Trial Outcomes & Findings for Positive Expiratory Pressure for the Treatment of Acute Asthma in Children (NCT NCT02494076)

Results Overview

The primary outcome was the change in asthma severity as determined by change in Pulmonary Asthma Score (PAS) before and after administration of intervention (or control). The same trained, blinded physician assessor, who was not involved in the care of the patient, assessed PAS scores for study subjects before intervention (or control), and 15 minutes after completion of administration. The PAS is a pediatric asthma severity scoring system adapted from previously validated scores, and includes measures of respiratory rate, oxygen saturation, auscultory findings, retractions, and dyspnea. Values from each category are summed producing a total score between 5 and 15. Total scores \< 7 correspond with mild asthma exacerbations, while scores ≥ 7 and \< 12 indicate moderate asthma, and scores ≥12 to 15 indicate severe asthma. The primary outcome was determined by subtracting the post-intervention score from the pre-intervention score.

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

52 participants

Primary outcome timeframe

0-30 minutes

Results posted on

2017-12-08

Participant Flow

Participant milestones

Participant milestones
Measure	EzPAP Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care
Overall Study STARTED	26	26
Overall Study COMPLETED	26	26
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Positive Expiratory Pressure for the Treatment of Acute Asthma in Children

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	EzPAP n=26 Participants Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care n=26 Participants Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care	Total n=52 Participants Total of all reporting groups
Age, Categorical <=18 years	26 Participants n=5 Participants	26 Participants n=7 Participants	52 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	8.7 years STANDARD_DEVIATION 4.3 • n=5 Participants	6.9 years STANDARD_DEVIATION 4.1 • n=7 Participants	7.8 years STANDARD_DEVIATION 4.3 • n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	5 Participants n=7 Participants	15 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants	21 Participants n=7 Participants	37 Participants n=5 Participants
Region of Enrollment United States	26 Participants n=5 Participants	26 Participants n=7 Participants	52 Participants n=5 Participants

PRIMARY outcome

Timeframe: 0-30 minutes

Population: 2 patients in EzPAP group were excluded from analysis due to protocol error and missing data.

The primary outcome was the change in asthma severity as determined by change in Pulmonary Asthma Score (PAS) before and after administration of intervention (or control). The same trained, blinded physician assessor, who was not involved in the care of the patient, assessed PAS scores for study subjects before intervention (or control), and 15 minutes after completion of administration. The PAS is a pediatric asthma severity scoring system adapted from previously validated scores, and includes measures of respiratory rate, oxygen saturation, auscultory findings, retractions, and dyspnea. Values from each category are summed producing a total score between 5 and 15. Total scores \< 7 correspond with mild asthma exacerbations, while scores ≥ 7 and \< 12 indicate moderate asthma, and scores ≥12 to 15 indicate severe asthma. The primary outcome was determined by subtracting the post-intervention score from the pre-intervention score.

Outcome measures

Outcome measures
Measure	EzPAP n=24 Participants Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care n=26 Participants Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care
Change in Pulmonary Asthma Score (PAS)	0.9 mean PAS score Standard Deviation 1.2	0.4 mean PAS score Standard Deviation 1.2

SECONDARY outcome

Timeframe: participants will be followed for the duration of ED stay, an expected average of 6-8 hours

Population: 2 subjects excluded from analysis due to missing data

Second line therapies include: continuous albuterol, intravenous magnesium sulfate, subcutaneous or intravenous terbutaline, non-invasive ventilation (BiPAP or CPAP), and supplemental oxygen. The need for these second line therapies will be assessed by the child's treating team in the Emergency Department.

Outcome measures

Outcome measures
Measure	EzPAP n=24 Participants Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care n=26 Participants Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care
Number of Participants Requiring Second Line Therapies Including Continuous Albuterol, Subcutaneous Terbutaline, IV Magnesium and Supplemental Oxygen After Administration of Intervention or Control Continuous albuterol	11 Participants	15 Participants
Number of Participants Requiring Second Line Therapies Including Continuous Albuterol, Subcutaneous Terbutaline, IV Magnesium and Supplemental Oxygen After Administration of Intervention or Control Intravenous magnesium	1 Participants	3 Participants
Number of Participants Requiring Second Line Therapies Including Continuous Albuterol, Subcutaneous Terbutaline, IV Magnesium and Supplemental Oxygen After Administration of Intervention or Control Subcutaneous or intravenous terbutaline	0 Participants	1 Participants
Number of Participants Requiring Second Line Therapies Including Continuous Albuterol, Subcutaneous Terbutaline, IV Magnesium and Supplemental Oxygen After Administration of Intervention or Control Supplemental oxygen	2 Participants	7 Participants

SECONDARY outcome

Timeframe: After intervention or control and until follow-up phone call 72 hours after disposition

Population: 2 patients excluded due to missing data and protocol error

Number of patients in each group requiring hospital admission

Outcome measures

Outcome measures
Measure	EzPAP n=24 Participants Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care n=26 Participants Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care
Rate of Inpatient Hospitalization	10 Participants	13 Participants

Adverse Events

EzPAP

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Standard Care

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	EzPAP n=26 participants at risk Patients randomized to the EzPAP arm will receive first-line therapies and PEP therapy via EzPAP. All patients will receive 4 cycles with 12 breaths per cycle. EzPAP	Standard Care n=26 participants at risk Patients randomized to the control arm will receive first-line therapies and standard therapy. Standard Care
Skin and subcutaneous tissue disorders Facial redness	3.8% 1/26 • Number of events 1 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.	0.00% 0/26 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.
Nervous system disorders Light-headedness	3.8% 1/26 • Number of events 1 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.	0.00% 0/26 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.
Respiratory, thoracic and mediastinal disorders Shortness of breath	3.8% 1/26 • Number of events 1 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.	0.00% 0/26 • Adverse events were monitored and collected during the total ED course. The definition of adverse event does not differ from the clinicaltrials.gov definitions.

Additional Information

Nidhya Navanandan, MD

University Colorado School of Medicine, Chidren's Hospital Colorado

Phone: 847-636-0817

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place