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Table 3 Estimated individual characteristics of biomarker response to ERT for the 197 patients

From: Modeling changes in biomarkers in Gaucher disease patients receiving enzyme replacement therapy using a pathophysiological model

  Median (range) at steady state Normalization n (%)
Half-life of response (years) 0.5 (0.01-3.1)  
Time to 95% of response (years) 2.1 (0.4-13.4)  
Ratio (level at steady state/level at ERT initiation)   
  Ferritin (%) 38.1 (2.1-101.5)  
  Chitotriosidase (%) 10.1 (0.8-116.7)  
  Hemoglobin (%) 117.3 (88.6-173.1)  
  Platelets (%) 200.4 (83.5-437.8)  
Predicted value at steady state   
  Ferritin (μg/L) 202.0 (16.5-1280.1) 115 (58.4)
  Chitotriosidase (nmol/h.mL) 820 (8–18183) 131 (66.5)
  Hemoglobin (g/dL) 13.5 (8.1-17.2) 174 (88.3)
  Platelets (/mm3) 172 000 (48 000–526 000) 126 (64.0)
  1. Normalization was defined by: ferritin <250 μg/L, chitotriosidase <100 nmol/mL/h, hemoglobin >12 g/dL, platelet count >150 000/mm3.