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Better therapies making CF more manageable

By Liz Jacques - 25th Jun 2023

Ms Liz Jacques, Regional Development Officer, Cystic Fibrosis Ireland, reports on the organisation’s recent Annual Conference

It is widely recognised that there has been more progress in cystic fibrosis (CF) care in Ireland over the past decade than any other decade since the formation of Cystic Fibrosis Ireland (CFI) in 1963. At CFI’s 2023 Annual Conference celebrating 60 years of the charity, held on 1 April, in the Dunboyne Castle Hotel, Co Meath, Mr Philip Watt, CFI CEO, and keynote speaker Prof Charles Gallagher, Consultant Respiratory Physician, St Vincent’s University Hospital (SVUH), Dublin, both highlighted the specific progress made and future priorities for care. There was a consistently positive message throughout the conference – that for many people living with CF (PWCF), it has become a more manageable disease with better therapies and improved care.

In 2016 a significant milestone in CF care was reached with the creation of the National Clinical Programme for Cystic Fibrosis (NCPCF). Prof Gallagher, who chairs the NCPCF, explained the intention of the NCPCF is to enlist the broad expertise of CF care across Ireland from all disciplines to make real and effective changes in CF care, in partnership with the HSE. Most crucially is the need to ‘future-proof’ the guidance delivered by the NCPCF in recognition that the CF population will continue to grow and evolve in years to come.

Prof Gallagher highlighted the future priorities of the NCPCF, which include a strong emphasis on the importance of CF psychology services. The NCPCF has recommended a national CF psychology service be developed in response to the increasing need for CF-specialist mental health support. In addition, there is a drive to ensure a CF psychologist is available within each CF centre. The risk of, and impact of, depression and anxiety in PWCF was a repeated theme throughout the conference agenda and a clear priority for all clinicians present. 

Treatment adherence, mental health, and quality-of-life in CF

International guest speaker, Dr Alexandra Quittner, Clinical Psychologist and Epidemiologist from Joe DiMaggio Children’s Hospital, Florida, US, presented a passionate and knowledgeable talk on her work in self-management, mental health, and quality-of-life for PWCF. Dr Quittner has over 30 years’ experience working in the field of CF, and strongly feels the new era of modulator therapies in CF is bringing increasing opportunities for improving adherence and managing mental health.  

Facilitating treatment adherence requires a multifaceted approach, taking into account an individual’s knowledge of disease and management, skill level, treatment plan, barriers to adherence, and problem-solving ability. Among the key tips for improving adherence is problem-solving, particularly among adolescent patient groups. Dr Quittner recommended problem-solving tools and techniques becoming part of the psychological interventions provided during clinic. These could be delivered within a tailored cognitive behavioural therapy (CBT) programme (Quittner et al, JCF 2019).

The rise of Kaftrio (elexacaftor, tezacaftor, ivacaftor (ETI)) and other modulator therapies may bring increased opportunities for personalised prescribing and a reduction in treatment burden for PWCF. However, a recurrent theme of both Dr Quittner’s and Consultant Respiratory Physician at SVUH Prof Ed McKone’s presentations was the reduced adherence to inhaled medications in patients who are prescribed ETI. Factors that influence adherence to any medication are varied, but may include age and developmental stage, knowledge of treatments, skill level, a patient’s mental health status, and the overall treatment burden – the latter of which is well recognised in CF. 

Dr Quittner clearly demonstrated the links between mental health and treatment adherence in CF. Research shows that rates of depression and anxiety are two-to-three times higher for PWCF compared to people without CF (Quittner et al, Thorax 2014). Additionally, the International Depression Epidemiological Study (TIDES Study, 2014) found elevated rates of screened positivity for depression and anxiety among PWCF, and a person with CF who reports one single elevated depression score is thought to have a doubling of mortality risk within five years (Schechter et al, JCF 2021). This is possibly due to patients’ depressed mood leading to reduced treatment adherence and increased risk of lung function decline (further research is ongoing). Treatment adherence in children with CF may also be impacted by parental depression scores. A 2016 study examining enzyme adherence in children with CF using electronic pill-cap bottles found that children of parents with symptoms of depression were less adherent than those without depressive symptoms (Barker and Quittner. Paediatrics, 2016). 

Dr Quittner described this as being a “call to action” for CF researchers to rebalance and refocus on the impact of mental health and adherence on physical wellbeing in PWCF. 

The US clinical model of assessing and treating depression and anxiety in CF involves the regular administration of screening tools in clinic, both during annual assessments or when patients present with clinical indicators. Published in Thorax in 2016, the model provides a flexible, stepped-care approach for clinicians working in CF (see Figure 1). Dr Quittner confirmed her preference for the PHQ-9 (Patient Health Questionnaire-9) and GAD-7 (Generalised Anxiety Disorder Assessment 7) tools over the HADS (Hospital Anxiety and Depression Scale), of which the latter is still commonly utilised in Irish CF clinics. 

CF-specific mental health interventions are being developed and trialled in the US and have been shown to have positive effects for CF patients. Two pilot studies have been completed on CF-specific CBT and the CALM (Coping and Learning to Manage Stress with CF) tool, which have demonstrated benefit for the small pilot groups, and larger randomised trials are underway. It is a promising development that, for the first time, shows that the unique mental health needs for PWCF are being proactively addressed, and the association between mental health and physical health is now well understood. 

CF Registry of Ireland celebrates 20 years

The Cystic Fibrosis Registry of Ireland (CFRI) has celebrated its 21st year anniversary in 2023. The registry was developed in 2001 as a response to the lack of centrally collected data on CF patients, treatments, and health outcomes in Ireland, and was originally aligned with, and located within, the CF Association of Ireland (now known as CF Ireland). To celebrate this important milestone, Dr Laura Kirwan from the CFRI presented an overview of the CFRI and its achievements, at the conference. 

The CFRI was established in 2001 and the first CFRI annual report was published in 2002, albeit with limited data. By 2008, the registry had reached 90 per cent coverage of the CF population in Ireland. This high consent rate has remained, and by 2022, 90.3 per cent of CF patients in Ireland were registered with the CFRI, representing 1,328 adults and children with CF. According to Dr Kirwan, the 10 per cent who are not registered include newborns yet to have the opportunity to consent to the registry, and PWCF who have chosen not to participate in the registry.

By reviewing Irish CF data for over 20 years, the impact of improved healthcare treatments, technology and policy is evident. A stark example of this is the introduction of the National Newborn Screening Programme for CF in 2011. Dr Kirwan presented a graph on its impact, which showed the immediate drop in median age at diagnosis for children with CF after 2011.

The CFRI has partnered with a number of different academic and clinical research groups over the 21 years. An example of this is the Irish Comparative Outcomes Study (ICOS), where the registry collaborated with Prof Patricia Fitzpatrick and researchers at University College Dublin, and in 2020  published an article examining the clinical impact that early diagnosis has on the health of PWCF. The ICOS research showed that following the introduction of the Newborn Screening Programme in 2011, there is evidence of improved growth in children with CF, reduced hospitalisation for acute exacerbations, and delayed Pseudomonas aeruginosa acquisition up to the age of three years in the newborn screening cohort. This partnership is continuing to this date with the ICOS2 follow-up study.

Data collected by the CFRI on several other topics was highlighted at the conference, as the registry is an important source of real-world evidence on clinical outcomes. An increase in lung function over the last 20 years in PWCF in Ireland was noted (Figure 2), with a notable uptick in PWCF aged 10 years and older from 2020 onwards. This is likely due to  a combined impact of the introduction of ETI in Ireland alongside the increased isolation during the Covid-19 pandemic. 

A 20-year summary of the median age of death, cause of death, and median predicted age of survival data was also presented and explained. The median predicted survival age for PWCF in Ireland is now 51.8 years compared to 38.3 years in the 2010-2014 period. Crucially, CFRI data identified for the first time in 20 years that the most common cause of death in PWCF was recorded as ‘non-CF related’ in the last year, rather than respiratory causes, transplant related, or liver disease/liver failure (Figure 3). This is a remarkable reflection on improvements in care and treatments for PWCF over the past two decades. 

The 20-year landscape of medical complications/co-morbidities of CF can also be examined using CFRI data. Dr Kirwan demonstrated in her presentation that while there has been a significant decline in chronic infections in CF (such as Pseudomonas aeruginosa and Staphylococcus aureus) (Figure 4), there has been a noted increase in certain CF complications such as diabetes, liver disease, and liver cirrhosis (Figure 5). This shows the changing landscape of CF care over the past 20 years and gives us an indication of future directions and priorities for CF care and research. 

An important part of the CFRI annual data collection is to complete an annual census of patients. This is a data request that is sent to all CF centres once a year, so as to create a complete picture of population coverage of the CF registry. This also provides an excellent snapshot of CF patients each year. 

The future of CFRI data collection is important and ever-changing, especially in the era of modulator therapy. Priorities for CFRI data collection include data on pregnancy in CF, discontinuation of long-term medications (such as inhaled medications), chronic infection trends, and patient-reported outcome measurements (PROMs). The CFRI has trialled a patient portal, named CF View, which is a smartphone app that allows CF patients to view their clinic data in real-time. The next step in this piece of work for CFRI is to explore the possibility of CF patients not only viewing their data on the portal, but also enabling patients to upload their own self-reported outcomes, such as quality-of-life measures. The CF View project provided important findings and learning, which continues to inform ongoing work in this area for the CFRI. 

A renewed sense of hope

There was a palpable sense of hope for the future throughout the CFI conference. A panel discussion on ageing and CF between Dr Cedric Gunaratnam, CF and Respiratory Consultant, Beaumont Hospital, Dublin, and two adults living with CF, centred on living well and discussing various age-related issues that would never have been a concern to CF patients in years prior. Dr Gunaratnam expressed this optimism by stating: “It is our great hope with current treatments that a lot, if not most, [CF patients] not only live with their CF, but go on to have full lives.” 

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