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Table 5 Summary of patient’s eligibility to be included (% included) based on different ASIS thresholds compared with three clinical trials based on age inclusion (upper panel). The lower panel summarise between group comparisons showing the percentage of patients included and excluded when comparing ASIS inclusion criteria versus age inclusion criteria

From: Annual severity increment score as a tool for stratifying patients with Niemann-Pick disease type C and for recruitment to clinical trials

  Inclusion criterion % Included  
ASIS-defined 0.5–2 63.2  
  0.75–2 50.0  
  1–2 34.2  
  0.5–2.5 65.8  
  0.75–2.5 52.6  
  1–2.5 36.8  
Age-defined
 Miglustat trial criteria [3] n = 39 >  12 76.3  
 Vtesse/Sucampo trial criteria n = 44 4–21 73.7  
 Orphazyme trial criteria n = 39 2–18 57.9  
  Between groups comparison (% Patients excluded, included)
  Age-defined   
ASIS-defined >  12 4–21 2–18
 0.5–2 15.8, 55.26 10.5, 47.4 10.5, 31.6
 0.75–2 15.8, 42.1 18.4, 42.1 21.1, 28.9
 1–2 18.4, 28.9 23.7, 31.6 31.6, 23.7
 0.5–2.5 13.2, 55.3 10.5, 50.0 10.5, 34.2
 0.75–2.5 13.2, 42.1 18.4, 44.7 21.1, 31.6
 1–2.5 15.8, 28.9 23.7, 34.21 31.6, 26.3
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