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
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 |