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.

Randomised cross-over trial evaluating the short-term effects of non-invasive ventilation as an adjunct to airway clearance techniques in adults with cystic fibrosis.

Introduction: Non-invasive ventilation (NIV) is used in cystic fibrosis (CF) to support airway clearance techniques (ACTs) by augmenting tidal volumes and reducing patient effort. However, the evidence base for this is limited. We hypothesised that NIV, in addition to usual ACT, would increase sputum clearance. In addition, we investigated ease of sputum clearance (EoC), work of breathing (WoB) and NIV tolerability. Methods: Adults with CF (16+ years) at the end of hospitalisation for a pulmonary exacerbation were randomised to a cross-over trial of NIV-supported ACT or ACT alone in two consecutive days. No other changes to standard care were made. The primary outcome was the total 24-hour expectorated sputum wet weight after the intervention. Spirometry was completed pre-treatment and post-treatment. Oxygen saturations were measured pre-treatment, during treatment and post-treatment. EoC and WoB were assessed using Visual Analogue Scale. Results: 14 subjects completed the study (7 male, mean age 35 [SD 17] years, mean forced expiratory volume in 1 s [FEV1] 49 [20] % predicted). The difference between treatment regimens was -0.98 g sputum (95% CI -11.5 to 9.6, p=0.84) over 24 hours. During treatment oxygen saturations were significantly higher with NIV-supported ACT (mean difference 2.0, 95% CI 0.9 to 2.6, p=0.0004). No other significant differences were found in post-treatment FEV1, EoC, WoB, oxygen saturations or subject preference. Conclusions: There was no difference in treatment effect between NIV-supported ACT and ACT alone, although the study was underpowered. Oxygen saturations were significantly higher during NIV-supported ACT, but with no effect on post-treatment saturations. NIV was well tolerated. Trial registration number: NCT01885650.

Optimising inhaled mannitol for cystic fibrosis in an adult population.

ABSTRACT: There has been remarkable progress in the treatment of cystic fibrosis (CF) patients over the past 20 years. However, limitations of standard therapies have highlighted the need for a convenient alternative treatment to effectively target the pathophysiologic basis of CF-related disease by improving mucociliary clearance of airway secretions and consequently improve lung function and reduce respiratory exacerbations. Mannitol is an osmotic agent available as a dry powder, dispensed in a convenient disposable inhaler device for the treatment of adult patients with CF. Inhalation of mannitol as a dry powder is thought to change the viscoelastic properties of airway secretions, increase the hydration of the airway surface liquid and contribute to increased mucociliary and cough clearance of retained secretions. In two large phase 3 studies [1, 2], long-term use of inhaled mannitol resulted in a significant and clinically meaningful improvement in lung function relative to control in adult CF subjects and had an acceptable safety profile. Clinical experience with inhaled mannitol confirms that it is safe and effective. A minority of patients are unable to tolerate the medication. However, through training in proper inhaler technique and setting clear expectations regarding therapeutic effects, both the tolerance and adherence necessary for long term efficacy can be positively influenced. EDUCATIONAL AIMS: To discuss the importance of airway clearance treatments in the management of cystic fibrosis.To describe the clinical data that supports the use of mannitol in adult patients with cystic fibrosis.To highlight the role of mannitol tolerance testing in screening for hyperresponsiveness.To provide practical considerations for patient education in use of mannitol inhaler. KEY POINTS: Inhaled mannitol is a safe and effective option in adult patients with cystic fibrosis.Mannitol tolerance testing effectively screens for hyperresponsiveness prior to initiation of therapy.Physiotherapists and respiratory therapists play an integral role in the introduction and maintenance of dry powder inhalation therapy.Patient training and follow-up is important for optimising longer term adherence.

Inhaled therapy in cystic fibrosis: agents, devices and regimens.

KEY POINTS: There have been significant advances in both inhalation medicines and delivery devices with "intelligent nebulisers" and "dry-powder inhalers" becoming commonplace in CF care.Inhaled medicines generate high levels of a drug within the airways with limited systemic effects, offering safe and convenient antibiotic and mucolytic therapy for individuals with CF.Variations in adherence are not unique to CF; however, treatment burden is high and therefore fast inhaled drug delivery devices may assist individuals in completing the prescribed treatment regimes.Prescribers of inhaled medicines have a responsibility to consider, in addition to efficacy, the appropriated drug/device combination for each individual in order to promote adherence and achieve the desired clinical benefit. SUMMARY: The recognised mainstay daily treatments for cystic fibrosis (CF) focus on inhaled and oral medications, airway clearance and optimised nutrition. This review discusses recent advances in inhaled therapies for the management of CF, including devices such as intelligent nebulisers, drug formulations and supporting evidence for inhaled antibiotics (for the management of chronic Pseudomonas aeruginosa) and muco-active drugs. We include practical advice for clinicians regarding the optimisation of inhalation technique and education. The influence of adherence on the use of inhaled therapies in CF is also reviewed. EDUCATIONAL AIMS: To inform readers about the history and progression of inhaled therapies for people with CF with reference to the literature supporting current practice.To highlight the factors that may impact the success of inhaled therapies, including those which are device specific such as drug deposition and those which influence adherence.

Positive pressure--analysing the effect of the addition of non-invasive ventilation (NIV) to home airway clearance techniques (ACT) in adult cystic fibrosis (CF) patients.

BACKGROUND: There is no published literature on the frequency of use of non-invasive ventilation (NIV) with airway clearance techniques (ACT) throughout the cystic fibrosis (CF) population; 3.9% (191 people of 5062 registered) of the United Kingdom CF population older than 16 years are reported to use NIV in registry data; however, it is not specified if this is for ACT or respiratory failure. Using NIV with ACT decreases work of breathing and fatigue during in-patient admissions for CF patients. We hypothesised these effects could be replicated at home, potentially reducing hospital admissions. METHOD: Fourteen adult patients with CF scored ease of clearance and breathlessness with ACT before and after addition of NIV to normal ACT routine using a visual analog scale. Patient views on NIV with ACT were collected via a structured interview. Number of home intravenous (IV) antibiotic courses and days in hospital was collected for one year pre- and post-NIV provision. RESULTS: Patients reported statistically significant improvements in ease of clearance (p = 0.011) and reduced breathlessness during ACT using NIV (p = 0.011). Structured interview results indicated patient reports of sputum clearance improved. In-patient days were lower, while home IV days were higher after NIV was set up, although not statistically significant. CONCLUSION: This study is limited by small numbers; however, trends towards less hospital admissions and greater patient ease while using NIV with ACT warrant further investigation.

Cystic fibrosis-Children and adults Tai Chi study (CF CATS2): Can Tai Chi improve symptoms and quality of life for people with cystic fibrosis? Second phase study protocol.

BACKGROUND: Cystic fibrosis (CF) is a genetic disorder affecting respiratory and digestive systems. People with CF experience physical symptoms; cough, poor lung ventilation, recurrent infections, poor weight gain, diarrhoea, and malnutrition, as well as lower quality of life. Tai Chi, a Chinese form of meditative movement, may help with the symptoms of CF and help people with CF to exercise. However, there is very little research in this area. OBJECTIVE: To evaluate the feasibility of studying Tai Chi for CF and to compare the effectiveness of Tai Chi to standard care and face-to-face Tai Chi with online Tai Chi for people with CF. METHODS: This is a comparative effectiveness trial with 72 people with CF over 6 years old and a patient at the Royal Brompton Hospital, London, UK. Participants receive 8 Tai Chi sessions, then home practice with a DVD. Sessions are one-on-one for group A, online for group B. Group B is a no treatment standard care control (first 3 months). At baseline, 3, 6 and 9 months, questionnaires will be used to measure quality of life, mindfulness and sleep, and medical data health and respiratory function. At Tai Chi sessions and at 4 follow-up points, the Borg Scale and healthcare use data will be collected. At 9 months online focus groups will assess participants' experience, Tai Chi feasibility, perceived health impact, and study participation. Recruitment will use adverts in hospital clinics and website, and letter/phone for non-regularly attending patients. Block randomisation will use random number tables. The two groups will be compared for: weeks 1 to 12 (Tai Chi vs. standard care); before and after intervention (differences in delivery method); week 1 (of intervention) to month 9 (long-term impact). Qualitative data will use Framework analysis. DISCUSSION: We believe this is the first trial of Tai Chi for CF. Tai Chi may help with the physiological symptoms of CF and increase levels of exercise by providing a self-management technique and low stress activity. This study will provide data on the feasibility of a randomized controlled trial of Tai Chi for CF, including data for a sample size calculation and will inform future study design.

Chest physiotherapy techniques in bronchiectasis.

Bronchiectasis is a persistent and progressive condition characterized by inflammation and infection causing damage that potentiates impaired mucociliary clearance. The rationale for promoting airway clearance is so that purulent secretions are removed from the airways, ameliorating the inflammation and improving control of symptoms such as cough and sputum plugging. Physiotherapists aim to teach patients one of a variety of airway clearance techniques to regularly perform as part of their daily management.

Evaluation of the Target Inhalation Mode (TIM) breathing maneuver in simulated nebulizer therapy in patients with cystic fibrosis.

BACKGROUND: Adaptive Aerosol Delivery (AAD) systems provide efficient drug delivery and improved lung deposition over conventional nebulizers by combining real-time analyses of patient breathing patterns and precisely timed aerosol delivery. Delivery and deposition are further enhanced by breathing techniques involving slow, deep inhalations. METHODS: This exploratory study assessed the acceptability of slow, deep inhalations in 20 patients with cystic fibrosis (CF) during up to eight simulated nebulizer treatments with the I-neb AAD System. The breathing maneuver, Target Inhalation Mode (TIM) breathing, involved the lengthening of the patient's inhalation time over successive breaths with guidance from auditory and tactile (vibratory) feedback from the device. RESULTS: At the end of the first treatment, most patients felt that the instructions were easy to understand (90%) and that the vibratory feedback was pleasant (65%). Half of the patients found the procedure to be comfortable. At the end of the final treatment, most patients felt that the breathing maneuver was easy to understand (90%) and use (80%), but that the duration of the breath was too long (100%). Logged data revealed that 90% of patients were able to comply with the breathing maneuver. The two patients unable to comply had a forced vital capacity of <1.75 L. The average treatment time decreased from 288.4 to 141.6 sec during the first and final treatments, respectively. CONCLUSIONS: This study provides preliminary evidence of the acceptability of the TIM breathing maneuver in patients with CF and their ability to perform repeated TIM breathing during simulated nebulizer therapy with the I-neb AAD System.

Predicting survival in end-stage cystic fibrosis.

The natural history of cystic fibrosis (CF) is unpredictable and the optimal timing for lung transplantation in end-stage disease uncertain. Predicting survival based on FEV1 alone remains controversial and therefore the aim of this study was to assess the value of walk test performance in pre-transplant assessment. Retrospective review of adult patients with end-stage CF who underwent transplant assessment between 1988 and 2004 including a documented walk test on room air, but who died before transplant. The six-minute walk test (6MWT) was used between 1988 and 1993 and the shuttle walk test (SWT) thereafter, the two cohorts were therefore individually assessed. A total of 121 patients were identified. The median (IQR) survival in patients performing SWT (n=77) and 6MWT (n=44) was 363 days (226, 566) and 433 days (232, 844), respectively, with survival in both cohorts significantly associated with pre-test (resting) heart rate (HR) (p<0.03), but not distance walked, pre-test SpO2, FEV1 or BMI. It was predicted that 85% of patients performing SWT with a resting HR of 120 bpm, 70% of those with a HR of 109 bpm (cohort median) but only 25% with a HR of 72 bpm would die within 500 days. Distance walked in the SWT was significantly related to pre-test HR (p<0.01), SpO2 (p<0.01) and Borg score (p=0.016) when performing linear regression. Only pre-test HR remained significant when performing multiple regression. Resting heart rate was the only consistent parameter in this study at predicting a high risk of dying on the transplant waiting list.