[Show abstract][Hide abstract] ABSTRACT: Studies measuring psychological distress in individuals with cystic fibrosis (CF) have found high rates of both depression and anxiety. Psychological symptoms in both individuals with CF and parent caregivers have been associated with decreased lung function, lower body mass index, worse adherence, worse health-related quality of life, more frequent hospitalisations and increased healthcare costs. To identify and treat depression and anxiety in CF, the CF Foundation and the European CF Society invited a panel of experts, including physicians, psychologists, psychiatrists, nurses, social workers, a pharmacist, parents and an individual with CF, to develop consensus recommendations for clinical care. Over 18 months, this 22-member committee was divided into four workgroups: Screening; Psychological Interventions; Pharmacological Treatments and Implementation and Future Research, and used the Population, Intervention, Comparison, Outcome methodology to develop questions for literature search and review. Searches were conducted in PubMed, PsychINFO, ScienceDirect, Google Scholar, Psychiatry online and ABDATA by a methodologist at Dartmouth. The committee reviewed 344 articles, drafted statements and set an 80% acceptance for each recommendation statement as a consensus threshold prior to an anonymous voting process. Fifteen guideline recommendation statements for screening and treatment of depression and anxiety in individuals with CF and parent caregivers were finalised by vote. As these recommendations are implemented in CF centres internationally, the process of dissemination, implementation and resource provision should be closely monitored to assess barriers and concerns, validity and use.
[Show abstract][Hide abstract] ABSTRACT: Objective:
Our objective was to quantify the effect of different statistical techniques, inclusion/exclusion criteria and missing data on the predicted median survival age.
Study design and setting:
Using the Canadian CF registry (CCFR), the median age of survival was calculated using both the Cox Proportional Hazards (PH) and the life-table methods. Through simulations, we examined how the median age of survival would change when: 1) patients were excluded, 2) death dates were inaccurate, 3) patients were lost to follow-up, 4) entire years with no clinic visits were excluded even if the patient had a visit in subsequent years, and 5) censoring patients at their date of transplant. Simulations were run assuming 5% to 35% of data were affected by each scenario.
Over the period 2009 to 2013, there were 4,666 individuals in the CCFR with 240 deaths. The observed median age of survival calculated by the Cox PH method was 50.9 (95% CI: 47.4-54.3) and 50.5 from the life-table method (95% CI: 47.5-53.5). Censoring patients at their transplant date overestimated the median age of survival by 7.2 years (58.1, 95% CI: 53.3-64.7). Simulations determined that by missing just 15% of deaths, the median age of survival can be overestimated by 3.5 years (54.4, 95% CI: 54.2, 56.1), and having 25% of patients lost to follow-up can underestimate the median age of survival by 3.3 years (47.6, 95% CI: 46.8-47.7).
We present several recommendations to assist national CF registries in calculating and reporting the median age of survival in a standardized fashion. It is imperative to state the statistical method used as well as the proportion lost to follow-up and the treatment of missing data and transplanted patients. Registries must be diligent in their data collection as incomplete data can lead to over and underestimation of survival.
Journal of clinical epidemiology 10/2015; DOI:10.1016/j.jclinepi.2015.08.026 · 3.42 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Background:
Studies in cystic fibrosis (CF) report late attention to advance care planning (ACP). The purpose of this study was to examine ACP with patients receiving care at US adult CF care programs.
Chart abstraction was used to examine ACP with adults with CF dying from respiratory failure between 2011 and 2013.
We reviewed 210 deaths among 67 CF care programs. Median age at death was 29years (range 18-73). Median FEV1 in the year preceding death was 33% predicted (range 13-100%); 68% had severe lung disease with FEV1<40% predicted. ACP was documented for 129 (61%), often during hospitalization (61%). Those with ACP had earlier documentation of treatment preferences, before the last month of life (73% v. 35%; p=<0.01). Advance directives were completed by 93% of those with ACP versus 75% without (p<0.01); DNR orders and health care proxy designation occurred more often for those with ACP. Patients awaiting lung transplant had similar rates of ACP as those who were not (67% v. 61%; p=0.55). The frequency of ACP varied significantly among the 29 programs contributing data from four or more deaths.
ACP in CF often occurs late in the disease course. Important decisions default to surrogates when opportunities for ACP are missed. Provision of ACP varies significantly among adult CF care programs. Careful evaluation of opportunities to enhance ACP and implementation of recommended approaches may lead to better practices in this important aspect of CF care.
Journal of cystic fibrosis: official journal of the European Cystic Fibrosis Society 09/2015; DOI:10.1016/j.jcf.2015.08.004 · 3.48 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Monitoring potential changes in the epidemiology of cystic fibrosis (CF) pathogens furthers our understanding of the potential impact of interventions.
We performed a retrospective analysis using data reported to the CF Foundation Patient Registry (CFFPR) from 2006-2012 to determine the annual percent changes in the prevalence and incidence of selected CF pathogens. Pathogens included P. aeruginosa, methicillin-susceptible S. aureus (MSSA), MRSA, Haemophilus influenzae, B. cepacia complex, Stenotrophomonas maltophilia and Achromobacter xylosoxidans. Changes in nontuberculous mycobacteria (NTM) prevalence were assessed from 2010-2012 when the CFFPR collected NTM species.
In 2012, the pathogens of highest prevalence and incidence were MSSA and P. aeruginosa, followed by MRSA. The prevalence of A. xylosoxidans and B. cepacia complex were relatively low. From 2006-2012, the annual percent change in overall (as well as in most age strata) prevalence and incidence significantly decreased for P. aeruginosa and B. cepacia complex, but significantly increased for MRSA. From 2010-2012, the annual percent change in overall prevalence of NTM and M. avium complex increased.
The epidemiology of CF pathogens continues to change. The causes of these observations are most likely multifactorial and include improvements in clinical care and infection prevention and control. Data from this study will be useful to evaluate the impact of new therapies on CF microbiology.
[Show abstract][Hide abstract] ABSTRACT: Treatment of pulmonary exacerbations (PEx) in cystic fibrosis (CF) varies widely with no consensus on management practices or best indicators of therapeutic success. To design trials evaluating PEx treatment factors, we characterise the heterogeneity of PEx care in adults and paediatrics, and correlate it with measures of clinical response including short-term and long-term lung function changes, change in symptom severity score and time to next intravenous antibiotic therapy.
Data were used from a prospective observational study of patients with CF ≥10 years of age enrolled at six sites between 2007 and 2010. All were started on intravenous antibiotics for a clinically diagnosed PEx. Analysis of variance, logistic and Cox regression were used to examine the association of treatment factors with short-term and long-term clinical response.
Of 123 patients with CF (60% women, aged 23.1±10.2 years), 33% experienced <10% relative improvement in FEV1 during treatment, which was associated with failing to recover baseline lung function 3 months after treatment (OR=7.8, 95% CI 1.9 to 31.6, p=0.004) and a longer time to next intravenous antibiotic (HR=0.48, 95% CI 0.27 to 0.85, p=0.011). Symptom improvement was observed but was not associated with subsequent lung function or time to next antibiotic therapy, which had a median recurrence time of 143 days.
Immediate symptomatic or respiratory response to PEx treatment did not have a clear relationship with subsequent outcomes such as lung function or intravenous antibiotic-free interval. These results can inform future research of treatment regimens for PEx in terms of interventions and outcome measures.
Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.
[Show abstract][Hide abstract] ABSTRACT: Introduction:
In 2012 and 2013, 30 adult and 40 pediatric CF Foundation-accredited programs across the United States recruited patients and families to complete an experience of care survey. This paper reports the positive attributes and the opportunities for improvement in CF care from the perspective of individuals with CF and families.
Patients and families completed the survey by web, interactive voice response, or with the help of a telecommunication professional. Funnel plot was used to determine positive attributes and improvement opportunities. Chi-square tests and 95% confidence intervals were used to determine differences between group and logistic regression models were used to determine factors associated with the experience of "best" care.
2090 adults with CF or parents of children with CF, 29% of the 7113 potential respondents, completed a survey. Both the adult and pediatric survey respondents reported the same 5 positive attributes of experience of care: courtesy and respect shown, easy to understand explanations, involved in decision-making, their questions were answered, and enough time with providers. Potential areas for improvement included assessing mental health and improving inpatient hospital staff's knowledge of CF. In general, results from the pediatric survey were significantly better than the adult survey. Variables predictive of "best" care experience from both adult and pediatric respondents were treatments always working and two self-care factors of finding information and working out solutions.
The CF Foundation developed an experience of care survey to systematically collect and learn directly from individuals with CF and families about their impressions and observations of CF health care delivery. Respondents reported positive and respectful experiences and improvement opportunities were identified, which can help programs target specific areas to enhance the care experience.
Journal of cystic fibrosis: official journal of the European Cystic Fibrosis Society 01/2015; 14(4). DOI:10.1016/j.jcf.2014.12.011 · 3.48 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Background:
Psychological morbidity in individuals with cystic fibrosis (CF) and their caregivers is common. The Cystic Fibrosis Foundation (CFF) and European Cystic Fibrosis Society (ECFS) Guidelines Committee on Mental Health sought the views of CF health care professionals concerning mental health care delivery.
An online survey which focused on the current provision and barriers to mental health care was distributed to CF health care professionals.
Of the 1454 respondents, many did not have a colleague trained in mental health issues and 20% had no one on their team whose primary role was focused on assessing or treating these issues. Insufficient resources and a lack of competency were reported in relation to mental health referrals. Seventy-three percent of respondents had no experience with mental health screening. Of those who did, they utilized 48 different, validated scales.
These data have informed the decision-making, dissemination and implementation strategies of the Mental Health Guidelines Committee sponsored by the CFF and ECFS.
[Show abstract][Hide abstract] ABSTRACT: Objectives:
The purpose of this study was to characterize the utilization of antibiotics for chronic methicillin-resistant Staphylococcus aureus (MRSA) infection in cystic fibrosis (CF) patients with acute pulmonary exacerbations (PEx).
An anonymous national cross-sectional survey of CF Foundation accredited care programs was performed using an electronic survey tool.
Fifty-eight percent (152/261) CF Foundation accredited programs completed the survey. Ninety-eight percent (149/152) of respondents reported using antibiotics (oral or intravenous) against MRSA. Variability exists in the use of antibiotics amongst the programs and in the dosages utilized. For oral outpatient treatment, sulfamethoxazole/trimethoprim was the most commonly utilized antibiotic by both pediatric (109/287, 38%) and adult (99/295, 34%) respondents, of which, ten percent of reported to use it in combination with rifampin. For inpatient treatment, linezolid (both intravenous (IV) and oral) was most commonly utilized in both pediatric (IV 35/224, 16%; oral 41/224, 18%), and adult (IV 44/235, 19%; oral 38/235, 16%) respondents for inpatient treatment. IV vancomycin was the second most commonly utilized antibiotic by pediatric (70/224, 31%) and adult (71/235, 30%) respondents. Most respondents reported dose titration to achieve a vancomycin trough level of 15-20 mg/L (150/179, 84%). Topical or inhaled antibiotic utilization was reported to be an uncommon practice with approximately 70% of pediatric and adult respondents reporting to use them either rarely or never. The concomitant use of anti-MRSA and anti-pseudomonal antibiotics was common with 96% of pediatric and 99% of adult respondents answering in the affirmative.
We conclude that anti-MRSA antibiotics are utilized via various dosage regimens by a majority of CF Foundation accredited care programs for the treatment of chronic MRSA in PEx, and there is no consensus on the best treatment approach.
[Show abstract][Hide abstract] ABSTRACT: The Cystic Fibrosis (CF) Foundation developed clinical care guidelines for the prevention of Pseudomonas aeruginosa infection, the treatment of initial P. aeruginosa infection, and the use of bronchoscopy to obtain routine airway cultures in individuals with CF.
A multidisciplinary committee developed questions about the prevention and treatment of initial P. aeruginosa infection and the use of bronchoscopy to obtain routine airway cultures. The outcome measure of interest was cultures without P. aeruginosa growth. Systematic reviews of PubMed, EMBASE, and the Cochrane Central Register of Controlled Trials were conducted in May 2012 and August 2013. Searches combined controlled vocabulary terms and text words for CF and terms relevant to each question. The entire committee reviewed the evidence, and final recommendation statements were graded using the U.S. Preventive Services Task Force system. Recommendation 1: The CF Foundation strongly recommends inhaled antibiotic therapy for the treatment of initial or new growth of P. aeruginosa from an airway culture (certainty of net benefit, high; estimate of net benefit, substantial; grade of recommendation, A). The favored antibiotic regimen is inhaled tobramycin (300 mg twice daily) for 28 days. Recommendation 2: The CF Foundation recommends against the use of prophylactic antipseudomonal antibiotics to prevent the acquisition P. aeruginosa (certainty of net benefit, moderate; estimate of net benefit, zero; grade of recommendation, D). Recommendation 3: The CF Foundation recommends routine oropharyngeal cultures rather than bronchoalveolar lavage cultures obtained by bronchoscopy in individuals with CF who cannot expectorate sputum to determine if they are infected with P. aeruginosa (certainty of net benefit, moderate; estimate of net benefit, moderate; grade of recommendation, B).
[Show abstract][Hide abstract] ABSTRACT: Background:
The purpose of this study was to identify the extent to which pediatric and adult cystic fibrosis (CF) care teams implement best practices in adherence assessment and counseling.
All US CF Foundation accredited programs were invited to participate in a web-based survey; 80% (92/115) of pediatric and 40% (38/95) of adult centers participated. Health care providers reported on current approaches and barriers to implementing adherence promotion practices.
64% discussed adherence at every clinic visit while only 8% used an objective assessment of adherence. Most centers reported frequent use of strategies to increase knowledge; behavioral and support strategies were used less regularly. Several barriers to adherence promotion were reported.
Many opportunities exist for care teams to improve consistency in adherence practices and integrate a greater repertoire of effective counseling strategies into clinic visits. Adherence promotion practices should be considered for quality improvement (QI) projects.
[Show abstract][Hide abstract] ABSTRACT: Background People with cystic fibrosis (CF) are managed differently in the USA and UK providing an opportunity to learn from differences in practice patterns.
Objectives To compare cross-sectional demographics, practice patterns and clinical outcomes between US and UK CF patients.
Methods This was a cross-sectional study using 2010 data from patients in the US Cystic Fibrosis Foundation and the UK Cystic Fibrosis patient registries. The a priori outcome measures of interest were lung function and nutritional status. Descriptive statistics and two sample comparisons were performed. Stratification and multivariable linear regression were used to adjust for confounding.
Results The study cohort included 13 777 children and 11 058 adults from the USA and 3968 children and 3965 adults from the UK. In children, mean body mass index centiles were similar. Lung function (FEV1 and FVC% predicted) was significantly higher in US patients ages 6–25 years of age. In a regression model adjusted for only age, FEV1% predicted was on average 3.31% of predicted (95% CI 2.65 to 3.96) higher in the USA compared with the UK. When adjusted for age, age at diagnosis, gender, pancreatic insufficiency and genotype, FEV1% predicted was on average 3.03% of predicted (95% CI 2.37 to 3.69) higher in the USA compared with the UK These differences persisted despite adjustment for possible confounders. Hypertonic saline and dornase alfa were much more commonly prescribed in US children.
Conclusions Children and young adults with CF have better lung function in the USA compared with the UK despite similar nutritional status.
[Show abstract][Hide abstract] ABSTRACT: Background: Advances in treatments for cystic fibrosis (CF) continue to extend survival. An updated estimate of survival is needed for better prognostication and to anticipate evolving adult care needs. Objective: To characterize trends in CF survival between 2000 and 2010 and to project survival for children born and diagnosed with the disease in 2010. Design: Registry-based study. Setting: 110 Cystic Fibrosis Foundation-accredited care centers in the United States. Patients: All patients represented in the Cystic Fibrosis Foundation Patient Registry (CFFPR) between 2000 and 2010. Measurements: Survival was modeled with respect to age, age at diagnosis, gender, race or ethnicity, F508del mutation status, and symptoms at diagnosis. Results: Between 2000 and 2010, the number of patients in the CFFPR increased from 21 000 to 26 000, median age increased from 14.3 to 16.7 years, and adjusted mortality decreased by 1.8% per year (95% CI, 0.5% to 2.7%). Males had a 19% (CI, 13% to 24%) lower adjusted risk for death than females. Median survival of children born and diagnosed with CF in 2010 is projected to be 37 years (CI, 35 to 39 years) for females and 40 years (CI, 39 to 42 years) for males if mortality remains at 2010 levels and more than 50 years if mortality continues to decrease at the rate observed between 2000 and 2010. Limitations: The CFFPR does not include all patients with CF in the United States, and loss to follow-up and missing data were observed. Additional analyses to address these limitations suggest that the survival projections are conservative. Conclusion: Children born and diagnosed with CF in the United States in 2010 are expected to live longer than those born earlier. This has important implications for prognostic discussions and suggests that the health care system should anticipate greater numbers of adults with CF.
Annals of internal medicine 08/2014; 161(4):233-241. DOI:10.7326/M13-0636 · 17.81 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: In summary, epidemiologic studies have shown that pathogens, other than Burkholderia spp., can be transmitted among individuals with CF, resulting in adverse clinical outcomes, including increased morbidity and mortality. The updated guideline is a response to new knowledge and new challenges in both IP&C and CF. The primary objective of the guideline is to provide recommendations to reduce the risk of transmission and acquisition of CF pathogens by individuals with CF and to provide a more comprehensive understanding of effective strategies to optimize safety for this unique population.
Infection Control and Hospital Epidemiology 08/2014; 35(S1):S1-S67. DOI:10.1086/676882 · 4.18 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: BACKGROUND
Bringing new therapies to patients with rare diseases depends in part on optimizing clinical trial conduct through efficient study start-up processes and rapid enrollment. Suboptimal execution of clinical trials in academic medical centers not only results in high cost to institutions and sponsors, but also delays the availability of new therapies. Addressing the factors that contribute to poor outcomes requires novel, systematic approaches tailored to the institution and disease under study.
To use clinical trial performance metrics data analysis to select high-performing cystic fibrosis (CF) clinical research teams and then identify factors contributing to their success.
Mixed-methods research, including semi-structured qualitative interviews of high-performing research teams.
CF research teams at nine clinical centers from the CF Foundation Therapeutics Development Network.
Survey of site characteristics, direct observation of team meetings and facilities, and semi-structured interviews with clinical research team members and institutional program managers and leaders in clinical research.
Critical success factors noted at all nine high-performing centers were: 1) strong leadership, 2) established and effective communication within the research team and with the clinical care team, and 3) adequate staff. Other frequent characteristics included a mature culture of research, customer service orientation in interactions with study participants, shared efficient processes, continuous process improvement activities, and a businesslike approach to clinical research.
Clinical research metrics allowed identification of high-performing clinical research teams. Site visits identified several critical factors leading to highly successful teams that may help other clinical research teams improve clinical trial performance.
Journal of General Internal Medicine 07/2014; 29(3). DOI:10.1007/s11606-014-2896-8 · 3.45 Impact Factor
[Show abstract][Hide abstract] ABSTRACT: Benchmarking is the process of using outcome data to identify high-performing centres and determine practices associated with their outstanding performance. The US Cystic Fibrosis Foundation (CFF) Patient Registry contains centre-specific outcomes data for all CFF-certified paediatric and adult cystic fibrosis (CF) care programmes in the USA. The CFF benchmarking project analysed these registry data, adjusting for differences in patient case mix known to influence outcomes, and identified the top-performing US paediatric and adult CF care programmes for pulmonary and nutritional outcomes. Separate multidisciplinary paediatric and adult benchmarking teams each visited 10 CF care programmes, five in the top quintile for pulmonary outcomes and five in the top quintile for nutritional outcomes. Key practice patterns and approaches present in both paediatric and adult programmes with outstanding clinical outcomes were identified and could be summarised as systems, attitudes, practices, patient/family empowerment and projects. These included: (1) the presence of strong leadership and a well-functioning care team working with a systematic approach to providing consistent care; (2) high expectations for outcomes among providers and families; (3) early and aggressive management of clinical declines, avoiding reliance on 'rescues'; and (4) patients/families that were engaged, empowered and well informed on disease management and its rationale. In summary, assessment of practice patterns at CF care centres with top-quintile pulmonary and nutritional outcomes provides insight into characteristic practices that may aid in optimising patient outcomes.
[Show abstract][Hide abstract] ABSTRACT: Remarkable biomedical research advances have led to innovative and increasingly effective therapies. We highlight several scientific milestones in elucidating the pathophysiology of cystic fibrosis (CF) and review the therapies that have become available over the past 20 years.
In 2002, the CF Foundation launched a multifaceted quality improvement initiative to accelerate improvement in CF care. We present evidence of substantial improvement in process measures, such as more consistent outpatient follow-up, and key medical outcomes, including survival, pulmonary function and nutritional status.
We offer our perspective on factors critical to the success of the quality improvement initiative, including a compelling strategic plan and the commitment of the CF Foundation to its implementation; the investment in building improvement capacity at CF care centres; the engagement of people with CF and their families as partners; and the integration of quality improvement into the existing CF care framework.
In addition to a continued investment in building and sustaining improvement capacity at CF care centres, and deeper patient engagement, we will address the oppressive treatment burden. We will also complement the measurement of clinical outcomes with patient reported outcomes and healthcare costs for a balanced assessment of the quality and value of care.
Major advances in basic science and therapeutic development coupled with improvements in healthcare delivery have resulted in striking gains in medical outcomes for people with CF.
[Show abstract][Hide abstract] ABSTRACT: Preparation of this supplement, Ten years of improvement innovation in cystic fibrosis care, tested a strategy to support writing and scholarly publication by cystic fibrosis (CF) healthcare improvement professionals.
Critical elements of the writing initiative included: a request for abstracts that was distributed to over 2000 professionals in the Cystic Fibrosis Foundation-supported improvement community to identify promising work; continuous peer review of manuscripts by co-authors and writing tutors; three webinars and a 2-day face-to-face writing retreat that addressed the challenges of successful scholarly healthcare improvement writing and publication; and finally, journal submission and formal external peer review. The SQUIRE Publication Guidelines provided content framework for manuscripts.
47 abstracts were submitted from which reviewers selected nine for participation. The 28 co-authors of these abstracts took part in the writing initiative. Authors' self-assessment showed that half had previously published fewer than five papers, while 80% considered themselves insufficiently prepared to write for the scholarly improvement literature. Eventually all of the nine abstracts led to full manuscripts, which were submitted to the journal for formal peer review. Of these, seven were accepted for publication and are included in this supplement.
A formal initiative to develop and support scholarly writing-while resource-intensive-offers opportunities for wider publication by healthcare improvement professionals.