Table 2.
Frequency of Disease Historical Clinical Manifestations at Baseline
| Symptom | Patient Groups, n (&%)
|
||
|---|---|---|---|
| Miglustat (n = 21) | No Miglustat Treatment (n = 9) | Overall (n = 30) | |
| Horizontal supranuclear gaze palsy | 21 (100) | 9 (100) | 30 (100) |
| Hepatomegaly | 17 (81) | 9 (100) | 26 (87) |
| Splenomegaly | 17 (81) | 9 (100) | 26 (87) |
| Spinal alignment abnormalities | 13 (62) | 6 (67) | 19 (63) |
| Cognitive impairment | 12 (57) | 2 (22) | 14 (47) |
| Thrombocytopeniaa | 12 (57) | 7 (78) | 19 (63) |
| Dysarthria | 9 (43) | 2 (22) | 11 (37) |
| Anemia | 9 (43) | 6 (67) | 15 (50) |
| Seizures | 5 (24) | 1 (11) | 6 (20) |
| Ataxia | 5 (24) | 6 (67) | 11 (37) |
| Bone crisis | 4 (19) | 1 (11) | 5 (17) |
| Swallowing difficulties | 3 (14) | 2 (22) | 5 (17) |
| Pyramidal tract dysfunction | 3 (14) | 2 (22) | 5 (17) |
a
At the start of the study, there were three patients with mild thrombocytopenia at baseline: Patients 116 (144 × 109/L) and 117 (132 × 109/L) in the miglustat group, and Patient 113 (149 × 109/L) in the no miglustat treatment group.