North American CF Conference 2024
The team once again travelled to the US for NACFC. This year, the conference was held in Boston. The team presented three talks and displayed a poster.
Paul McNally presented the 12+ liver data. This was the first time the team looked at liver outcomes. The data confirmed that CF liver disease is a complex area and not easy to analyse. There were no obvious signs that ETI causes significant liver injury, and in the two years of data collection, we didn’t see improvements in liver status. As liver disease takes a long time to manifest, it is too early to see if ETI causes reduction or progression of liver disease.
Building on from the abdominal symptom data, our co-Principal Investigator Jane Davies presented updated data for the 6-11 cohort. Using the CFAbd-Score, we saw that improvement in abdominal symptoms on ETI are maintained for 12 months in children with CF. Unlike in older people with CF who had abnormal levels of gut inflammation at baseline, baseline levels in the 6-11 cohort were closer to the normal range and did not change significantly with treatment. Analysis of abdominal symptom scores and stool samples is ongoing.
Paul presented on how clinical outcomes of the 12+ cohort are effected by adherence. He showed that the self-report questionnaires overestimate adherence compared to medication possession rates and that ETI adherence is lower in the second year of treatment.
Paul looked at the medication pick up rates of ETI vs outcomes, which showed a strong link between adherence and sweat chloride and LCI scores. We will look at whether this is seen in the 6-11 cohort when that data has been collected. The data highlighted the need for a useable, practical adherence measuring device.
In the 6-11 cohort, we saw sustained and significant improvements in outcomes measured over a 2 year period. However, there were no significant differences seen in outcomes between 12 and 24 months. With better treatments like ETI being used in children, traditional clinical outcome measures may not detect changes in disease. Here we demonstrate that, even with mild disease, some pulmonary endpoints still demonstrate clinical and significant changes. Nutritional endpoints in this cohort were non-significant however, this is likely evidence of a ceiling effect. Future studies, such as the ENHANCE study will deal with the absence of signal from disease outcome measures and start to focus on measurement of prevention.
