULTS: There were five children (four girls), median age 15.0 years; three had S. apiospermum and two had L. prolificans. Treatment duration: median 5 months (range: 5-18 m). In no patient was eradication achieved, with the follow-up range being 6 months to 4 years. Effect on lung function was variable but encouraging. No adverse effects were reported, one child had transient elevation of liver enzymes. CONCLUSIONS: While the combination therapy was well tolerated, it was unsuccessful at eradication.

Prescribing emergency oral steroids in asthma clinics.

We retrospectively reviewed children who had been prescribed emergency oral corticosteroids (OCS) in a routine tertiary paediatric respiratory clinic appointment. We subsequently assessed adherence from prescription uptake of inhaled corticosteroids or combination inhalers in the 6 months prior to the episode. In 2 years, 25 children received 32 courses of prednisolone. Median adherence was 33%, but 28% for those with repeated OCS prescriptions. Prescribing acute OCS in a routine clinic is a red flag for potential poor adherence to preventer therapies, and may also indicate the child has poor perception of the severity of their symptoms. An assessment of adherence should be carried out and help given to the child and their family to improve poor adherence when detected.

Minimal change in structural, functional and inflammatory markers of lung disease in newborn screened infants with cystic fibrosis at one year.

BACKGROUND: With the widespread introduction of newborn screening for cystic fibrosis (CF), there has been considerable emphasis on the need to develop objective markers of lung health that can be used during infancy. We hypothesised that in a newborn screened (NBS) UK cohort, evidence of airway inflammation and infection at one year would be associated with adverse structural and functional outcomes at the same age. METHODS: Infants underwent lung function testing, chest CT scan and bronchoscopy with bronchoalveolar lavage (BAL) at 1 year of age when clinically well. Microbiology cultures were also available from routine cough swabs. RESULTS: 65 infants had lung function, CT and BAL. Mean (SD) lung clearance index and forced expiratory volume in 0.5 s z-scores were 0.9(1.2) and -0.6(1.1) respectively; median Brody II CF-CT air trapping score on chest CT =0 (interquartile range 0-1, maximum possible score 27). Infants isolating any significant pathogen by 1 yr of age had higher LCI z-score (mean difference 0.9; 95%CI:0.4-1.4; p = 0.001) and a trend towards higher air trapping scores on CT (p = 0.06). BAL neutrophil elastase was detectable in 23% (10/43) infants in whom BAL supernatant was available. This did not relate to air trapping score on CT. CONCLUSIONS: In this UK NBS cohort at one year of age, lung and airway damage is much milder and associations between inflammation, abnormal physiology and structural changes were at best weak, contrary to our hypothesis and previously published reports. Continued follow-up will clarify longer term implications of these very mild structural, functional and inflammatory changes.

Diagnosing allergic bronchopulmonary aspergillosis in children with cystic fibrosis.

Allergic bronchopulmonary aspergillosis (ABPA) is an important complication of cystic fibrosis. It is a hypersensitivity reaction to Aspergillus fumigatus, leading to a Th2 CD4 response mediated by the release of specific IgE. If ABPA is not treated early, it can cause severe impairment in lung function and long-term lung damage. Hence, early recognition with a prompt diagnosis is important. Due to clinical and radiological features of ABPA overlapping with those of bacterial or viral pulmonary exacerbations in cystic fibrosis, diagnosis can sometimes be difficult. Specific criteria for making the diagnosis of ABPA have been suggested. Newer serological tests, such as specific IgE to recombinant allergens and the detection of thymus- and activation-regulated chemokine, are being developed to improve early detection and monitoring of ABPA with greater sensitivity and specificity.

Inhaled corticosteroids for cystic fibrosis.

BACKGROUND: Reduction of lung inflammation is one of the goals of cystic fibrosis (CF) therapy. Inhaled corticosteroids (ICS) are often used to treat children and adults with CF. The rationale for this is their potential to reduce lung damage arising from inflammation, as well as their effect on symptomatic wheezing. It is important to establish the current level of evidence for the risks and benefits of ICS, especially in the light of their known adverse effects on growth. OBJECTIVES: To assess the effectiveness of taking regular ICS, compared to not taking them, in children and adults with CF. SEARCH STRATEGY: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register, comprising references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We requested information from pharmaceutical companies manufacturing inhaled corticosteroids and authors of identified trials.Date of most recent search of the Group's Trials Register: June 2008. SELECTION CRITERIA: Randomised or quasi-randomised trials, published and unpublished, comparing ICS to placebo or standard treatment in individuals with CF. DATA COLLECTION AND ANALYSIS: Two independent authors assessed methodological quality of trials using established criteria and extracted data using standard pro formas. MAIN RESULTS: Thirty citations were identified by the searches, of which 25, representing 13 trials were eligible for inclusion. These 13 trials reported the use of ICS in 506 people with CF aged between 6 and 55 years. One trial was a withdrawal study in individuals who were already taking ICS. Methodological quality was difficult to assess from published information. Inclusion criteria varied between trials, as did type and duration of treatment and timing of outcome assessments. Objective measures of airway function were reported in most trials but were often incomplete. Significant benefit has not been conclusively demonstrated. Four trials systematically documented adverse effects and growth was significantly affected in one study using high doses. AUTHORS' CONCLUSIONS: Evidence from these trials is insufficient to establish whether ICS are beneficial in CF, but withdrawal in those already taking them has been shown to be safe. There is some evidence they may cause harm in terms of growth. It has not been established whether long-term use is beneficial in reducing lung inflammation, which should improve survival, but it is unlikely this will be proven conclusively in a randomised controlled trial.

Domiciliary oxygen for children.

Domiciliary oxygen is used increasingly in pediatric practice, and the largest patient group to receive it is ex-premature babies with chronic neonatal lung disease. Because of a scarcity of good evidence to inform clinicians, there is a lack of consensus over many issues, even those as fundamental as the optimum target oxygen saturation. Nevertheless, many children benefit from receiving supplemental oxygen at home, particularly because it helps to keep them out of the hospital.