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Table 1 Patient characteristics for patients in survival analyses

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

  Kishnani (2009) Nicolino (2009) Erasmus MC
N 18 21 20
Deaths 5 (28%) 6 (29%) 4 (20%)
Age at end study in months [range] 34.5 [19.7-44.0] 41.0 [7.7-80.3] 60.9 [3.2-178.8]
Patients using ventilation 9 (50%) 7 (33%) 5 (25%)b
Dosage (every other week)    
15 mg/kg 0 0 2c, d
20 mg/kg 9 21a 8e
40 mg/kg 9 0 10d
  1. aEight patients switched to 40 mg/kg after 26 weeks due to clinical deterioration; bTwo patients died; cBoth patients switched to 30 mg/kg and later to 40 mg/kg; dEvery week; eFive patients switched to 40 mg/kg.