NACFC 2023
North American CF Conference 2023
The project team made their way to Phoenix, Arizona for NACFC 2023. Paul McNally presented pooled data from the RECOVER study, which takes place in Ireland and the UK, and the PROMIS study, which takes place in the US. This presentation showed that female and younger people with CF had the lowest sweat chloride levels after taking ETI. It also showed that targeting both genes is important for achieving normal levels of sweat chloride.

Paul also presented the below data that looked at abdominal symptoms in participants aged 6-11. The data was taken from the Abdominal symptom questionnaires that participants filled out at baseline, 1 and 6 months. The data showed an improvement in pain, reflux and bowel movement problems after 6 month of ETI. Data collection in this cohort is continuing.

Sharon Sutton, the study pharmacist presented data on the adherence to medications of the 12+ cohort. This data compared the adherence of participants at 12 months compared to 24 months. Building on from the presentation at NACFC 2022, Sharon once again showed that adherence is over-estimated by the self-reported questionnaires. The medication possession rates showed that adherence dropped from 12 to 24 months. Based on this data, Sharon setup focus groups and workshops with the CF community to try and understand attitudes to adherence.

In addition to the presentation, four posters were displayed. The 6-11 1-year CT data demonstrated significant improvements in the overall percentage disease scores. Our collborators are currently looking into using AI to analyse the CT images.

Updated 12+ cohort data was displayed. The 24month visits are ongoing so the complete dataset was not ready to be shared. The data collected so far showed significant and sustained improvements in all outcome measures over 2 years. No significant differences were seen in outcomes between 12 and 24 months.

Gavin Stone displayed a poster on sino-nasal MRI and nasal symptoms in the 6-11 age group. Numbers for this study were relatively low, with roughly 25 patients having an MRI scan. The data showed that ETI improves nasal MRI appearance but not nasal symptoms in participants. Swabs from these participants will be analysed to see if different or less bugs are in the nose on ETI treatment.

Another poster on display was on exhaled Nitric Oxide (FeNO). Here, we saw that those on ETI in the 12+ cohort who were homozygous for F508del had a significant increase in FeNO. No such change in FeNO was seen in the 6-11 cohort. This data may suggest that the observed changes in FeNO are not directly related to correction of CFTR dysfunction. Further biomarker studies are needed to help explain the effects of ETI on airway Nitric Oxide metabolism.

Our collaborator Sam Nolan who is pursuing a PhD at Queens University Belfast presented data looking at infections in participants, and inflammation markers in nasal lavage and sputum samples. The data showed that ETI is associated with significant decreases in overall chronic infections. The study team found it difficult to collect sputum sample from participants, this is most likely a result of patients becoming healthier and non-productive. The team are looking at whether nasal lavage can be used as a technique to gather data on infection instead.
