Before initiation of ERT | After initiation of ERT | P value | |
FEV1, mL | −32.4 (−43.5, –21.4) | −26.3 (39.7, –13.0) | 0.12 |
FEV1, z-score | −0.010 (−0.029, 0.009) | −0.010 (−0.036, 0.015) | 0.99 |
FVC, mL | −14.7 (−29.3, –10.0) | −14.3 (−30.8, 2.1) | 0.11 |
FVC, z-scores | 0.157 (−0.155, 0.470) | 0.077 (−0.182, 0.337) | 0.42 |
FEV1/FVC, z-score | −0.045 (−0.075, –0.014) | −0.015 (−0.036, 0.006) | 0.014 |
FEF25%–75%, mL | −45.1 (−64.7, –25.5) | −43.3 (−56.5, –30.1) | 0.81 |
FEF25%–75%, z-score | −0.004 (−0.030, 0.022) | 0.002 (−0.014, 0.018) | 0.56 |
DLCO, % predicted | 0.07 (−0.32, 0.47) | −0.09 (−0.36, 0.17) | 0.34 |
Values are estimated slopes (95% CI) of annual pulmonary function change, whereas median (IQR) spirometric follow-up time was 7.7 (3.7, 10.1) years.
DLCO, CO diffusion capacity of the lung; ERT, enzyme replacement therapy; FEF25%–75%, forced expiratory flow between 25% and 75% of FVC; FEV1, forced expiratory volume in the first second; FVC, forced (expiratory) vital capacity.