Pediatric Pulmonology最新文献

Background: Flexible bronchoscopy (FB) is widely used in the management of children with Cystic Fibrosis (CF) to visualize airway abnormalities, assess inflammation and detect infection. While previous scoring systems have been proposed to quantify visual airway findings in general pediatric populations, no standardized tool exists for assessing airway inflammation specific to CF.

Methods: We modified the previously proposed pediatric bronchoscopy scoring tool by adding four features relevant to CF pathology: mucus plugging, secretion viscosity, bleeding, and vascular drawing (abnormal or enhanced visualization of airway mucosal vessels, reflecting neovascular remodeling associated with inflammation). Eighty bronchoscopy recordings (50 CF, 30 non-CF) were retrospectively scored by four raters blinded to the clinical information, and ten visual features were assessed: six from the previously proposed score (secretion amount and color, mucosal edema, erythema, ridging, and pallor) and the four CF-specific additions. Inter-rater reliability was assessed using Gwet's AC2 coefficient.

Results: Agreement between raters varied across features. Mucus plugging (AC2 = 0.94) and bleeding (0.80), both CF-specific, were among the most reliably scored features, while secretion viscosity (0.47) and vascular drawing (0.50) showed the lowest agreement. The expanded score demonstrated comparable or improved reliability for overlapping features from earlier scoring systems.

Conclusion: The modified bronchoscopy score demonstrated moderate to excellent inter-rater reliability and added clinically relevant features specific to CF. It may serve as a standardized method to assess bronchoscopy for pediatric CF lung disease, although further validation is needed for features with lower inter-rater reliability.

Objectives: CFTR modulators have revolutionized cystic fibrosis (CF) management by targeting the defective protein rather than its consequences. Their impact on quality of life (QoL) have been studied in numerous trials, but few data are available on QoL in patients receiving Elexacaftor-Tezacaftor-Ivacaftor (ETI), notably in children given its recent authorization for this age group. We aimed to assess the impact of ETI on children's QoL.

Methods: This prospective observational study included children with CF (6-17 years), assessing QoL using the CF Questionnaire Revised (CFQ-R) before (baseline) and 3 months after (M3) starting ETI treatment for children and their caregivers.

Results: We included 23 children (median [range]) age 10.2 [6-17.2] years, 13 (57%) with homozygous F508del genotype. The total QoL score at baseline ([mean (SD)] children: 74.07 [10.86]; caregivers: 73.21 [10.38]) reflected severe disease impact, particularly regarding treatment burden in the children's perspective (63.28 [21.04]) and digestive domains in the caregivers' perspective (digestive symptoms: 66.67 [17.37]; eating disorder: 67.54 [32.14]; weight: 61.40 [33.82]). At M3, there was a significant increase in reported QoL (p = 0.0001), particularly regarding physical domains. Emotional/social/school domains barely showed improvement. Although QoL mean scores were comparable between children and caregivers' groups, they were poorly correlated within the same family. Homozygous F508del genotype was associated with better QoL improvement at M3 compared to composite heterozygous genotypes (p < 0.001).

Conclusion: ETI treatment has a significant impact on children's QoL, particularly in physical health domains. Other QoL domains that are not improved by ETI need to be addressed, in particular, psycho-social components. Both children's and caregivers' perspectives must be considered for a holistic picture of children's QoL.

Background: While adult patients with interstitial lung disease (ILD) commonly experience poor sleep quality characterized by abnormal sleep architecture, increased fragmentation, and sleep-disordered breathing (SDB), children's interstitial lung disease (chILD)-a heterogeneous group of diffuse parenchymal lung diseases-remains less studied in terms of its impact on sleep.

Objective: We aimed to assess sleep quality and the prevalence of SDB, and to investigate potential associations between SDB and clinical, functional, and radiological parameters.

Materials and methods: This prospective cross-sectional study included children diagnosed with ILD. Sleep questionnaire scores, polysomnography (PSG) parameters, and SDB outcomes were recorded and compared between patients with and without lung fibrosis.

Results: Of 30 patients (36.7% female, mean age 115 ± 59.3 months), 77% were diagnosed with OSAS (63.3% mild, 13.3% moderate). Sleep efficiency was below 85% in 20% of cases, and REM sleep was reduced in 90%. Median AHI was 1.8. According to sleep questionnaires, 80% of patients had impaired sleep quality, and 60% showed an increased risk for SDB. No significant association was found between pulmonary fibrosis and SDB.

Conclusion: Contrary to common belief, SDB is not rare in chILD, and PSG alongside sleep questionnaires should be used in follow-up to improve screening and detection.

Background: Spirometry (FEV₁) is often measured as part of ongoing asthma management, but little is understood about what merits a clinically meaningful change in FEV₁.

Metholology: Data were collated from five clinical trials where FEV₁ was measured at 3-month intervals. Change in FEV₁ over 3-months was expressed as: FEV₁ change score (Zc), absolute change in %FEV₁ (∆FEV₁%) and absolute change in FEV₁ z score (∆FEV₁z). The association between change in FEV₁ over 3 months and asthma outcomes in the following 3 months was estimated using logistic regression.

Results: Data from 1264 children were analysed. Declines in in Zc between 1.1 and 2.9, in ∆FEV₁% between 12% and 23% and in ∆FEV₁z between 0.9 and 2.0, were associated with increased odds ratio (OR) for later asthma exacerbation of between 1.3 and 2.3. Unexpectedly, rises in Zc of 1.7-1.9 and in ∆FEV₁% of 14 and 18 were also associated with increased OR for a future exacerbation of between 1.3 and 1.6. Among children with controlled asthma symptoms, declines in Zc of 1.7, in ∆FEV₁% of 11 and 14 and in ∆FEV₁z of 0.8-1.1 were associated with increases in OR for future loss of control of between 1.5 and 1.7. Rises in all three indices of change in FEV₁ were associated with reduced OR between 0.4 and 0.7 for future loss of control. A lower initial FEV₁ z score was associated with higher variability in longitudinal FEV₁ z-score measurements.

Conclusions: FEV₁ variability, and not simply a fall in FEV₁, identifies children at increased risk for future asthma exacerbations. The clinical relevance of a single FEV₁ may be uncertain.

Introduction: While chloral hydrate (CH) has been standard for infant pulmonary function testing (iPFT) sedation, CH shortages are necessitating use of different sedation approaches. We aimed to compare the safety, test duration, and test quality of alternative sedation strategies for iPFT.

Methods: We conducted a retrospective chart review of iPFT conducted at our center from January 2019 to December 2021. We manually abstracted patient demographics, sedation medications given, adverse events, and iPFT type (raised volume-rapid thoracic compression, plethysmography, bronchodilator response, and/or multiple breath washout), duration (induction, procedure, recovery, and total times), and quality (satisfactory vs unsatisfactory), then compared features of tests conducted with CH to tests conducted with ketamine and midazolam (KM), dexmedetomidine (DX), or multiple agents (polypharmacy, PP) using bivariate and multivariable analysis.

Results: Sixty-six children had iPFT (CH n = 42, KM n = 10, PP n = 8, and DX n = 6). Testing types and proportion of satisfactory tests did not significantly differ between CH and the other sedation strategies. In the multivariable analysis, compared to CH, we found that procedure time was shorter for KM, induction time was shorter for DX, and recovery time was longer for DX, yet total testing duration did not differ between CH and KM (p = 0.61) or DX (p = 0.22). In adjusted analyses, total testing time was longer for PP compared to CH (β = 12.0 min, p = 0.047). Adverse events, all of which were mild, occurred in three patients (PP n = 2, DX n = 1).

Conclusions: Our findings provide preliminary evidence that KM, DX, and PP may be safe and effective alternatives to CH for iPFT sedation.

The management of cystic fibrosis (CF) has significantly improved with the approval of the CF transmembrane conductance regulator (CFTR) modulators. Elexacaftor/tezacaftor/ivacaftor (ETI) is approved for treatment in people with CF (pwCF) over the age of 2 years with eligible mutations. We report 2 cases highlighting improved clinical outcomes following exposure to ETI in an off-label use, including enhanced nutrition status, decrease in respiratory support, exacerbation rates, and need for antibiotics in one of the infants and resolution of echogenic bowel that was diagnosed in utero in the second.

Objective: To compare the effect of the palatal expansion with Hyrax and Hyrax + Up-Locker on sleep behavior, snoring and sleep architecture in children with Sleep Disordered Breathing (SDB).

Materials and methods: This prospective, single-blind, randomized controlled clinical trial enrolled 34 children aged 6-8 years. Participants were randomly assigned using a computer-generated randomization process to receive treatment with either the Hyrax expansion appliance alone or in combination with the Up-Locker vacuum activator. Sleep architecture was evaluated via polysomnography and sleep behavior through the Children's Sleep Habits Questionnaire (CSHQ). Data were analyzed with Shapiro-Wilks for homogeneity and comparisons were performed with either t-test or Mann-Whitney.

Results: Children in the Hyrax + Up-Locker exhibited superior outcomes than in the Hyrax group, including statistically significant reduction in sleep onset delay (p = 0.04) and night wakings (p = 0.02), as well as a notable decrease in snoring (p < 0.001). Children in the Hyrax + Up-Locker vacuum activator group experienced more significant improvements in sleep latency, total sleep time, REM sleep, AHI in REM and nREM sleep, and arousal indices compared to the Hyrax group (p < 0.05) after treatment.

Conclusion: Combined treatment with Hyrax + Up-Locker vacuum activator yielded superior outcomes in sleep architecture and sleep behavior.

Trial registration: ClinicalTrials.gov identifier: NCT06986343.