Abstract
BackgroundA randomized controlled trial of adults with empyema recently demonstrated decreased length of stay in hospital in patients treated with intrapleurally administered dornase alfa and fibrinolytics compared to fibrinolytics alone. Whether this treatment strategy is safe and effective in children remains unknown.Methods/designThis study protocol is for a superiority, placebo-controlled, parallel-design, multicenter randomized controlled trial. The participants are previously well children admitted to a children’s hospital with a diagnosis of empyema requiring chest tube insertion and fibrinolytics administered intrapleurally. Children will be randomized after the treating physician has decided that pleural drainage is required but prior to chest tube insertion. After chest tube insertion, participants in the treatment group will receive intrapleurally administered tissue plasminogen activator (tPA) 4 mg followed by dornase alfa 5 mg. Participants in the placebo group will receive tPA 4 mg followed by normal saline. Study treatments will be administered once daily for 3 days. All participants, parents or caregivers, clinicians, and research personnel will remain blinded. The primary outcome is length of stay from chest tube insertion to discharge from hospital. Secondary outcomes include time to meeting discharge criteria, chest tube duration, fever duration, need for additional procedures, adverse events, hospital readmission, cost of hospitalization, and mortality.DiscussionThis multicenter randomized controlled trial will assess the safety, effectiveness, and cost-effectiveness of combined treatment with dornase alfa and fibrinolytics compared to fibrinolytics alone for the treatment of empyema in children.Trial registrationClinicalTrials.gov: NCT01717742. Registered on 8 October 2012.
Highlights
A randomized controlled trial of adults with empyema recently demonstrated decreased length of stay in hospital in patients treated with intrapleurally administered dornase alfa and fibrinolytics compared to fibrinolytics alone
This multicenter randomized controlled trial will assess the safety, effectiveness, and cost-effectiveness of combined treatment with dornase alfa and fibrinolytics compared to fibrinolytics alone for the treatment of empyema in children
In our previous prospective cohort study of children with empyema, we found that by 6 months virtually all patients were asymptomatic, radiographs had normalized in 95% of patients, and pulmonary function tests were normal in 96% [38]
Summary
There are important gaps in the scientific literature regarding the optimal therapy for pleural empyema in children [13, 14, 16]. Over the past 20 years, the number of adult randomized controlled trials published in leading general and subspecialty medical journals has increased substantially, while the number of pediatric trials has increased only modestly [41, 42] This problem is relevant in pharmaceutical trials, and has undoubtedly led to the widespread use of pharmaceutical products in children without sufficient data on effectiveness or safety [43]. Data from a randomized controlled trial in adults with empyema suggests that adding dornase alfa may be superior to using fibrinolytics alone This treatment strategy requires further study in a pediatric setting since differences in the underlying disease may affect response to treatment.
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