PMC4590953_01
BMI trend. There was a significant overall improvement in BMI (P < 0.01). The adult patient (patient number 5) was obese to start with who lost some weight to a healthier BMI.
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The mean BMI among pediatric patients was 16.2 kg/m2 at baseline which improved to 20.9 kg/m2 at 15 months (Figure 1).
FEV1 trend. There was significant overall improvement (P < 0.01) in FEV1, however more so in the younger patients. Patient number 5 (46-year-old female) who already had established bilateral bronchiectasis had a modest improvement. Patient number 4, a 14-year-old male who also had bilateral diffuse bronchiectasis and multiple pulmonary exacerbations prior to starting Ivacaftor, demonstrated significant improvement in his FEV1 presumably due to decreased mucus impaction in the airways (see chest CT scans in Figure 4).
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The mean FEV1 changed from 56.2 to 99.7 percent predicted (Figure 2) and the mean FEF25-75 from 40.2 to 90 percent predicted (Figure 3).
FEF25-75 trend. Similar to trend in FEV1 (see Figure 2), there was significant improvement in FEF 25-75% (P < 0.01). The adult patient (number 5) had only modest improvement suggesting that the patient already had irreversible lung damage.
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The mean FEV1 changed from 56.2 to 99.7 percent predicted (Figure 2) and the mean FEF25-75 from 40.2 to 90 percent predicted (Figure 3).
CT of the chest before and after Ivacaftor. Two representative chest CT slices of patient number 4 ((a) and (b), 16-year-old male) at the same anatomic region before and after Ivacaftor therapy. They demonstrate decreased mucus impaction in the latter scan despite the continued presence of bronchiectasis. Decreased mucus impaction probably explains improved lung function (Figures 2 and 3) despite irreversible nature of bronchiectasis. In the bottom panels ((c) and (d)) are the chest CT of another patient (14-year-old female) who already had established bronchiectasis in the peripheral airways before Ivacaftor (c) which has been resolved 18 months after Ivacaftor therapy (d).
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Ivacaftor. In two patients, where pre- and post-chest CT scans were available, there was significant improvement in the radiology findings, which showed decreased mucus impaction, improved bronchial wall thickening, and partial reversal of bronchiectasis despite established lung disease (Figure 4).