Clinical Outcome Measure | Natural course mean pp/y (95% CI) P-value | Treatment course mean pp/y (95% CI) P-value | Difference* mean pp/y (95% CI) P- value | |||
---|---|---|---|---|---|---|
MRC sumscore Total study population (N = 69, M = 558) | 1.4 (0.8 to 2.1) | <0.001 | ||||
MRC sumscorePatients with pre-ERT + ERT follow-up (N = 49, M = 523) | −1.2 (−2.1 to −0.4) | 0.006 | 2.1 (1.2 to 3.0) | <0.001 | 3.3 (1.9 to 4.7) | <0.001 |
HHD sumscore Total study population (N = 64, M = 503) | 4.0 (2.5 to 5.6) | <0.001 | ||||
HHD sumscore Patients with pre-ERT + ERT follow-up (N = 42, M = 435) | −2.8 (−4.2 to −1.3) | <0.001 | 5.1 (3.0 to 7.3) | <0.001 | 7.9 (5.0 to 10.7) | <0.001 |
QMFT score Total study population (N = 69, M = 553) | 0.7 (−0.2 to 1.7) | 0.14 | ||||
FVC in upright position Total study population (N = 62, M = 475) | 0.1 (−1.0 to 1.1) | 0.92 | ||||
FVC in upright position Patients with pre-ERT + ERT follow-up (N = 46, M = 480) | −2.0 (−3.1 to −0.8) | 0.001 | −0.2 (−1.6 to 1.2) | 0.76 | 1.8 (−0.2 to 3.7) | 0.08 |
FVC in supine position Total study population (N = 54, M = 411) | −1.1 (−2.1 to −0.1) | 0.03 | ||||
FVC in supine position Patients with pre-ERT + ERT follow-up (N = 42, M = 436) | −1.8 (−2.9 to −0.7) | 0.002 | −1.0 (−2.3 to 0.3) | 0.12 | 0.8 (−0.9 to 2.4) | 0.38 |