NHLBI TOPMed: Pediatric Asthma Controller Trial (PACT)

After a 2-4 week assessment/characterization run-in period, 6-14 year-old children who met NAEPP criteria for mild-moderate persistent asthma specifically based on symptom criteria and methacholine PC20 ≤ 12.5 mg/ml and FEV1 ≥ 80% were randomized to one of the three active treatment arms for 12 months. Randomization was stratified according to clinical center, bronchodilator response (< 12% or ≥ 12%), race (Caucasian or non-Caucasian), and methacholine PC20 (< 2 or ≥ 2 mg/ml). The primary outcome variable was the proportion of asthma-free days during the 12-month treatment period. Secondary outcomes included other measures of asthma control (percentage of rescue-free days, albuterol-free days, and episode-free days; the number of asthma exacerbations requiring prednisone therapy and the time to the first asthma exacerbation), forced oscillation and spirometry, reversibility (FEV1 pre- and post 2 puffs of albuterol MDI), methacholine PC20, exhaled nitric oxide, and asthma-related quality of life.]]>
TOPMed Whole Genome Sequencing Methods: Freeze 8TOPMed Whole Genome Sequencing Methods: Freeze 9Children with mild-moderate persistent asthma, as defined by symptoms and positive methacholine challenge. Inclusion criteria were physician-diagnosed asthma, age 6 to less than 14 years, ability to perform reproducible spirometry,17 an FEV1 (measured more than 4 hours since the most recent use of a bronchodilator) ≥ 80% predicted normal at screening and ≥ 70% predicted normal at randomization, and a methacholine FEV1 PC20 ≤ 12.5 mg/mL. All children had mild-moderate persistent asthma, as defined by diary-reported symptoms or β-agonist use (not including pre-exercise) or peak flows <80% calculated from the mean of morning and evening peak flows obtained during the final week of the run-in period, on average at least 3 times per week. Exclusion criteria included other lung diseases; respiratory tract infection, asthma exacerbation, or systemic corticosteroid use within 4 weeks; 2 or more asthma hospitalizations in the past year; history of a life-threatening asthma exacerbation; ≥ 4 courses of systemic corticosteroids in the past year; cigarette smoking within the past year; pregnancy or lactation; failure to practice abstinence or use a medically acceptable birth control method; and history of adverse reactions to the PACT medications. Enrolled children were not on controller medications for at least 2 weeks before randomization. Participants were excluded if they were unable to use the study drug delivery systems or to adhere with ≥75% of doses during the run-in.]]>