Pediatric Pulmonology最新文献

Background: People with cystic fibrosis (pwCF) and primary ciliary dyskinesia (pwPCD) are particularly vulnerable to the harmful effects of air pollution due to the impairment of mucociliary clearance (MCC). Despite growing evidence supporting the use of indoor air purification in common lung conditions, its role in CF and PCD remains unexplored.

Methods: The Clean Air for Rare MCC dIsorders (CARMUCI) study is a randomized, double-blind, cross-over, sham-controlled trial aiming to evaluate the impact of indoor air purification on the respiratory health of pwCF and pwPCD (children and adults) in Cyprus. Sixty-two participants will receive two 3-month intervention periods using identical-looking air purifiers: one 3-month period with active filtration via high-efficiency filters and one 3-month period with a sham unit. Primary outcome will be a mean difference of at least 10% change in lung clearance index, and secondary outcomes will include spirometry, pulmonary exacerbations, inflammatory biomarkers, and health-related quality of life. Exposure to indoor particulate matter (PM) will be continuously monitored using device operation logs and wearable activity trackers.

Results: By August 2025, 24 pwCF and 26 pwPCD (20 females, age: 31 [16.1] years) have been enrolled. CARMUCI continues enrollment and will start data collection in September 2025. Results are expected to provide novel evidence on the efficacy of air purification in reducing indoor PM exposure and improving respiratory outcomes in people with chronic MCC disorders.

Conclusion: CARMUCI trial first results are expected in late-2027 and may inform future clinical guidelines and environmental health strategies for managing rare MCC disorders.

Background: Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators revolutionized the care of people with Cystic fibrosis (pwCF) by addressing the underlying genetic defect rather than merely managing symptoms. Elexacaftor/Tezacaftor/Ivacaftor (ETI) has shown significant clinical benefits in pwCF carrying specific CFTR mutations; however, data on its safety and efficacy in young children with CF (ycwCF), aged 2-6 years, remain sparse.

Methods: We conducted a real-world, retrospective study that included all Israeli ycwCF who were genetically eligible for and treated with ETI. Body mass index (BMI) z-score, forced expiratory volume in one second (FEV₁) percent predicted, rate of pulmonary exacerbations (PEx), sweat conductivity levels, bacterial sputum isolates, clinic visits frequency, and sputum samples collected before and after ETI initiation were evaluated. Additionally, adverse effects were assessed.

Results: Sixteen ycwCF, aged 2-6 years, received ETI therapy for a mean duration of 13 months (range 4-25 months), including three pancreatic-sufficient patients. Following ETI initiation, significant improvements were observed. BMI z-scores increased from -0.37 ± 1.32 to -0.01 ± 1.14 (p = 0.005), and in the subset of patients tested (7/16), FEV₁ improved from 91 ± 12 to 113 ± 18 percent predicted (p = 0.018). The rate of PEx decreased from 0.7  ±  1.1 to 0.2  ±  0.4 (p = 0.058) and sweat conductivity levels dropped significantly from 112  ±  38 to 48  ±  30 mmol/L (p = 0.002). There was a marked reduction in bacterial colonization in sputum: Pseudomonas aeruginosa declined from 9/16 to 2/14 (p = 0.04), and Staphylococcus aureus from 12/16 to 2/14 (p = 0.01). Notably, the total number of sputum cultures sent also decreased by 35%, from 116 before ETI to 75 after treatment initiation. This reduction paralleled decreased CF clinic visits, from 7.3  ±  3 per year to 4.7  ±  3 (p = 0.001). Reported adverse effects were minimal, and no treatment discontinuations were required.

Conclusions: These real-world findings support the safety and effectiveness of ETI in ycwCF. Robust, long-term studies involving larger populations are essential to confirm these results.

Background: Poor indoor air quality (IAQ) contributes to asthma morbidity, particularly affecting children and young people (CYP) from socioeconomically deprived backgrounds. Air purification devices may improve IAQ and asthma symptoms, but real-world evidence remains limited.

Aim: To assess the feasibility, acceptability, and potential clinical impact of installing an air purifier in the homes of CYP with severe asthma.

Methods: An unblinded feasibility study recruiting children ≤ 15 years with a confirmed diagnosis of asthma attending a regional severe asthma service. Participants selected by computer-generated random allocation. An air purifier (manufactured by Rensair Ltd.) was installed in each home, with instructions to operate continuously on "quiet mode" for 1-year, and operating costs covered. Data on asthma management and symptoms, quality of life, and qualitative feedback were collected and analysed using thematic analysis.

Results: Twenty CYP were recruited (mean age: 11.2 (SD: 2.68), 60% boys). Thematic analysis of feedback from CYP and families indicated high acceptability and perceived improvements in night-time symptoms and IAQ. Over this 12-month study period, the mean annual number of systemic corticosteroid courses decreased from 4.2 to 2.15 (p = 0.006) from the previous year, and unscheduled primary care visits fell from 4.0 to 1.8 (p = 0.02). Trends towards marginal improvements in asthma control test mean scores (13.3 to.14.2 for < 12 years (p = 0.2), 15.5 to 16.8 for ≥12 years (p = 0.6) and quality of life scores were observed (54.6 to 62.4 for total score (p = 0.08), 17.2 to 19.8 for emotional domain (p = 0.07) and 13.6 to 13.9 for activity domain (p = 0.7)), with a statistically significant increase in the symptom domain of the Mini-PAQLQ (23.8 to 28.8 p = 0.04).

Conclusions: Air purifiers may offer a feasible, acceptable, and potentially beneficial intervention for children with severe asthma, particularly in settings with poor IAQ. Larger controlled trials are warranted to confirm these findings and inform implementation.

Trial registration: NCT05817357.

Quality of life (QOL) measures are understudied outcomes in the pediatric transplant population, specifically in pediatric lung transplant recipients. Dr. Martin and colleagues from the Medical University of Vienna have recently published on post-transplant outcomes in children who have undergone lung transplant using the EuroQol questionnaire, a unique study in this population. While lung transplantation in children has been shown to improve functional status based on international data and other small volume QOL studies, pediatric data remains sparse and often relies on extrapolation from adult measures that have not been validated in children. In this editorial, we are pleased to highlight Dr. Martin et al.'s study, present a broader view of QOL measures in pediatric lung transplant, and compare and contrast various QOL outcomes that have been proposed for use in this population. This includes a review of the PedsQL Transplant Module, KIDSCREEN-52, CHIP, KINDL, PROMIS/PSC, and others that use pediatric-specific, transplant-focused tools. There is a need for multicenter collaboration in this population, and we encourage the international community to view this as a global initiative moving forward.

Aim: Preeclampsia (PE) is characterized by antenatal hypoxia and inflammation, which may impair neonatal respiratory control. However, its association with apnea of prematurity (AOP) in late preterm infants remains unclear. This study aimed to test the hypothesis that maternal PE is an independent risk factor for AOP in late preterm infants.

Methods: This retrospective cohort study was conducted at Yamanashi Central Hospital between January 2017 and June 2025. Singleton pregnancies delivered between 34^ + 0 and 36^ + 6 weeks of gestation were included. Early-onset PE was defined as diagnosis before 34 weeks of gestation, and late-onset PE as diagnosis at or after 34 weeks. Multivariable logistic regression analyses were performed to evaluate the association between PE and AOP, adjusting for potential confounders. In Model 1, PE was analyzed as a binary variable. In Model 2, subjects were categorized into three groups: early-onset, late-onset, and no PE.

Results: A total of 470 mother-infant pairs were analyzed. Among them, 23 women experienced early-onset PE, and 34 experienced late-onset PE. The overall incidence of AOP was 27.9% (131/470). In Model 1, PE was associated with a higher AOP risk (adjusted odds ratio [aOR], 2.44; 95% confidence interval [CI]: 1.21-4.90). In Model 2, early-onset PE was significantly associated with AOP (aOR: 3.42; 95% CI: 1.20-9.72), whereas late-onset PE was not (aOR: 1.88; 95% CI: 0.75-4.73).

Conclusion: PE, particularly early-onset, was significantly associated with AOP. Incorporating maternal PE status into perinatal risk stratification may help identify late preterm infants at elevated AOP risk and guide timely intervention.

Background: Acute bronchiolitis is a frequent cause of hospitalization in early childhood and has been associated with subsequent respiratory morbidity. However, its long-term impact on mental health has not been extensively studied.

Methods: This population-based cohort study used data from the National Health Insurance Service (NHIS) in South Korea, including 985,957 children born between 2002 and 2003, followed until December 2021. Children hospitalized for acute bronchiolitis (n = 25,550) were compared to a matched unexposed cohort (n = 102,220). The primary outcomes were 24 predefined mental health disorders, categorized into early-onset (before 10 years) and late-onset (after 10 years) conditions. Hazard ratios (HRs) were estimated using proportional hazards models, adjusting for potential confounders. The modifying effects of asthma and atopic dermatitis were also evaluated.

Results: The median age at hospitalization for bronchiolitis was 8 months (IQR, 3-20 months). Over a mean follow-up of 15 years, the incidence rate of any mental health disorder was 187.9 and 153.9 per 10,000 person-years in the bronchiolitis and unexposed cohorts, respectively. The adjusted hazard ratio (aHR) for any mental health disorder in the bronchiolitis cohort was 1.20 (95% CI, 1.16-1.23). Early-onset developmental disorders showed aHR of 1.34 (95% CI, 1.26-1.43), and late-onset psychiatric disorders had aHR of 1.18 (95% CI, 1.15-1.22). There was a progressive increase in risk for multiple mental health disorders (p < 0.01). Asthma (p = 0.77) and atopic dermatitis (p = 0.48) did not significantly modify this risk.

Conclusions: Children hospitalized for acute bronchiolitis showed an increased risk of developing mental health disorders. These findings provide epidemiologic evidence supporting the need for long-term mental health surveillance and supportive care in this population.

Background: In managing certain clinical chronic diseases, the sense of coherence (SOC) in salutogenic theory is continuously assessed. However, no studies have yet focused on childhood asthma, a clinical condition closely associated with effective caregiver involvement. Therefore, we aimed to assess the role of maternal SOC in children's psychological stress and other modifiable factors affecting asthma control.

Methods: This observational, case-control study included 193 children with asthma (6-10 years) and their parents. The individuals were grouped into controlled (n = 97) and uncontrolled (n = 96) asthma groups using the Childhood Asthma Control Test. Additionally, validated questionnaires were administered to the mother-child dyad. We assessed maternal SOC as a moderator of the child's psychological stress, treatment adhesion, inhalation technique, and environmental control. We used multivariate logistic regression analysis to confirm the association of these variables with asthma control, and the Breslow-Day test assessed the effect of maternal SOC.

Results: High maternal SOC was observed in 81/97 (83.5%) and 8/96 (8.3%) patients in the controlled and uncontrolled asthma groups, respectively. Multivariate analysis indicated that high maternal SOC, early phases of psychological stress, and low exposure to aeroallergens were associated with clinical asthma control. The Breslow-Day test identified a significant interaction between high maternal SOC and children's psychological stress tolerance, treatment adhesion, and ideal inhalational technique.

Conclusion: High maternal SOC affected psychological stress in children and primary modifiable factors, such as treatment adhesion and inhalational technique, for managing asthma in infancy.