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Recombinant human DNase in children with airway malacia and lower respiratory tract infection
Ruben Boogaard1, Johan C de Jongste, Anja A P H Vaessen-Verberne
1Division of Pulmonology, Department of Pediatrics, Erasmus MC-Sophia Children's Hospital, Erasmus University Medical Centre, Rotterdam, The Netherlands. r.boogaard@erasmusmc.nl
Insights
Recombinant human deoxyribonuclease (rhDNase) did not improve respiratory symptoms or reduce antibiotic use in children with airway malacia and lower respiratory tract infection. This study found no significant benefits from rhDNase treatment.
Area of Science:
- Pediatric Pulmonology
- Respiratory Medicine
- Clinical Trials
Background:
- Children with airway malacia experience recurrent respiratory infections due to impaired mucociliary clearance from dynamic airway collapse.
- Lower respiratory tract infections (LRTI) in these children often lead to prolonged illness.
Purpose of the Study:
- To evaluate the efficacy of nebulized recombinant human deoxyribonuclease (rhDNase) in improving recovery from LRTI in pediatric patients with airway malacia.
- To assess the impact of rhDNase on respiratory symptom scores and the need for antibiotic treatment.
Main Methods:
- A randomized, double-blind, placebo-controlled trial involving 40 children diagnosed with airway malacia and LRTI.
- Participants received either nebulized rhDNase (2.5 mg) or placebo twice daily for two weeks.
- Primary endpoint was the change in cough diary score; secondary endpoints included symptom scores, antibiotic use, and lung function.
Main Results:
- No statistically significant difference was observed in the change of cough diary scores between the rhDNase and placebo groups.
- Proportions of patients requiring antibiotics and changes in other symptom scores and lung function parameters did not differ significantly between the groups.
Conclusions:
- Two weeks of nebulized rhDNase treatment does not accelerate recovery from LRTI in children with airway malacia.
- rhDNase did not reduce the need for antibiotic therapy in this patient population.
Background:
Children with airway malacia often have protracted courses of airway infections, because dynamic airway collapse during coughing results in impaired mucociliary clearance. The aim of this study was to determine the effect of the mucolytic drug recombinant human deoxyribonuclease (rhDNase) on the recovery of respiratory symptoms in children with airway malacia and lower respiratory tract infection (LRTI).
Methods:
In a randomized double-blind controlled clinical trial, 40 children with airway malacia and LRTI were randomly assigned to receive either 2.5 mg nebulized rhDNase or placebo twice daily for 2 weeks. The primary endpoint was the change in the cough diary score (CDS) (scale 0-5) from baseline to the second week of treatment. Secondary endpoints were VAS symptom scores for cough, dyspnea, and difficulty in expectorating sputum, need for an antibiotic course, and lung function data (FVC, FEV(1), FEF(75), R(int(e))).
Results:
There was no significant difference in the mean change in CDSs from baseline between the rhDNase group and the placebo group (mean difference for daytime 0.19 (95% CI -0.53 to 0.90); for nighttime 0.38 (95% CI -0.30 to 1.05). Proportions of patients requiring antibiotics, and the mean changes in symptom scores and lung function from baseline did not significantly differ between both groups.
Conclusion:
Treatment with 2 weeks of nebulized rhDNase does not enhance recovery or reduce the need for antibiotics in children with airway malacia and LRTI. (Controlled-trials.com number, ISRCTN85366144).
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