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®.