Paediatric Pulmonology

BACKGROUND Rituximab (RTX) is a chimeric IgG monoclonal antibody directed against the CD20 antigen expressed on normal and malignant B cells. It is used for treating a variety of neoplastic, autoinflammatory, and autoimmune pathologies, including disorders occurring in the pediatric population. It has been proven that rituximab can induce pulmonary complications including interstitial lung disease, bronchiectasis and pulmonary toxicities, predominantly in adults. By describing this case, we aim...

CONCLUSION: This study has demonstrated a predominance of low anthropometric measurements and restrictive abnormality on spirometry in children with SCD in Ghana. Females and those with higher BMI z-scores had significantly lower odds of having a restrictive lung function pattern compared to normal lung function.

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Most children and adolescents recover rapidly from SARS-CoV-2 infection, yet a subset develops paediatric long COVID (LC). How immune ontogeny shapes LC biology and heterogeneity remains unclear. We deeply phenotype a two-visit cohort with severe LC (n = 74) and controls (n = 27) spanning up to 3.2 years post index infection. Symptom burden remains high and neurofilament light chain (NfL) percentiles inversely associate with functional status (Bell score; r = -0.3536, P = 0.0060)....

CONCLUSIONS: NPIs were associated with significant changes in the incidence of pediatric SAEs. This unexpected benefit suggests that public health interventions targeting respiratory viruses may reduce the burden of SAEs in children. The mid- and long-term impact of lifting NPIs on SAEs and on the use of healthcare resources should be carefully monitored.

CONCLUSIONS: In this exploratory study of young, modulator-naïve children with CF, ETI initiation was associated with changes in the lower airway microbiology composition assessed by IS. These findings highlight potential shifts in lower airway microbiology and the importance of age-appropriate lower airway sampling in future paediatric studies.

CONCLUSIONS: Pulmonary function deficit was frequent in childhood ALL survivors, especially after stem cell transplantation or pulmonary disease. Tailored long-term pulmonary monitoring, including small airway function and diffusing capacity, may aid in timely detection and intervention.

The number of paediatric patients evaluated for long-term home respiratory support has risen sharply in recent years, reflecting both improved survival of children with complex chronic conditions and expanded therapeutic indications. This population is characterized by substantial clinical heterogeneity, encompassing obstructive, restrictive, or central ventilatory disorders. Contributing comorbidities - such as severe obesity, chronic aspiration with secondary lung disease, neuromuscular...

Cystic fibrosis (CF) is one of the most common rare, genetic diseases. Newly available, highly effective modulator therapy (HEMT) improves many domains of CF pathophysiology. Thereby, HEMT challenges current standards of care and prediction models of disease outcomes.We established a public patient involvement (PPI) group including experts and caregivers of children with CF and collated a narrative review on CF management. This review is based on the views of healthcare professionals and...

Cystic fibrosis (CF) care has advanced rapidly, yet diagnosis and inclusion in patient registries remain severely limited in low- and middle-income countries (LMICs). Barriers include restricted newborn screening, limited availability of sweat chloride testing, and underrepresentation of non-European CFTR variants in standard panels. Sparse registries further impede epidemiological insight and resource planning. Also, access to comprehensive CF care remains a major challenge in LMICs, where...

CONCLUSION: Short and long-term CF-specific survivorship care plans should be developed based on both disease entities in anticipation of compounded risks for early cardio-respiratory disease and an increased awareness of secondary cancer screening. Research into the copresentation of these diseases should be promoted.

INTRODUCTION: Lung-function outcomes among preterm-born children referred for pulmonology care are highly heterogeneous, and early determinants remain incompletely defined. We aimed to identify early-life factors associated with school-age lung function after preterm birth and to examine whether school-age spirometry patterns relate to subsequent lung-function trajectories.

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Sustainable development of neonatal and paediatric lung organoid research requires effective regulatory and collaborative frameworks. Ethically compliant tissue sources should be prioritised; robust ethical governance on sensitive sources is essential. https://bit.ly/4b9qeLf

Submerged cultures of undifferentiated or transformed epithelial cells are widely used in respiratory research due to their ease of use and scalability. However, these systems fail to capture the cellular diversity of the human airway epithelium. Here, we describe a submerged differentiation model using cryopreserved human nasal epithelial cells obtained via minimally invasive brushings. By targeting Notch and BMP signaling with small molecule inhibitors, we differentiate these cells into...

TREC-NBS identifies patients with inborn errors of immunity (IEI) and syndromic features, but uncertainty remains regarding their immunological management. To address this, syndromic patients detected by TREC-NBS in Germany between August 2019 and April 2024 were systematically analyzed, including phenotype, treatment, and outcomes. National registries were screened, and data were completed by the treating centres. A total of 77 syndromic patients were identified, with 22 different gene defects...

CONCLUSIONS: In a diverse global post-transplant CF population, the timing of transplantation was not significantly associated with severe outcomes following SARS-CoV-2 infection. Those with more severe lung disease were at increased risk for worse outcomes and should be monitored closely.

CONCLUSIONS: We identified several socioeconomic disparities that were increased in children with severe asthma and severe acute asthma at the PICU in the Netherlands. Comprehensive assessment and mitigation of these determinants may improve health equity in paediatric severe asthma and enhancement of asthma care.

CONCLUSIONS: Three-year adherence to ETI and IVA based on pharmacy refill data was high, yet declined over time.

COPA syndrome is a rare monogenic autoinflammatory disease due to heterozygous mutations in COPA, encoding the coatomer subunit α. COPA syndrome demonstrates phenotypic overlap with STING-associated vasculopathy with onset in infancy (SAVI), the latter due to gain-of-function mutations in STING1. Indeed, STING activation is a key driver of the pathogenesis of COPA syndrome, and a recent report suggested that the presence of the common HAQ STING allele confers complete protection against the...

In this article the curmudgeonly authors take a broad strokes reflection upon the misuse of language in the medical literature through the lens of paediatric pulmonology. The paper addresses the cult of conciseness as reflected in the explosion of ambiguous acronyms, the dismantling of the Latin and Greek origins of medical terminology, and the insatiable desire to change taxonomy seemingly for change sake. The paper serves as a call to arms for those seasoned clinicians of a certain age to push...

CONCLUSION: Current evidence supports a model in which neutrophilic inflammation, driven by dysregulated cytokine responses and potentially sustained by microbial dysbiosis, is central to the pathophysiology of paediatric NCFB. Key gaps remain regarding dysbiosis, adaptive immunity and the longitudinal trajectories of immune mediators. Addressing these may support biomarker development and targeted therapies.

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Bronchiectasis in children is a heterogeneous, chronic respiratory condition for which standardised, evidence-based management is essential. While international guidelines have improved diagnostic and therapeutic consistency, the treatable traits (TT) approach offers a complementary precision-medicine framework aimed at identifying all clinically relevant, measurable, and modifiable factors in individual patients. Through a comprehensive literature review, we identified 40 potential TT in...

Expiratory variability index is not a specific or fully validated gold standard for small airway disease. It works best as a screening or follow-up tool, but should be combined with other techniques such as oscillometry or MBW for confirmation. https://bit.ly/4n15use.

BACKGROUND: Persistent tachypnea of infancy (PTI), also known as neuroendocrine cell hyperplasia of infancy (NEHI), represents one of the most common childhood interstitial lung diseases. Despite its frequency, standardized management protocol is lacking, and long-term outcome data remain limited.

CONCLUSIONS: Evidence of small airway dysfunction was found in almost one-third of subjects who have been ventilated for life-threatening RSV disease, although not always accompanied by respiratory symptoms.

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Continuous glucose monitoring (CGM) is now central to diabetes management, yet variation in how respective medical products are evaluated limits meaningful comparison between CGM systems. Three barriers currently constrain reliable interpretation of glucose-derived measures. The first is limited transparency: in several regulatory settings, particularly those using Conformité Européenne marking, clinical-study reports, reference-method information and analytical documentation required for market...

CONCLUSION: Integrating real-time FEV(1) analysis into the Swedish CF registry provides clinicians and patients with a practical tool for continuous evaluation of short- and long-term lung function trends, facilitating the monitoring of disease progression and supporting clinical decision-making.

Cystic fibrosis (CF) care has been revolutionized by CFTR modulators, particularly the triple combination elexacaftor/tezacaftor/ivacaftor (ETI). However, a subset of people with CF (pwCF) carrying ETI-unresponsive variants still lack effective therapies. A nextgeneration modulator combination, vanzacaftor/tezacaftor/deutivacaftor (VTD), shows promise in addressing this gap. Phase 3 trials report superior efficacy of VTD in reducing sweat chloride and suggest potential benefit for 31 CFTR...

CONCLUSIONS: We report a large European cohort of patients with COPA syndrome. While confirming the core organ features (lung, joint, and kidney) of the disease, our data expand the phenotype to include cardiac, skin, and gastrointestinal features, further demonstrating the clinical overlap with SAVI and other type I interferonopathies.

CONCLUSION: In this national cohort of children with repaired PAH-CHD, mortality is significant. This novel, simple risk score has been developed and validated specifically for children with repaired PAH-CHD, useful at the time of post-operative assessment to predict outcome and direct management.

CONCLUSIONS: Participants with AD, especially moderate-to-severe disease, are more likely to experience (nonatopic) multimorbidity and showed unique patterns of nonatopic multimorbidity with regard to orofacial and cardiometabolic diseases. Our findings highlight the importance of promoting awareness for interdisciplinary approaches to managing patients with AD. An author video to accompany this article is available online.