TABLE 2.
Description of crude death rates for miglustat‐treated vs untreated patients
| Patients in the miglustat‐treated period a | Patients in the miglustat‐untreated period b | |||||||
|---|---|---|---|---|---|---|---|---|
| Age‐at‐neurological‐onset sub‐group | No. of patients in the analysis, n | Total PY | Death rate (per 100 PY) | No. (%) of patients who died | No. of patients in the analysis, n | Total PY | Death rate (per 100 PY) | No (%) of patients who died |
| Neurological onset group | ||||||||
| All ages | 353 | 942.9 | 5.51 | 52 (14.7) | 406 | 3174.8 | 5.61 | 178 (43.8) |
| Early infantile (<2 years) | 43 | 88.9 | 21.37 | 19 (44.2) | 65 | 226.3 | 11.93 | 27 (41.5) |
| Late infantile (2 to <6 years) | 96 | 247.5 | 5.25 | 13 (13.5) | 139 | 1016.6 | 7.87 | 80 (57.6) |
| Juvenile (6 to <15 years) | 108 | 320.8 | 2.81 | 9 (8.3) | 111 | 1035.8 | 4.54 | 47 (42.3) |
| Adolescent/adult (≥15 years) | 106 | 285.7 | 3.85 | 11 (10.4) | 91 | 896.2 | 2.68 | 24 (26.4) |
| Diagnosis group | ||||||||
| All ages | 307 | 880.8 | 5.56 | 49 (16.0) | 353 | 1846.3 | 9.42 | 174 (49.3) |
| Early infantile (<2 years) | 38 | 82.2 | 20.67 | 17 (44.7) | 60 | 161.5 | 16.72 | 27 (45.0) |
| Late infantile (2 to <6 years) | 88 | 239.7 | 5.42 | 13 (14.8) | 136 | 706.6 | 11.04 | 78 (57.4) |
| Juvenile (6 to <15 years) | 91 | 298.2 | 2.68 | 8 (8.8) | 100 | 647.3 | 7.26 | 47 (47.0) |
| Adolescent/adult (≥15 years) | 90 | 260.6 | 4.22 | 11 (12.2) | 57 | 330.8 | 6.65 | 22 (38.6) |
a
Patients received miglustat at any time.
b
Patients either never received miglustat or were initially untreated after their onset of neurological manifestations.
Abbreviation: PY, patient‐years.