cystic fibrosis foundation patient registry annual data report 2014

What is the CF Foundation patient registry?

The CF Foundation Patient Registry collects information on the health status of people with cystic fibrosis who receive care in CF Foundation-accredited care centers and agree to participate in the Registry.

What do CF Foundation-accredited care centers do?

CF Foundation-accredited care centers play an important role in research. They collect information on the health status of their patients with CF who agree to participate, and report that data to the CF Foundation Patient Registry.

Are people with cystic fibrosis living longer lives?

People with cystic fibrosis continue to live longer and healthier lives, and the Patient Registry data support this general trend. To understand what this means for our community, however, it is important to understand how these numbers are calculated and what they represent.

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How do you cite the cystic fibrosis Foundation?

Cite This ItemChicago citation style: Cystic Fibrosis Foundation. Cystic Fibrosis Foundation . United States, 2002. ... APA citation style: Cystic Fibrosis Foundation. (2002) Cystic Fibrosis Foundation . United States. ... MLA citation style: Cystic Fibrosis Foundation. Cystic Fibrosis Foundation . United States, 2002.

What is the cystic fibrosis Registry?

The CF Foundation Patient Registry collects information on the health status of people with cystic fibrosis who receive care in CF Foundation-accredited care centers and agree to participate in the Registry.

How many people in the US have cystic fibrosis?

According to the Cystic Fibrosis Foundation Patient Registry, in the United States: There are close to 40,000 children and adults living with cystic fibrosis in the United States (and an estimated 105,000 people have been diagnosed with CF across 94 countries).

How common is cystic fibrosis Worldwide?

Worldwide, about 70,000 to 100,000 people have cystic fibrosis.

Who is the most famous person with cystic fibrosis?

9 Famous Cystic Fibrosis PatientsAlexandra Deford. Alexandra Deford is the daughter of the sports writer Frank Deford and she died in 1980 at just eight-years-old. ... Alice Martineau. ... Andrew Simmons. ... Bob Flanagan. ... Fredric Chopin. ... Gregory Lemarchal. ... Gunnar Esiason. ... Celine Dion's niece Karine.More items...•

What is the 6 foot rule with cystic fibrosis?

Germs can spread as far as 6 feet when someone coughs or sneezes, landing on surfaces or in another person's eyes, nose, or mouth. That's why it's important for people with CF to stay at least 6 feet away from others with CF and anyone with a cold, flu, or infection.

What country has the most cases of cystic fibrosis?

CF in the U.S. The U.S. is among countries with the highest incidence of CF, with about 30,000 people currently living with the disease.

Which state has the highest rate of cystic fibrosis?

As of 2017, the U.S. states with the largest numbers of people living with CF are California (2,386), Texas (2,048), New York (1,644), and Florida (1,599). Cystic fibrosis is most common among Caucasians. In the U.S., the chances of being a carrier of a CFTR mutation are: 1 in 29 Caucasian-Americans.

What gender is cystic fibrosis most common in?

How Are Men Affected by Cystic Fibrosis? Males account for slightly more than 50 percent of all cases of cystic fibrosis (CF) but generally have better outcomes than females until about age 20.

What are 3 interesting facts about cystic fibrosis?

Top 5 Things You (Probably) Don't Know About Cystic FibrosisPeople with CF can't be together. ... CF and Tay Sachs are tied as the most fatal Jewish genetic diseases. ... Our skin is super salty. ... We are master deceptors. ... The nickname for CF is 65 roses.

What celebrities have cystic fibrosis?

List of people diagnosed with cystic fibrosisNameLifeChristopher Davies(1978—)Alexandra Deford(1971–1980)Gunnar Esiason(1991—)Bob Flanagan(1952–1996)25 more rows

Who has lived the longest with cystic fibrosis?

Marlene Pryson, 86, probably one of the oldest individuals living with cystic fibrosis, certainly possesses all of these characteristics.

Is cystic fibrosis testing mandatory?

Healthcare providers screen newborns for cystic fibrosis and several other conditions via a blood test while the baby is still in the hospital. This blood test is mandatory for all babies born in New York State and usually occurs when the infant is one day old.

What is the longest someone with cystic fibrosis has lived?

Thanks to advances in DNA testing, doctors are identifying more and more people with CF for the first time well into their 50s, 60s, and 70s. The oldest person diagnosed with CF for the first time in the U.S. was 82, in Ireland was 76, and in the United Kingdom was 79.

What is a chronic disease registry?

A disease registry is a special database that contains information about people diagnosed with a specific type of disease. Most disease registries are either hospital based or population based.

Are you considered disabled if you have cystic fibrosis?

People living with cystic fibrosis may apply for Social Security Disability Insurance or Supplemental Security Income, programs that serve as a source of income and health insurance coverage for people who are unable to work due to their health status.

What is the UK CF registry?

The UK CF Registry has been sponsored and hosted by the Cystic Fibrosis Trust since 2007. It is a database of consenting people with CF in the UK. The Registry collects demographic, treatment, and health outcomes data. You can find a full list of the data items we collect at

Who is included in the CF report?

This includes people with CF, their families and clinical teams, healthcare managers, commissioners, and policy makers.

What is the RSC in the UK?

The Registry Steering Committee (RSC) (Appendix 3) is responsible for making sure that the UK CF Registry is compliant with legislation like the Data Protection Act 1998, and its Research Ethics Study Protocol. The RSC also assesses applications for data, and makes recommendations about the future development of the Registry.

How old were people in 2014 when they were diagnosed?

The table below shows the age of people aged 16 and over in 2014 when they were diagnosed. These people were born before newborn screening was done routinely across the UK. There were some regions with newborn screening prior to 2007.

What is the cause of cystic fibrosis?

When the gene is faulty, the lungs can become clogged with mucus over time . This damages the lungs. Around 85% of people with cystic fibrosis also have difficulty digesting food.

When did Ivacaftor start?

Ivacaftor is a drug that began being prescribed as a treatment for cystic fibrosis in patients aged 6 years and over with at least one copy of the genotype G551D, in June 2012. The table shows ivacaftor use and outcomes from June 2012 – December 2014.

Is cystic fibrosis related to diabetes?

Cystic fibrosis-related diabetes (CFRD) is common in adults and adolescents with cystic fibrosis. This is because, for many people with CF, the pancreas does not work properly. This can mean that not enough insulin is produced, causing CFRD. CFRD is different from type 1 and type 2 diabetes, but has features of both.

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.

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 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.

What does CF stand for in medical terms?

Definition of abbreviations: CF = cystic fibrosis; MRSA = methicillin-resistant Staphylococcus aureus; MSSA = methicillin-sensitive Staphylococcus aureus. An audit of Cystic Fibrosis Foundation Patient Registry (CFFPR) data suggests high accuracy and low missingness of most variables compared with the medical record.

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.

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 PEx in CF?

Pulmonary exacerbations (PEx) in cystic fibrosis (CF) are a frequent cause of hospitalisations and lead to long-term decline in pulmonary function. Successful CF inpatient care requires the coordination of multiple providers and complex therapies. Children's Hospital of Wisconsin (CHW) and Children's Healthcare of Atlanta (CHoA) independently identified PEx inpatient care for focused improvements, with emphasis on improving care coordination and patient outcomes. Both centres began by forming multidisciplinary workgroups, including patient and family representatives. CHW's specific aim was to eliminate delays in the time to initial intravenous antibiotics. A written handoff tool was developed to allow more efficient ordering. Efforts at CHoA focused on coordination and consistent care delivery. A written schedule and patient incentive programme were devised to ensure proper administration of treatments and promote patient adherence. At CHW, interventions decreased the mean antibiotic order time by 59% with resultant decrease in administration time by 25%. At CHoA, improvements led to a 42% decrease in the proportion of hospitalisations unsuccessful in returning lung function back to within 90% of baseline. Inpatient CF PEx care is complex and requires multiple competing activities and treatments. Consistent and timely delivery of these treatments is challenging. Our improvements used the skills and insights of providers and patients to improve, standardise and synchronise care, and to develop tools to coordinate hand offs. With these improvements, applicable to hospital treatment of many other conditions, both centres were successfully able to deliver treatments in a more consistent and timely manner with improved outcomes.

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.

Does tobramycin help with CF?

Though tobramycin inhalation solution has been used for over a decade to improve lung function and reduce exacerbations in patients with cystic fibrosis ( CF), its effects on mortality have not been well-described. This study aimed to assess the association between use of tobramycin inhaled solution and mortality in patients with CF and chronic Pseudomonas aeruginosa (PA) infection. Longitudinal logistic regression was used to assess the association between current-year reported use of tobramycin inhalation solution and subsequent-year mortality of patients meeting recommended criteria for tobramycin inhalation solution use in the United States Cystic Fibrosis Foundation's Patient Registry (1996-2008). Among 12,740 patients meeting inclusion criteria, 2,538 deaths were observed during a median follow-up of 6 years. After regression adjustment, use of tobramycin inhaled solution was associated with a 21% reduction in the odds of subsequent year mortality (odds ratio (95% CI): 0.79 (0.72-0.88), P < 0.001). In our model, use of dornase alfa was also associated with a 15% reduction in the odds of subsequent year mortality (odds ratio (95% CI): 0.85 (0.76-0.95), P = 0.005). Underweight for age, CF-related diabetes, female gender, worse lung function and cultures positive for Pseudomonas aeruginosa or Burkholderia cepacia complex, among multiple other patient characteristics, were associated with significantly increased mortality. Adjusted mortality rates for patients reporting tobramycin inhalation solution use in all versus none of the follow-up years were 1.3% versus 2.1% at 2 years, 5.2% versus 8.0% at 5 years, and 9.9% versus 15.0% at 10 years. After adjustment for multiple patient characteristics and known risk factors, use of tobramycin inhalation solution was associated with significantly reduced mortality among patients with CF.