Michael S. Schechter

The Cystic Fibrosis Foundation Patient Registry as a tool for use in quality improvement

The US Cystic Fibrosis Foundation (CFF) began in 1955 with a mission to support the development of new drugs to fight the disease, improve the quality of life for those with cystic fibrosis (CF), and ultimately to find a cure for this disease.1 The CFF does this by supporting basic science and clinical research in CF, supporting the care of CF patients through accredited CF centres nationwide and advocating for CF patients at the state and national level. Recognising the critical role of data collection and measurement of outcomes to better understand the natural history of CF, the CFF created a patient registry in 1966, the CFF Patient Registry (CFFPR).2 The CFFPR has evolved over the years from a few demographic variables including vital status to a comprehensive database that gives healthcare providers, researchers, policy makers and change agents data to support epidemiological and clinical research as well as efforts to improve quality of care. The specific purpose of this commentary is to describe the CFFPR and primarily to focus on how the CFFPR and its associated tools are being used for quality improvement (QI) activities, with the hope that it may help CF healthcare teams in the USA who are not familiar with the registrys capabilities, CF providers outside the USA with registries at various stages of development, and others interested in how a patient registry has been used to improve care. The CFFPR contains detailed demographic and diagnostic data dating back to 1986 with current annual and encounter-based data on over 300 unique variables including outcomes (eg, microbiology, lung function and nutritional metrics, CF complications) and care processes (eg, hospitalisations, medications, surveillance measures) for each of its more than 27 000 participants in 2012; in all, there are over 46 000 unique individuals’ data in the registry.3 …

The National Spina Bifida Patient Registry: profile of a large cohort of participants from the first 10 clinics.

OBJECTIVE To use data from the US National Spina Bifida Patient Registry (NSBPR) to describe variations in Contexts of Care, Processes of Care, and Health Outcomes among individuals with spina bifida (SB) receiving care in 10 clinics. STUDY DESIGN Reported here are baseline cross-sectional data representing the first visit of 2172 participants from 10 specialized, multidisciplinary SB clinics participating in the NSBPR. We used descriptive statistics, the Fisher exact test, χ(2) test, and Wilcoxon rank-sum test to examine the data. RESULTS The mean age was 10.1 (SD 8.1) years with slightly more female subjects (52.5%). The majority was white (63.4%) and relied upon public insurance (53.5%). One-third had sacral lesions, 44.8% had mid-low lumbar lesions, and 24.9% had high lumbar and thoracic lesions. The most common surgery was ventricular shunt placement (65.7%). The most common bladder-management technique among those with bladder impairment was intermittent catheterization (69.0%). Almost 14% experienced a pressure ulcer in the last year. Of those ages 5 years or older with bowel or bladder impairments, almost 30% were continent of stool; a similar percentage was continent of urine. Most variables were associated with type of SB diagnosis. CONCLUSION The NSBPR provides a cross section of a predominantly pediatric population of patients followed in specialized SB programs. There were wide variations in the variables studied and major differences in Context of Care, Processes of Care, and Health Outcomes by type of SB. Such wide variation and the differences by type of SB should be considered in future analyses of outcomes.

Risk factors for lung function decline in a large cohort of young cystic fibrosis patients

To identify novel risk factors and corroborate previously identified risk factors for mean annual decline in FEV1% predicted in a large, contemporary, United States cohort of young cystic fibrosis (CF) patients.

Key findings of the US Cystic Fibrosis Foundation's clinical practice benchmarking project

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.

Antibiotic treatment of signs and symptoms of pulmonary exacerbations: A comparison by care site

Antibiotic treatment of cystic fibrosis pulmonary exacerbations is inconsistent. Previous research has indicated that intravenous antibiotics are used more frequently at sites with better pulmonary function but it is not clear under what circumstances they are prescribed.

Design and Methodological Considerations of the Centers for Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida

PURPOSE Care of children with spina bifida has significantly advanced in the last half century, resulting in gains in longevity and quality of life for affected children and caregivers. Bladder dysfunction is the norm in patients with spina bifida and may result in infection, renal scarring and chronic kidney disease. However, the optimal urological management for spina bifida related bladder dysfunction is unknown. MATERIALS AND METHODS In 2012 the Centers for Disease Control and Prevention convened a working group composed of pediatric urologists, nephrologists, epidemiologists, methodologists, community advocates and Centers for Disease Control and Prevention personnel to develop a protocol to optimize urological care of children with spina bifida from the newborn period through age 5 years. RESULTS An iterative quality improvement protocol was selected. In this model participating institutions agree to prospectively treat all newborns with spina bifida using a single consensus based protocol. During the 5-year study period outcomes will be routinely assessed and the protocol adjusted as needed to optimize patient and process outcomes. Primary study outcomes include urinary tract infections, renal scarring, renal function and bladder characteristics. The protocol specifies the timing and use of testing (eg ultrasonography, urodynamics) and interventions (eg intermittent catheterization, prophylactic antibiotics, antimuscarinic medications). Starting in 2014 the Centers for Disease Control and Prevention began funding 9 study sites to implement and evaluate the protocol. CONCLUSIONS The Centers for Disease Control and Prevention Urologic and Renal Protocol for the Newborn and Young Child with Spina Bifida began accruing patients in 2015. Assessment in the first 5 years will focus on urinary tract infections, renal function, renal scarring and clinical process improvements.

Early Life Growth Trajectories in Cystic Fibrosis are Associated with Pulmonary Function at Age 6 Years

OBJECTIVE To determine whether severity of lung disease at age 6 years is associated with changes in nutritional status before age 6 within individual children with cystic fibrosis (CF). STUDY DESIGN Children with CF born between 1994 and 2005 and followed in the CF Foundation Patient Registry from age ≤2 through 7 years were assessed according to changes in annualized weight-for-length (WFL) percentiles between ages 0 and 2 years and body mass index (BMI) percentiles between ages 2 and 6 years. The association between growth trajectories before age 6 and forced expiratory volume in 1 second (FEV1)% predicted at age 6-7 years was evaluated using multivariable linear regression. RESULTS A total of 6805 subjects met inclusion criteria. Children with annualized WFL-BMI always >50th percentile (N = 1323 [19%]) had the highest adjusted mean (95% CI) FEV1 at 6-7 years (101.8 [100.1, 103.5]). FEV1 at 6-7 years for children whose WFL-BMI increased >10 percentile points by age 6 years was 98.3 (96.6, 100.0). This was statistically significantly higher than FEV1 for children whose WFL-BMI was stable (94.4 [92.6, 96.2]) or decreased >10 percentile points (92.9 [91.1, 94.8]). Among children whose WFL-BMI increased >10 percentile points, achieving and maintaining WFL-BMI >50th percentile at younger ages was associated with significantly higher FEV1 at 6-7 years. CONCLUSIONS Within-patient changes in nutritional status in the first 6 years of life are significantly associated with FEV1 at age 6-7 years. The establishment of a clear relationship between early childhood growth measurements and later lung function suggests that early nutritional interventions may impact on eventual lung health.

Long-term Effects of Pregnancy and Motherhood on Disease Outcomes of Women with Cystic Fibrosis

RATIONALE Studies of pregnancy in cystic fibrosis (CF) have shown no short-term harmful effects, but there are no long-term studies on the impact of motherhood. OBJECTIVES This study sought to evaluate longer-term physiologic and functional outcomes in women with CF reporting a pregnancy, with the intent of assessing how the demands of parenting impacted on disease course. METHODS Using 1994 to 2005 Epidemiologic Study of Cystic Fibrosis data, we developed a propensity score to match women reporting a pregnancy at a 1:10 ratio with never-pregnant control subjects and compared clinical outcomes, health-related quality of life, and health care use. MEASUREMENTS AND MAIN RESULTS One hundred nineteen pregnant women presumed to have become mothers were matched with 1,190 control subjects, a median of 6.0 years (range 1.8-11.1 yr) from the pregnancy. No differences were found in annualized change from baseline FEV1 and body mass index, in respiratory signs and symptoms, or in prescribed chronic therapies. Women who had been pregnant were treated for more pulmonary exacerbations and had more illness-related clinic visits but showed no increase in prescribed chronic therapies. They also reported lower health-related quality-of-life scores for Respiratory Symptoms, Physical Functioning, Vitality, and Health Perceptions. CONCLUSIONS Pregnancy and motherhood do not appear to accelerate disease progression but lead to more illness-related visits, pulmonary exacerbations, and a decrease in some domains of quality of life. These differences presumably reflect the impact of the physical and emotional challenges of early motherhood on disease self-management.

Risk factors for mortality before age 18 years in cystic fibrosis

Understanding early‐life risk factors for childhood death in cystic fibrosis (CF) is important for clinical care, including the identification of effective interventions.

Rate of Uptake of Ivacaftor Use after U.S. Food and Drug Administration Approval among Patients Enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry.

RATIONALE Chronic cystic fibrosis (CF) therapies have variable rates of prescribed use, and therapies are rarely prescribed to more than 80% of eligible patients. Ivacaftor was approved in the United States in January 2012 for patients ages 6 years and older with a G551D mutation in their CF gene. OBJECTIVES To examine the rate of uptake and patterns of documented ivacaftor use among U.S. patients with CF during the first year after approval, to compare eligible patients with and without reported use, and to describe characteristics of early adopters of ivacaftor use. METHODS A cross-sectional study of patients in the U.S. Cystic Fibrosis Foundation Patient Registry in 2012 with at least one encounter in which ivacaftor use was documented. Ivacaftor-eligible patients were defined as any individual 6 years of age or older with a G551D mutation. We performed bivariate and multivariate regression analyses, stratified by age group, to compare clinical and demographic characteristics of (1) eligible patients with and without documented ivacaftor use in 2012 and (2) early (February-June) versus late (July-December) adopters in 2012. MEASUREMENTS AND MAIN RESULTS A total of 1,087 patients with CF with G551D mutations were in the U.S. Cystic Fibrosis Foundation Patient Registry in 2012. By June 2012, 64% of eligible patients had documented ivacaftor use, which increased to 81% by the end of 2012. Among eligible patients younger than 18 years of age, 85% were prescribed ivacaftor, with significantly lower odds among those with higher BMI percentile, fewer clinical encounters in 2011, and later age at diagnosis. Among eligible patients age 18 years or older, 79% were prescribed ivacaftor, with significantly lower odds in nonwhite patients and those with later age at diagnosis. Documented prescriptions of ivacaftor also varied by state of residence, with a range of 42-100% of eligible patients across states. The only association with early adoption of ivacaftor in 2012 was a decreased likelihood in adults with fewer than four encounters in 2011. CONCLUSIONS Uptake of ivacaftor use among eligible patients in the United States was rapid, with the majority of use initiated within 4 months of regulatory approval. Differences in ivacaftor prescriptions appear to be related to patient age, older age at diagnosis, and less frequent clinical encounters. Nutritional status also appears to play a role in children, and race seems to have an association in adults.