cystic fibrosis foundation patient registry annual data report

What is the cystic fibrosis patient registry?

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 people with cystic fibrosis were diagnosed in 2018?

Therefore the number of patients identified in 2018 is higher (167) in this report than was recorded in the previous. 887 (14.9%)of adults with CF in the Registry in 2019 were diagnosed at age 16 or over. In 2019, 28people aged 16 or over were newly diagnosed with cystic fibrosis.

What data is included in the CF Foundation patient registry?

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.

Where is the CF Foundation annual data report based?

ABOUT THIS REPORT The Annual Data Report is based on data entered in the CF Foundation Patient Registry through our online portal, PortCF©. Data are entered by teams of dedicated health professionals in our nationwide network of more than 120 CF Foundation-accredited Care Centers. Inclusion Criteria

What is CF 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 much is the CF Foundation worth?

For $3.3 billion. Suddenly, the CF Foundation was the largest disease-focused charity in the country as measured by net assets. Most medical charities don't get any money from the research they fund, and none had ever gotten a windfall so big.

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 Port CF?

Port CF is a New Zealand data registry owned by CFNZ. It uses anonymous patient data to analyse trends in CF care. These reports can be compared to CF registries in the UK and Australia to help determine how NZ compares to other countries. They also provide information to help CFNZ lobby for new treatments. Download.

Is Cystic Fibrosis Foundation a good charity?

(Bethesda, Md.) -- The Cystic Fibrosis Foundation announced today it has received a coveted 4-star rating for sound fiscal management from Charity Navigator, the largest charity evaluator in the United States. The Foundation earned four out of four possible stars for achieving excellence in fiscal responsibility.

Is CFF org a nonprofit?

The Cystic Fibrosis Foundation (CFF) is a voluntary, non-profit organization. Its mission is to assure the development of the means to cure and control cystic fibrosis (CF) and to improve the quality of life for those with the disease.

Who is the CEO of Cystic Fibrosis Foundation?

Michael Boyle, MD, is president and chief executive officer of the Cystic Fibrosis Foundation. Prior to his appointment as president and CEO, Dr. Boyle served as the Foundation's senior vice president, therapeutics development from 2015-2019.

Is there a cure for cystic fibrosis 2022?

FRIDAY, Feb. 18, 2022 (HealthDay News) -- Hundreds of new proteins that may be linked to cystic fibrosis have been identified by researchers and could point the way to better treatments for people with the genetic disease. There is no cure for cystic fibrosis, which affects more than 90,000 people worldwide.

When was the Cystic Fibrosis Foundation website made?

Cystic Fibrosis FoundationFoundedDecember 16, 1955Employees (2016)731Volunteers (2016)250,000Websitewww.cff.orgFormerly calledNational Cystic Fibrosis Research Foundation11 more rows

What percentage of the population has cystic fibrosis?

1 in 29 Caucasian-Americans. 1 in 46 Hispanic-Americans. 1 in 65 African-Americans.

What is the life expectancy of cystic fibrosis?

Outlook (Prognosis) Lung disease eventually worsens to the point where the person is disabled. Today, the average life span for people with CF who live to adulthood is about 44 years. Death is most often caused by lung complications.

Why do CF patients have ports?

Ports can be used by people with CF who require long-term, frequent intravenous (IV) antibiotic treatment or who have poor venous access ('difficult veins'). Ports are not usually considered for people with CF who have short-term treatment needs or who have infrequent courses of IV antibiotics.

What is the Cystic Fibrosis Trust?

© Cystic Fibrosis Trust 2020. Registered as a charity in England and Wales (1079049) and in Scotland (SC040196). A company limited by guarantee, registered in England and Wales number 3880213.

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 www.cysticfibrosis.org.uk/registry.

How many copies of F508del are there in tezacaftor?

Tezacaftor/ivacaftor is licenced for use in patients aged 12 and over who have two copies of the F508del mutation, or a single copy of F508del and one of 14 specified ‘residual function’ mutations. Managed access agreements were agreed to make tezacaftor/ivacaftor available on the NHS in Scotland from September 2019, followed by England, Wales and Northern Ireland from November 2019.

When was Ivacaftor first approved?

Ivacaftor was first approved for use on the NHS in England in January 2013. Soon after, it was made available in Wales, Scotland and Northern Ireland. Since this time, ivacaftor’s license has expanded across age ranges and mutation types. As of 2019, ivacaftor was approved for use on the NHS across the UK for people aged one and older with at least one copy of 9 specified CFTR ‘gating’ mutations, and for people aged 18 and over with the R117H mutation. In June 2020, the marketing authorisation was extended to cover anyone aged 6 months and over with the R117H mutation.

What is a genotype in CF?

Genotypes are part of the genetic makeup of an individual that usually control a particular characteristic, known as a phenotype. For people with CF, their genotype reveals which mutations of the CF gene cause their cystic fibrosis. Everyone living with CF has two mutations of the gene for CFTR; one on each allele. One is inherited from their mother, and one from their father. If both mutations (or genotypes) are the same, the person is said to be homozygous. Someone who has two different variants is heterozygous.

How many people died from CF in 2019?

The table below shows the ages of the 137 people with CF who died in 2019. In 2019 the median age of the 114 people who died was 31. Median age of death is based on the people with CF who died in any given year.

What is the Venn diagram for CF?

The Venn diagram shows how many people with CF are on one or more inhaled therapy and the combinations they take. 1960 (19.5%) people are on no inhaled therapies.

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.

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 ).

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.

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 …