Table 4

Annual pulmonary function changes before and after initiation of enzyme replacement therapy

Before initiation of ERTAfter initiation of ERTP 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-scores0.157 (−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, % predicted0.07 (−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.