Developments in Cystic Fibrosis Paediatric Care

by Philip Watt, CEO Cystic Fibrosis Ireland

“Heath research remains a vital part of shaping clinical services. As part of the HRCI/HRB joint funding scheme, CFI is currently funding 2 major CF related research projects focussing on the transition form paediatric to adult services and CF related liver disease”

This is a time of hope and increased expectation for the health and future all people with cystic fibrosis in Ireland, especially young people and children. In my capacity as CEO of Cystic Fibrosis Ireland I have been meeting up with our many voluntary branches around the country in recent weeks as COVID-19 subsides, but still remains a concern. The general feedback of parents from these meetings is recognition of the significant progress in CF care in Ireland in recent years, with still more to do.

Much of the initial push for the concept of CF paediatric services in the 1960’s came from Professor Colman Saunders and his daughter Anne O’Dwyer, who was then a mother of twins with CF. Anne became the first chairperson of the CF Association of Ireland (CFAI) in 1963 – an early example of partnership in effective patient advocacy, a concept that remains important to this day.

Paediatric CF services commenced in Ireland in 1969 with the opening of a modest CF unit in the Children’s Hospital in Crumlin, with initial seed funding provided by Cystic Fibrosis Ireland. As this centre proved effective, it gained support beyond the board of Crumlin hospital. Because of support from the Department of Health, the first CF paediatric consultant in Ireland, Dr. Edward Tempany, was appointed in 1971.

Because of the severity of CF and poor expectations of survival during this period, it was not until 1976 that the first CF adult consultant was appointed – Dr. Muiris Fitzgerald in St Vincent’s Hospital, Dublin. These two much respected clinicians are fondly remembered to this day. The challenges they faced in delivering CF services at this time were immense because of major gaps in facilities and medications and CF research was still at an early stage.

Major developments in CF paediatric care in more recent years includes: The addition of CF to the newborn screening Programme (2011) and the emergence of a network of specialised and shared care paediatric centres around the country that followed on from the recommendations of the CFI commissioned the Pollock Report that was subsequently updated and replaced with the Model of Care for CF in 2019 published by the National Clinical Programme for CF, and its steering group, which includes CFI.

Of further crucial importance in the early years of CF paediatric care in Ireland was the emergence the first range of effective medications. These drug therapies include: Creon which is an enzyme that aids the digestion of food (1992); Pulmozyme, which helps thins CF related mucus (1994) and the nebulised Tobramycin that was the first effective antibiotic against Pseudomonas (1998).

While these medications remain vital in CF care to this day, most focus and expectations are now on a range of drug therapies from Boston based company Vertex. These CFTR modulators commenced with Kalydeco (2013) and Orkambi (2017) and more recently Kaftrio (2020). Kaftrio in particular has demonstrated very significant increases in lung function and a reduction in hospitalisations that have been confirmed through real world data.

Thanks to a range of factors over the past decade, CF is now a much more manageable long term disease. The outlook for those with CF, particularly young people with CF, has never being so positive. This is reflected in the increasing positive data from the CF Registry of Ireland set up by CFI in 2002 and which became independent in 2005 and is based in UCD.

These improvements have come about through the partnership of clinicians; HSE funded CF specialised and shared care hospitals; the CF related health research undertaken and supported by many.

Cystic Fibrosis Ireland, the national patient organisation has played a significant role in all these developments since our formation in 1963, as have at times individual patient advocates/spokespeople.

The new exciting CFTR Modulators address the underlying cause of cystic fibrosis, not just the symptoms. With some younger patients in particular, the impact of these drug therapies is so powerful that the sweat test used to diagnose CF at birth can sometimes return a negative result.

It is distressing that as a result of a dispute between Vertex and the HSE, 35 children with CF remain excluded from the drug therapy kaftrio that has helped revolutionise CF care in Ireland in recent years. This exclusion stems from a difference in interpretation of the innovative 2017 portfolio agreement for CFTR modulators and CFI continues to advocate resolution as soon as possible.

The next key infrastructure development in CF paediatric care will be the opening of the forthcoming Children’s Hospital Ireland in the St James campus.

CHI will host the merger of the 3 existing Dublin CF centres in Tallaght, Temple St and Crumlin and will herald in another new era if CF care for children and young people with CF.

Heath research remains a vital part of shaping clinical services. As part of the HRCI/HRB joint funding scheme, CFI is currently funding 2 major CF related research projects focussing on the transition form paediatric to adult services and CF related liver disease. CFI is also a partner in the CF Recover research project looking at real world outcomes from CFTR modulators, led by Professor Paul McNally (CHI Crumlin).

Finally, CFI wishes to thank the excellent CF clinical teams; the National Clinical Programme for CF; the HSE and most of all the thousands of people who support our work as an association as we look forward to celebrating 60 years of advocacy for better CF services and outcomes for people with CF in Ireland.

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