Patient-reported outcome measures and value-based medicine in paediatrics: a timely review.

Healthcare delivery is moving towards a more personalised and patient-centric approach. There is now an appropriate emphasis on providing value in our healthcare system. Patient-reported outcome measures (PROMs) assess our patients' perceptions of the status of their health and quality of life, measured over a period of time. PROM is an integral component of a value-driven and value-based healthcare system and is key if we want to practise value-based medicine. In paediatrics and child health, PROMs, if implemented well with appropriate measurement tools that are regularly updated and validated in a self-learning healthcare ecosystem, will help to enhance personalised healthcare delivery and collectively improve the health of the community at large. This review covers the role of PROMs in paediatrics, as well as their role in value-based medicine.

Trimethoprim-sulfamethoxazole-induced lung injury: a case report.

Background: Trimethoprim-sulfamethoxazole (TMP-SMX) is a commonly used antibiotic. While cutaneous adverse drug reactions associated with TMP-SMX are commonly recognized, lung toxicity induced by TMP-SMX is an unusual condition, with scattered reports of hypersensitivity pneumonitis, acute fibrinous organizing pneumonia, interstitial lung disease and acute respiratory distress syndrome. Reports of TMP-SMX-associated drug-induced lung injury (DLI) are rare in the pediatric population and its pathogenesis is not well understood. Diagnosis of DLI remains a challenge, given the wide range of clinical presentations that overlap with other conditions and the lack of diagnostic tests. In this report, we describe a case of TMP-SMX-induced lung injury in an eight-year-old child. Case Description: An eight-year-old girl presented in respiratory failure with acute symptoms of shortness of breath, fever, maculopapular rash and vomiting. This was associated with pneumonitis, pneumothorax, pneumomediastinum and subcutaneous emphysema on imaging. She had been on 25 days of TMP-SMX for treatment of Group D Salmonella bacteremia and osteomyelitis that was diagnosed prior to this current presentation. TMP-SMX was discontinued on admission due to concerns of possible drug reaction. Extensive infective, autoimmune and immunologic workup did not reveal the cause of the respiratory failure. Considering the absence of an alternative explanation for her clinical presentation and similarities in clinical courses to other reported cases, she was eventually diagnosed with TMP-SMX-associated DLI. She received a course of corticosteroids with subsequent clinical improvement and was weaned off home oxygen therapy a few months after her discharge from the hospital. Conclusions: Diagnosis of DLI can be challenging. The early identification of DLI and discontinuation of culprit drug is essential in its management. Further understanding of the underlying pathophysiology and risk factors for TMP-SMX-associated DLI is required.

Epidemiology and risk factors for sleep disturbances in children and youth with cerebral palsy: An ICF-based approach.

OBJECTIVES AND BACKGROUND: Children with cerebral palsy are at risk for sleep disorders, and there is a complex relationship between sleep and physical, environmental and functional factors in such children. The WHO International Classification of Functioning, Disability and Health model serves as a universal framework for describing and organizing functioning and disability. This study aimed to describe sleep disturbances in Singaporean children and youth with cerebral palsy, and develop a holistic framework for evaluating risk factors and potential management strategies for poor sleep. METHODS: A cross-sectional analysis was conducted on 151 children and youth in a nationwide registry for cerebral palsy. The WHO International Classification of Functioning, Disability and Health for Cerebral Palsy Questionnaire was used to identify sleep disturbances. Risk factors analyzed were age, gender, ethnic background, financial assistance, the dominant motor feature of cerebral palsy, functional status, and comorbidities such as active epilepsy, hearing and visual impairments, generalized pain, muscle tone and involuntary contractions. RESULTS: 46% had difficulty with sleep, with similar proportions having difficulty with amount, onset, maintenance and quality of sleep. On multivariate regression analysis, higher functional gross motor impairment as indicated by a GMFCS level of V (adjusted OR 4.24; 95% CI 1.09-19.0) and difficulty with involuntary contractions (aOR 2.80; 1.20-6.71) were significant factors for sleep difficulties. CONCLUSION: An ICF-based framework was useful in identifying possible contributory factors and strategies for managing poor sleep. Further studies with objective sleep measures would allow for better characterization of sleep disturbances in children and youth with cerebral palsy, and guide management.

Keeping Pace with Adolescent Asthma: A Practical Approach to Optimizing Care.

Known for their pre-occupation with body image, self-identity creation, peer acceptance, and risk-taking behaviors, adolescents with asthma face unique challenges. Asthma is a heterogeneous disease and accurate diagnosis requires assessment through detailed clinical history, examination, and objective tests. Diagnostic challenges exist as many adolescents can present with asthma-like symptoms but do not respond to asthma treatment and risk being mis-diagnosed. Under-recognition of asthma symptoms and denial of disease severity must also be addressed. The over-reliance on short-acting beta-agonists in the absence of anti-inflammatory therapy for asthma is now deemed unsafe. Adolescents with mild asthma benefit from symptom-driven treatment with combination inhaled corticosteroids (ICS) and long-acting beta-agonist (LABA) on an as-required basis. For those with moderate-to-persistent asthma requiring daily controller therapy, maintenance and reliever therapy using the same ICS-LABA controller simplifies treatment regimes, while serving to reduce exacerbation risk. A developmentally staged approach based on factors affecting asthma control in early, middle, and late adolescence enables better understanding of the individual's therapeutic needs. Biological, psychological, and social factors help formulate a risk assessment profile in adolescents with difficult-to-treat and severe asthma. Smoking increases risks of developing asthma symptoms, lung function deterioration, and asthma exacerbations. Morbidity associated with e-cigarettes or vaping calls for robust efforts towards smoking and vaping cessation and abstinence. As adolescents progress from child-centered to adult-oriented care, coordination and planning are required to improve their self-efficacy to ready them for transition. Frequent flare-ups of asthma can delay academic attainment and adversely affect social and physical development. In tandem with healthcare providers, community and schools can link up to help shoulder this burden, optimizing care for adolescents with asthma.

Assessing the accuracy of artificial intelligence enabled acoustic analytic technology on breath sounds in children.

Interpretation of breath sounds by auscultation has high inter-observer variability, even when performed by trained healthcare professionals. This can be mitigated by using Artificial Intelligence (AI) acoustic analysis. We aimed to develop and validate a novel breath sounds analysis system using AI-enabled algorithms to accurately interpret breath sounds in children. Subjects from the respiratory clinics and wards were auscultated by two independent respiratory paediatricians blinded to their clinical diagnosis. A novel device consisting of a stethoscope head connected to a smart phone recorded the breath sounds. The audio files were categorised into single label (normal, wheeze and crackles) or multi-label sounds. Together with commercially available breath sounds, an AI classifier was trained using machine learning. Unique features were identified to distinguish the breath sounds. Single label breath sound samples were used to validate the finalised Support Vector Machine classifier. Breath sound samples (73 single label, 20 multi-label) were collected from 93 children (mean age [SD] = 5.40 [4.07] years). Inter-rater concordance was observed in 81 (87.1%) samples. Performance of the classifier on the 73 single label breath sounds demonstrated 91% sensitivity and 95% specificity. The AI classifier developed could identify normal breath sounds, crackles and wheeze in children with high accuracy.

HIDEA syndrome: A rare cause of congenital hypoventilation in a premature infant.

BACKGROUND: HIDEA (hypotonia, hypoventilation, intellectual disability, dysautonomia, epilepsy and eye abnormalities) syndrome is a rare and novel disease. We describe a premature patient who required extensive work up for his hypoventilation with a diagnosis of HIDEA syndrome. CASE DESCRIPTION: The patient was born to a pair of consanguineous parents at 32-week gestation. His intermittent bradypnoea requiring significant respiratory support during his postnatal clinical course was atypical for bronchopulmonary dysplasia and this required further extensive work up to look for a cause for his hypoventilation. A trio whole exon sequencing was done which identified homozygous variants in P4HTM, in keeping with the diagnosis of autosomal recessive HIDEA syndrome. He is currently doing well on BiPAP 18 cm H2O / 8 cm H2O, Rate 30 breaths per minute in room air and full nasogastric feeding. He also has cortical blindess and severe global developmental delay. CONCLUSION: Early diagnosis is crucial to optimise adequate ventilatory management including early tracheostomy as many require lifelong continuous or intermittent ventilation. This minimises the complications of chronic hypoxia and reduces mortality risk.

Necrotizing pneumonia requiring prolonged extracorporeal membrane oxygenation: Pushing the boundaries in pediatric ECMO.

Extracorporeal membrane oxygenation (ECMO) is a life-saving rescue therapy used in acute respiratory failure refractory to invasive mechanical ventilation. Recent studies on positive outcomes of extended ECMO therapy are promising. We describe a case of a 2-year 8-month-old female child with necrotizing pneumonia secondary to Streptococcus pneumoniae, Influenza A, and Mycoplasma pneumoniae, who survived with intact neurological function and no long-term adverse outcomes after a prolonged ECMO run of 86 days. To the best of our knowledge, this is one of the longer durations of ECMO with transplant-free survival in a pediatric patient requiring respiratory support with good recovery and a good functional outcome. Allowing time for native lung recovery is pivotal for optimal recovery, despite significant lung injury due to the underlying disease process. With evolving ECMO experience, clinicians may need to re-consider the conventional maximum duration of ECMO in children with severe respiratory failure on a case-by-case basis.

Approach to the snoring child.

Sleep is an important component in a child's growth and development. Snoring is common in children and often perceived as benign, but habitual snoring may be an indication of obstructive sleep apnoea (OSA). OSA can have health, developmental and cognitive consequences. The three common risk factors for paediatric OSA are tonsillar and/or adenoidal hypertrophy, obesity and allergic rhinitis. Primary care providers are well-placed to identify children at risk by screening for habitual snoring and associated OSA risk factors during routine consultations. Physician awareness of OSA symptoms/signs facilitates diagnosis, management and referral decisions. A trial of medical treatment may be considered for habitual snoring with mild symptoms/signs before referral. Overnight polysomnography is the gold standard investigation utilised by paediatric sleep specialists to diagnose OSA. Adenotonsillectomy is the first-line management for OSA with adenotonsillar hypertrophy, but residual/recurrent OSA may occur, so follow-up by primary care providers is important after surgery.

Predictive equation for optimal continuous positive airway pressure in children with obstructive sleep apnoea.

AIM: A subgroup of children with obstructive sleep apnoea (OSA) requires treatment with continuous positive airway pressure (CPAP). This study's aims were: 1) to determine if the optimal CPAP for the treatment of OSA in children correlates with body mass index (BMI); 2) to determine the correlation between polysomnographic variables and optimal CPAP in children with OSA; and 3) to develop a CPAP predictive equation for children with OSA. METHODS: This was a retrospective study of children with OSA who underwent CPAP titration studies. Patients with craniofacial abnormalities (except Down syndrome) and neuromuscular diseases were excluded. Polysomnograms were done using Sandman Elite. Correlations between optimal CPAP, clinical and polysomnographic variables were analysed. A multivariable linear regression model for optimal CPAP was developed. RESULTS: 198 children (mean±sd age 13.1±3.6 years) were studied. Optimal CPAP had a significant positive correlation with age (rho=0.216, p=0.002), obstructive apnoea-hypopnoea index (rho=0.421, p<0.001), 3% oxygen desaturation index (rho=0.417, p<0.001), rapid eye movement respiratory disturbance index (rho=0.378, p<0.001) and BMI z-score (rho=0.160, p=0.024); and a significant negative correlation with arterial oxygen saturation measured by pulse oximetry nadir (rho= -0.333, p<0.001). The predictive equation derived was:Optimal CPAP (cmH2O)=6.486+0.273·age (years)-0.664·adenotonsillectomy(no=1, yes=0)+2.120·Down syndrome (yes=1, no=0)+0.280·BMI z-score. CONCLUSION: The equation developed may help to predict optimal CPAP in children with OSA. Further studies are required to validate this equation and to determine its applicability in different populations.