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Patient disposition, n (%)
| Enrolled | Year 1 | Year 2 | Year 3 | Year 4 | Year 5 | |
|---|---|---|---|---|---|---|
| USA | 207 | 181 (87) | 171 (83) | 121 (59) | 54 (26) | 44 (21) |
| Outside USA | 60 | 48 (80) | 39 (65) | 0 (0) | 0 (0) | 0 (0) |
| Treated: 267 (100) | ||||||
| Completed: 188 (70) | ||||||
| Discontinued: 79 (30) | ||||||
| Patient, parent, or caregiver request: 33 (12) | ||||||
| Lack of efficacy: 15 (6) | ||||||
| Adverse event: 10 (4) | ||||||
| Death: 9 (3) | ||||||
| Other reasons: 12 (4) | ||||||
