Pediatric Pulmonology最新文献

Background: Severe asthma (SA) in children is a complex condition with high morbidity and healthcare costs. Mepolizumab, an anti-interleukin-5 biologic, is approved for severe eosinophilic asthma (SEA) in patients ≥ 6 years, yet long-term real-world pediatric data remain limited.

Aim: To assess the long-term safety and effectiveness of mepolizumab in pediatric SEA, focusing on lung function, oral corticosteroid (OCS) use, and exacerbation rates.

Methods: This retrospective, multicenter study examined the medical records of 33 patients with SEA (aged 6-17 years) who received mepolizumab treatment at three tertiary centers in Turkiye. Inclusion criteria included GINA-defined SA, ≥ 2 severe exacerbations in the previous year requiring OCS, high-dose ICS plus a second controller, and elevated eosinophil counts. Data on exacerbations, pulmonary function tests (PFTs), OCS use, ACT scores, eosinophils, and adverse events were collected over 24 months.

Results: At baseline, patients showed poor asthma control (median ACT: 13), impaired lung function (FEV1%: 62%), and frequent exacerbations (median: 7/year). Mepolizumab significantly reduced exacerbation rates (median: 7 to 0-0.05 at 12/24 months, p = 0.005) and OCS use (87.9% OCS-free by 3 months). ACT scores improved (median: 13-25, p < 0.001), as did FEV1% (62% to 89%, p < 0.001) at 24 months. Eosinophil counts decreased markedly (460 to 50 cells/µL, p < 0.001). The treatment was well-tolerated; one patient discontinued due to anaphylaxis and four due to lack of efficacy.

Conclusion: Mepolizumab showed sustained effectiveness and good tolerability in pediatric SEA, significantly reducing exacerbations and OCS use while improving asthma control and lung function. These findings support its real-world utility and underscore the need for careful patient selection and monitoring.

Background: The primary risk factor determining the progression of tuberculosis (TB) infection is the host's immune status. However, reports of TB cases in children diagnosed with primary immunodeficiency (PID), also referred to as inborn errors of immunity (IEI), remain scarce. In this study, we describe the impact of PID/IEI on childhood TB disease.

Methods: In this retrospective cohort study, data of patients aged 1 month to 18 years who were diagnosed with TB between January 2012 and January 2025 were collected. TB patients were compared according to PID status. Additionally, radiological, histopathological, and microbiological diagnostic findings, as well as clinical features and treatments of TB patients with PID, were evaluated.

Results: A total of 217 TB patients were included, with a median age of 118 months (IQR: 42-169.5). PID was detected in 5.5% (n = 12) of the patients. In 6 (50%) of the PID patients, the immunodeficiency was not known before the TB diagnosis. The median age of patients with PID was 17 months (IQR: 10.3-58.5), which was significantly lower compared to other patients (p = 0.001). The diagnosis of extrapulmonary TB was significantly more common among PID patients (p = 0.049). Treatment durations in patients with PID ranged from 6 to 24 months, and no mortality was observed.

Conclusion: Investigating PID in children diagnosed with TB may be a critical step in enabling early diagnosis and treatment before the development of potentially fatal complications. We also believe that expanding immunological investigations will contribute to a better understanding of childhood TB pathogenesis.

Introduction: Respiratory syncytial virus (RSV) is a leading cause of lower respiratory tract infections (LRTIs) in young children. Additionally, RSV is associated with long-term respiratory morbidities. This study evaluates acute and long-term healthcare utilization (HCU) in infants hospitalized with RSV-associated LRTI (RSV-LRTI) compared to non-RSV LRTI (Non-RSV-LRTI) in a nationwide cohort.

Methods: A retrospective case-control study used data from Clalit Healthcare Services (CHS), Israel's largest healthcare provider. Infants born between 2015 and 2023, admitted before 12 months of age with LRTI during the RSV season, were included. PCR confirmed RSV-LRTI cases, while controls had negative RSV PCR and positive PCR for other respiratory viruses. Acute HCU was assessed within 30 days post-discharge. Long-term respiratory-related HCU was evaluated up to 6 years of age. Statistical analyses included Poisson regression, adjusting for demographic and clinical potential confounders.

Results: A total of 8626 infants were included, with 4,951 in the RSV-LRTI group and 3675 in the Non-RSV-LRTI group. The adjusted acute HCUs were higher in RSV-LRTI cases with increased systemic steroid use (IRR = 2.25, 95%CI: 1.94-2.62, p < 0.001) and short-acting beta-agonist use (IRR = 3.80, 95%CI: 3.36-4.30, p < 0.001). The adjusted long-term HCU trends persisted, with significantly higher use of respiratory-related medications and pediatric pulmonologist visits (IRR = 1.21, 95%CI: 1.06-1.40, p = 0.006) in the RSV-LRTI group.

Conclusion: This nationwide study highlights the substantial acute and long-term HCU burden associated with RSV-LRTI compared to Non-RSV-LRTI, underscoring the need for effective preventive strategies for all infants, especially in the first months of life.

Background: Mental health was identified as a top research priority of the cystic fibrosis (CF) community. The CF Foundation formed a Mental Health Research Priorities Working Group and sought input into specific mental health topics for research prioritization.

Methods: A survey of adults with CF, caregivers/family members, CF Foundation staff, and multidisciplinary CF healthcare providers elicited feedback regarding prioritization of a range of mental health research topics. We compared quantitative and qualitative responses based on respondent type and demographic characteristics, and analyzed group means using two-sided T-tests with Bonferroni adjustment for multiple comparisons (p < 0.1). We summarized progress to date in addressing the identified research priorities.

Results: Community (n = 693) and provider (n = 352) respondents agreed on the top six research priorities rated as "very important" or "a top priority": (1) anxiety, including procedural anxiety; (2) depression/mood disorders; (3) effects of mental health on physical health; (4) effects of treatment burden on mental health; (5) understanding risk factors and prevalence of mental health conditions in people with CF (PWCF); (6) effects of CF on the family, including family planning. Providers rated substance misuse and disordered eating/body image "very important/top priority" more often than community members. Community members rated mental health side effects of CF medications, PTSD/medical trauma, grief/bereavement, and survivor's guilt "very important/top priority" more often than providers.

Conclusions: The CF Foundation Mental Health Research Prioritization survey yielded a compelling roadmap for CF mental health research. These priorities are shaping initiatives to improve the mental health and well-being of PWCF and their families.

Rationale: The lung is uniquely positioned for chemical inhalation exposures considering its direct communication with the environment. Childhood interstitial lung diseases (chILD) syndrome is a rare and heterogeneous group of pediatric lung diseases. Despite common inhalation exposures, few studies have associated chemical inhalation exposures with chILD. The purpose of this review was to assess for chILD syndromes following toxic chemical inhalation exposures.

Methods: PubMed and Embase databases were searched with the following inclusion criteria: (1) toxic chemical inhalation exposure, (2) pediatric subjects, and (3) chILD syndrome. Studies were excluded due to incorrect (1) study design, (2) patient population, and/or (3) outcome.

Results: Two hundred and one studies were identified, of which 142 articles were retrieved, with 74 articles included after inclusion/exclusion criteria were applied by two independent reviewers. Most of the evidence stemmed from two pandemics: humidifier disinfectant associated lung disease (HD-ILD; n = 27) and e-cigarette, or vaping product associated, lung injury (EVALI; n = 45). Common signs and symptoms included cough, shortness of breath, hypoxemia, and inspiratory crackles. Common radiographic findings included centrilobular nodules and ground-glass opacities with subpleural sparing. Histopathologic features included airway-centric injury/inflammation with foamy macrophages, as well as subpleural sparing. Oxidized lipids were common plasma biomarkers associated with both HD-ILD and EVALI. Long-term pulmonary function testing suggests a restrictive phenotype in HD-ILD but variable phenotypes in EVALI.

Conclusions: ChILD syndromes secondary to toxic chemical inhalation exposures manifest with common radiographic and histopathologic findings of airway-centric disease with subpleural sparing. Long-term monitoring is under-reported in toxic chemical inhalation chILD syndromes but suggests persistent lung function impairment, especially after high-dose, repeated exposures. Additional monitoring, evaluation, and reporting of chILD syndromes secondary to chemical inhalation exposure are needed to better understand its complex pathogenesis and long-term lung function implications.