Table 2. Effects of elexacaftor/tezacaftor/ivacaftor treatment on lung function, BMI, and sweat chloride concentrations at baseline and at the end of the follow-up period of the study.
| Sample or subgroup | Outcome | Baseline | End of follow-up | Mean difference | p |
|---|---|---|---|---|---|
| Overall (n = 24) | FEV1, % predicted | 50.36 (42.27-58.46) | 65.60 (56.14-75.05) | 15.23 (10.51-19.95) | < 0.001 |
| FEV1, L | 1.75 (1.41-2.10) | 2.29 (1.87-2.71) | 0.54 (0.36-0.72) | < 0.001 | |
| Sweat chloride, mmol/L | 71.59 (56.29-86.88) | 35.65 (25.89-45.40) | −35.94 (−49.65 to −22.24) | < 0.001 | |
| BMI, kg/m2 | 19.72 (18.98-20.45) | 21.03 (20.23-21.82) | 1.31 (0.84-1.78) | < 0.001 | |
| Homozygous (n = 13) | FEV1, % predicted | 51.82 (39.52-64.18) | 65.92 (51.62-80.22) | 14.10 (5.33-22.87) | 0.005 |
| FEV1, L | 1.79 (1.27-2.31) | 2.29 (1.66-2.92) | 0.50 (0.18-0.82) | 0.006 | |
| Sweat chloride, mmol/L | 67.88 (40.49-95.26) | 36.00 (17.14-54.86) | −31.88 (−55.42 to −8.33) | 0.015 | |
| BMI, kg/m2 | 19.46 (18.26-20.65) | 20.76 (19.51-22.03) | 1.31 (0.71-1.90) | < 0.001 | |
| Heterozygous (n = 11) | FEV1, % predicted | 48.91 (36.15-61.67) | 65.27 (50.23-80.32) | 16.36 (10.96-21.77) | < 0.001 |
| FEV1, L | 1.72 (1.18-2.26) | 2.30 (1.62-2.97) | 0.58 (0.10-0.33) | < 0.001 | |
| Sweat chloride, mmol/L | 74.89 (53.07-96.71) | 35.33 (22.60-48.07) | −39.56 (−59.84 to −19.27) | 0.002 | |
| BMI, kg/m2 | 20.03 (19.07-20.99) | 21.34 (20.22-22.46) | 1.31 (0.43-2.19) | 0.008 | |
| Advanced lung diseasea (n = 8) | FEV1, % predicted | 32.98 (27.81-38.14) | 46.25 (32.31-60.19) | 13.28 (3.97-22.58) | 0.012 |
| FEV1, L | 1.07 (0.93-1.20) | 1.49 (1.12-1.86) | 0.42 (0.16-0.68) | 0.006 | |
| Sweat chloride, mmol/L | 62.50 (33.87-91.13) | 33.67 (16.02-51.31) | −28.83 (−63.05 to 5.38) | 0.083 | |
| BMI, kg/m2 | 18.83 (17.48-20.18) | 20.96 (19.43-22.49) | 2.13 (1.27-2.98) | 0.001 |
a
Defined as FEV1 < 40% of the predicted value.