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BackgroundPhysical activity levels are known to decline following hospitalisation for people with cystic fibrosis (pwCF). However, optimal physical activity promotion strategies are unclear. This study investigated the effect of a web-based application (ActivOnline) in promoting physical activity in young pwCF.MethodsMulticentre randomised controlled trial with assessor blinding and qualitative evaluation. People with CF (12–35 years) admitted to hospital for a respiratory cause were eligible and randomised to the 12-week ActivOnline intervention (AO) or usual care (UC). The primary outcome was change in device-based time spent in moderate-to-vigorous physical activity (MVPA) from baseline to post-intervention. Follow-up was at 6 months from hospital discharge when qualitative evaluation was undertaken.Results107 participants were randomised to AO (n=52) or UC (n=55). Sixty-three participants (59%) contributed to the intention-to-treat analysis. Mean (SD) age was 21 (6) years (n=46, ConclusionA web-based application, including individualised goal setting, real-time feedback and motivation for behavioural change, was no better than usual care at promoting physical activity in young pwCF following hospital discharge. High levels of baseline physical activity levels in both groups, and limited engagement with the intervention, suggest alternative strategies may be necessary to identify and support young pwCF who would benefit from enhanced physical activity.Trial registration numberACTRN12617001009303, 13 July 13 2017.

Rationale: The prognostic value of cardiopulmonary exercise testing (CPET) for survival in cystic fibrosis (CF) in the context of current clinical management, when controlling for other known prognostic factors, is unclear. Objectives: To determine the prognostic value of CPET-derived measures beyond peak oxygen uptake (VO 2 peak) following rigorous adjustment for other predictors. Methods: Data from 10 CF centers in Australia, Europe, and North America were collected retrospectively. A total of 510 patients completed a cycle CPET between January 2000 and December 2007, of which 433 fulfilled the criteria for a maximal effort. Time to death/lung transplantation was analyzed using Cox proportional hazards regression. In addition, phenotyping using hierarchical Ward clustering was performed to characterize high-risk subgroups. Measurements and Main Results: Cox regression showed, even after adjustment for sex, FEV1% predicted, body mass index (z-score), age at CPET, Pseudomonas aeruginosa status, and CF-related diabetes as covariates in the model, that VO 2 peak in % predicted (hazard ratio [HR], 0.964; 95% confidence interval [CI], 0.944-0.986), peak work rate (% predicted; HR, 0.969; 95% CI, 0.951-0.988), ventilatory equivalent for oxygen (HR, 1.085; 95% CI, 1.041-1.132), and carbon dioxide (HR, 1.060; 95% CI, 1.007-1.115) (all P < 0.05) were significant predictors of death or lung transplantation at 10-year follow-up. Phenotyping revealed that CPET-derived measures were important for clustering. We identified a high-risk cluster characterized by poor lung function, nutritional status, and exercise capacity. Conclusions: CPET provides additional prognostic information to established predictors of death/lung transplantation in CF. High-risk patients may especially benefit from regular monitoring of exercise capacity and exercise counseling.

Spirometry is usually performed under the supervision of a trained respiratory scientist to ensure acceptability and repeatability of results. To evaluate the quality of spirometry performance by adult cystic fibrosis (CF) patients with and without observation by a trained respiratory scientist, an observational, single centre study was conducted between February to December 2020. 74 adults were recruited and instructed to perform spirometry without supervision within 24 h of their remote CF clinic consultation. Spirometry was repeated at their consultation, supervised by a respiratory scientist using video conferencing. The majority of patients achieved grade A (excellent) or B (very good) spirometry quality with (95%) and without supervision (93%) independent of lung function severity. Similarly, forced expiratory volume in 1 second demonstrated no significant differences with paired spirometry performed within a 24 hour period. For a large proportion of adult CF patients, unsupervised portable spirometry produces acceptable and repeatable results.

Several agents are used to clear secretions from the airways of people with cystic fibrosis. Mannitol increases mucociliary clearance, but its exact mechanism of action is unknown. The dry powder formulation of mannitol may be more convenient and easier to use compared with established agents which require delivery via a nebuliser. Phase III trials of inhaled dry powder mannitol for the treatment of cystic fibrosis have been completed and it is now available in Australia and some countries in Europe. This is an update of a previous review.To assess whether inhaled dry powder mannitol is well tolerated, whether it improves the quality of life and respiratory function in people with cystic fibrosis and which adverse events are associated with the treatment.We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register which comprises references identified from comprehensive electronic databases, handsearching relevant journals and abstracts from conferences. Date of last search: 12 December 2019.All randomised controlled studies comparing mannitol with placebo, active inhaled comparators (for example, hypertonic saline or dornase alfa) or with no treatment.Authors independently assessed studies for inclusion, carried out data extraction and assessed the risk of bias in included studies. The quality of the evidence was assessed using GRADE.Six studies (reported in 36 unique publications) were included with a total of 784 participants. Duration of treatment in the included studies ranged from 12 days to six months, with open-label treatment for an additional six months in two of the studies. Five studies compared mannitol with control (a very low dose of mannitol or non-respirable mannitol) and the final study compared mannitol to dornase alfa alone and to mannitol plus dornase alfa. Two large studies had a similar parallel design and provided data for 600 participants, which could be pooled where data for a particular outcome and time point were available. The remaining studies had much smaller sample sizes (ranging from 22 to 95) and data could not be pooled due to differences in design, interventions and population. Pooled evidence from the two large parallel studies was judged to be of low to moderate quality and from the smaller studies was judged to be of low to very low quality. In all studies, there was an initial test to see if participants tolerated mannitol, with only those who could tolerate the drug being randomised; therefore, the study results are not applicable to the cystic fibrosis population as a whole. While the published papers did not provide all the data required for our analysis, additional unpublished data were provided by the drug's manufacturer and the author of one of the studies. Pooling the large parallel studies comparing mannitol to control, up to and including six months, lung function (forced expiratory volume at one second) measured in both mL and % predicted was significantly improved in the mannitol group compared to the control group (moderate-quality evidence). Beneficial results were observed in these studies in adults and in both concomitant dornase alfa users and non-users in these studies. In the smaller studies, statistically significant improvements in lung function were also observed in the mannitol groups compared to the non-respirable mannitol groups; however, we judged this evidence to be of low to very low quality. For the comparisons of mannitol and control, we found no consistent differences in health-related quality of life in any of the domains except for burden of treatment, which was less for mannitol up to four months in the two pooled studies of a similar design; this difference was not maintained at six months. It should be noted that the tool used to measure health-related quality of life was not designed to assess mucolytics and pooling of the age-appropriate tools (as done in some of the included studies) may not be valid so results were judged to be low to very low quality and should be interpreted with caution. Cough, haemoptysis, bronchospasm, pharyngolaryngeal pain and post-tussive vomiting were the most commonly reported side effects in both treatment groups. Where rates of adverse events could be compared, statistically no significant differences were found between mannitol and control groups; although some of these events may have clinical relevance for people with CF. For the comparisons of mannitol to dornase alfa alone and to mannitol plus dornase alfa, very low-quality evidence from a 12-week cross-over study of 28 participants showed no statistically significant differences in the recorded domains of health-related quality of life or measures of lung function. Cough was the most common side effect in the mannitol alone arm but there was no occurrence of cough in the dornase alfa alone arm and the most commonly reported reason of withdrawal from the mannitol plus dornase alfa arm was pulmonary exacerbations. In terms of secondary outcomes of the review (pulmonary exacerbations, hospitalisations, symptoms, sputum microbiology), evidence provided by the included studies was more limited. For all comparisons, no consistent statistically significant and clinically meaningful differences were observed between mannitol and control treatments (including dornase alfa).There is moderate-quality evidence to show that treatment with mannitol over a six-month period is associated with an improvement in some measures of lung function in people with cystic fibrosis compared to control. There is low to very low-quality evidence suggesting no difference in quality of life for participants taking mannitol compared to control. This review provides very low-quality evidence suggesting no difference in lung function or quality of life comparing mannitol to dornase alfa alone and to mannitol plus dornase alfa. The clinical implications from this review suggest that mannitol could be considered as a treatment in cystic fibrosis; but further research is required in order to establish who may benefit most and whether this benefit is sustained in the longer term. Furthermore, studies comparing its efficacy against other (established) mucolytic therapies need to be undertaken before it can be considered for mainstream practice.پیشینه: عوامل دارویی متعددی برای پاک کردن ترشحات از مجاری هوایی مبتلایان به فیبروز سیستیک استفاده می‌شوند. مانیتول پاکسازی موکوسیلیاری را افزایش می‌دهد، اما مکانیسم دقیق عملکرد آن ناشناخته است. فرمولاسیون پودر خشک مانیتول در مقایسه با عوامل تائید شده شده که توسط نبولایزر تجویز می‌شوند، ممکن است برای استفاده، راحت‌تر و آسان‌تر باشد. کارآزمایی‌های فاز III پودر خشک استنشاقی مانیتول برای درمان فیبروز سیستیک به پایان رسیده و هم اکنون در استرالیا و برخی از کشورهای اروپایی در دسترس قرار دارد. این یک به‌روزرسانی از یک مرور کاکرین است که قبلا منتشر شده است. اهداف: ارزیابی اینکه آیا پودر خشک استنشاقی مانیتول به خوبی تحمل می‌شود، آیا کیفیت زندگی و عملکرد تنفسی را در افراد مبتلا به فیبروز سیستیک بهبود می‌بخشد یا اینکه چه عوارض جانبی با درمان همراه است. روش‌های جست‌وجو: ما پایگاه ثبت کارآزمایی‌های گروه فیبروز کیستیک و اختلالات ژنتیکی در کاکرین را جست‌وجو کردیم که شامل منابع شناسایی‌شده از طریق جست‌وجوهای جامع در پایگاه‌های اطلاعاتی الکترونیکی، جست‌وجوهای دستی در مجلات مربوطه، کتاب‌های چکیده و مجموعه مقالات کنفرانس‌ها است. تاریخ آخرین جست‌وجو: 12 دسامبر 2019. معیارهای انتخاب: تمام مطالعات تصادفی‌سازی و کنترل شده که به مقایسه مانیتول با دارونما (placebo)، مقایسه کننده‌های استنشاقی فعال (به عنوان مثال، سالین هیپرتونیک یا دورناز آلفا) یا عدم درمان پرداختند. گردآوری و تجزیه‌وتحلیل داده‌ها: نویسندگان به‌طور مستقل از هم مطالعات را برای ورود ارزیابی کردند، داده‌‌ها را استخراج کرده و خطر سوگیری (bias) را در مطالعات وارد شده ارزیابی کردند. کیفیت شواهد با استفاده از GRADE ارزیابی شد. نتایج اصلی: شش مطالعه (گزارش شده در 36 مقاله تکی) با 784 شرکت‌کننده وارد شدند. مدت زمان درمان در مطالعات وارد شده، از 12 روز تا شش ماه متغیر بود، با درمان برچسب باز به مدت شش ماه بیشتر در دو مطالعه. پنج مطالعه مانیتول را با کنترل (دوز بسیار کمی از مانیتول یا مانیتول غیرقابل تنفس) مقایسه کرده و مطالعه نهایی به مقایسه مانیتول با دورناز آلفا به تنهایی و مانیتول به اضافه دورناز آلفا پرداخت. دو مطالعه بزرگ از یک طراحی موازی مشابه برخوردار بوده و داده‌ها را برای 600 شرکت‌کننده ارائه کردند، که می‌توانست در جایی که داده‌ها برای یک پیامد و نقطه زمانی خاص در دسترس بودند، تجمیع شوند. مطالعات باقیمانده حجم نمونه‌های بسیار کمتری داشتند (از 22 تا 95) و به دلیل تفاوت‌ها در طراحی مطالعه، مداخلات و جمعیت، داده‌ها قابل تجمیع نبودند. شواهد تجمعی از دو مطالعه موازی بزرگ، كیفیت پایین تا متوسطی داشته و شواهد به دست آمده از مطالعات كوچكتر به نظر می‌رسید كه از كیفیت پائین تا بسیار پائینی برخوردار بودند. در تمام مطالعات، آزمایش اولیه‌ای وجود داشت تا مشخص شود که شرکت‌کنندگان قادر به تحمل مانیتول بودند یا خیر، فقط کسانی که می‌توانستند دارو را تحمل کنند، تصادفی‌سازی شدند؛بنابراین، نتایج مطالعه برای کل جمعیت مبتلا به فیبروز سیستیک کاربرد ندارند. در حالی که مقالات منتشر شده تمام داده‌های مورد نیاز را برای تجزیه‌و‌تحلیل ما ارائه نمی‌دهند، داده‌های منتشر نشده اضافی توسط سازنده دارو و نویسنده یکی از مطالعات ارائه شد. تجمیع مطالعات موازی بزرگ که به مقایسه مانیتول با کنترل، تا شش ماه و شامل 6 ماه پرداختند، نشان دادند که عملکرد ریه (حجم اجباری بازدمی در یک ثانیه)، اندازه‌گیری شده به هر دو صورت میلی‌لیتر و درصد پیش‌بینی شده، در گروه مانیتول نسبت به گروه کنترل به‌طور قابل توجهی بهبود یافت (شواهد با کیفیت متوسط). در این مطالعات نتایج مفیدی در بزرگسالان و در هر دو گروه مصرف کننده همزمان دورناز آلفا و کسانی که از آن استفاده نکردند، مشاهده شد. در مطالعات کوچکتر نیز پیشرفت‌های معنی‌داری از نظر آماری در عملکرد ریه در گروه مانیتول در مقایسه با گروه‌های مانیتول غیرقابل تنفس مشاهده شد. با این حال، این شواهد از کیفیت پایین تا بسیار پایینی برخوردار بودند. برای مقایسه‌های مانیتول و کنترل، ما تفاوت‌های پایداری را در کیفیت زندگی مرتبط با سلامت در هیچ یک از حوزه‌ها به جز بار (burden) درمان پیدا نکردیم، که برای مانیتول تا چهار ماه، در دو مطالعه تجمیع شده با طراحی مشابه، کمتر بود؛ این اختلاف در شش ماه باقی نماند. لازم به ذکر است که ابزار مورد استفاده برای سنجش کیفیت زندگی مرتبط با سلامت، که برای ارزیابی موکولیتیک‌ها و ادغام ابزارهای مناسب سن (همانطور که در برخی از مطالعات وارد شده انجام شد) طراحی نشده بود، ممکن است معتبر نباشد، بنابراین نتایج با کیفیت پایین تا بسیار پایین داوری شدند و باید با احتیاط تفسیر شوند. سرفه، هموپتیزی، برونکواسپاسم، درد فارنگولارنژیال و استفراغ پس از سرفه، شایع‌ترین عوارض جانبی در هر دو گروه درمانی بودند. در جایی که میزان عوارض جانبی با هم قابل مقایسه بودند، از نظر آماری تفاوت معنی‌داری بین مانیتول و گروه کنترل مشاهده نشد؛ اگرچه برخی از این حوادث ممکن است ارتباط بالینی برای افراد مبتلا به CF داشته باشند. برای مقایسه‌های مانیتول با دورناز آلفا به تنهایی و مانیتول به علاوه دورناز آلفا، شواهدی با کیفیت بسیار پایین از یک مطالعه متقاطع 12 هفته‌ای با 28 شرکت‌کننده به دست آمد که هیچ تفاوت آماری معنی‌داری را در حوزه‌های ثبت شده کیفیت زندگی مرتبط با سلامت یا معیارهای عملکرد ریه نشان ندادند. سرفه شایع‌ترین عارضه جانبی در بازوی مانیتول به تنهایی بود اما هیچ موردی از وقوع سرفه در بازوی دورناز آلفا به تنهایی وجود نداشت و شایع‌ترین دلیل خروج از بازوی مانیتول به علاوه دورناز آلفا، تشدید حملات ریوی گزارش شد. از نظر پیامدهای ثانویه مرور (تشدید حملات ریوی، بستری در بیمارستان، علائم، میکروبیولوژی خلط)، شواهد ارائه شده توسط مطالعات وارد شده، محدودتر بودند. برای همه مقایسه‌ها، هیچ تفاوتی معنی‌دار آماری پایداری و تفاوت بالینی قابل‌توجهی بین درمان‌های مانیتول و کنترل (از جمله دورناز آلفا) مشاهده نشد. نتیجه‌گیری‌های نویسندگان: شواهدی با کیفیت متوسط وجود دارد که نشان می‌دهد درمان با مانیتول در طول یک دوره شش ماهه، با بهبود برخی معیارهای عملکرد ریه در افراد مبتلا به فیبروز سیستیک در مقایسه با کنترل همراه است. شواهدی با کیفیت پایین تا بسیار پایین وجود دارد که نشان می‌دهد هیچ تفاوتی در کیفیت زندگی شرکت‌کنندگان تحت درمان با مانیتول در مقایسه با کنترل، وجود ندارد. این مرور شواهدی را با کیفیت بسیار پائین نشان می‌دهد که هیچ تفاوتی در عملکرد ریه یا کیفیت زندگی در مقایسه مانیتول با دورناز آلفا به تنهایی و با مانیتول به همراه دورناز آلفا وجود ندارد. کاربردهای بالینی این مرور پیشنهاد می‌کند که مانیتول می‌تواند به عنوان درمانی در فیبروز سیستیک در نظر گرفته شود؛ اما تحقیقات بیشتری لازم است تا مشخص شود چه کسی بیشترین نفع را از آن می‌برد و اینکه این سود در طولانی‌مدت پایدار است یا خیر. علاوه بر این، پیش از آنکه مانیتول در طبابت بالینی به کار گرفته شود، مطالعات مربوط به مقایسه اثربخشی آن در برابر سایر روش‌های درمانی موکولیتیک (اثبات شده) باید انجام شوند.

Cystic fibrosis (CF) lung disease is characterised by reduced mucus clearance, resulting in retention of thick mucus, creating a nidus for chronic infection and progressive lung damage. A cornerstone of the medical management of CF lung disease is daily airway clearance therapy (ACT), tailored to the individual, such as active cycle of breathing technique (ACBT), autogenic drainage, positive expiratory pressure (PEP) and oscillating PEP devices, postural drainage, percussion and high-frequency chest wall oscillations.1 There is widespread agreement among people with CF and healthcare professionals that exercise may also improve airway clearance2 and many people with CF already use exercise as a substitute for ACT.3 Whether exercise is a satisfactory substitute for traditional ACTs in people with CF has been identified as a top 10 research priority,4 yet none of the many systematic reviews on the various ACTs have answered this question, as in the five systematic reviews5 only one study was eligible for inclusion, and the systematic review on exercise training did not specifically address airway clearance.6 People with CF are encouraged to regularly exercise,1 due to the known benefits on exercise capacity and potential improvements in lung function and quality of life.6 If exercise could adequately replace traditional ACTs for people with CF, it may also reduce …

Background and objective: No published studies have examined the long-term effects of non-invasive ventilation (NIV) in cystic fibrosis (CF). Our primary aim was to determine if adults with CF and sleep desaturation were less likely to develop hypercapnia with NIV ± O compared to low-flow oxygen therapy (LFO) or meet the criteria for failure of therapy over 12 months. We studied event-free survival, hospitalizations, lung function, arterial blood gases (ABG), sleep quality and health-related quality of life. Methods: A prospective, randomized, parallel group study in adult patients with CF and sleep desaturation was conducted, comparing 12 months of NIV ± O to LFO. Event-free survival was defined as participants without events. Events included: failure of therapy with PaCO > 60 mm Hg, or increase in PaCO > 10 mm Hg from baseline, increases in TcCO > 10 mm Hg, lung transplantation or death. Outcomes were measured at baseline, 3, 6 and 12 months, including lung function, ABG, Pittsburgh Sleep Quality Inventory (PSQI), SF36 and hospitalizations. Results: A total of 29 patients were randomized to NIV ± O (n = 14) or LFO (n = 15) therapy for 12 months. Of the 29 patients, 18 met the criteria for event-free survival over 12 months. NIV ± O group had 33% (95% CI: 5–58%) and 46% (95% CI: 10–68%) more event-free survival at 3 and 12 months than LFO group. No statistically significant differences were seen in spirometry, ABG, questionnaires or hospitalizations. Conclusion: NIV ± O during sleep increases event-free survival over 12 months in adults with CF. Further studies are required to determine which subgroups benefit the most from NIV.

Objectives Conduct an elicitation study, using the Theory of Planned Behaviour framework, to identify salient beliefs about exercise participation in adults with cystic fibrosis (CF). Specifically, identify attitudes on advantages and disadvantages of exercise (behavioural beliefs); individuals and groups who apply social pressure to exercise (normative beliefs); and perceived control over facilitators and barriers to exercise (control beliefs) for adults with CF. Design Qualitative interviews using open-ended, structured questions. Setting Adult CF clinic in a large Australian hospital. Participants Sixteen adults with CF, three relatives/friends of adults with CF and six CF clinic staff. Results The most common positive attitudes about exercise were to keep fit and healthy (68%) and feel better and happier (60%), and negative attitude was to feel breathless (36%). Social pressure to exercise mainly came from parents/family (72%) and friends (52%), and 60% of participants reported that no-one discourages exercise. Having someone to exercise with (44%) and be encouraged (36%) were the most common facilitators of perceived control to exercise, whereas being unwell (96%) and not having sufficient time (56%) were the most common barriers. Conclusions Attitudes, social pressure and perceived control to exercise for adults with CF were similar to beliefs previously reported by the general population and some patient groups. A number of CF-specific exercise beliefs, mainly related to pulmonary function, were also reported. These findings can help develop questionnaires for larger groups of adults with CF, interpret relationships between exercise beliefs and participation, and inform clinicians to target interventions to increase exercise participation.

Background: Cystic Fibrosis Related Diabetes (CFRD) is a significant complication of Cystic Fibrosis (CF) with prevalence increasing with age. Regular physical activity (PA) is important for people with CF conferring improved clinical and health outcomes. To date, the relationship between levels of PA and the development and management of CFRD is limited in CF. Aim: To describe PA levels in adults with CF, with and without CFRD. Methods: Retrospective review of data collected at 3 tertiary CF centres in Australia. Adults with CF ≥17 years, in stable health, contributed data for this analysis. CFRD status (CFRD; Impaired glucose tolerance (GT); Indeterminate GT; and normal GT) were classified according to standard guidelines. Adherence to PA guidelines was classified as reporting ≥150mins of at least moderate PA/week with PA frequency ≥5. Results: 154 adults with CF (n=85 male) mean (SD) age 28 (9) years and FEV1 64 (22) %predicted provided self-report of PA. Thirty-nine individuals (n=23 male; FEV1 56 (21)%predicted) had CFRD; n=9 impaired GT; n=4 indeterminate GT, and n=102 normal GT. 61 individuals (40%) reported undertaking PA of sufficient frequency and duration to meet PA guidelines. Only n=12 (31%) individuals with CFRD self-reported PA sufficient to meet guidelines. FEV1 was not different between those with CFRD who did and did not report PA to meet guidelines (p=0.9) Conclusions: A minority of individuals with CF met guidelines for PA. Only a third with CFRD reported sufficient PA to meet guidelines. Further analysis of the impact of PA on the development of glucose intolerance (impaired GT and CFRD) may provide empirical evidence for providing advice on PA.

Cystic fibrosis transmembrane conductance regulator (CFTR) is expressed in human skeletal muscle cells. Variations of CFTR dysfunction among patients with cystic fibrosis may be an important determinant of maximal exercise capacity in cystic fibrosis. Previous studies on the relationship between CFTR genotype and maximal exercise capacity are scarce and contradictory. This study was designed to explore factors influencing maximal exercise capacity, expressed as peak oxygen uptake (V.O2peak), with a specific focus on CFTR genotype in children and adults with cystic fibrosis. In an international, multicenter, cross-sectional study, we collected data on CFTR genotype and cardiopulmonary exercise tests in patients with cystic fibrosis who were ages 8 years and older. CFTR mutations were classified into functional classes I–V. The final analysis included 726 patients (45% females; age range, 8–61 yr; forced expiratory volume in 1 s, 16 to 123% predicted) from 17 cystic fibrosis centers in North America, Europe, Australia, and Asia, all of whom had both valid maximal cardiopulmonary exercise tests and complete CFTR genotype data. Overall, patients exhibited exercise intolerance (V.O2peak, 77.3 ± 19.1% predicted), but values were comparable among different CFTR classes. We did not detect an association between CFTR genotype functional classes I–III and either V.O2peak (percent predicted) (adjusted β = −0.95; 95% CI, −4.18 to 2.29; P = 0.57) or maximum work rate (Wattmax) (adjusted β = −1.38; 95% CI, −5.04 to 2.27; P = 0.46) compared with classes IV–V. Those with at least one copy of a F508del-CFTR mutation and one copy of a class V mutation had a significantly lower V.O2peak (β = −8.24%; 95% CI, −14.53 to −2.99; P = 0.003) and lower Wattmax (adjusted β = −7.59%; 95% CI, −14.21 to −0.95; P = 0.025) than those with two copies of a class II mutation. On the basis of linear regression analysis adjusted for relevant confounders, lung function and body mass index were associated with V.O2peak. CFTR functional genotype class was not associated with maximal exercise capacity in patients with cystic fibrosis overall, but those with at least one copy of a F508del-CFTR mutation and a single class V mutation had lower maximal exercise capacity.

Question: During an acute exacerbation of cystic fibrosis, is non-invasive ventilation beneficial as an adjunct to the airway clearance regimen? Design: Randomised controlled trial with concealed allocation and intention-to-treat analysis. Participants: Forty adults with moderate to severe cystic fibrosis lung disease and who were admitted to hospital for an acute exacerbation. Intervention: Comprehensive inpatient care (control group) compared to the same care with the addition of non-invasive ventilation during airway clearance treatments from Day 2 of admission until discharge (experimental group). Outcome measures: Lung function and subjective symptom severity were measured daily. Fatigue was measured at admission and discharge on the Schwartz Fatigue Scale from 7 (no fatigue) to 63 (worst fatigue) points. Quality of life and exercise capacity were also measured at admission and discharge. Length of admission and time to next hospital admission were recorded. Results: Analysed as the primary outcome, the experimental group had a greater rate of improvement in forced expiratory volume in 1 second (FEV 1 ) than the control group, but this was not statistically significant (MD 0.13% predicted per day, 95% CI –0.03 to 0.28). However, the experimental group had a significantly higher FEV 1 at discharge than the control group (MD 4.2% predicted, 95% CI 0.1 to 8.3). The experimental group reported significantly lower levels of fatigue on the Schwartz fatigue scale at discharge than the control group (MD 6 points, 95% CI 1 to 11). There was no significant difference between the experimental and control groups in subjective symptom severity, quality of life, exercise capacity, length of hospital admission or time to next hospital admission. Conclusion: Among people hospitalised for an acute exacerbation of cystic fibrosis, the use of non-invasive ventilation as an adjunct to the airway clearance regimen significantly improves FEV 1 and fatigue. Trial registration: ANZCTR 12605000437662. [Dwyer TJ, Robbins L, Kelly P, Piper AJ, Bell SC, Bye PTP (2015) Non-invasive ventilation used as an adjunct to airway clearance treatments improves lung function during an acute exacerbation of cystic fibrosis: a randomised trial. Journal of Physiotherapy 61: 142–147]

This chapter briefly describes the airway surface liquid (ASL) and the critical role of its hydration status and reviews the pathophysiology initiated by insufficient hydration of ASL. It then presents detailed evidence about medications intended to restore ASL and their effectiveness in people with CF. Inhaled osmotic agents are one class of ASL-augmenting medications. Nebulized hypertonic saline significantly improves lung function, reduces exacerbations, and improves the quality of life of people with CF. Dry powder mannitol also significantly improves lung function and reduces the incidence of exacerbations in this population, but it does not significantly improve quality of life. Ion transport modifiers are another class of inhaled medications that augment the ASL. These medications address the underlying ion transport defects by either inhibiting sodium absorption (e.g., amiloride) or by stimulating chloride secretion (e.g., denufosol). Despite promising preliminary research, ion transport modifiers have not demonstrated clinical benefits to date. In summary, osmotic agents, such as hypertonic saline and dry powder mannitol, have clinical worthwhile benefits in people with CF and are currently used in clinical practice.

Exercise improves mucus clearance in people without lung disease and those with chronic bronchitis. No study has investigated exercise alone for mucus clearance in cystic fibrosis (CF). The aim of this study was to compare the effects of treadmill exercise to resting breathing and airway clearance with positive expiratory pressure (PEP) therapy on mucus clearance in adults with CF.This 3-day randomised, controlled, crossover trial included 14 adults with mild to severe CF lung disease (forced expiratory volume in 1 s % predicted 31–113%). Interventions were 20 min of resting breathing (control), treadmill exercise at 60% of the participant's peak oxygen consumption or PEP therapy (including huffing and coughing). Mucus clearance was measured using the radioaerosol technique and gamma camera imaging.Treadmill exercise improved whole lung mucus clearance compared to resting breathing (mean difference 3%, 95% CI 2–4); however, exercise alone was less effective than PEP therapy (mean difference −7%, 95% CI −6– −8). When comparing treadmill exercise to PEP therapy, there were no significant differences in mucus clearance from the intermediate and peripheral lung regions, but significantly less clearance from the central lung region (likely reflecting the huffing and coughing that was only in PEP therapy).It is recommended that huffing and coughing are included to maximise mucus clearance with exercise.

SummaryObjectiveDetermine the SenseWear Pro3 Armband (SWA) accuracy for estimating energy expenditure (EE) and step count during treadmill walking in cystic fibrosis (CF) compared to healthy adults.HypothesisSWA estimation of EE would be less accurate for CF, than for healthy subjects, due to interference with the SWA skin sensors caused by the high salt concentration in the sweat of CF subjects.Methods17 CF (mean age 26yr; FEV1 54% predicted) and 17 age-matched control subjects walked slightly faster than their comfortable pace on a treadmill for 20min, whilst simultaneously wearing the SWA and breathing through an open-circuit indirect calorimetry (IC) system. Subjects' steps were manually counted.ResultsCombined EE data from all subjects showed no significant difference in EE measured by IC (6.0±3.4kcalmin−1) compared to the SWA estimate (6.3±2.5kcalmin−1), however the SWA significantly overestimated EE at low exercise intensities and underestimated EE at high exercise intensities. Correlations between EE values, estimated by the SWA and measured by IC, were greater than 0.85 (p

Sarah J. Nolan *, Judith Thornton , Clare S. Murray , Tiffany Dwyer 4 Department of Biostatistics, The University of Liverpool, Liverpool, UK Centre for Clinical Practice, National Institute for Health and Care Excellence, Manchester, UK Centre for Respiratory Medicine and Allergy, Institute of Inflammation and Repair, University of Manchester and University Hospital of South Manchester, Manchester, UK 4 Sydney Medical School, University of Sydney, Sydney, Australia

Physiotherapy management is a key element of care for people with cystic fibrosis (CF) throughout the lifespan. Although considerable evidence exists to support physiotherapy management of CF, there is documented variation in practice. The aim of this guideline is to optimize the physiotherapy management of people with CF in Australia and New Zealand. A systematic review of the literature in key areas of physiotherapy practice for CF was undertaken. Recommendations were formulated based on National Health and Medical Research Council (Australia) guidelines and considered the quality, quantity and level of the evidence; the consistency of the body of evidence; the likely clinical impact; and applicability to physiotherapy practice in Australia and New Zealand. A total of 30 recommendations were made for airway clearance therapy, inhalation therapy, exercise assessment and training, musculoskeletal management, management of urinary incontinence, managing the newly diagnosed patient with CF, delivery of non‐invasive ventilation, and physiotherapy management before and after lung transplantation. These recommendations can be used to underpin the provision of evidence‐based physiotherapy care to people with CF in Australia and New Zealand.

Objective: The effect of muscle length on strength and dexterity after stroke was investigated. The aim was to determine if poor function at a particular muscle length could be attributed solely to differential weakness at this joint angle or whether an additional problem of differential dexterity exists. Design: This descriptive research study measured elbow flexor and extensor strength as well as dexterity at three elbow joint angles: 30°, 60° and 90° flexion. Dexterity was measured independently of strength. Subjects: Fifteen (seven female, eight male) chronic stroke patients (mean age 67 years) who could actively flex and extend their affected elbow participated. Ten neurologically normal control subjects (mean age 67 years) acted as controls. Main outcome measures: Strength was measured as peak elbow flexor and extensor torque at three angles; and dexterity was measured as coherence for slow and fast tracking also at three angles. Results: Dexterity was not affected by muscle length but strength was and this finding was the same for both stroke and controls. While the magnitude of the torque-angle curves was not significantly different between stroke and controls, the shape of torque-angle curves was altered after stroke so that both the elbow flexors ( p < 0.05) and extensors ( p < 0.05) tested weaker in the testing position where they were shortest. Conclusion: Since there was no differential loss of dexterity, it appears that differential loss of strength, especially in the shortened range, may explain the clinical observation of poorer function at one muscle length than another after stroke. Specific training to strengthen the muscles in these ranges is therefore of clinical importance for rehabilitation.

Purpose of review Consensus statements about the care of people with cystic fibrosis (CF) recommend exercise as part of a wider management strategy. Many of these recommendations are based on high-quality evidence that regular exercise improves some important clinical outcomes, such as lung function and quality of life. However, the evidence about the effect of exercise on other clinical outcomes is less extensive or lower in quality. This article will review the physiological effects of exercise on a range of outcomes in people with CF, the mechanisms by which exercise may improve these outcomes and the quality and findings of clinical research into the effects of exercise in the management of CF. Recent findings Substantial evidence confirms that exercise significantly reduces the rate of decline in lung function in people with CF, at least in part by increasing mucus clearance. Regular exercise training over 6 months improves aerobic exercise capacity. Bone health is often poor in people with CF, but only indirect evidence supports that increasing the amount of exercise will have a beneficial effect on bone density. CF-related diabetes is also a common sequela of the disease, but again only evidence from type-2 diabetes exists to support exercise as a way of managing it. Summary Although its effects on some outcomes are unclear, the overall effect of exercise on quality of life is substantially beneficial and the evidence available for other specific outcomes is directly or indirectly supportive, so it appears appropriate to recommend it in clinical practice.

Background Treadmill exercise and airway clearance with the Flutter® device have previously been shown to improve mucus clearance mechanisms in people with cystic fibrosis (CF) but have not been compared. It is therefore not known if treadmill exercise is an adequate form of airway clearance that could replace established airway clearance techniques, such as the Flutter®. The aim of this study was to evaluate respiratory flow, sputum properties and subjective responses of treadmill exercise and Flutter® therapy, compared to resting breathing (control). Methods Twenty-four adults with mild to severe CF lung disease (FEV1 28–86% predicted) completed a three-day randomised, controlled, cross-over study. Interventions consisted of 20 min of resting breathing (control), treadmill exercise at 60% of the participant’s peak oxygen consumption and Flutter® therapy. Respiratory flow was measured during the interventions. Sputum properties (solids content and mechanical impedance) and subjective responses (ease of expectoration and sense of chest congestion) were measured before, immediately after the interventions and after 20 min of recovery. Results Treadmill exercise and Flutter® resulted in similar significant increases in peak expiratory flow, but only Flutter® created an expiratory airflow bias (i.e. peak expiratory flow was at least 10% higher than peak inspiratory flow). Treadmill exercise and Flutter® therapy resulted in similar significant reductions in sputum mechanical impedance, but only treadmill exercise caused a transient increase in sputum hydration. Treadmill exercise improved ease of expectoration and Flutter® therapy improved subjective sense of chest congestion. Conclusions A single bout of treadmill exercise and Flutter® therapy were equally effective in augmenting mucus clearance mechanisms in adults with CF. Only longer term studies, however, will determine if exercise alone is an adequate form of airway clearance therapy that could replace other airway clearance techniques. Trial registration Australian and New Zealand Clinical Trials Registry, Registration number #ACTRN12609000168257, Retrospectively registered (Date submitted to registry 26/2/2009, First participant enrolled 27/2/2009, Date registered 6/4/2009). Electronic supplementary material The online version of this article (doi:10.1186/s12890-016-0360-8) contains supplementary material, which is available to authorized users.