A comparison of peak cough flow and peak expiratory flow in children with neuromuscular disorders.

Spirometry and peak cough flow testing (PCF) are commonly used in the respiratory assessment of children with a neuromuscular disorder (NMD). Testing uses two different machines, increases laboratory time, costs and resource utilisation. No studies have assessed the correlation between peak expiratory flow (PEF) obtained from spirometry and PCF in children with NMD using one device. An audit of children with a NMD managed at the Children's Hospital at Westmead in 2022-2024 aged < 20 years who performed spirometry and PCF testing on the same device (Vyaire Body BoxTM, Ultrasonic flow meter-based, or Vyaire PneumotachographTM, Pneumotach flow meter-based; Germany) was conducted to assess the correlation between PCF and PEF. Fifty-one sets of testing were identified, and 40 subjects (9F) had reproducible testing and were included. Median (range) age was 14.95 (7.20-19.00) years. Median PEF (L/min) was 4.05 (1.22-10.26) and median PCF (L/min) was 4.29 (1.69-10.82). PEF and PCF had a strong Pearson's correlation coefficient, (R = 0.97, p = 0.03). The coefficient of determination was 0.93. If laboratory resources permit, spirometry should be the test of choice for children with NMD. On average, spirometry required multiple practices to achieve reproducibility to meet ATS/ERS standards. PCF testing can be utilised for children where performing technically acceptable spirometry is not possible.

Can postural changes in spirometry in children with Duchenne muscular dystrophy predict sleep hypoventilation?

AIM: To explore the relationship between postural changes in lung function and polysomnography (PSG) in children with Duchenne muscular dystrophy (DMD). METHODS: In this prospective cross-sectional study, children with DMD performed spirometry in sitting and supine positions. A control group of age and gender matched healthy children also underwent postural lung function testing. PSG was performed within six months of spirometry. RESULTS: Seventeen children with DMD, aged 12.3 ± 3 years performed sitting spirometry. 14 (84%) performed acceptable spirometry in the supine position. Mean FEV1sit and FVCsit were 77% (SD ± 22) and 74% (SD ± 20.4) respectively, with mean% ΔFVC(sit-sup) 9% (SD ± 11) (range 2% to 20%), and was significantly greater than healthy controls 4% (n = 30, SD ± 3, P < 0.001). PSG data on the 14 DMD children with acceptable supine spirometry showed total AHI 6.9 ± 5.9/hour (0.3 to 29), obstructive AHI 5.2 ± 4.0/hour (0.2 to 10), and REM AHI 14.1 ± -5.3/hour (0.1 to 34.7). ΔFVC(sit-sup) had poor correlation with hypoventilation on polysomnography. CONCLUSION: Children with DMD and mild restrictive lung disease showed greater postural changes in spirometry than healthy controls but lower supine spirometry was not predictive of sleep hypoventilation.

Respiratory and sleep outcomes in children with SMA treated with nusinersen - real world experience.

It is unclear how improvements in peripheral motor function in children with spinal muscular atrophy (SMA), treated with nusinersen, translate into clinically significant respiratory/sleep outcomes. A retrospective chart review of SMA children at the Sydney Children's Hospital Network was undertaken looking at 2 years before and after receiving their first dose of nusinersen. Polysomnography (PSG), spirometry and clinical data were collected and analysed using paired and unpaired t-tests for PSG parameters and generalised estimating equations for longitudinal lung function data. Forty-eight children (10 Type 1, 23 Type 2, 15 Type 3) at mean age 6.98 yrs (SD 5.25) for nusinersen initiation were included. There was a statistically significant improvement in oxygen nadir during sleep in individuals post nusinersen (mean of 87.9% to 92.3% (95%CI 1.24 - 7.63, p = 0.01)). Based on clinical and PSG findings, 6/21 patients (5 Type 2, 1 Type 3) ceased nocturnal NIV post nusinersen. Non-significant improvements were demonstrated in mean slope for FVC% predicted, FVC Z-score and mean FVC% predicted. Within 2 years of commencing nusinersen, stabilisation of respiratory outcomes occurred. Whilst some of the SMA type 2/3 cohort ceased NIV, there were no statistically significant improvements lung function and most PSG parameters.

Exercise testing for young athletes.

With increasing competitiveness across the sporting landscape, there is a need for more research into monitoring and managing the young athlete, as the needs of a young athlete are vastly different to those of an older athlete who is already established in their respective sport. As the age of sports specialisation seems to decrease, exercise testing in the younger cohort of athletes is crucial for safety and long-term success. This article provides a comprehensive summary of available testing and monitoring methods that can be used to assist young athletes as they mature and attempt to excel in their chosen sport.

Applying lessons learnt from research of child pneumonia management in Vietnam.

Pneumonia is the leading cause of paediatric hospitalisation in Vietnam, placing a huge burden on the health care system. Pneumonia is also the main reason for antibiotic use in children. Unfortunately many hospital admissions for child pneumonia in Vietnam are unnecessary and inappropriate use of antibiotics is common, as in the rest of Asia, with little awareness of its adverse effects. We explored the value of an alternative approach that, instead of focusing on the identification of children with severe bacterial pneumonia, focuses on the identification of children with 'unlikely bacterial pneumonia' to improve patient care and rational antibiotic use. Implementing improved models of care require pragmatic management algorithms that are well validated, but it is ultimately dependent on financial structures, management support and evidence-based training of healthcare providers at all relevant levels. Apart from better case management, sustained reductions in the pneumonia disease burden also require increased emphasis on primary prevention.

European and United Kingdom COVID-19 pandemic experience: The same but different.

The global healthcare landscape has changed dramatically and rapidly in 2020. This has had an impact upon paediatricians and in particular respiratory paediatricians. The effects in Europe, with its mature healthcare system, have been far faster and greater than most authorities anticipated. Within six weeks of COVID-19 being declared a public health emergency by the World Health Organisation [WHO] in China, Europe had become the new epicentre of disease. A pandemic was finally declared by the WHO on March 11th 2020. Continued international travel combined with the slow response of some political leaders and a variable focus on economic rather than health consequences resulted in varying containment strategies in response to the threat of the initial wave of the pandemic. It is likely that this variation has contributed to widely differing outcomes across Europe. Common to all countries was the stark lack of preparations and initial poor co-ordination of responses between levels of government to this unforeseen but not unheralded global health crisis. In this article we highlight the impact of the first wave of the COVID-19 pandemic in Italy, Austria, Germany, and the United Kingdom.

Pro con debates in clinical medicine infection prevention and control in cystic fibrosis: One size fits all? The argument in favour.

This article advocates for a universal approach to infection control measures in cystic fibrosis. The central tenets of infection control include hand hygiene, contact precautions, regular microbiological surveillance and adopting inpatient, outpatient, domestic and social practices to minimise acquisition of common CF pathogens. Infection control measures should be proactive and prospective, assuming all patients harbour aggressive pathogens, and not relying on past culture results. The challenges of implementing these policies include cost, equipment, education, consistency, meticulousness all whilst balancing additional procedures to a busy clinical workload.

Use of non-invasive ventilation in children with congenital tracheal stenosis.

INTRODUCTION: Congenital tracheal stenosis (CTS) is a rare airway condition characterized by complete tracheal rings. Most patients undergo a slide tracheoplasty, which greatly reduces mortality but significant morbidity remains. The assessment of sleep disordered breathing (SDB) and use of non-invasive ventilation (NIV) in these children has not been described. AIM: To describe the presence of SDB and use of NIV in children diagnosed with CTS over a 10-year period (2005-2015). DESIGN: Retrospective case series at a tertiary children's hospital. RESULTS: There were 16 patients identified with CTS with a median [range] age at diagnosis of 2.5 months (0-9 months). One child died in the immediate post-operative period following a slide tracheoplasty, leaving 15 survivors. There were no later deaths during follow-up while using NIV for up to 3 years after surgery. Slide tracheoplasty was undertaken in (12/15) with long-segment tracheal stenosis. 3/15 patients had a short-segment tracheal stenosis and were managed conservatively. The use of NIV occurred in 10/15 (66.67%) patients, all of whom had long-segment CTS. Pre-operative polysomnography (PSG) showed a median (±SD) obstructive apnoea/hypopnoea index (OAHI) of 14.6/hr (±6.2) which reduced to 7.2/hour (±4.2) on NIV prior to slide tracheoplasty. The median oxygen desaturation index (ODI) before NIV use was 15.3 (±19.4) episodes/hour, which reduced to 6.3 (±11) on NIV. The median period of NIV use was 5 [1-24 months] months. CONCLUSION: Patients with CTS have obstructed sleep disordered breathing. Trials of NIV are well-tolerated and improve sleep disordered breathing.

Abnormal preschool Lung Clearance Index (LCI) reflects clinical status and predicts lower spirometry later in childhood in cystic fibrosis.

BACKGROUND: Clinical and prognostic value of preschool Multiple Breath Washout (MBW) remains unclear. METHODS: Initial MBW results (Exhalyzer® D, EcoMedics AG) in preschool Cystic Fibrosis (CF) subjects (age 2-6 years) at a time of clinical stability were compared to (1) concurrent clinical status measures and (2) later spirometry outcomes. Abnormal Lung Clearance Index (LCI) was defined using published reference data (ULN for LCI 8.0). RESULTS: LCI was abnormal in 56% (28/50), with mean (SD) LCI 8.61(1.85) at age 4.71(1.3) years. Abnormal LCI was associated with higher dornase alfa use, previous positive bacterial cultures and pF508.del homozygous genotype. Later spirometry (n = 44; mean (SD) 2.3(0.5) years after MBW) demonstrated that abnormal initial preschool LCI was a strong predictor of lower later spirometry outcomes. CONCLUSION: Abnormal preschool LCI was associated with concurrent measures of clinical status and later spirometry deficits, suggesting early prognostic utility of MBW testing in this age range.

Can daytime measures of lung function predict respiratory failure in children with neuromuscular disease?

Neuromuscular disorders in children are a heterogeneous group of conditions with a variable age of presentation and overlapping clinical manifestations, many of which have progressive respiratory morbidity. Respiratory insufficiency occurs as a consequence of an imbalance between demands on the respiratory system and respiratory muscle capacity. Daytime measures of pulmonary function are used routinely in these children to assess respiratory status and monitor the consequences of the progression of muscle weakness. This review describes the current evidence for daytime pulmonary function tests and their ability to predict imminent respiratory morbidity.

Expert Consensus on the Management of Adverse Events from EGFR Tyrosine Kinase Inhibitors in the UK.

Epidermal growth factor receptor (EGFR) tyrosine kinase inhibitors (TKIs) such as gefitinib, erlotinib, and afatinib are standard-of-care for first-line treatment of EGFR-mutant advanced non-small cell lung cancer (NSCLC). These drugs have a proven benefit in terms of higher response rate, delaying progression and improvement of quality of life over palliative platinum-based chemotherapy. The most common adverse events (AEs) are gastrointestinal (GI) (diarrhoea and stomatitis/mucositis) and cutaneous (rash, dry skin and paronychia). These are usually mild, but if they become moderate or severe, they can have a negative impact on the patient's quality of life (QOL) and lead to dose modifications or drug discontinuation. Appropriate management of AEs, including prophylactic measures, supportive medications, treatment delays and dose reductions, is essential. A consensus meeting of a UK-based multidisciplinary panel composed of medical and clinical oncologists with a special interest in lung cancer, dermatologists, gastroenterologists, lung cancer nurse specialists and oncology pharmacists was held to develop guidelines on prevention and management of cutaneous (rash, dry skin and paronychia) and GI (diarrhoea, stomatitis and mucositis) AEs associated with the administration of EGFR-TKIs. These guidelines detail supportive measures, treatment delays and dose reductions for EGFR-TKIs. Although the focus of the guidelines is to support healthcare professionals in UK clinical practice, it is anticipated that the management strategies proposed will also be applicable in non-UK settings.

Neural response during attentional control and emotion processing predicts improvement after cognitive behavioral therapy in generalized social anxiety disorder.

BACKGROUND: Individuals with generalized social anxiety disorder (gSAD) exhibit attentional bias to salient stimuli, which is reduced in patients whose symptoms improve after treatment, indicating that mechanisms of bias mediate treatment success. Therefore, pre-treatment activity in regions implicated in attentional control over socio-emotional signals (e.g. anterior cingulate cortex, dorsolateral prefrontal cortex) may predict response to cognitive behavioral therapy (CBT), evidence-based psychotherapy for gSAD. METHOD: During functional magnetic resonance imaging, 21 participants with gSAD viewed images comprising a trio of geometric shapes (circles, rectangles or triangles) alongside a trio of faces (angry, fearful or happy) within the same field of view. Attentional control was evaluated with the instruction to 'match shapes', directing attention away from faces, which was contrasted with 'match faces', whereby attention was directed to emotional faces. RESULTS: Whole-brain voxel-wise analyses showed that symptom improvement was predicted by enhanced pre-treatment activity in the presence of emotional face distractors in the dorsal anterior cingulate cortex and dorsal medial prefrontal cortex. Additionally, CBT success was foretold by less activity in the amygdala and/or increased activity in the medial orbitofrontal gyrus during emotion processing. CONCLUSIONS: CBT response was predicted by pre-treatment activity in prefrontal regions and the amygdala. The direction of activity suggests that individuals with intact attentional control in the presence of emotional distractors, regulatory capacity over emotional faces and/or less reactivity to such faces are more likely to benefit from CBT. Findings indicate that baseline neural activity in the context of attentional control and emotion processing may serve as a step towards delineating mechanisms by which CBT exerts its effects.

Screening for obstructive sleep apnea in preschool children with cleft palate.

OBJECTIVE: The objective of this study was to explore the prevalence, range of reported symptoms, and clinical risk factors of obstructive sleep apnea in preschool children with cleft lip and/or palate. DESIGN: Questionnaires were distributed to parents/guardians of all children from birth to 5 years of age who were followed by the cleft clinic. RESULTS: Questionnaire data and cleft classification were available for 248 children, with a mean age of 33.4 months. Obstructive sleep apnea was identified in 31.4% of the children. Only 29.5% of children with obstructive sleep apnea had undergone an investigation of these symptoms. The three most common symptoms reported in children with a questionnaire diagnosis of obstructive sleep apnea were (1) "heavy or loud breathing," (2) "easily distracted," and (3) "on the go" or "driven by a motor." The only clinical risk factor associated with a questionnaire diagnosis of obstructive sleep apnea was the presence of a syndrome (chi(2) = 3.5, p = .05). There were no significant differences in risk of obstructive sleep apnea by age, cleft classification, and surgical status. CONCLUSION: Preschool children with cleft lip and/or palate have a risk of obstructive sleep apnea that is as much as five times that of children without cleft. Obstructive sleep apnea appears to be underrecognized in this group of children. Further research is needed to investigate important risk factors for obstructive sleep apnea in children with cleft lip and/or palate.

The identification of children with cleft palate and sleep disordered breathing using a referral system.

BACKGROUND: Cleft palate is associated with an increased risk of sleep disordered breathing (SDB) but the magnitude of this risk and specific risk factors are unclear. A better understanding of these components of risk will aid the early identification of SDB in this group of children. OBJECTIVE: To describe the clinical characteristics and results of sleep studies undertaken in a cohort of children with cleft palate. Clinical features will be examined to determine potential associations with SDB in this group. METHOD: A retrospective chart review was undertaken to ascertain sleep study results and clinical data for all children with cleft palate. Clinical features of interest included age, gender, syndrome diagnosis, cleft classification, and surgical status. RESULTS: A total of 99 sleep studies were available from 62 children. The sample included a select group of children with cleft palate with features predictive of a high risk of SDB. Baseline sleep study results were consistent with SDB for 87% of children and 28% (15 of 54) of these children demonstrated severe SDB. Uni-variate analysis showed that age, syndrome, and surgical status had significant association with the severity of SDB. On multi-variate analysis only surgical status maintained this association, such that pre-palatoplasty/pharyngoplasty was associated with more severe SDB. Follow-up studies were completed in one-third of the cohort. CONCLUSION: Children with cleft palate appear to have a significant risk of SDB. A prospective study of a population of children with cleft palate is needed to further define the characteristics of this risk and important risk factors.

Outcome of noninvasive ventilation in children with neuromuscular disease.

OBJECTIVE: To assess the effect of institution of noninvasive ventilation (NIV) on clinical outcome and quality of life (QOL) in a cohort of children with severe neuromuscular disorders. METHODS: We reviewed records and obtained clinical data from the year prior to commencing NIV and annually thereafter. Data obtained included diagnosis, patient symptoms, mortality, NIV adverse effects, pulmonary function tests, polysomnographic data, length of hospitalizations, and health care costs. Patients and parents completed questionnaires assessing QOL with NIV and recalling QOL before NIV. RESULTS: Fourteen of 17 (82%) suitable patients were enrolled. Follow-up ranged from 6 to 84 months (median 30). Symptoms of daytime sleepiness (p = 0.003) and headache (p = 0.046) improved after initiation of NIV. Sleep quality assessed by polysomnography also improved. Hospitalization rates (p = 0.002) and health care costs (p = 0.003) decreased. QOL remained stable after NIV, despite disease progression. CONCLUSION: Treatment of respiratory failure, in children with neuromuscular disease, with noninvasive ventilation results in a reduction in symptoms, hospitalizations, and health care costs without adverse effects on quality of life.

Arnold Chiari type 1 malformation presenting with sleep disordered breathing in well children.

Healthy children, aged 3, 9, and 13 years referred with sleep disordered breathing had marked central apnoea and bradypnoea on polysomnography, necessitating the use of non-invasive bilevel ventilation in two cases. Each had normal neurological examinations and an Arnold Chiari type 1 malformation was confirmed on magnetic resonance imaging. All underwent urgent posterior fossa decompression which normalised their sleep breathing.

Positioning for acute respiratory distress in hospitalised infants and children.

BACKGROUND: Because of the association between prone positioning and sudden infant death syndrome SIDS) it is recommended that young infants be placed on their backs (supine). However, the supine position might not be the most appropriate position for infants and children hospitalised with acute respiratory distress. Positioning patients has been proposed as a non-invasive way of increasing oxygenation in adult patients with acute respiratory distress. But, because of substantial differences in respiratory mechanics between adults and children and the risk of SIDS in young infants, a specific review of positioning for infants and young children with acute respiratory distress is warranted. OBJECTIVES: To compare the effects of different body positions in hospitalised infants and children with acute respiratory distress. SEARCH STRATEGY: We searched the Cochrane Central Register of Controlled Trials (CENTRAL) (The Cochrane Library Issue 3, 2004); MEDLINE (January 1966 to October Week 3, 2004); EMBASE (1980 to week 24, 2004); and CINAHL (1982 to October Week 3, 2004). SELECTION CRITERIA: All randomised or systematically-allocated controlled clinical trials comparing two or more positions in the management of infants and children hospitalised with acute respiratory distress. DATA COLLECTION AND ANALYSIS: Data were extracted from each study independently by two authors. Differences were resolved by consensus or referral to a third author. Continuous outcomes were analysed using a weighted mean difference and 95% confidence interval. No bivariate outcomes were available. All but one included study reported crossover data therefore this data was used for meta-analysis. Fixed-effect models were used unless heterogeneity was significant (p value equal to or less than 0.1), in which case a random-effects model was used. MAIN RESULTS: Forty-nine papers were selected for this review of which 21 studies (22 publications) were included. These studies compared prone, supine, lateral, elevated, and flat positions. Prone positioning was significantly more beneficial than supine positioning in terms of oxygen saturation, partial pressure of arterial oxygen, oxygenation index, thoraco-abdominal synchrony, and episodes of desaturation. There were no statistically significant differences between any other positions. AUTHORS' CONCLUSIONS: The prone position was significantly superior to the supine position in terms of oxygenation. However, as most patients included in the meta-analysis were ventilated, preterm infants the benefits of prone positioning may be most relevant to these infants. In addition, although placing infants and children in the prone position may improve respiratory function, the association of sudden infant death with prone positioning means that infants should only be placed in this position if continuous cardiorespiratory monitoring is used.