Table 1.
Patient characteristics for patients in survival analyses
| Kishnani (2009) | Nicolino (2009) | Erasmus MC | |
|---|---|---|---|
| N |
18 |
21 |
20 |
| Deaths |
5 (28%) |
6 (29%) |
4 (20%) |
| Age at end study in months [range] |
34.5 [19.7-44.0] |
41.0 [7.7-80.3] |
60.9 [3.2-178.8] |
| Patients using ventilation |
9 (50%) |
7 (33%) |
5 (25%)b |
| Dosage (every other week) |
|
|
|
| 15 mg/kg |
0 |
0 |
2c, d |
| 20 mg/kg |
9 |
21a |
8e |
| 40 mg/kg | 9 | 0 | 10d |
aEight patients switched to 40 mg/kg after 26 weeks due to clinical deterioration; bTwo patients died; cBoth patients switched to 30 mg/kg and later to 40 mg/kg; dEvery week; eFive patients switched to 40 mg/kg.