5 hours ago 6 Cystic Fibrosis Foundation Patient Registry Annual Data Report 2020 INTRODUCTION The CF Foundation Patient Registry (CFFPR) is composed of data collected via the CF Foundation Care Center Network, including participant demographic characteristics, routine clinical measurements, therapeutic history, hospitalizations, transplant, and vital status. >> Go To The Portal
The CF Foundation Patient Registry (CFFPR) is composed of data collected via the CF Foundation Care Center Network, including participant demographic characteristics, routine clinical measurements, therapeutic history, hospitalizations, transplant, and vital status.
The Patient Registry is an invaluable tool for researchers conducting observational studies about people with CF in the U.S. About 50,000 individuals have been followed in the Registry since its inception in 1986; many of them have been included for over 20 years. 4 min read
Information is reported by CF care teams following encounters with physicians, respiratory therapists, social workers, dietitians, and other team members as well as hospitalizations and home-based IV treatment.
The CFFPR contains data on almost 50,000 unique patients and has been used for research reported in over 120 peer-reviewed manuscripts, in addition to numerous quality improvement and benchmarking initiatives.
The CFFPR data are collected through a web-based portal, PortCF, which contains five electronic data capture forms: demographic, diagnosis, encounter, care episode, and annual review forms. All data are entered by staff at the care center programs from the data available in the medical record or in forms completed by patients or families. CFFPR questionnaires are available in the annual reports ( 2 ).
The CFFPR has been recognized as an example of a leading disease registry in the United States ( 24 ). We estimate there were approximately 33,292–34,327 individuals with CF in the United States in 2012. Approximately 81–84% of this estimated population was seen at a CF Foundation–accredited care center in 2012 and consented to have data included in the CFFPR.
Cystic fibrosis (CF) is a genetic disease that affects multiple organs in the body. It is the most common life-shortening recessive genetic disorder among white persons and occurs in approximately 1 in 3,500 births in the United States ( 1 ). Mutations in the cystic fibrosis transmembrane conductance regulator ( CFTR) gene lead to malfunctioning or absent CFTR protein, resulting in impaired mucosal clearance mechanisms that lead to recurring infections and obstruction in the lungs. As of 2014, the median predicted survival had increased to almost 40 years of age for individuals in the CF Foundation Patient Registry (CFFPR) from approximately 28 years in the late 1980s ( 2 ).
The Cystic Fibrosis Foundation Patient Registry was established in the 1960s and has continually evolved to keep pace with changes in technology and regulations, as well as improvements in the treatment of cystic fibrosis (CF).
Registries such as the CFFPR are important tools for collection of real-world data and are thus an ideal tool for conducting comparative effectiveness research (CER). Compared with randomized controlled trials, registry studies offer large study populations, long observational periods, and are less expensive, especially in a rare disease setting ( 36, 37 ). The CFFPR is beginning to be used for both CER research and pragmatic clinical trials ( 38 ). Increased interest in CER, linkages to other data sources, and international comparisons will continue to drive the need for high-quality observational data such as that included in the CFFPR.
Methods: Inclusion criteria for the CFFPR include diagnosis with CF or a CFTR-associated disorder, care at an accredited care center program, and provision of informed consent. Data from clinic visits and hospitalizations are collected through a secure website. Loss to follow-up and generalizability were examined using several methods. The accuracy of CFFPR data was evaluated with an audit of 2012 CFFPR data compared to the medical record.
In 2014, there were 28,676 people with CF included in the CFFPR, of whom 51.6% were male, 93.9% were white, 4.6% were African American, 3.1% were of another race, and 8.2% were Hispanic (of any race). An average of 4.5 clinic visits and 0.7 pulmonary exacerbations per patient were reported. A detailed summary of the 2014 registry population is available in the Patient Registry Annual Data Report ( 2 ).