METHODS
All cases gave their informed consent to complete CPET and device-based PA assessments at baseline and 6 weeks after receiving Kaftrio®. All participants were pancreatic insufficient and prescribed regular nebulised rhDNase. Cases 2 and 3 were also receiving nebulised antibiotics, having had recurrent isolates of Pseudomonas aeruginosa. Case 3 had more severe CF lung disease as a result of previous non-tuberculous mycobacteria infection. Case 1 self-reported high levels of baseline PA (device-based data was unavailable due to poor adherence), whereas Case 2 was less physically active and Case 3 had low PA levels (Table 1).
Before and after treatment with Kaftrio®, participants underwent CPET as previously described8-10. Briefly, breath-by-breath changes in \(\dot{V}\)O2, carbon dioxide production (\(\dot{V}\)CO2) and minute ventilation (\(\dot{V}\)E) were measured during incremental cycling to exhaustion. Additionally, device-based daily PA of Cases 2 and 3 was assessed using GENEActiv™ accelerometers (Active Insights, Kimbolton, Cambridgeshire, UK) worn on the non-dominant wrist for 7 consecutive days. Data was downloaded using the manufacturer’s software, and validated threshold values were used to classify movement as light-, moderate- or vigorous-intensity11.
Case 3, who had CF-related diabetes (CFRD), also wore a Freestyle Libre Pro™ (Abbott, Chicago, USA) continuous glucose monitoring (CGM) sensor for 14 days before and after treatment with Kaftrio®.