Theresa W. Guilbert

Background: Achieving and maintaining pediatric asthma control depends on effective and regular monitoring. While a wide range of monitoring tools are available, how and why clinicians use these in routine practice has not been formally characterized. Here we describe distinct real‐world monitoring patterns among healthcare professionals ( HCPs ) caring for children with asthma in an international convenience survey, examining the geographic, professional, and institutional factors associated with them. Methods: Data from the international cross‐sectional Pediatric Asthma in Real Life ( PeARL ) HCP survey, administered to clinicians with expertise in childhood asthma, were analyzed. Monitoring patterns were derived by Ward agglomerative hierarchical clustering on four standardized domains: clinical evaluation, spirometry, fractional exhaled nitric oxide ( FeNO ), and home‐monitoring. Independent associations between cluster membership and region, specialty, sector, and level of care were estimated by multinomial logistic regression ( MLR ). Results: 951 HCPs from 78 countries were included. Four monitoring patterns emerged: Low‐intensity ( n = 88, 9.3%), lower overall monitoring intensity but moderate clinical assessment; Clinical ( n = 449, 47.2%), predominantly clinical assessment with moderate spirometry but infrequent FeNO and home monitoring; Clinical & Objective ( n = 308, 32.4%) with additional regular spirometry with moderate FeNO use and Comprehensive ( n = 106, 11.1%), also providing home monitoring. In MLR ( n = 933), region and care level were significantly associated with cluster membership ( p < .001), and specialty ( p = .012) and sector ( p = .039) also contributed independently. The most pronounced gradients involved objective in‐clinic testing: specialists were 3.5‐fold (95% CI 1.68–7.37) more likely than generalists, and tertiary‐care HCPs 3.3‐fold (95% CI 1.99–5.58) more likely than primary‐care HCPs, to belong to the Clinical & Objective pattern, with routine use of objective measures concentrated in Western and Northern European practices. Home monitoring showed no consistent gradient across the measured characteristics. Conclusion: Real‐world pediatric asthma monitoring follows recognizable patterns, distinguished by the use of objective in‐clinic testing and between‐visit home‐based practices. Nearly half of HCPs relied predominantly on clinical assessment with limited use of FeNO and home‐based monitoring, and only a minority integrated home‐monitoring tools. Objective in‐clinic testing concentrated in specialist services and Western/Northern European practices, while home‐monitoring adoption showed no consistent gradient. These findings identify factors associated with pediatric asthma monitoring and suggest system‐level factors that warrant direct evaluation in dedicated implementation studies. image

Background: Asthma phenotype has implications for presentation and treatment selection. Methods: This was a retrospective study of patients attending a pediatric pulmonary clinic (2009–2015) with EHR data available for phenotyping (allergic, eosinophilic/non‐allergic or non‐allergic/non‐eosinophilic). Patients were classified by age. A multinomial regression model examined factors associated with phenotype. A logistic regression model examined factors associated with a change in phenotype (present/absent). Results: There were 2042 patients included. Approximately 44% of participants were allergic, 17% eosinophilic/non‐allergic and 39% non‐allergic/non‐eosinophilic. Among children 0–4 years, age of asthma onset (aOR 1.42, 95% CI 1.21, 1.65) and mean exacerbations (aOR 1.26 95% CI 1.09, 1.44) were associated with higher odds of an allergic phenotype (vs. non‐allergic/non‐eosinophilic). Among those aged 5–11 and 12–21, male sex and mild obstruction were positively associated, and age was negatively associated, with an allergic phenotype. Moderate/severe obstruction on spirometry (aOR 6.67, 95% CI 2.00, 22.29) and smoke exposure (aOR 1.77, 95% CI 1.17, 2.67) were also positively associated with an allergic phenotype in patients 12–21 years. Factors positively associated with a change in phenotype included index phenotype (eosinophilic/non‐allergic: aOR 5.31, 95% CI 3.46, 8.14) and mean exacerbations (aOR 1.34, 95% CI 1.16, 1.55) ( n = 1379). The allergic phenotype was most stable (> 95% predicted probability of remaining allergic); an eosinophilic/non‐allergic phenotype had the greatest predicted probability of change (48%). Conclusions: Factors associated with asthma phenotype vary by age. The allergic phenotype was the most stable, while an eosinophilic/non‐allergic phenotype was most prone to change.

Background and Objective: Individuals from historically marginalized populations face structural and social determinants of health that create barriers to receiving specialty asthma care. School‐based specialist care delivered via telehealth offers a potential solution for children with uncontrolled asthma. This study evaluated the reach, implementation, and effectiveness of school‐based telehealth asthma clinics led by pediatric pulmonologists in historically marginalized neighborhoods. Methods: Patients completed at least one visit with an asthma specialist via telehealth, including assessments of asthma control, lung function, exacerbations, and healthcare utilization. Descriptive analyses and means comparison tests of electronic health record data were conducted to examine outcomes. Results: Asthma specialists provided telehealth care for 54 patients ( M _age = 12.23, SD = 3.71) between May 2019 and May 2023. The clinics reached a high percentage of individuals from historically marginalized populations (90.7% Black or African American, 92.6% public insurance) who had not previously received specialty asthma care (51.9%). Twenty‐three patients (42.6%) were seen more than once in the school‐based telehealth asthma clinics ( M = 3.43 visits, SD = 1.53). Asthma control significantly improved for those patients, t (19) = 2.61, p = 0.017, Cohen's d = 0.58, with 30% improving from poorly controlled to well‐controlled. Exacerbations and healthcare utilization decreased. Conclusions: Specialist‐delivered, school‐based telehealth asthma clinics effectively reached patients from historically marginalized backgrounds and improved clinical outcomes. This school‐based telehealth model can be applied across settings to reach patients who might not otherwise have access to specialty care.

Purpose: Dupilumab, a fully human monoclonal antibody, blocks the shared receptor component for interleukins-4/-13, key and central drivers of type 2 inflammation in multiple diseases. This post hoc analysis of the Phase 3 LIBERTY ASTHMA VOYAGE study (NCT02948959) evaluated the efficacy of dupilumab in children aged 6 to 11 years with moderate-to-severe asthma with a type 2 inflammatory phenotype (blood eosinophil count ≥ 150 cells/μL or fractional exhaled nitric oxide [FeNO] ≥ 20 ppb) and a history of 1, 2, or ≥ 3 prior exacerbations. The impact of baseline type 2 biomarker levels on the efficacy of dupilumab in this population was also investigated. Patients and Methods: Patients were stratified by the number of exacerbations in the prior year (1, 2, or ≥ 3) and level of FeNO or blood eosinophil count at baseline. Endpoints included rate of severe exacerbations, percentage of non-exacerbators, and change from baseline in both lung function parameters (pre- and post-bronchodilator [BD] percent predicted forced expiratory volume in 1 s (ppFEV₁) and ppFEV₁/forced vital capacity [FVC] ratio) and Asthma Control Questionnaire 7 Interviewer-Administered (ACQ-7-IA) score. Results: A total of 350 patients were included in this analysis. Across patients with 1, 2, or ≥ 3 prior exacerbations and different levels of type 2 biomarkers, dupilumab reduced the risk of severe asthma exacerbations vs placebo by 53.0– 96.0% and improved both pre-BD ppFEV₁ and pre-BD FEV₁/FVC ratio at Week 52. Dupilumab led to significant reductions in ACQ-7-IA scores in all groups of patients by Week 52. Conclusion: In children with uncontrolled, moderate-to-severe asthma with a type 2 phenotype, dupilumab consistently reduced the risk of asthma exacerbations, improved lung function, and reduced ACQ-7-IA scores, regardless of exacerbation history.

Background: Severe, uncontrolled asthma and asthma exacerbations in children are associated with abnormal lung function and airway development, and increased risk of chronic obstructive lung disease in adulthood. The rationale for this post hoc analysis was to explore the relationship between changes in asthma exacerbation rates and lung function in children treated with dupilumab. Methods: This post hoc analysis included children aged 6 to 11 years with uncontrolled, moderate-to-severe type 2 asthma (blood eosinophils >= 150 cells/mu L or fractional exhaled nitric oxide >= 20 ppb) who received dupilumab or placebo in the phase 3 LIBERTY ASTHMA VOYAGE study (NCT02948959). Endpoints were the proportion of patients achieving clinically meaningful improvements (>= 5% or >= 10%) in pre-bronchodilator percent-predicted forced expiratory volume in 1 second (ppFEV(1)) by Week 12, annualized severe asthma exacerbation rates from Week 12-52, and mean change from baseline in ppFEV(1) to Week 12. Results: At Week 12 of VOYAGE, 141/236 (60%) of children treated with dupilumab and 57/114 (50%) of children receiving placebo showed improvements of >= 5% in ppFEV(1); 106/236 (45%) children receiving dupilumab and 36/114 (32%) receiving placebo achieved improvements in ppFEV(1) >= 10%. During the Week 12-52 treatment period, dupilumab vs placebo significantly reduced severe exacerbation rates in all subgroups by 52-60% (all P= 5%. Conclusion: Dupilumab vs placebo significantly improved pre-bronchodilator ppFEV(1), with a higher proportion of patients achieving a clinically meaningful response at Week 12. Dupilumab also significantly reduced severe exacerbation rates, independent of pre-bronchodilator ppFEV(1) response at Week 12.

Introduction: Persistent lung function impairment in children with asthma is associated with impaired quality of life and risk of exacerbation. In phase 3 LIBERTY ASTHMA VOYAGE (NCT02948959), add-on dupilumab (DPL) was generally well tolerated and significantly reduced the annualized rate of severe exacerbations (AER) and improved lung function in children. Aims and objectives: To evaluate lung function responder rates at Week (Wk)12 and Wk52 of VOYAGE. Methods: Children 6–11 years with uncontrolled, moderate-to-severe, type 2 asthma (baseline [BL] blood eosinophils ≥150 cells/μL or fractional exhaled nitric oxide ≥20 ppb) were randomized to dupilumab 100mg/200mg (based on bodyweight) q2w or volume-matched placebo for 52 wks. Endpoints: least squares mean (LSM) change from BL in % predicted pre-BD FEV1 (ppFEV1) at Wk12 and 52; proportion of patients with ≥10% improvement in ppFEV1 at Wk12 and 52; and AER in those responders. Results: ppFEV1 was significantly improved with DPL vs placebo (PBO) at Wk12 (LSM difference [95% CI] 5.20 [2.14–8.26]; P= 0.0009) and Wk52 (7.79 [4.36–11.22]; P<0.0001). A ≥10% ppFEV1 improvement was achieved at Wk12 in 44.9% (n=106; [38.5–51.5]) vs 31.6% (n=36; [23.2–40.9]; P=0.0173) and at Wk52 in 47.5% (n=112; [40.9–54.0]) vs 28.1% (n=32; [20.1–37.3]; P=0.0006) of patients receiving DPL vs PBO, respectively. DPL vs PBO significantly decreased the AER in patients achieving ≥10% ppFEV1 improvement, 0.304 (n=106; [0.185–0.500]) vs 0.744 (n=36; [0.424–1.308]; P=0.0103), respectively. Conclusions: At Wk12 and 52, DPL significantly improved ppFEV1. More patients treated with DPL vs PBO met the ppFEV1 responder criteria and experienced significantly lower AER.