CYSTIC FIBROSIS LUNG SOUNDS TRIAL

The recommendations of the Preferred Reporting Items for Systematic reviews and Meta-Analyses (PRISMA) statement were followed to ensure a systematic approach and minimize bias. In addition, recommendations for future research to validate and develop new tools are provided, considering that no ‘gold standard’ outcome measure related to respiratory physiotherapy is available. The aim of this review is to assist researchers in their choice of outcome measures by considering the qualities and limitations of the techniques. Besides behavioral challenges, the respiratory disease will often be in an early stage, which means sufficient sensitivity of the outcome is needed to detect any treatment effects. Outcome measures suitable for pediatrics require a specific approach. This systematic review provides an overview of all outcomes that have been used to evaluate ACTs in the pediatric population and whether or not significant changes were found. A wide variety of outcome measures have been used in previous studies, but it is not clear which outcomes are appropriate to measure the acute and/or long-term effects related to airway clearance.

CYSTIC FIBROSIS LUNG SOUNDS TRIAL

Unfortunately, for studies evaluating the effectiveness of airway clearance related interventions, a number of these clinical and surrogate endpoints are difficult to evaluate, as most studies only investigate short-term effects and the use of a control group (not receiving any physiotherapy) in a long-term trial is not accepted in most countries. For pulmonary diseases, the accepted surrogate endpoints for drug development by the European Medicines Agency (EMA) are currently lung function parameters and imaging. When it is not feasible to directly measure the clinical impact, surrogate endpoints are used to establish therapeutic efficacy and to predict clinical benefit. For example: pulmonary exacerbations, intravenous antibiotic usage, hospitalizations, quality of life and ultimately survival. Ī clinical endpoint in research has been defined as a characteristic or variable that reflects how a patient feels, functions, or survives. In order to evaluate the effectiveness of the therapy, it is necessary to have reliable and valid outcome measures that have sufficient discriminative capability. One of the barriers is the lack of adequate outcome measures specifically related to respiratory physiotherapy. Although ACTs are an important part of the treatment of our pediatric respiratory population, a sound evidence base is lacking and airway clearance strategies are largely based on clinical expertise. The main goals of airway clearance techniques (ACTs) are to enhance mucus mobilization, reduce airway resistance, improve ventilation and gas exchange and to reduce work of breathing. Physiotherapy focusing on augmenting mucociliary clearance and facilitating expectoration of excessive mucus is widely prescribed in chronic obstructive respiratory diseases. An overview of important considerations has been provided to assist researchers in their choice of outcomes in future studies. Since no ‘gold standard’ method could be identified, a combination of different outcome measures is recommended to gain a better understanding and to identify the potential effects of ACTs. The synthesis of results showed a high discrepancy between studies due to differences in study design, population and the application of techniques. 48 (98%) studies performed pulmonary function tests, 19 (39%) assessed expectorated sputum, 10 (20%) parameters related to disease exacerbation, 8 (16%) oxygenation, 8 (16%) patient-reported outcomes, 5 (10%) exercise capacity and 5 (10%) applied imaging techniques. Results and conclusionsĭata are summarized according to the type of outcome measure. After screening, 49 articles remained for further analysis. Studies were independently selected by the investigators according to the eligibility criteria.

Search terms included chronic obstructive lung diseases and ACTs. Data sourcesĪ systematic literature search was performed in PubMed, Web of Science and EMBASE databases. This review discusses all outcome measures applied in previous research in the pediatric population to provide a baseline for future studies. One of the reasons for the limited evidence is the lack of appropriate outcome measures specifically related to the effectiveness of ACTs. Unfortunately, a sound evidence base is lacking and airway clearance strategies are largely based on clinical expertise. Airway clearance techniques (ACTs) are an important aspect of the treatment of children with chronic obstructive lung diseases.

Cystic fibrosis lung sounds