Small Airway Deposition of Dornase Alfa During Exacerbations in Cystic Fibrosis; a Randomized Controlled Clinical Trial

被引:9
作者
Bakker, E. M. [1 ]
Volpi, S. [2 ]
Salonini, E. [2 ]
Muellinger, B. [3 ]
Kroneberg, P. [3 ]
Bakker, M. [4 ]
Hop, W. C. J. [5 ]
Assael, B. M. [2 ]
Tiddens, H. A. W. M. [1 ]
机构
[1] Erasmus MC Sophia Childrens Hosp, Dept Paediat Pulmonol & Allergol, NL-3000 CB Rotterdam, Netherlands
[2] Azienda Osped Verona, Cyst Fibrosis Ctr, Verona, Italy
[3] Activaero GmbH, Gemunden, Germany
[4] Erasmus MC, Dept Pulmonol, Rotterdam, Netherlands
[5] Erasmus MC, Dept Biostat, Rotterdam, Netherlands
关键词
aerosol; nebulizer; nocturnal oxygen saturation; respiratory tract exacerbation; spirometry; RECOMBINANT HUMAN DNASE; ACUTE PULMONARY EXACERBATIONS; LUNG-FUNCTION; AEROSOL DEPOSITION; CONTROLLED INHALATION; PULSE OXIMETRY; YOUNG-CHILDREN; DRUG-DELIVERY; DISEASE; INFANTS;
D O I
10.1002/ppul.22800
中图分类号
R72 [儿科学];
学科分类号
100202 ;
摘要
IntroductionSmall airway obstruction is important in the pathophysiology of cystic fibrosis (CF) lung disease. Additionally, many CF patients lose lung function in the long term as a result of respiratory tract exacerbations (RTEs). No trials have been performed to optimize mucolytic therapy during a RTE. We investigated whether specifically targeting dornase alfa to the small airways improves small airway obstruction during RTEs. MethodsIn a multi-center, double-blind, randomized controlled trial CF patients hospitalized for a RTE and on maintenance treatment with dornase alfa were switched to a smart nebulizer. Patients were randomized to small airway deposition (n=19) or large airway deposition (n=19) of dornase alfa for at least 7 days. Primary endpoint was forced expiratory flow at 75% of forced vital capacity (FEF75). Main ResultsSpirometry parameters improved significantly during admission, but the difference in mean change in FEF75 between treatment groups was not significant: 0.7 SD, P=0.30. FEF25-75, FEV1, nocturnal oxygen saturation and diary symptom scores also did not differ between groups. ConclusionsThis study did not detect a difference if inhaled dornase alfa was targeted to small versus large airways during a RTE. However, the 95% confidence interval for the change in FEF75 was wide. Further studies are needed to improve the effectiveness of RTE treatment in CF. Pediatr Pulmonol. 2014; 49:154-161. (c) 2013 Wiley Periodicals, Inc.
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收藏
页码:154 / 161
页数:8
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