CF Foundation Research Articles

Design and Implementation of a Novel Electronic Health Record Tool to Enhance the Care of Individuals with Cystic Fibrosis: The Cystic Fibrosis Note Template

AbstractCystic fibrosis (CF) is a genetic disease in which dysfunction of a single protein channel leads to organ damage, resulting in chronic health problems and premature death. In the United States, medical care of individuals living with CF is delivered by care centers accredited and subsidized by the CF Foundation. CF outcomes have improved significantly through the use of collaborative networks, registry data, and research. CF clinicians are perpetually challenged to assimilate and act upon large quantities of data generated by the care of these individuals. CF Foundation accreditation also requires care centers to enter patient-level data from clinical encounters into the CF Foundation Patient Registry (CFFPR). Commercially available electronic health record systems often lack tools with sufficient context specificity and ease of use to facilitate productive interactions between clinicians and patients. We describe a CF-specific NoteWriter template built and implemented in Epic, which captures discrete data and simultaneously generates clinical documentation during ambulatory encounters. Unlike other examples of note templates in CF, this project involves SmartData Elements (SDEs) using the NoteWriter tool in Epic, which enables data to be entered in the exact way in which the CFFPR captures data. By conducting a pre-/poststudy of its use in our health system, we found that the template can expedite note completion when clinicians have adequate time to become familiar with the tool. We anticipate that the NoteWriter template will become a vehicle for delivering standardized, structured patient data to the CFFPR.

Tobacco Smoke Exposure and Socioeconomic Factors are Independent Predictors of Pulmonary Decline in Pediatric Cystic Fibrosis

Background: Few studies have attempted to disentangle the effects of tobacco smoke exposure and socioeconomic factors on lung function decline in pediatric CF. The current study evaluates their contributions longitudinally across the entire U.S. CF care network population. Methods: Data from the CF Foundation Patient Registry were obtained on all individuals who at the end of 2016 were 6-18 years old. Lung function measures (median, highest, and lowest ppFEV1) for each person were calculated at each attained age. Multivariable analyses used mixed modeling to assess the impact of smoke exposure and socioeconomic factors on both initial lung function and change over time. Findings: The sample included 10,692 individuals contributing 57,888 person years. At age 6, ppFEV1 of smoke-exposed children was 4% lower than among unexposed. The deficit persisted through age 18. In fully adjusted mixed models, smoke exposure and socioeconomic factors had independent, additive associations with lung function. Median ppFEV1 declined 2% with smoke exposure, 3% with lower paternal education, 1% with public insurance, and increased 0.2% with each $10,000 annual household income. The effect of smoke exposure on ppFEV1 was larger in disadvantaged children compared to privileged counterparts (2.5% vs 1.5%). Interpretation: Smoke exposure and socioeconomic factors are independent risk factors for decreased ppFEV1 in pediatric CF. Strategies to reduce smoke exposure should be introduced early. Interventions may be prioritized in disadvantaged children, among whom the exposure has a disproportionately large effect. Funding Statement: This study was supported by grants from NIH (P30DK72482, K08HL140190), AHRQ (K12HS023009), and CFF (CC032-14). Declaration of Interests: The authors have no conflicts of interest. Ethics Approval Statement: Written informed consent was obtained from all participants. The study was approved by the Institutional Review Board of the University of Alabama at Birmingham (protocol number 300002076).

Rate and predictors of prescription of lumacaftor – Ivacaftor in the 18 months following approval in the United States

RationaleLumacaftor-ivacaftor (LUM-IVA) was approved in the US in 2015 for patients with CF aged >12 homozygous for the delF508 mutation, and patients aged 6 to 12 in 2016. ObjectivesTo examine the rate of initial LUM-IVA prescriptions following approval. MethodsWe compared patients eligible for LUM-IVA in the CF Foundation Patient Registry with and without prescriptions in 2015–2016. Results5534 (53%) eligible patients had reported prescriptions. Prescription rate in children ages 6–11 was 19% and 61% among patients ≥12 years old. Individuals ≥12 with prescriptions more likely observed among those with private insurance, clinical trial participation, ages 18–30, FEV1 < 90%, more pulmonary exacerbations, and more use of chronic medications. ConclusionsLUM-IVA uptake was less rapid than what was previously observed for ivacaftor, a CFTR modulator approved for a different population. Age, insurance status, disease severity and use of other therapies differed in those prescribed LUM-IVA in the initial post-approval period.

Advances in the Diagnosis and Management of Cystic Fibrosis in the Genomic Era.

Cystic fibrosis (CF) is a complex autosomal recessive disease that continues to present unique diagnostic challenges. Because CF was first described in 1938, there has been a substantial growth of genetic and phenotypic information about the disorder. During the past few years, as more evidence has become available, a consortium of international experts determined that the 2008 guidelines from the CF Foundation needed to be reviewed and updated. The goal of this review is to highlight the latest advances in CF multidisciplinary care, together with the recent updates to the 2017 CF Foundation diagnostic guidelines. Data from newborn screening programs, patient registries, clinical databases, and functional research have led to a better understanding of the CF transmembrane conductance regulator (CFTR) gene. Recent consensus guidelines have provided recommendations for clinicians and laboratorians to better assist with interpretation of disease status and related CF mutations. The highly recommended Clinical and Functional Translation of CFTR project should be the first resource in the evaluation of disease severity for CF mutations. Screen-positive newborns and patients with high clinical suspicion for CF are always recommended to undergo confirmatory sweat chloride testing with interpretations based on updated reference intervals. Every patient diagnosed with CF should receive genotyping, as novel molecular therapies are becoming standard of practice. The future of CF management must consider healthcare system disparities as CF transitions from a historically childhood disease to a predominantly adult epidemic.

Understanding of safety monitoring in clinical trials by individuals with CF or their parents: A qualitative analysis

BackgroundRecruiting both pediatric and adult participants for clinical trials in CF is currently of paramount importance as numerous new therapies are being developed. However, recruitment is challenging as parents of children with CF and adults with CF cite safety concerns as a principal barrier to enrollment. In conjunction with the CF Foundation (CFF) Data Safety Monitoring Board (DSMB), a pilot brochure was developed to inform patients and parents of the multiple levels of safety monitoring; the CFF simultaneously created an infographic representing the safety monitoring process. This study explores the attitudes and beliefs of CF patients and families regarding safety monitoring and clinical trial participation, and elicits feedback regarding the educational materials. MethodsSemi-structured interviews were conducted using a pre-tested interview guide and audio-recorded during routine CF clinic visits. Participants included 5 parents of children with CF <16years old; 5 adolescents and young adults with CF 16–21years old; and 5 adults with CF ≥22years old from pediatric and adult CF centers. The study team performed systematic text condensation analysis of the recorded interviews using an iterative process. ResultsFour major thematic categories with subthemes emerged as supported by exemplar quotations: attitudes toward clinical trials, safety values, conceptualizing the safety monitoring process, and priorities for delivery of patient education. Participant feedback was used to revise the pilot brochure; text was shortened, unfamiliar words clarified (e.g., “pipeline”), abbreviations eliminated, and redundancy avoided. ConclusionsQualitative analysis of CF patient and family interviews provided insights into barriers to participation in clinical trials, safety concerns, perspectives on safety monitoring and educational priorities. We plan a multicenter study to determine if the revised brochure reduces knowledge, attitude and practice barriers regarding participation in CF clinical trials.

Longitudinal development of initial, chronic and mucoid Pseudomonas aeruginosa infection in young children with cystic fibrosis

BackgroundWhile the emergence of chronic and mucoid Pseudomonas aeruginosa (Pa) infection are both associated with poorer outcomes among CF patients, their relationship is poorly understood. We examined the longitudinal relationship of incident, chronic and mucoid Pa in a contemporary, young CF cohort in the current era of Pa eradication therapy. MethodsThis retrospective cohort was comprised of patients in the U.S. CF Foundation Patient Registry born 2006–2015, diagnosed before age 2, and with at least 3 respiratory cultures annually. Incidence and age-specific prevalence of Pa infection stages (initial and chronic [≥ 3Pa+cultures in prior year]) and of mucoid Pa were summarized. Transition times and the interaction between Pa stage and acquisition of mucoid Pa were examined via Cox models. ResultsAmong the 5592 CF patients in the cohort followed to a mean age of 5.5years, 64% (n=3580) acquired Pa. Of those, 13% (n=455) developed chronic Pa and 17% (n=594) cultured mucoid Pa. Among those with mucoid Pa, 36% (211/594) had it on their first recorded Pa+culture, while mucoid Pa emerged at or after entering the chronic stage in 12% (73/594). Mucoidy was associated with significantly increased risk of transition to chronic Pa infection (HR=2.59, 95% CI 2.11, 3.19). ConclusionsTwo-thirds of early-diagnosed young children with CF acquired Pa during a median 5.6years of follow up, among whom 13% developed chronic Pa and 17% acquired mucoid Pa. Contrary to our hypothesis, 87% of young children who developed mucoid Pa did so before becoming chronically infected.

Data that empower: The success and promise of CF patient registries.

In this article, we describe existing CF registries with a focus on US registry data collected through the CF Foundation Patient Registry (CFFPR) and the Epidemiologic Study of CF (ESCF); highlight what registries have taught us regarding epidemiology of CF; showcase the impact of registries on research and clinical care; and discuss future directions. This manuscript complements the plenary address given by Dr Wayne Morgan at the 2016 North American CF Conference by summarizing the key points from the presentation and providing additional detail and information.

Poor recovery from cystic fibrosis pulmonary exacerbations is associated with poor long-term outcomes.

People with CF treated with IV antibiotics for a pulmonary exacerbation (PEx) frequently fail to recover to baseline FEV1 . The long-term impact of these events has not been studied. To determine if a patient's spirometric recovery after a PEx is associated with time to next PEx within 1 year, the spirometric recovery after the next PEx, and/or the number of PEx episodes in the next 3 years. We used data from the CF Foundation Patient Registry from 2004 to 2011. We randomly selected one PEx per patient that met inclusion/exclusion criteria. Patients were defined as Non-Responders if their best FEV1 (in liters) recorded in the 3 months after the PEx was <90% of the best FEV1 (in liters) in the 6 months before the PEx. We compared Responders and Non-Responders using multivariable regression models. We randomly chose 13 954 PEx episodes that met inclusion/exclusion criteria. A total of 2 762 (19.8%) patients were classified as Non-Responders. Non-Responders had a shorter median time to the next PEx, 235 (95%CI 218, 252) days, versus >365 days for Responders. Thirty-four percent of Non-Responders at the initial PEx were also Non-Reponders at the next PEx, versus 20% of Responders at the initial PEx. Non-Responders had more PEx episodes over the next 3 years, 4.99 (95%CI 4.84, 5.13), than Responders, 3.46 (95%CI 3.41, 3.51). Poor recovery after a PEx is associated with a shorter time to the next PEx, increased risk of poor recovery at a second PEx, and more frequent subsequent PEx treatments.

Poor recovery from a pulmonary exacerbation does not lead to accelerated FEV1 decline

BackgroundPatients with CF treated for pulmonary exacerbations (PEx) may experience faster subsequent declines in FEV1. Additionally, incomplete recovery to baseline FEV1 occurs frequently following PEx treatment. Whether accelerated declines in FEV1 are preceded by poor PEx recovery has not been studied. MethodsUsing 2004 to 2011 CF Foundation Patient Registry data, we randomly selected one PEx among patients ≥6years of age with no organ transplantations, ≥12months of data before and after the PEx, and ≥1 FEV1 recorded within the 6months before and 3months after the PEx. We defined poor PEx recovery as the best FEV1 in the 3months after the PEx <90% of the best FEV1 in the 6months before the PEx. We calculated mean (95% CI) hazard ratios (HR) of having >5% predicted/year FEV1 decline and poor PEx recovery using multi-state Markov models. ResultsFrom 13,954 PEx, FEV1 declines of >5% predicted/year were more likely to precede poor spirometric recovery, HR 1.17 (1.08, 1.26), in Markov models adjusted for age and sex. Non-Responders were less likely to have a subsequent fast FEV1 decline, HR 0.41 (0.37, 0.46), than patients who recovered to >90% of baseline FEV1 following PEx treatment. ConclusionsAccelerated declines in FEV1 are more likely to precede a PEx with poor recovery than to occur in the following year. Preventing or halting declines in FEV1 may also have the benefit of preventing PEx episodes.

Nominal Carbonic Anhydrase Activity Mitigates Airway‐Surface Liquid pH Changes During the Respiratory Cycle

The airway‐surface liquid pH (pHASL) is slightly acidic relative to the plasma pH and becomes more acidic in airway disease (e.g., cystic fibrosis, asthma, and COPD). Pathological pHASL may dysregulate ASL viscosity, mucociliary transport, and bacterial killing. Under constant 5% CO2, porcine large airways are more acidic (~7.0) than small airways (~7.4). However, the small airways are exposed to a constant 5% CO2, whereas the large airways are exposed to wide swings in pCO2 during respiration. Therefore, pHASL in the large airway may oscillate and be more alkaline during respiration. In this study, we investigated how oscillating CO2 affects the large airway pHASL for differentiated porcine tracheal epithelia cultured at the airway‐liquid interface. The apical surface of epithelia was initially exposed to 5% CO2. Apical CO2 was then rapidly switched to 0.04% CO2, allowed to equilibrate, and was then returned to 5% CO2. The pH was monitored by SNARF‐1 dextran fluorescence. Relative to a breath (~4 s), the time constant for pH changes were unexpectedly slow (τ = 51.9 ± 5.8 s, n = 12). We hypothesize that this slow time constant might be due to low carbonic anhydrase activity, which catalyzes the carbonic buffering reaction. Consistent with this hypothesis, the carbonic anhydrase inhibitor acetazolamide did not appreciably change the time constant (τ = 64.1 ± 6.1 s, n = 10; p = 0.21 vs. non‐treated). In contrast, adding exogenous carbonic anhydrase strikingly decreased the mean time constant (τ = 17.3 ± 3.1 s, n = 16; p &lt; 0.01 vs. non‐treated). Curated mRNA microarray data indicated nominal expression for all extracellular and secreted carbonic anhydrase isoforms in large airway epithelia. To further understand how CO2 affects pHASL in the large airway, we modeled the pHASL during respiration using the pH‐dependent rates (dph/dt) obtained from our functional experiments. There were three major findings from our simulations. First, under physiological conditions (inspiratory:expiratory ratio = 1:2; respiration rate = 15 breathes per minute), the pHASL oscillations were small (peak‐to‐peak pHASL = 0.09 ± 0.02 pH units). In contrast, adding carbonic anhydrase increased the pHASL oscillations (peak‐to‐peak pHASL = 0.56 ± 0.16 pH units). Second, correcting the large airway steady‐state pHASL for the respiratory cycle still yields an acidic pHASL relative to the small airway (pHlarge 7.05 vs. pHsmall 7.45). Third, the inspiratory:expiratory ratio affected the average pHASL, whereas the respiration rate affected the magnitude of pHASL oscillations. Thus, minimal carbonic anhydrase activity in large airways prevents sizeable shifts in pHASL during respiration. Maintaining relatively constant pHASL may optimize airway host defense by antimicrobials and mucociliary transport.Support or Funding InformationNIH HL091842, NIH HL5167, CF Foundation LI1410

Cystic fibrosis newborn screening programs: implications of the CFTR variant spectrum in nonwhite patients.

Cystic fibrosis newborn screening (CFNBS) has been offered across the United States since 2010. However, as compared with white patients with CF, CFTR variant identification in nonwhite populations remains inequitable. Utilizing the recent characterization of the nonwhite CF variant spectrum, we examined the effectiveness of current CFNBS molecular panels in identifying affected nonwhite newborns. Based on a cross-sectional evaluation of genotyping data from the CF Foundation Patient Registry that compared 3,496 nonwhite with 22,206 white CF patients, the current CFNBS algorithms used in the 50 states and the District of Columbia were analyzed. We assessed the percentage of CF patients of Hispanic, African, Asian, and Native American heritage who would not be identified by the molecular panels most commonly used. Compared with whites, variant detection was significantly lower in Hispanic, black, and Asian newborns (P ≤ 0.0001 each), as well as in Native American newborns (P values ranged from 0.001 to 0.0003), for the most common CFNBS panels. This study provides a perspective on the applicability of current panels to a diverse population and enables CFNBS programs to consider more inclusive test approaches to facilitate diagnosis, timely clinical intervention, and enhanced prognosis for CF patients of nonwhite and mixed ethnicities.Genet Med 19 1, 36-44.

Improving performance in the detection and management of cystic fibrosis-related diabetes in the Mountain West Cystic Fibrosis Consortium

ObjectiveCystic fibrosis (CF)-related diabetes (CFRD) is associated with increased morbidity and mortality. Improved detection and management may improve outcomes; however, actual practice falls short of published guidelines. We studied efforts...

IV-treated pulmonary exacerbations in the prior year: An important independent risk factor for future pulmonary exacerbation in cystic fibrosis

BackgroundSingle-center analyses have suggested that the number of CF pulmonary exacerbations (PEx) treated with intravenous antibiotics an individual has experienced in the prior year is significantly associated with their future PEx hazard. MethodsWe studied Prior-year PEx association with future PEx hazard by Cox proportional hazards regression among CF Foundation Patient Registry patients who experienced PEx after Jan 1, 2010. ResultsAmong 13,579 patients, those with 1, 2, 3, or ≥4 Prior-year PEx treated with intravenous antibiotics were at 1.8, 2.9, 4.8, and 8.7 higher PEx hazard vs those without (P<.0001). Adjustment with significant demographic and clinical covariates (univariate P≤.0001) reduced Prior-year PEx hazard ratios to 1.6, 2.4, 3.6, and 6.0 (P<.0001). No other covariates had adjusted hazard ratios of >1.7. ConclusionsPrior-year PEx strongly associate with future PEx hazard and should be accounted for in prospective trials where treatment-associated change in PEx hazard is an efficacy outcome.

CF healthcare workers feel unprepared in providing suitable end of life care and desire more education: Results of a nationwide survey

IntroductionLittle is known about the depth of knowledge and preparedness of CF caregivers in delivering end of life and palliative care to CF patients and families. MethodNationwide survey questionnaires for CF care providers using the CF Foundation Listserv electronic web-based tool. ResultsThe majority of non-physician CF care providers (55%) had more than 15years of experience in their discipline and 84% of physician had greater than 15years of experience. The majority reported that they felt “somewhat” or “very” involved in palliative or end of life care in their current role. Yet, when asked whether they felt adequately prepared to deliver palliative and end of life care, only 18% reported that they were “fully prepared” and 45% felt that they were only “minimally” or “not” prepared. Further, only one third of respondents received more than 10h of education in general palliative or end-of-life care, while only 10% had received more than 10h of education specific to CF end of life care. The majority (73%) of CF healthcare providers preferred more education specific to CF end of life care. ConclusionCF healthcare providers are involved in CF end of life issues but a fair number did not understand their role and felt inadequately prepared in delivering suitable end of life and palliative care. Many desired more education in the provision of such care.

266 Experience of Mycobacterium abscessus eradication therapy at Aberdeen Royal Infirmary

Mycobacterium abscessus (MA) is emerging as a potential major pathogen in CF with potential implications for transplantation. Ongoing research shows that an aggressive attempt at eradication is the treatment choice. Three patients underwent eradication treatment at Aberdeen Royal Infirmary during 2014, following the Papworth eradication protocol. Patients underwent 4 weeks of multiple intravenous antibiotics followed by a long-term regime of nebulised and oral antibiotics. Close monitoring of sputum cultures was ongoing throughout this time. To date, all three patients have had 3 consecutive negative sputum cultures for MA following eradication therapy. The CF Foundation and ECFS draft guidelines state that eradication therapy should continue for one year after the first of 3 negative sputum cultures, thus within the next 3 months, all 3 patients will be able to discontinue their long-term eradication treatment and return to a routine CF clinic within the department. Patients A and B both dropped their FEV1 predicted values during eradication therapy (9.67% and 30.73% respectively) whilst Patient C experienced a slight increase in predicted FEV1 of 6.78%. Patient B has commenced anti-asthma therapy, which has led to an improvement in her most recent FEV1 recording. All 3 patients have gained weight, with Patient C gaining 13.8 kg over the last 8 months since commencing eradication treatment. Patients A and B have both gained just over 1 kg in weight in this time. Eradication treatment for MA is complex and difficult for some patients to tolerate. We believe that further research regarding treatment and cross infection is imperative.

WS07.1 Standardized treatment of pulmonary exacerbations (STOP) study: Treatment goals for pulmonary exacerbations

Objectives Pulmonary exacerbations (PEx) are common in individuals with CF, but there are no standard treatment recommendations. STOP collects information to define the primary endpoint for studies of treatment of PEx, and to access the feasibility for future trials to use the CF Foundation Patient Registry (CFFPR) for data capture. Methods STOP is an observational pilot study using the CFFPR and projected to enroll over 200 patients (age >12 years) at 10 sites admitted to the hospital for PEx treatment. A survey of admitting physicians was performed to ascertain treatment goals and physician willingness to enroll the patient in hypothetical interventional trials. Results After 12 months, 204 patients (56% female) have entered the study, with a mean age of 27.2±9.2 years and 53% homozygous F508del. The primary objective of treatment was recovery of lung function (53%) and improvement of symptoms (45%). The mean lung function recovery goal (target FEV1 – admission FEV1) was 13.7% predicted (range: 0–65.7%). For CF centers with a treatment protocol, the average planned treatment duration was 13.9±1.6 days. A majority of physicians were willing to enroll their patients in a trial of differing durations [10 days (71%) and 14 days (87%)], specified antibiotics (86%), and other treatments (83%), but less so for treatment of 7 days (30%). Conclusion The primary goals for treatment of PEx are recovery of lung function, targeting almost 14% recovery, and symptom improvement. Most sites express willingness to enroll patients into interventional trials of treatments. Early experience suggests the CFFPR can be used successfully for clinical trials. Supported by CFFT.

286 Emotional distress, coping and quality of life in children with cystic fibrosis: A cross-cultural study

Background The CF Foundation and European CF Society seek unified consensus on standards of care, yet differences remain and psychosocial disparities in patients may exist. Objective We compare anxiety, depression, coping strategies, and quality of life (QOL) in children with CF between Belgium and the USA. Method We recruited 20 Belgian and 17 American children with CF (ages 8–12), and a matched (nationality, age, & sex) control group of healthy children, from hospitals and schools in Belgium and USA. Using a cross-sectional design, all children completed the State-Trait Anxiety Inventory for Children (STAIC), Children's Depression Inventory (CDI), Kidcope, and Quality of Life Systemic Inventory for Children (QLSI-C; modular approach). Data collection in the United States is on going and a sample of 20–25 American children with CF is expected for this presentation. Results Though some results are statistical trends, Americans with CF had lower emotional distress (CDI: p = 0071) as well as better QOL ( p = 0028) than Belgians with CF and, surprisingly, healthy American children (STAIC: p = 0038; CDI: p = 0072; QOL: p = ns). For QOL, Americans with CF reported less emotional problems than Belgians ( p = 0078). Americans (47%) use more social withdrawal coping than Belgians with CF (10%) ( p = 0011). Emotional regulation coping is related to higher anxiety ( p = 0017) and worse QOL ( p = 0038) in Americans with CF. Conclusion These preliminary findings suggest cultural differences regarding psychosocial outcomes in children with CF. Future research should examine the impact of emotion-related differences on treatment adherence and medical outcomes in CF.

WS03.1 Positive effects of a coaching program for parents after the diagnosis of CF following newborn screening

Objectives Parents are emotionally severely disrupted after their child is diagnosed with CF following Newborn Screening. The Dutch CF Foundation (NCFS) started a coaching program for parents to assist them to regain control of their lives. Methods After the diagnosis, parents were offered a maximum of 4 individual sessions at home and one group session with other parents and an experienced family coach. At the start of the coaching program and at the end, both parents filled in questionnaires related to the perceived health status of their child (TAPQOL), anxiety and distress of the parents, and satisfaction about the coaching program. If applicable, paired non-parametric testing was performed for differences between the start and the end of the program. Results 48 parents participated in the program. Mean age of the child at start was 28 weeks. Almost all parents completed 3 or 4 individual sessions. Parental perception of the health status of their child improved significantly over time. Anxiety and distress of the parents significantly decreased. The coaching program was rated good or very good by 80% concerning information, by 90% concerning the coach, and by 75% concerning the content (with only lower scores for the amount of sessions, which were preferred to be higher). 36 parents participated in the group session. 90% rated this session as good or very good. 90% of the parents has reached their predefined goals and almost all parents advised to continue the coaching project in the home situation for future parents. Conclusion A coaching program for parents after the diagnosis CF is feasible and useful. Other groups also might benefit from a coaching program.

Experience of care from the perspective of individuals with cystic fibrosis and families: Results from 70 CF Foundation accredited programs in the USA

IntroductionIn 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. MethodsPatients 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. Results2090 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. ConclusionThe 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.

Early attained weight and length predict growth faltering better than velocity measures in infants with CF

BackgroundCF infants often do not grow as expected which adversely affects later clinical outcomes, thus sensitive early measures of growth deficiency are important. This study compared attained growth for age with velocity standards to determine which better predicts growth deficits at 24months of age. MethodsGrowth deficiency in infancy based on weight and length velocity, and attained growth was calculated for 1992 infants in the US CF Foundation National Registry using the World Health Organization (WHO) and US growth standards. One, two and three month increments were used for calculating velocity and pooled for each age interval. Sensitivity and specificity of early indicators to predict growth deficiency at 24months were calculated. ResultsObserved prevalence of weight deficiency (<10th percentile) during the first year of life was 26.8% higher (95% CI=(25.6, 28.1%), p<0.001) on average when measured by attained weight for age than velocity. Attained weight for age at four months was a more sensitive predictor of diminished weight for age (<10th percentile) at 24months (sensitivity=100%, 95% CI=(87, 100%)) than weight velocity (sensitivity=40%, 95% CI=(23, 59%)). Attained length at four months was more sensitive to detecting subsequent stunting (<10th percentile length for age) (77%, 95% CI=(62, 87%)) than length velocity (30%, 95% CI=(19, 45%)). ConclusionsIn CF infancy, attained weight or length is more sensitive than velocity-based definitions for predicting subsequent diminished growth.