RESULTS
Baseline data and changes after 6 weeks of treatment are shown in Table 1. \(\dot{V}\)O2peak, peak power output (Wpeak) and the anaerobic threshold (AT) were all improved following 6 weeks treatment with Kaftrio® in all cases. The magnitude of response was greater in those with moderate-to-severe lung disease at baseline (Table 1).
Improvements in exercise capacity were independent of changes in PA. The greatest increase in \(\dot{V}\)O2peak was achieved by Case 2, who had a substantial decrease in PA throughout the study period (Table 1). Changes in ventilatory function during exercise, measured as\(\dot{V}\)E/\(\dot{V}\)O2peak, improved in cases 2 and 3 who had low fitness levels and moderate-to-severe lung disease (Case 1: +7.7% vs. Case 2: -31.1% and Case 3: -15.6%). There were no consistent improvements in ventilatory drive (Case 1: +16.2%; Case 2: -0.9%; Case 3: -2.3%), breathing reserve (Table 1) or\(\dot{V}\)E/\(\dot{V}\)CO2peak (Case 1: -0.4%; Case 2: +2.3%; Case 3: +5.5%).
\(\dot{V}\)O2 per unit of power output was higher after treatment in the two participants with low baseline fitness levels and advanced lung disease (i.e. the \(\dot{V}\)O2-gain; Case 2: +36.0% and Case 3: +62.1%), but was negligible in Case 1 (+1.6%) who demonstrated greater baseline fitness and mild lung disease (Figure 1). Predicted maximal heart rates (180 bpm) were achieved by cases 1 and 2 pre- and post-treatment, whereas predicted heart rate maximum was not achieved by case 3 in either CPET, probably because of a greater degree of ventilatory limitation due to more severe lung disease. Glycaemic control was unchanged after treatment in Case 3 (Table 1).