17 hours ago Annual Data Report 2020 Cystic Fibrosis Foundation Patient Registry 1 September 2021 Dear Friends and Colleagues: We are pleased to share the 2020 Patient Registry Annual Data Report. The impact of two significant events is visible in this report: first, the impact of the COVID-19 pandemic and, second, the availability of elexacaftor/ >> Go To The Portal
CFF’s 2020 patient registry annual data report found a significant decline in lung transplants among CF patients — 91 people received a transplant in 2020 compared with a high of 244 in 2019. Results for the 2017–2018 and 2018–2019 academic years are provided for comparison.
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This Annual Report is a testament to the large amount of information gathered by Cystic Fibrosis Centres and the innovative science surrounding the treatment of cystic fibrosis (CF).
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
Symptoms Reported at CF Diagnosis All Individuals (%) Diagnosed in 2020 (%) Diagnosed in 2020 Age < 1 (%) Diagnosed in 2020 Age ≥ 1 (%) Number of Individuals (n) 31,411 708 522 186 Asymptomatic
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.
© 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.
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.
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.
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.
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.
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.
The Registry Steering Committee (RSC) is responsible for making sure that the UK CF Registry is compliant with data protection legislation, and its Research Ethics Committee-approved Study Protocol. It also makes recommendations about the future development of the Registry. A sub-committee of the RSC, the Registry Research Committee, assesses applications for data and guides the Registry research strategy.
In 2019, of the deaths with a recorded cause of death (41 deaths; 83.7%), 80.5% of deaths were reported as pulmonary/infection/cardiovascular complications. 29 (59.2%) individuals with cystic fibrosis who died in 2019 had never received a transplant (any organ). FIGURE 47.
Since being founded by parents in 1960, Cystic Fibrosis Canada has grown into a leading organization with a central role engaging people living with cystic fibrosis, parents and caregivers, volunteers, researchers and healthcare professionals, government and donors.
In addition to the physical effects of the disease, mental health concerns are emerging; anxiety and depression are common among this population. Individuals with cystic fibrosis may reach the point where they require a lung transplant; most fatalities of people with cystic fibrosis are due to lung disease.