Skip to main content

Table 1 Baseline characteristics of Pompe patients included in the cost-effectiveness analyses (n = 90)

From: Cost-effectiveness of enzyme replacement therapy with alglucosidase alfa in adult patients with Pompe disease

  Mean Median Range
Age at first visit 49.1 years 50.0 years 23.0–75.0 years
Disease duration (since diagnosis) 7.7 years 4.3 years 0.0–27.6 years
Female 48%   
Residual Enzyme activity (in fibroblasts) 12.0% 12.0% 0.5–19.9%
Wheelchair use 31%   
Ventilation use 27%   
Period at risk in ST survival analyses 3.5 years 3.5 years 0.0–8.9 years
Period at risk in ERT survival analyses 3.4 years 3.7 years 0.2–8.4 years