Single Dose Dexamethasone is as Effective as Two Doses in Mild to Moderate Pediatric Asthma Exacerbations in the Emergency Department
Phase: PHASE3
Conditions: Asthma
Sponsor: University at Buffalo

 
 
Official Title: 
Single Dose Dexamethasone is Not Inferior to Two Doses 
in Mild to Moderate Pediatric Asthma Exacerbations in the 
Emergency Department 
NCT number: 
NCT02192827 
Document Type:  
Study Protocol and Statistical Analysis Plan 
Date of the 
Document: 
November 9, 2017 
 
 
 

Methods 
We performed a single-site, prospective, parallel group, un-blinded randomized clinical trial which 
enrolled a convenience sample of patients aged 2-20 years with previously diagnosed asthma presenting 
to a single emergency department with a mild to moderate exacerbation of asthma. The hospital’s 
institutional review board approved the study and informed consent was obtained from eligible 
patients’ legal guardians or adult patients (18-20 years age).  In addition, patients 7 to 17 years of age 
provided written ascent.  
 
Study Setting and Participants:  
Children aged 2 to 20 years with a known history of asthma who presented to the Emergency 
Department at Women and Children’s Hospital of Buffalo/Oishei Children’s Hospital between April 2015 
and March 2018 with an acute exacerbation of mild or moderate asthma were eligible for the study. 
Asthma severity was defined by Pediatric Asthma Scores[2, 12] (PAS). Mild asthma is defined as: PAS of 
5 to 7; Moderate asthma is defined as: PAS of 8 to 11; Severe asthma is defined as: PAS of 12 or more. 
History of asthma is defined by physician diagnosis of at least one prior episode of wheezing which 
responded to beta agonists.  Patients were excluded from the study if they had signs of severe asthma 
exacerbation (PAS of 12 or more), had used oral steroids in the last 2 weeks, had chronic lung disease 
(e.g., cystic fibrosis), had been given  parenteral steroids, or if they vomited two doses of oral steroids in 
emergency department.  
Research assistants screened asthma patients through the electronic medical record between the hours 
of 8 a.m. and 11 p.m. 7 days a week. Patients were approached sequentially based on registration time. 
If a patient appeared to meet study criteria, his/her ED treating physician was approached by the 
research assistant for identifying patient’s eligibility.  If a patient was fully eligible, the legal guardian 
and/or patient were approached for consent.  
 
Study Protocol: 
Block randomization was used to generate a list to be used for subject assignment with a 1:1 ratio of 
allocation to the single dose group and the two dose group. Demographic information such as age, 
gender, race, duration of asthma symptoms, number of previous hospitalizations, and current 
medication was collected.  Pertinent exam findings such as patient’s vital signs, pulse oximetry, and PAS 
were also collected along with medications given in the ED.   
Group 1 was given 0.6mg/kg (max 16mg) of dexamethasone orally in the emergency department. Group 
2 was given the same dose in the emergency department, and then a prescription for a second dose was 
sent to their pharmacy to be filled and administered at home 24 hours later.  We worked with a nearby 
24-hour pharmacy to ensure medication availability; however, pharmacy preference was left up to the 
family. Both groups received asthma treatment following a standardized asthma care path.     
All patients were contacted by phone by a research assistant on the 6th day following the emergency 
department visit. Research assistants were not completely blinded during the study. They were initially 
blinded to the study group at the start of the phone interviews; however, during the course of the 

interview they did ask if the prescription for the second dose of dexamethasone was filled and if the 
medication was given to the patient.  Information collected during the phone interview included 
additional visits to medical providers (ED, primary care or urgent care) for continued asthma symptoms, 
information was collected on the reason for visit, whether symptoms continued or worsened, and if 
additional treatment was needed (scheduled visits and unrelated visits were excluded). School days 
missed due to asthma exacerbation; length of time symptoms persisted; compliance with the 
recommended steroid regimen; and any vomiting, side effects or medication administration problems 
caused by the steroids was also collected.   
 
Analytic Plan: 
Descriptive characteristics were computed for all patients combined and separately by dexamethasone 
group assignment.  Categorical variables were reported as proportions in percentage, and continuous 
level variables as means and standard deviations.  Separate independent t-tests were used to assess 
differences by group for post-discharge outcomes of interest including days for symptoms to resolve.  
Separate binary logistic regression adjusting for age, gender and severity of exacerbation were 
conducted to assess group differences in post-discharge outcomes including patients who had a return 
visit for unresolved asthma symptoms (ED, primary care or urgent care), days to symptom resolution, 
any missed school days between discharge and follow-up, rates of vomiting, and side-effects (changes in 
appetite, insomnia, mood swings); odds ratios with 95% confidence intervals were reported.  Similar 
analyses were conducted to compare events and side effects between Groups 1 and 2 by asthma 
severity, using an intention-to-treat approach.  Based on our clinical experience, we based our a-priori 
sample size calculation on an estimated non-inferiority limit of 11% and a rate of 12% return for asthma 
for each group.  With a power of 80% and alpha of 0.05, a minimum sample size of 216 (108 subjects per 
group) was determined. All statistical tests were two-tailed, and analyses conducted with SYSTAT 13 
(SYSAT Software, 2004).