Research investigating the actual situation of cystic fibrosis

DUBLIN, IRELAND, Thursday 30 July 2020 – Researchers from the RCSI School of Medicine and Health Sciences have launched a new study to investigate the real-world effectiveness of the latest cystic fibrosis drugs now available for people with CF. He is leading a new €3 million study.

The study will examine how the new triple cystic fibrosis treatment drug Kaftrio affects people in their daily lives, rather than in controlled clinical trial conditions. Kaftrio (known as TriKafta in the US) is the successor to Orkambi.

The project, called RECOVER, was awarded €2.85 million by the Cystic Fibrosis Foundation (USA), €112,000 from the Cystic Fibrosis Trust (UK) and €100,000 from Cystic Fibrosis Ireland.

In recent years, many promising new drugs have been developed to treat the underlying genetic defects of cystic fibrosis. Kaftrio, the latest triple-drug cystic fibrosis treatment, showed remarkable improvements in clinical trials in patients with the most common form of cystic fibrosis.

Pending final approval from the European Medicines Agency, which is expected to be approved in the coming weeks, these new medicines will be used to treat cystic fibrosis in Ireland and the UK as part of existing agreements between manufacturers and the states. will be made available for use by a large number of people.

The study will recruit patients from August 2020 and will begin investigating clinical outcomes over a two-year period across cystic fibrosis centers in Ireland and the UK. In addition to collecting routine health measurements, we will study detailed imaging, functional, biological and quality of life measurements that could not be collected in clinical trials.

“Our research allows us to discover in detail how this powerful new treatment affects the health and daily lives of people with cystic fibrosis, and to understand why different people respond differently to the drug. “It gives us insight into how this treatment affects patients themselves,'' says “The enormous treatment burden currently endured by cystic fibrosis patients.'' is a respiratory consultant and recovery researcher at Child Health Ireland.

“Clinical trials are conducted under very controlled conditions and exclude many people with cystic fibrosis. We will review the trial results and look at the detailed impact on the daily lives of all people with cystic fibrosis. “real world'' studies such as RECOVER are essential. . ”

In partnership with the Irish and UK Cystic Fibrosis Registries, RECOVER is led by an Irish research team and collaborates with researchers from 16 clinical and academic sites across the UK, Europe and North America.

Phillip Watt, CEO of Cystic Fibrosis Ireland, said: “Cystic Fibrosis Ireland provides a wealth of new detailed data on the impact of new and innovative CF treatments across Europe and North America. We are proud to support this large-scale study.”

Dr Lucy Allen, research director at the Cystic Fibrosis Trust, said: 'Clinical trial data shows that Kaftrio makes a huge difference to people's lives. However, CF affects many parts of the body. It is important to understand the full effects of this drug because of the complex symptoms it causes. The results of the study have the potential to accelerate access to Kaftrio worldwide in the short term, and in the long term, provide information on personalized care for individuals with cystic fibrosis. .

It will be led by RCSI and include collaborating institutions such as Imperial College, London; Children's Health Ireland (Crumlin, Tallaght, Temple Street, Connolly). National Child Research Center. St Vincent's University Hospital, Dublin. Limerick University Hospital; Belfast City Hospital. Royal Belfast Children's Hospital. Royal Brompton Hospital, London. Irish CF Registry, Dublin; UK CF Registry; Hospital for Sick Children (SickKids), Toronto. Eramsus MC, Rotterdam University Medical Center, Brandenburg Medical University, Germany. Queen's University Belfast. Teagasc Food Research Centre, Cork; Hospital for Sick Children, Toronto;

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Note to editor

• Paul McNally, Associate Professor of Pediatrics, RCSI School of Medicine and Health Sciences, is available for an interview.
• Photos can also be taken upon request.

About cystic fibrosis

• Cystic fibrosis (CF) is an inherited chronic disease that primarily affects the lungs and digestive system. The defective gene causes the body to produce abnormally thick mucus, which clogs the lungs, obstructs the pancreas, and shuts down the natural enzymes that help the body break down and absorb food.
• Ireland has the highest incidence of cystic fibrosis in the world, with approximately 1,400 people diagnosed with the disease. Some more severe forms of the disease also exist in Ireland.
• Approximately 1 in 19 people are thought to carry one copy of the altered gene that causes CF.
• People with CF experience a variety of symptoms, including frequent lung infections, persistent cough, wheezing, shortness of breath, and poor growth and weight gain despite having an appetite. Over time, CF patients accumulate significant damage and scar tissue in their lungs, leading to end-stage lung disease, possibly requiring a double lung transplant.
• According to the Irish Cystic Fibrosis Registry Annual Report 2018, between 2010 and 2018, an average of 40 people were diagnosed with CF each year.
• In 2018, there were 140 people with CF over the age of 40.
• In 2018, the median age of people with CF was 21 years. In other words, half of the 1,239 registrants were under the age of 21, and the other half were over the age of 21. However, an increasing number of people are now expected to live until their 30s, 40s, and beyond.
• The counties with the highest number of CF cases are Dublin (327), Cork (167), Limerick (73), Tipperary (60) and Galway (59). In 2018, there were a total of 1,129 cases and 529 people hospitalized with CF.

About RCSI University of Medicine and Health Sciences

• Ranked first in the world for health and wellbeing in the Times Higher Education (THE) University Impact Rankings 2020, RCSI School of Medicine and Health Sciences is an international not-for-profit university headquartered in Dublin.
• RCSI is dedicated to education and research to drive improved health for people around the world. It is included among the top 250 universities in the world in THE World University Rankings (2020) and its research ranks first in Ireland for citations. RCSI has achieved Athena Swan Bronze accreditation for positive gender practices in higher education.
• Visit the RCSI MyHealth Expert Directory to find out more about experts on a range of medical issues and concerns. These clinicians and researchers recognize their responsibility to share their knowledge and discoveries and provide information that improves human health outcomes, and they actively engage with the media in their areas of expertise.

About cystic fibrosis in Ireland

• Cystic Fibrosis Ireland (CFI) is the leading national organization dedicated to improving the care and facilities for people with CF and their families across Ireland. This organization is dedicated solely to the well-being of people with CF in Ireland.
• Its mission is to support the development of means to treat and control CF, to promote the interests and welfare of people with CF in Ireland, and to act as an advocate for people with CF and their needs.
• This is done through providing information and education, advice and advocacy to people and their families, carrying out medical and scientific research, promoting the development of lung transplantation in Ireland and providing free assistance to patients.
• CFI also funds many specialist CF clinical positions and provides capital funding for the development of state-of-the-art CF units across the country, including dedicated inpatient, day care, and outpatient facilities.
• CFI is comprised of 18 chapters and one local chapter, overseen by a national executive committee that includes representatives of CF patients and their families. Learn more here http://www.cfireland.ie

About Cystic Fibrosis Trust

• The Cystic Fibrosis Trust is the only UK-wide charity dedicated to fighting for the infinite lives of everyone living with cystic fibrosis.
• For confidential advice, support and information on all aspects of Cystic Fibrosis, including financial support, please contact the Cystic Fibrosis Trust Helpline on (+44) 0300 373 1000 or 020 3795 2184.
• Our work is only made possible by generous donations from our supporters. visit http://www.cysticfibrosis.org.uk Find out more about cystic fibrosis, the Trust's work and how you can support our fight for limitless lives.