Role of the Physical Therapist in Cystic Fibrosis Care.

In looking back on 2020 and 2021, this Perspective reflects on the monumental impacts of the rollout of cystic fibrosis (CF) transmembrane conductance regulator highly effective modulator therapies and the COVID-19 pandemic on the management of CF. Advancements in the clinical management of people with CF have been both enormous and rapid, and physical therapists specializing in the care of people with CF have been at the forefront of driving this evolution in care. This year sees the 30th anniversary of the UK Association of Chartered Physiotherapists in Cystic Fibrosis and, as is inevitable in reaching such milestones, thoughts have turned to origins, role, impacts, and the future. With the changing demographics of the population of people with CF after the introduction of highly effective modulator therapies, potentially with fewer secondary complications, the future role of the physical therapist who specializes in CF is in question. This Perspective reflects on and highlights the role of physical therapy within CF and provides insights into how physical therapists and respiratory therapists can evolve their roles to ensure relevance for the future.

Airway clearance and exercise for people with cystic fibrosis: Balancing longevity with life.

Airway clearance has been an integral part of cystic fibrosis (CF) care for almost as long as CF has been identified as a condition. From diagnosis as a neonate through to end-of-life care, airway clearance is an everyday aspect of life, adding a considerable treatment burden to the lives of people with CF. There are many different techniques used for airway clearance which have evolved over time with an aim to improve effectiveness, support adherence and, more recently, to consider the impact of burden. A popular thought is whether airway clearance could be replaced by exercise. With new precision therapies in the form of CFTR modulators available, the CF landscape is rapidly changing, raising the question of whether certain treatments are needed at all. Depending on factors such as CFTR mutation, age, and pre-existing lung damage before starting a CFTR modulator, individuals with CF may need different levels of intensity and type of maintenance treatment. Precision medicine is likely to lead to the need for increased precision and individualized management around other maintenance therapies such as airway clearance.

Using a learning health system to understand the mismatch between medicines supply and actual medicines use among adults with cystic fibrosis.

BACKGROUND: Studies in separate cohorts suggest possible discrepancies between inhaled medicines supplied (median 50-60%) and medicines used (median 30-40%). We performed the first study that directly compares CF medicine supply against use to identify the cost of excess medicines supply. METHODS: This cross-sectional study included participants from 12 UK adult centres with ≥1 year of continuous adherence data from data-logging nebulisers. Medicine supply was measured as medication possession ratio (MPR) for a 1-year period from the first suitable supply date. Medicine use was measured as electronic data capture (EDC) adherence over the same period. The cost of excess medicines was calculated as whole excess box(es) supplied after accounting for the discrepancy between EDC adherence and MPR with 20% contingency. RESULTS: Among 275 participants, 133 (48.4%) were females and mean age was 30 years (95% CI 29-31 years). Median EDC adherence was 57% (IQR 23-86%), median MPR was 74% (IQR 46-96%) and the discrepancy between measures was median 14% (IQR 2-29%). Even with 20% contingency, mean potential cost of excess medicines was £1,124 (95% CI £855-1,394), ranging from £183 (95% CI £29-338) for EDC adherence ≥80% to £2,017 (95% CI £1,507-2,526) for EDC adherence <50%. CONCLUSIONS: This study provides a conservative estimate of excess inhaled medicines supply cost among adults with CF in the UK. The excess supply cost was highest among those with lowest EDC adherence, highlighting the importance of adherence support and supplying medicine according to actual use. MPR provides information about medicine supply but over-estimates actual medicine use.

Optimising the care and quality of life of people with cystic fibrosis: the influence of cystic fibrosis transmembrane conductance regulator modulators.

Cystic fibrosis is a life-limiting, inherited, multi-organ disease which affects many systems of the body. Until recently, treatments were only able to ameliorate symptoms, but the introduction of precision medications which modulate the underlying defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene has changed this. Notably improvements in nutrition and lung function, reduced use of antibiotics and reduced occupation rates for hospital beds have been seen. This article summarises the discussion of a group of healthcare professionals from different specialties and an expert patient, representing their personal views and experience of treating patients who are using CFTR modulators. The discussion was sponsored by an unrestricted grant from Chiesi Limited (Manchester, UK).

What effective ways of motivation, support and technologies help people with cystic fibrosis improve and sustain adherence to treatment?

INTRODUCTION: "What effective ways of motivation, support and technologies help people with cystic fibrosis improve and sustain adherence to treatment?" was identified as one of the James Lind Alliance Priority Setting Partnership's top 10 research questions in cystic fibrosis (CF). Using electronic questionnaires, we aimed to gain a deeper understanding of this research priority. METHOD: The work was led by the steering group representative of the UK CF community consisting of patients, carers and healthcare professionals (HCPs). Electronic questionnaires were completed over a 4-week period and promoted via online forums such as Twitter, the UK CF Trust and US CF Foundation websites and via professional networks. Analysis of the closed questions was completed using Microsoft Excel, with keyword analysis and the final thematic analysis completed using NVivo software. RESULTS: There were 313 respondents; 176/313 (56%) were from people with CF and their families. HCPs comprised of 10 professional groups accounting for 137/313 (44%) of respondents, with global involvement of participants with the majority from the UK. Common themes identified as impacting on adherence included: having no time, treatment burden, competing life demands, fatigue and the patient's general health. Having a routine was identified as the most frequently used motivational strategy, valued by both the patient and professional community. However, some strategies were valued more by HCPs than used in practice by patients; these included the use of short-term goal setting and technology use. CONCLUSION: Adherence to treatment is crucial, however it is often suboptimal and strategies valued by HCPs to promote adherence are not always shared by patients. To promote adherence clinicians and researchers should be mindful that in a condition where treatment burden and time pressures are considerable, any interventions should focus on simplifying care and reducing treatment burden.

Characterising burden of treatment in cystic fibrosis to identify priority areas for clinical trials.

In a recent James Lind Alliance Priority Setting Partnership in cystic fibrosis (CF) the top priority clinical research question was: "What are effective ways of simplifying the treatment burden of people with CF?" We aimed to summarise the lived experience of treatment burden and suggest research themes aimed at reducing it. An online questionnaire was co-produced and responses subjected to quantitative and thematic analysis. 941 survey responses were received (641 from lay community). People with CF reported a median of 10 (interquartile range: 6-15) current treatments. Seven main themes relating to simplifying treatment burden were identified. Treatment burden is high, extending beyond time taken to perform routine daily treatments, with impact varying according to person-specific factors. Approaches to communication, support, evaluation of current treatments, service set-up, and treatment logistics (obtaining/administration) contribute to burden, offering scope for evaluation in clinical trials or service improvement.

The top 10 research priorities in cystic fibrosis developed by a partnership between people with CF and healthcare providers.

There remain many treatment uncertainties in cystic fibrosis (CF). With limited resources, research should focus on questions which are most important to the CF community. We conducted a James Lind Alliance Priority Setting Partnership in CF. Research questions were elicited and then prioritised in successive surveys. A workshop agreed the final top 10. Online methods avoided cross infection and widened participation. The elicitation survey had 482 respondents (1080 questions) and prioritisation survey 677 respondents. Participants were drawn equally from the patient and clinical communities globally. We have achieved a consensus on 10 research priorities which will be attractive to funders.

Nebuliser systems for drug delivery in cystic fibrosis.

BACKGROUND: Nebuliser systems are used to deliver medications to control the symptoms and the progression of lung disease in people with cystic fibrosis. Many types of nebuliser systems are available for use with various medications; however, there has been no previous systematic review which has evaluated these systems. OBJECTIVES: To evaluate effectiveness, safety, burden of treatment and adherence to nebulised therapy using different nebuliser systems for people with cystic fibrosis. SEARCH METHODS: We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register comprising references identified from comprehensive electronic database searches, handsearching of relevant journals and abstract books of conference proceedings. We searched the reference lists of each study for additional publications and approached the manufacturers of both nebuliser systems and nebulised medications for published and unpublished data. Date of the most recent search: 15 Oct 2012. SELECTION CRITERIA: Randomised controlled trials or quasi-randomised controlled trials comparing nebuliser systems including conventional nebulisers, vibrating mesh technology systems, adaptive aerosol delivery systems and ultrasonic nebuliser systems. DATA COLLECTION AND ANALYSIS: Two authors independently assessed studies for inclusion. They also independently extracted data and assessed the risk of bias. A third author assessed studies where agreement could not be reached. MAIN RESULTS: The search identified 40 studies with 20 of these (1936 participants) included in the review. These studies compared the delivery of tobramycin, colistin, dornase alfa, hypertonic sodium chloride and other solutions through the different nebuliser systems. This review demonstrates variability in the delivery of medication depending on the nebuliser system used. Conventional nebuliser systems providing higher flows, higher respirable fractions and smaller particles decrease treatment time, increase deposition and may be preferred by people with CF, as compared to conventional nebuliser systems providing lower flows, lower respirable fractions and larger particles. Nebulisers using adaptive aerosol delivery or vibrating mesh technology reduce treatment time to a far greater extent. Deposition (as a percentage of priming dose) is greater than conventional with adaptive aerosol delivery. Vibrating mesh technology systems may give greater deposition than conventional when measuring sputum levels, but lower deposition when measuring serum levels or using gamma scintigraphy. The available data indicate that these newer systems are safe when used with an appropriate priming dose, which may be different to the priming dose used for conventional systems. There is an indication that adherence is maintained or improved with systems which use these newer technologies, but also that some nebuliser systems using vibrating mesh technology may be subject to increased failures. AUTHORS' CONCLUSIONS: Clinicians should be aware of the variability in the performance of different nebuliser systems. Technologies such as adaptive aerosol delivery and vibrating mesh technology have advantages over conventional systems in terms of treatment time, deposition as a percentage of priming dose, patient preference and adherence. There is a need for long-term randomised controlled trials of these technologies to determine patient-focused outcomes (such as quality of life and burden of care), safe and effective dosing levels of medications and clinical outcomes (such as hospitalisations and need for antibiotics) and an economic evaluation of their use.

Accurate assessment of adherence: self-report and clinician report vs electronic monitoring of nebulizers.

BACKGROUND: People with cystic fibrosis have a high treatment burden. While uncertainty remains about individual patient level of adherence to medication, treatment regimens are difficult to tailor, and interventions are difficult to evaluate. Self- and clinician-reported measures are routinely used despite criticism that they overestimate adherence. This study assessed agreement between rates of adherence to prescribed nebulizer treatments when measured by self-report, clinician report, and electronic monitoring suitable for long-term use. METHODS: Seventy-eight adults with cystic fibrosis were questioned about their adherence to prescribed nebulizer treatments over the previous 3 months. Self-report was compared with clinician report and stored adherence data downloaded from the I-Neb nebulizer system. Adherence measures were expressed as a percentage of the prescribed regimen, bias was estimated by the paired difference in mean (95% CI) patient and clinician reported and actual adherence. Agreement between adherence measures was calculated using intraclass correlation coefficients (95% CI), and disagreements for individuals were displayed using Bland-Altman plots. RESULTS: Patient-identified prescriptions matched the medical record prescription. Median self-reported adherence was 80% (interquartile range, 60%-95%), whereas median adherence measured by nebulizer download was 36% (interquartile range, 5%-84.5%). Nine participants overmedicated and underreported adherence. Median clinician report ranged from 50% to 60%, depending on profession. Extensive discrepancies between self-report and clinician report compared with nebulizer download were identified for individuals. CONCLUSIONS: Self- and clinician-reporting of adherence does not provide accurate measurement of adherence when compared with electronic monitoring. Using inaccurate measures has implications for treatment burden, clinician prescribing practices, cost, and accuracy of trial data.

Physiotherapeutic management strategies for the treatment of cystic fibrosis in adults.

Physiotherapy has long been considered a cornerstone of condition management for people with cystic fibrosis (CF). The presentation of CF has changed over time with an increased life expectancy and increased expectations of people with CF to have a complete lifestyle. In turn, the scope of strategies used in physiotherapy for CF have also changed dramatically over the years, moving away from routine postural drainage and manual techniques toward an individualized regimen including the choice of many different forms of airway clearance, such as both independent and assisted, exercise, treatments to promote continence and good posture, inhalation therapy, oxygen, and noninvasive ventilation. This article describes the techniques and overall strategies used by physiotherapists in helping people with CF to manage the symptoms and progression of their condition.

Contextual assessment of couples therapy: the Clinical Discourse Q-Sets.

This article describes a method for creating highly contextual assessments of couples therapy interaction using concepts from Systemic Functional Linguistics (SFL). According to SFL, talk in interaction serves three primary functions: to mediate social relations, to negotiate representations of reality, and to organize and structure the event as coherent. These concepts are operationalized using observational q-methodology. The Clinical Discourse Q-Sets (CDQS) include three separate q-sets for use by trained observers in rating 12-min segments of couples therapy conversation. Each q-set captures one aspect of the communication process as defined by Systemic Functional theory. Good-to-high reliabilities for the q-set profiles over various (n = 16) couples therapy episodes were found. Preliminary evidence for the conceptual structure and clinical validity of the system was found.