Making the invisible visible: the availability and desirability of adherence data in routine CF care- findings from a national questionnaire survey.
ABSTRACT: Background: Inhaled medications for cystic fibrosis (CF) are effective but adherence is low. Clinicians find it difficult to estimate how much treatment people with CF (PWCF) take, whilst objective adherence measurement demonstrates that patients are poorly calibrated with a tendency to over-estimate actual adherence. The diagnostic approach to a PWCF with deteriorating clinical status and very low adherence is likely to be different to the approach to a deteriorating patient with optimal adherence. Access to objective adherence data in routine consultations could help to overcome diagnostic challenges for clinicians and people with CF. Attitudes of clinicians to the use and importance of routinely available adherence data is unknown. Methods: We conducted an online questionnaire survey with UK CF centres. We asked five questions relating to the current use and perception of objective measurements of adherence in routine care. Results: A total of eight CF centres completed the questionnaire. Few of the responding centres have adherence data readily available in routine clinics (13% of centres use medicines possession ratio; of centres with access to I-nebs® it was estimated that 17% of patients had I-neb data regularly available in clinics). All centres considered the availability of objectively measured adherence data to be important. Respondents identified that systems developed to provide adherence data in clinical practice must provide data to both clinicians and patients that is readily understood and easy to use. Conclusions: Centres perceived the availability of adherence data in routine care to be important but objective measures of adherence is rarely available at present.
Project description:OBJECTIVES:To undertake a process evaluation of an adherence support intervention for people with cystic fibrosis (PWCF), to assess its feasibility and acceptability. SETTING:Two UK cystic fibrosis (CF) units. PARTICIPANTS:Fourteen adult PWCF; three professionals delivering adherence support ('interventionists'); five multi-disciplinary CF team members. INTERVENTIONS:Nebuliser with data recording and transfer capability, linked to a software platform, and strategies to support adherence to nebulised treatments facilitated by interventionists over 5 months (± 1 month). PRIMARY AND SECONDARY MEASURES:Feasibility and acceptability of the intervention, assessed through semistructured interviews, questionnaires, fidelity assessments and click analytics. RESULTS:Interventionists were complimentary about the intervention and training. Key barriers to intervention feasibility and acceptability were identified. Interventionists had difficulty finding clinic space and time in normal working hours to conduct review visits. As a result, fewer than expected intervention visits were conducted and interviews indicated this may explain low adherence in some intervention arm participants. Adherence levels appeared to be >100% for some patients, due to inaccurate prescription data, particularly in patients with complex treatment regimens. Flatlines in adherence data at the start of the study were linked to device connectivity problems. Content and delivery quality fidelity were 100% and 60%-92%, respectively, indicating that interventionists needed to focus more on intervention 'active ingredients' during sessions. CONCLUSIONS:The process evaluation led to 14 key changes to intervention procedures to overcome barriers to intervention success. With the identified changes, it is feasible and acceptable to support medication adherence with this intervention. TRIAL REGISTRATION NUMBER:ISRCTN13076797; Results.
Project description:<h4>Background</h4>Preventative medication reduces hospitalisations in people with cystic fibrosis (PWCF) but adherence is poor. We assessed the feasibility of a randomised controlled trial of a complex intervention, which combines display of real time adherence data and behaviour change techniques.<h4>Methods</h4>Design: Pilot, open-label, parallel-group RCT with concurrent semi-structured interviews.<h4>Participants</h4>PWCF at two Cystic Fibrosis (CF) units. Eligible: aged 16 or older; on the CF registry. Ineligible: post-lung transplant or on the active list; unable to consent; using dry powder inhalers.<h4>Interventions</h4>Central randomisation on a 1:1 allocation to: (1) intervention, linking nebuliser use with data recording and transfer capability to a software platform, and behavioural strategies to support self-management delivered by trained interventionists (n?=?32); or, (2) control, typically face-to-face meetings every 3?months with CF team (n?=?32).<h4>Outcomes</h4>RCT feasibility defined as: recruitment of ??48 participants (75% of target) in four months (pilot primary outcome); valid exacerbation data available for ??85% of those randomised (future RCT primary outcome); change in % medication adherence; FEV<sub>1</sub> percent predicted (key secondaries in future RCT); and perceptions of trial procedures, in semi-structured interviews with intervention (n?=?14) and control (n?=?5) participants, interventionists (n?=?3) and CF team members (n?=?5).<h4>Results</h4>The pilot trial recruited to target, randomising 33 to intervention and 31 to control in the four-month period, June-September 2016. At study completion (30th April 2017), 60 (94%; Intervention?=?32, Control =28) participants contributed good quality exacerbation data (intervention: 35 exacerbations; control: 25 exacerbation). The mean change in adherence and baseline-adjusted FEV<sub>1</sub> percent predicted were higher in the intervention arm by 10% (95% CI: -5.2 to 25.2) and 5% (95% CI -2 to 12%) respectively. Five serious adverse events occurred, none related to the intervention. The mean change in adherence was 10% (95% CI: -5.2 to 25.2), greater in the intervention arm. Interventionists delivered insufficient numbers of review sessions due to concentration on participant recruitment. This left interventionists insufficient time for key intervention procedures. A total of 10 key changes that were made to RCT procedures are summarised.<h4>Conclusions</h4>With improved research processes and lower monthly participant recruitment targets, a full-scale trial is feasible.<h4>Trial registration</h4>ISRCTN13076797 . Prospectively registered on 07/06/2016.
Project description:<h4>Objectives</h4>Chest CT is increasingly used to monitor disease progression in children with cystic fibrosis (CF) but there is no national guideline regarding its use. Our objective was to assess the indications for undertaking chest CT and the protocols used to obtain scans.<h4>Design setting and participants</h4>An electronic questionnaire was developed to assess clinicians views on chest CT in children with CF. It included general questions on perceived benefits and specific questions about its role in five clinical scenarios. It was sent to the clinical lead in 27 UK paediatric CF centres. A separate questionnaire was developed to collect the technical details of chest CT in children with CF. It was sent to the superintendent radiographer at each of the 27 centres.<h4>Results</h4>Responses were obtained from 27 (100%) clinical leads and 22 (81%) superintendent radiographers. 93% clinicians reported chest CT useful in monitoring disease progression and 70% said it frequently altered management. Only 5 (19%) undertook routine scans. To aid diagnosis, 81% performed chest CT in non-tuberculous mycobacterial disease and 15% in allergic bronchopulmonary aspergillosis. There was wide variation in the perceived need for and/or timing of chest CT in children with reduced lung function with no benefit from intravenous antibiotics, new cystic changes on chest X-ray, and lobar collapse. The radiographers reported using a mixture of helical (volumetric) and axial scans depending on the clinical question, the age and the cooperation of the child. When indicated, 6 (27%) used sedation and 16 (73%) general anaesthetic. Only 1 (5%) used intravenous contrast routinely and 3 (14%) obtained expiratory images routinely.<h4>Conclusions</h4>There is marked variation in the use of chest CT in children with CF and in the scan protocols. The lack of a national guideline is likely to be contributing to this lack of standardisation.
Project description:<h4>Background</h4>Cystic fibrosis (CF) is a life-limiting genetic condition in which daily therapies to maintain lung health are critical, yet treatment adherence is low. Previous interventions to increase adherence have been largely unsuccessful and this is likely due to a lack of focus on behavioural evidence and theory alongside input from people with CF. This intervention is based on a digital platform that collects and displays objective nebuliser adherence data. The purpose of this paper is to identify the specific components of an intervention to increase and maintain adherence to nebuliser treatments in adults with CF with a focus on reducing effort and treatment burden.<h4>Methods</h4>Intervention development was informed by the Behaviour Change Wheel (BCW) and person-based approach (PBA). A multidisciplinary team conducted qualitative research to inform a needs analysis, selected, and refined intervention components and methods of delivery, mapped adherence-related barriers and facilitators, associated intervention functions and behaviour change techniques, and utilised iterative feedback to develop and refine content and processes.<h4>Results</h4>Results indicated that people with CF need to understand their treatment, be able to monitor adherence, have treatment goals and feedback and confidence in their ability to adhere, have a treatment plan to develop habits for treatment, and be able to solve problems around treatment adherence. Behaviour change techniques were selected to address each of these needs and were incorporated into the digital intervention developed iteratively, alongside a manual and training for health professionals. Feedback from people with CF and clinicians helped to refine the intervention which could be tailored to individual patient needs.<h4>Conclusions</h4>The intervention development process is underpinned by a strong theoretical framework and evidence base and was developed by a multidisciplinary team with a range of skills and expertise integrated with substantial input from patients and clinicians. This multifaceted development strategy has ensured that the intervention is usable and acceptable to people with CF and clinicians, providing the best chance of success in supporting people with CF with different needs to increase and maintain their adherence. The intervention is being tested in a randomised controlled trial across 19 UK sites.
Project description:Identification of mechanisms promoting neutrophil trafficking to the lungs of patients with cystic fibrosis (CF) is a challenge for next generation therapeutics. Cholesterol, a structural component of neutrophil plasma membranes influences cell adhesion, a key step in transmigration. The effect of chronic inflammation on neutrophil membrane cholesterol content in patients with CF (PWCF) remains unclear. To address this we examined neutrophils of PWCF to evaluate the cause and consequence of altered membrane cholesterol and identified the effects of lung transplantation and ion channel potentiator therapy on the cellular mechanisms responsible for perturbed membrane cholesterol and increased cell adhesion.PWCF homozygous for the ?F508 mutation or heterozygous for the G551D mutation were recruited (n=48). Membrane protein expression was investigated by mass spectrometry. The effect of lung transplantation or ivacaftor therapy was assessed by ELISAs, and calcium fluorometric and ?-calpain assays.Membranes of CF neutrophils contain less cholesterol, yet increased integrin CD11b expression, and respond to inflammatory induced endoplasmic reticulum (ER) stress by activating ?-calpain. In vivo and in vitro, increased ?-calpain activity resulted in proteolysis of the membrane cholesterol trafficking protein caveolin-1. The critical role of caveolin-1 for adequate membrane cholesterol content was confirmed in caveolin-1 knock-out mice. Lung transplant therapy or treatment of PWCF with ivacaftor, reduced levels of circulating inflammatory mediators and actuated increased caveolin-1 and membrane cholesterol, with concurrent normalized neutrophil adhesion.Results demonstrate an auxiliary benefit of lung transplant and potentiator therapy, evident by a reduction in circulating inflammation and controlled neutrophil adhesion.
Project description:Background:The benefits of physical activity (PA) for people with cystic fibrosis (pwCF) are widely accepted, yet how PA is promoted and utilised by pwCF is unclear. Method:An online questionnaire to explore attitudes, practices and promotion of PA in cystic fibrosis was completed by healthcare providers (HCP), pwCF and parents/caregivers. Results:351 respondents (105 HCP, 120 pwCF, and 126 parents/caregivers) from 12 countries completed the survey. Importance of PA was rated highly by the majority of respondents. Physical (e.g. health), psychological (e.g. enjoyment) and social (e.g. social interaction) factors were motives for PA for 82%, 49% and 37% of pwCF, respectively, irrespective of country. Common barriers to PA included time (49% and 36%) and tiredness (61% and 7%) for pwCF and parents/carers, respectively. pwCF also reported psychosocial barriers (e.g. stigma, demoralisation), while parents/caregivers reported structural barriers (e.g. cost). Clinical teams varied substantially in terms of the emphasis placed on PA, facilities available, staff and training, and advice given to pwCF. Conclusion:Despite the majority of participants rating the importance of PA highly, substantial variability was evident regarding the facilities and clinical support available to them, as well as why and how people were active. There remains a need to identify what constitutes "best practice" for PA promotion within clinics.
Project description:The cystic fibrosis (CF) community seeks to explain heterogeneous outcomes of pulmonary exacerbation (PEX) treatment. Serum and sputum inflammatory mediators may identify people with CF (PwCF) at risk for suboptimal responses. However, lack of an established association between response phenotypes and these mediators limits clinical application. In this pilot study, we prospectively characterized treatment response phenotypes by assessing health-related quality-of-life (HRQoL) during PEX. We also measured lung function and iron-related biochemical parameters in serum and sputum. We classified subjects as sustained symptom-responders (SRs) or non-sustained symptom-responders (NSRs) based on the absence or presence, respectively, of worsened symptom scores after initial improvement. We used linear mixed models (LMMs) to determine whether trends in lung function, hematologic, serum, and sputum indices of inflammation differed between response cohorts. In 20 PwCF, we identified 10 SRs and 10 NSRs with no significant differences in lung function at PEX onset and treatment durations. SRs had better model-predicted trends in lung function than NSRs during PEX. Non-linear trends in serum and sputum iron levels significantly differed between SRs and NSRs. In adults with cystic fibrosis, PEX treatment response phenotypes may be correlated with distinctive trends in serum and sputum iron concentrations.
Project description:There are controversies about the most effective treatment to eradicate first growth of Pseudomonas aeruginosa (P aeruginosa) from the lower airways of patients with cystic fibrosis (CF). UK guidelines recommend oral treatment, but some advocate intravenous (IV) treatment. The objective of this study was to assess the feasibility of conducting a randomised controlled trial comparing two treatment strategies to eradicate P aeruginosa in CF patients.Two surveys were conducted. Survey  included clinicians who were responsible for the treatment of individuals with CF, to assess their clinical practice, opinions and numbers of potentially eligible patients. Survey  included adults and young people aged 13 years or more with CF and parents of children with CF aged less than 13 years, identified at six UK CF centres, who fulfilled eligibility criteria for the proposed clinical trial, to assess their views about the interventions and their willingness to participate in the trial. Generally clinicians treat first or new growth of P aeruginosa with oral antibiotics, but 90% reported that they would consider IV treatment of first isolation of P aeruginosa. 74% of clinicians would consider recruiting their patients and 45% of consumers would consider entry for themselves or their children into a trial comparing oral with intravenous antibiotics. The median rate per annum for first or new growths of P aeruginosa in adults was 3% (range 1% to 9%) and in children was 10% (range 3% to 23%). If the trial was conducted across the UK, with a consent rate of 45%, then the number of eligible patients per annum who would be willing to take part in a study would be approximately 41 adults and 203 children.This work demonstrates the importance of feasibility studies in preparation for multicentre clinical trials. It confirmed the uncertainty amongst clinicians and patients about the clinical question, enabled assessment of the number of potentially eligible patients, the proportion of patients and clinicians prepared to participate and aspects of trial design which might encourage this. It showed that a clinical trial was feasible, but only if patients were recruited from across United Kingdom.
Project description:Adherence with tobramycin inhalation solution (TIS) during routine cystic fibrosis (CF) care may differ from recommended guidelines and affect health care utilization.We analyzed 2001-2006 healthcare claims data from 45 large employers. Study subjects had diagnoses of CF and at least 1 prescription for TIS. We measured adherence as the number of TIS therapy cycles completed during the year and categorized overall adherence as: low ≤ 2 cycles, medium >2 to <4 cycles, and high ≥ 4 cycles per year. Interquartile ranges (IQR) were created for health care utilization and logistic regression analysis of hospitalization risk was conducted by TIS adherence categories.Among 804 individuals identified with CF and a prescription for TIS, only 7% (n = 54) received ≥ 4 cycles of TIS per year. High adherence with TIS was associated with a decreased risk of hospitalization when compared to individuals receiving ≤ 2 cycles (adjusted odds ratio 0.40; 95% confidence interval 0.19-0.84). High adherence with TIS was also associated with lower outpatient service costs (IQR: $2,159-$8444 vs. $2,410-$14,423) and higher outpatient prescription drug costs (IQR: $35,125-$60,969 vs. $10,353-$46,768).Use of TIS did not reflect recommended guidelines and may impact other health care utilization.
Project description:BACKGROUND:The 2007 Guidelines for the Diagnosis and Management of Asthma provide evidence-based recommendations to improve asthma care. Limited national-level data are available about clinician agreement and adherence to these guidelines. OBJECTIVE:To assess clinician-reported adherence with specific guideline recommendations, as well as agreement with and self-efficacy to implement guidelines. METHODS:We analyzed 2012 National Asthma Survey of Physicians data for 1412 primary care clinicians and 233 asthma specialists about 4 cornerstone guideline domains: asthma control, patient education, environmental control, and pharmacologic treatment. Agreement and self-efficacy were measured using Likert scales; 2 overall indices of agreement and self-efficacy were compiled. Adherence was compared between primary care clinicians and asthma specialists. Logistic regression models assessed the association of agreement and self-efficacy indices with adherence. RESULTS:Asthma specialists expressed stronger agreement, higher self-efficacy, and greater adherence with guideline recommendations than did primary care clinicians. Adherence was low among both groups for specific core recommendations, including written asthma action plan (30.6% and 16.4%, respectively; P < .001); home peak flow monitoring, (12.8% and 11.2%; P = .34); spirometry testing (44.7% and 10.8%; P < .001); and repeated assessment of inhaler technique (39.7% and 16.8%; P < .001). Among primary care clinicians, greater self-efficacy was associated with greater adherence. For specialists, self-efficacy was associated only with increased odds of spirometry testing. Guideline agreement was generally not associated with adherence. CONCLUSIONS:Agreement with and adherence to asthma guidelines was higher for specialists than for primary care clinicians, but was low in both groups for several key recommendations. Self-efficacy was a good predictor of guideline adherence among primary care clinicians but not among specialists.