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Table 2 Clinical outcome measures during enzyme replacement therapy and relative to the natural course of disease

From: Effect of enzyme therapy and prognostic factors in 69 adults with Pompe disease: an open-label single-center study

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

  1. ERT = Enzyme Replacement Therapy; pp/y = percentage points per year; 95% CI = 95% Confidence Interval; MRC sumscore = Medical Research Council sumscore; QMFT score = Quick Motor Function Test score; HHD sumscore = Hand-Held Dynamometry sumscore; FVC = Forced Vital Capacity; N = Number of patients; M = Number of Measurements. Data shown are mean changes in percentage points per year as calculated by repeated-measures ANOVA.
  2. * Difference between the course of disease during treatment and the natural course.