Pediatrics:

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In a global landscape defined by polycrisis, children are being failed. To address this failure, we ask an ambitious yet fundamental question: how do we create child-inclusive societies where every child thrives and has the best start in life, where intergenerational disadvantage is redressed, and where child poverty is ended? Building on the power of the social determinants of health in advancing equity and human wellbeing, we argue that child inclusiveness requires three foundational actions linked to the political, commercial, and social determinants of health: (1) prioritising implementation of transformative collaboration between policy makers, public bodies, and communities to improve outcomes for children; (2) reclaiming the public good through child-centred regulatory frameworks that aim to deliver health care and improve wellbeing; and (3) valuing the time to care for children and to build meaningful and responsive relationships with them. With innovative thinking about our societies and their core values, we can design child-inclusive interventions and derive relevant metrics and indicators to track progress.

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The Australian Paediatric Surveillance Unit (APSU), established in 1993 to address the paucity of national data on rare childhood disorders, has become an invaluable research resource. It facilitates prospective, active surveillance for a variety of rare disorders, with monthly reporting by ~1500 paediatricians, who are invited to notify incident cases and provide demographic and clinical data. APSU is highly collaborative (used by >400 individuals/organisations), patient-informed and productive (>300 publications). In 30 years, 72 studies have been initiated on rare infections, and genetic, psychological and neurological disorders, and injuries. Return rates of monthly report cards were >90% for 30 years and paediatricians have provided data for >90% of notified cases. Although there are limitations, including case underascertainment in remote regions, APSU often provides the only available national data. APSU has assisted the government in reporting to the WHO, developing national strategies, informing inquiries and investigating disease outbreaks. APSU data have informed paediatrician education, practice, policy, and service development and delivery. APSU was integral in establishing the International Network of Paediatric Surveillance Units (INoPSU) and supporting development of other units. APSU's expanded remit includes one-off surveys, hospital audits, systematic reviews, studies on the impacts of rare disorders on families, surveillance evaluations, and joint studies with INoPSU members. Paediatricians value the APSU, reporting that APSU data inform their practice. They must be congratulated for an outstanding collective commitment to the APSU, in providing unique data that contribute to our understanding of rare disorders and support optimal, evidence-based care and improved child health outcomes.

CONCLUSIONS: Reductions in the use of medications for bronchiolitis achieved in 2019 through the implementation of our integrated clinical pathway have been sustained over the three subsequent waves.

The rapid and accurate diagnosis of rare diseases is paramount in directing clinical management. In recent years, the integration of multi-omics approaches has emerged as a potential strategy to overcome diagnostic hurdles. This review examines the application of multi-omics technologies, including genomics, epigenomics, transcriptomics, proteomics, and metabolomics, in relation to the diagnostic journey of rare diseases. We explore how these combined approaches enhance the detection of pathogenic genetic variants and decipher molecular mechanisms. This review highlights the groundbreaking potential of multi-omics in advancing the precision medicine paradigm for rare diseases, offering insights into future directions and clinical applications. IMPACT: This review discusses using current tests and emerging technologies to diagnose pediatric rare diseases. We describe the next steps after inconclusive molecular testing and a structure for using multi-omics in further investigations. The use of multi-omics is expanding, and it is essential to incorporate it into clinical practice to enhance individualized patient care.

Abstract: Apremilast (Otezla) is an inhibitor of phosphodiesterase-4 (PDE-4) being developed by Amgen. It is approved in multiple countries worldwide, including the USA and those of the EU, for the treatment of adults with psoriatic arthritis, plaque psoriasis, or oral ulcers associated with Behçet's disease. In April 2024, based on clinical data in patients aged 6-17 years, apremilast received its first pediatric approval in the USA for the treatment of pediatric patients aged ≥ 6 years and weighing ≥ 20 kg with moderate to severe plaque psoriasis who are candidates for phototherapy or systemic therapy. Apremilast was also approved in the EU in October 2024 for the treatment of moderate to severe plaque psoriasis in children and adolescents from the age of 6 years and weighing ≥ 20 kg who are candidates for systemic therapy. This article summarizes the milestones in the development of apremilast leading to the first pediatric approval for plaque psoriasis.

Respiratory monitoring is an essential part of routine clinical care of the newborn. Recent technological developments have improved respiratory monitoring and allowed for a two-way interaction between the monitored parameter and the level of the provided respiratory support. We herein discuss applications of monitoring by neurally adjusted ventilatory assist, closed-loop oxygen control, tidal capnography, lung ultrasound, diaphragmatic electromyography and lung magnetic resonance imaging.

Cerebral/cortical visual impairment (CVI) is a leading cause of pediatric visual impairment in nations with developed economies and is increasing in those with developing economies. Because vision is the predominant sense used for learning, delay in diagnosis of CVI can negatively affect education, making early detection and management important. The American Academy of Pediatrics has published the policy statement "Visual System Assessment in Infants, Children, and Young Adults by Pediatricians" and an accompanying clinical report that are based on identifying potential causes of ocular visual impairment in children. Yet, routine vision screening may not accurately identify the brain-based visual impairment in children with CVI. Moreover, children with CVI often have medical complexity with other neurocognitive impairments and serious medical conditions that can make the diagnosis of CVI more difficult. Strategies are necessary for early identification of CVI to promote early diagnosis and referral for vision services that may allow a child with CVI to engage more fully in school, activities of daily living, vocational pursuits, and recreational activities. Knowledge of the characteristics of CVI as well as risk factors for CVI will assist the pediatrician in identifying children with CVI. This clinical report is complementary to previous vision screening policies, allowing both ocular and brain-based visual impairments in children to be identified and addressed. Pediatricians, other primary care physicians, pediatric ophthalmologists, neurologists, and other specialized pediatric eye care clinicians can identify children with CVI and coordinate effective evaluation, diagnosis, and referrals for vision services for these children.

CONCLUSIONS: The detection rate of depressive symptoms in this study aligns closely with global rates for children and adolescents. High school students exhibit a higher prevalence of depressive symptoms compared to other age groups, highlighting the need for effective interventions targeted at this population. There was no clear temporal trend in the prevalence of depressive symptoms. Additionally, the choice of measurement tool is a critical aspect of epidemiological research; standardizing these measurements is essential for enhancing data comparability across studies. Trial Registration International Prospective Register of Systematic Reviews: No. CRD42023455604.

BACKGROUND: Diagnosing anaphylaxis in children within the first 2 years of life can be difficult due to the often confusing and nonspecific signs and symptoms.

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CONCLUSIONS: Our findings suggest that for febrile children <36 months of age undergoing bladder catheterization for suspected UTI, pyuria will be absent in ∼20% of children who are eventually shown to have pure growth of a pathogen on a culture. This raises questions about the appropriateness of requiring pyuria for the diagnosis of UTIs.

OBJECTIVE: The relationship between low income and adverse perinatal outcomes, such as low birth weight and developmental delays, is well established making the search for protective factors important. One such factor may be neighbourhood greenspace. This study elucidates the role of urban neighbourhood greenspace in the relationship between income and perinatal outcomes in a nationally representative birth cohort from the UK.

CONCLUSIONS: Language ability at arrival and immigration category are associated with virtual mental healthcare utilisation. Whether findings reflect user preference or inequities in accessibility, particularly for refugees and those without CLA at arrival, warrants further study.

CONCLUSIONS: Our findings emphasise the importance and urgency of long-term control of air pollution and pollution-related diseases, especially among children and adolescents. There is a need for further research employing more homogeneous methodologies for assessing exposure and outcome measurements, in order to enable systematic reviews with meta-analysis.

CONCLUSIONS: Approximately 1% of patients with non-refractory KD may develop CA sequelae. Our findings highlight the importance of initial echocardiographic assessment and early initiation of IVIG treatments for patients with KD.

CONCLUSIONS: This study and its results are aligned with, and supports, the needs and preferences of caregivers of children with neurological conditions, promoting information provision, healthcare engagement and clinical decision-making. These resources will form a foundation for accurate and contemporary scientific knowledge that is distilled and available to a wide range of stakeholders.