cystic fibrosis foundation patient registry: 2013 annual data report. bethesda, maryland

What is in the annual data report for cystic fibrosis?

This Annual Data Report contains data from individuals diagnosed with CF who (a) have consented to participate in the Registry, and (b) were seen in a CF care center during the 2019 calendar year, including those who were born, diagnosed, or died in the year.

What is the cystic fibrosis patient registry?

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. Patient Registry Data Requests | Cystic Fibrosis Foundation

What is the average age of people with cystic fibrosis?

Annual Data Report 2019Cystic Fibrosis Foundation Patient Registry 9 The median age of people with CF currently in the Registry is 20.3 years. The range is from birth to 89.7 years.

What is the prevalence of mycobacterial infections in cystic fibrosis (CF)?

Annual Data Report 2019Cystic Fibrosis Foundation Patient Registry 35 Of the 15,497 individuals who had a mycobacterial culture performed in 2019, 2,149 (13.9 percent) had a mycobacterial species isolated one or more times.

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What is the Breathe Deep for Cystic Fibrosis event?

Celebrating ongoing success, the Breathe Deep for CF social event has established awareness of Cystic Fibrosis and the importance of exercise in both the Boston and northern New Jersey area. An impressive 100% of funds raised in 2013 through admission, general donations and silent auction have been used to award Recreation Grants to people with CF across the country. In the five years the event has occurred in Boston, and two years in NJ, Breathe Deep for CF has generated over $40,000 for the CFLF.

Where is Rod from Cystic Fibrosis?

Rod was born and raised in Hawaii, and was diagnosed with CF at the age of three months. He received a Bachelors degree from the University of Hawaii, and lived in Chicago, where he earned an MBA from DePaul University. He now work as a Business Analyst at Bank of Hawaii. Rod is am extremely proud to display the talents of CF patients through the website Cystic Fibrosis Artists, a space dedicated to publicizing the gifts of those of us in this community. This website is intended to show the world that people with CF are a group to be recognized and respected, despite their challenges. Rod understands the importance of exercise for those who have CF, and how a faithful regimen can lead to a healthy, productive, and fulfilling life. He is excited to work with this team at CFLF to promote this lifestyle throughout the CF community.

How many states have been helped by CFLF?

People in 44 states and Canada have been helped by CFLF. Click on the upper right menu box in the map to see the locations of recipients by year since 2007!

How much did the Flatley Foundation raise in 2012?

The commitment of the Flatley Foundation with such a sizable matching grant pushed the CFLF fundraising efforts into high gear to surpass the 2012 fundraising and grant income by more than $58,000.

When was the Flatley Foundation matching challenge?

With the Flatley Foundation leading the charge on this amazing matching challenge for 2013, the ongoing support of philanthropists, grassroots fundraising and grants from foundations and corporations was able to surpass all previous years. The match was received in February 2013, and was met by the CFLF in October, only eight months into the challenge.

Does CFLF give anonymously?

CFLF would also like to extend acknowledgement and gratitude to the many other organizations that generously contribute financial and in-kind support anonymously. In particular, the majority of professionally printed materials was donated 100% through an anonymous corporate contribution.

When was the Cystic Fibrosis Foundation established?

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).

How many patients were seen at CF Foundation in 2012?

As reported by the care centers during the annual reaccreditation process, there were 1,875 patients who were seen at accredited care centers in 2012 who did not provide consent to participate in the registry. This represents 6.3% of patients seen at CF Foundation–accredited care centers in 2012.

How is CFFPR used?

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 captures a substantial portion of the U.S. patient population with CF and has robust and high-quality data in key variables of interest, such as lung function, nutritional status and hospitalizations. Data in the registry have been used for many years to compare center-level variation in care and outcomes. As CF registries are implemented in other countries, the CFFPR has also been used to compare treatment and outcomes between the United States and other countries ( 35 ). International comparisons leverage variation in availability and physician preference of therapies and can be facilitated with standardization of data collection across registries.

How many people in the US have CF in 2012?

On the basis of the two methods of estimating the number of persons with CF in the United States using national birth and death data, we derived estimates of 33,292 and 34,327 individuals with CF in the United States in 2012, respectively. In 2012, the CFFPR contained 27,804 individuals. Thus, approximately 81–84% of persons with CF were captured in the CFFPR in 2012, the most recent year for which national birth and mortality data were available.

How is CFFPR data collected?

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 ).

What is CF Foundation?

All individuals diagnosed with CF and associated disorders (CFTR-related metabolic syndrome and CFTR-related disorders) who are seen at CF Foundation–accredited care center programs and provide informed consent are eligible to participate in the CFFPR. The CF Foundation has developed and sustains a network of 121 accredited CF care centers (comprised of 121 pediatric care programs and 105 adult care programs) and 51 affiliate programs across the United States ( see additional information available in the online supplement). All accredited care programs are required to participate in the CFFPR. A portion of care center funding is based on the number of patients enrolled in the CFFPR and the completeness of their records. Each program obtains institutional review board approval and written informed consent and assent, as appropriate, from participants and/or their legal guardians. The CF Foundation provides online user manuals, data entry guidelines, training sessions, and user support for care center staff who enter data into the CFFPR. The CF Foundation serves as the coordinating center for data collection and data analysis.

When was CFFPR established?

The CFFPR was established in the 1960s to collect information on patient demographics and survival ( 3 ). Regular updates have been made to comply with changes in regulations, improvements in data collection, and advances in new technology ( Figure 1) ( 4 ).

How many people have CF?

Background Cystic fibrosis (CF) affects >70,000 people worldwide, yet the microbiologic trigger for pulmonary exacerbations (PExs) remains unknown. The objective of this study was to identify changes in bacterial metabolic pathways associated with clinical status. Methods Respiratory samples were collected at hospital admission for PEx, end of intravenous (IV) antibiotic treatment, and follow-up from 27 hospitalized children with CF. Bacterial DNA was extracted and shotgun DNA sequencing was performed. MetaPhlAn2 and HUMAnN2 were used to evaluate bacterial taxonomic and pathway relative abundance, while DESeq2 was used to evaluate differential abundance based on clinical status. Results The mean age of study participants was 10 years; 85% received combination IV antibiotic therapy (beta-lactam plus a second agent). Long-chain fatty acid (LCFA) biosynthesis pathways were upregulated in follow-up samples compared to end of treatment: gondoate ( p = 0.012), oleate ( p = 0.048), palmitoleate ( p = 0.043), and pathways of fatty acid elongation ( p = 0.012). Achromobacter xylosoxidans and Escherichia sp. were also more prevalent in follow-up compared to PEx ( p < 0.001). Conclusions LCFAs may be associated with persistent infection of opportunistic pathogens. Future studies should more closely investigate the role of LCFA production by lung bacteria in the transition from baseline wellness to PEx in persons with CF. Impact Increased levels of LCFAs are found after IV antibiotic treatment in persons with CF. LCFAs have previously been associated with increased lung inflammation in asthma. This is the first report of LCFAs in the airway of persons with CF. This research provides support that bacterial production of LCFAs may be a contributor to inflammation in persons with CF. Future studies should evaluate LCFAs as predictors of future PExs.

What is the CFF?

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 registry's 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 …

What is CF in Cyprus?

Background Specialized clinical care for cystic fibrosis (CF) in Cyprus, a small island country, has been implemented since the 1990s. However, only recently, a national CF patient registry has been established for the systematic recording of patients’ data. In this study, we aim to present data on the epidemiological, genotypic and phenotypic features of CF patients in the country from the most recent data collection in 2019, with particular emphasis on notable rare or unique cases. Results Overall, data from 52 patients are presented, 5 of whom have deceased and 13 have been lost to follow-up in previous years. The mean age at diagnosis was 7.2 ± 12.3 years, and the mean age of 34 alive patients by the end of 2019 was 22.6 ± 13.2 years. Patients most commonly presented at diagnosis with acute or persistent respiratory symptoms (46.2%), failure to thrive or malnutrition (40.4%), and dehydration or electrolyte imbalance (32.7%). Sweat chloride levels were diagnostic (above 60 mmol/L) in 81.8% of examined patients. The most common identified mutation was p.Phe508del (F508del) (45.2%), followed by p.Leu346Pro (L346P) (6.7%), a mutation detected solely in individuals of Cypriot descent. The mean BMI and FEV1 z-scores were 0.2 ± 1.3 and − 2.1 ± 1.7 across all age groups, respectively, whereas chronic Pseudomonas aeruginosa colonization was noted in 26.9% of patients. The majority of patients (74.5%) were eligible to receive at least one of the available CFTR modulator therapies. In 25% of patients we recovered rare or unique genotypic profiles, including the endemic p.Leu346Pro (L346P), the rare CFTR-dup2, the co-segregated c.4200_4201delTG/c.489 + 3A > G, and the polymorphism p.Ser877Ala. Conclusions CF patient registries are particularly important in small or isolated populations, such as in Cyprus, with rare or unique disease cases. Their operation is necessary for the optimization of clinical care provided to CF patients, enabling their majority to benefit from evolving advances in precision medicine.