Standards for the care of people with cystic fibrosis; establishing and maintaining health.

This is the second in a series of four papers updating the European Cystic Fibrosis Society (ECFS) standards for the care of people with CF. This paper focuses on establishing and maintaining health. The guidance is produced using an evidence-based framework and with wide stakeholder engagement, including people from the CF community. Authors provided a narrative description of their topic and statements, which were more directive. These statements were reviewed by a Delphi exercise, achieving good levels of agreement from a wide group for all statements. This guidance reinforces the importance of a multi-disciplinary CF team, but also describes developing models of care including virtual consultations. The framework for health is reinforced, including the need for a physically active lifestyle and the strict avoidance of all recreational inhalations, including e-cigarettes. Progress with cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapy is reviewed, including emerging adverse events and advice for dose reduction and interruption. This paper contains guidance that is pertinent to all people with CF regardless of age and eligibility for and access to modulator therapy.

Airway clearance physiotherapy and health-related quality of life in cystic fibrosis.

OBJECTIVE: Airway clearance physiotherapy is recommended in cystic fibrosis, but limited evidence exists to suggest how much treatment is enough. As a secondary analysis of a prior study investigating the safety, efficacy, and participants' perceptions of a novel airway clearance technique, specific cough technique (SCT) compared to forced expiration technique (FET), we aimed to evaluate whether the intervention was associated with changes in health-related quality of life (HRQoL). METHODS: We conducted randomised, controlled individual trials with six adults (N-of-1 RCTs). Each trial included eight weeks of treatment, twice a week, using saline inhalation in horizontal positions, one with SCT and one with FET, in random order. Efficacy was measured by sputum wet weight (g) after each session. Perceived usefulness and preference were self-reported at the end of the study. Lung function was assessed at baseline and at the end of study. HRQoL was measured using the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at baseline (week 1) and at completion of the study (week 8). Individual HRQoL scores (0-100) were coded and analysed using CFQ-R Software Program, version 2.0. RESULTS: Patient-reported outcomes were completed by all subjects. Individual CFQ-R-Respiratory Symptoms Scores (CFQ-R-RSS) showed a positive change, meeting the minimal important difference (MID) ≥ 4 points in five participants and a negative change in one individual. A strong correlation (r = 0.94 (p<0.01) was found between total sputum weight (g) and the positive changes in CFQ-R-RSS, and between changes in lung function and CFQ-R-RSS (r = 0.84 (p = 0.04). CONCLUSION: The airway clearance intervention was associated with clinically meaningful changes in patient-reported symptoms on the CFQ-R in the majority of the participants. This finding warrants further investigation regarding treatment, duration and frequency. A long-term study may reveal beneficial effects on other clinically meaningful endpoints, such as pulmonary exacerbations, high-resolution computed tomography scores and HRQoL. TRIAL REGISTRATION: The study was registered in ClinicalTrials.gov, under the number NCT0 1266473.

Evaluation of a novel technique in airway clearance therapy - Specific Cough Technique (SCT) in cystic fibrosis: A pilot study of a series of N-of-1 randomised controlled trials.

OBJECTIVES: The aim of this pilot study was to evaluate the efficacy, safety and participants' perception of a novel technique in airway clearance therapy - specific cough technique in cystic fibrosis. METHODS: We conducted randomised controlled individual trials (N-of-1 randomised controlled trials) in six adults. Each trial included 8 weeks of treatment with two interventions each week, one with specific cough technique and one with forced expiration technique. The efficacy was investigated by a blinded assessor measuring wet weight of sputum (g) after each session. Perceived usefulness and preference was self-reported at the end of study. Additional measurements included oxygen saturation and heart rate before and after each session and lung function (week 2). RESULTS: Three of six participants produced significantly higher mean sputum weight when using specific cough technique, differences being 21%, 38% and 23%, respectively. In three of the six participants, mean sputum weight was lower after forced expiration technique than after specific cough technique in each of the eight treatment pairs. Participant-reported outcomes were completed in all participants. Specific cough technique was reported to be easier to use in daily treatments and more normalising in everyday life. CONCLUSION: Specific cough technique was well tolerated and accepted by the participants with cystic fibrosis. Specific cough technique was non-inferior to forced expiration technique in terms of sputum production, thus specific cough technique appears to represent a promising alternative for clearing sputum in airway clearance therapy.