Evolution with and without HCT: nontransplanted patients
| Patient ID . | Nontransplanted patients . | |||||||
|---|---|---|---|---|---|---|---|---|
| MLD type . | Follow-up, months . | GMF at diagnosis . | GMF latest assessment* . | Cognition at diagnosis (age in years) . | MRI at diagnosis . | MRI at latest assessment* . | Deceased (age in years) . | |
| MLD-8 | Late- infantile | 39 | 6 | 6 | NA | 4 | NA | Yes (5 y) |
| MLD 20 | Late-infantile | 36 | 6 | 6 | NA | 10 | NA | Yes (6 y) |
| MLD-26 | Late-infantile | 72 | 5 | 6 | NA | 17 | NA | Yes (8 y) |
| MLD-34 | Late-infantile | 36 | 6 | 6 | NA | 12 | NA | Yes (4 y) |
| MLD-40 | Late-infantile | 19 | 6 | 6 | NA | 8 | NA | No |
| MLD-57 | Late-infantile | 29 | 4b | 6 | NA | 13 | NA | No |
| MLD-5 | Juvenile | 42 | 1 | 6 | 71 (7 y) | 22 | 26 | No |
| MLD-6 | Juvenile | 42 | 1 | 6 | 65 (9 y) | 21 | 23 | No |
| MLD-10 | Juvenile | 84 | 1 | 5 | NA | 18 | NA | No |
| MLD-11 | Juvenile | 84 | 1 | 5 | NA | 14 | NA | No |
| MLD-12 | Juvenile | 93 | 1 | 6 | 68 (6 y) | 12 | NA | No |
| MLD-17 | Juvenile | 72 | 1 | 6 | 66 (6 y) | 14 | NA | Yes (12 y) |
| MLD-18 | Juvenile | 48 | 1 | 6 | 75 (6 y) | 14 | NA | Yes (12 y) |
| MLD-22 | Juvenile | 44 | 1 | 6 | 65 (15 y) | 22 | NA | No |
| MLD-29 | Juvenile | 22 | 1 | 6 | 61 (7 y) | 18 | NA | Yes (8 y) |
| MLD-33 | Juvenile | 68 | NA | 6 | NA | 15 | 25 | Yes (11 y) |
| MLD-38 | Juvenile | 29 | 1 | 5 | NA | 17 | NA | No |
| MLD-39 | Juvenile | 33 | 1 | 4b | 63 (7 y) | 15 | NA | No |
| MLD-54 | Juvenile | 87 | 1 | 5 | NA | 17 | 19 | No |
| MLD-25 | Adult | 103 | 1 | 3 | 50 (22 y) | 19 | 23 | No |
| MLD-32 | Adult | 70 | 0 | 0 | NA | 16 | 16 | No |
| MLD-56 | Adult | 38 | 1 | 2 | 53 (32 y) | 13 | 17 | No |
| Patient ID . | Nontransplanted patients . | |||||||
|---|---|---|---|---|---|---|---|---|
| MLD type . | Follow-up, months . | GMF at diagnosis . | GMF latest assessment* . | Cognition at diagnosis (age in years) . | MRI at diagnosis . | MRI at latest assessment* . | Deceased (age in years) . | |
| MLD-8 | Late- infantile | 39 | 6 | 6 | NA | 4 | NA | Yes (5 y) |
| MLD 20 | Late-infantile | 36 | 6 | 6 | NA | 10 | NA | Yes (6 y) |
| MLD-26 | Late-infantile | 72 | 5 | 6 | NA | 17 | NA | Yes (8 y) |
| MLD-34 | Late-infantile | 36 | 6 | 6 | NA | 12 | NA | Yes (4 y) |
| MLD-40 | Late-infantile | 19 | 6 | 6 | NA | 8 | NA | No |
| MLD-57 | Late-infantile | 29 | 4b | 6 | NA | 13 | NA | No |
| MLD-5 | Juvenile | 42 | 1 | 6 | 71 (7 y) | 22 | 26 | No |
| MLD-6 | Juvenile | 42 | 1 | 6 | 65 (9 y) | 21 | 23 | No |
| MLD-10 | Juvenile | 84 | 1 | 5 | NA | 18 | NA | No |
| MLD-11 | Juvenile | 84 | 1 | 5 | NA | 14 | NA | No |
| MLD-12 | Juvenile | 93 | 1 | 6 | 68 (6 y) | 12 | NA | No |
| MLD-17 | Juvenile | 72 | 1 | 6 | 66 (6 y) | 14 | NA | Yes (12 y) |
| MLD-18 | Juvenile | 48 | 1 | 6 | 75 (6 y) | 14 | NA | Yes (12 y) |
| MLD-22 | Juvenile | 44 | 1 | 6 | 65 (15 y) | 22 | NA | No |
| MLD-29 | Juvenile | 22 | 1 | 6 | 61 (7 y) | 18 | NA | Yes (8 y) |
| MLD-33 | Juvenile | 68 | NA | 6 | NA | 15 | 25 | Yes (11 y) |
| MLD-38 | Juvenile | 29 | 1 | 5 | NA | 17 | NA | No |
| MLD-39 | Juvenile | 33 | 1 | 4b | 63 (7 y) | 15 | NA | No |
| MLD-54 | Juvenile | 87 | 1 | 5 | NA | 17 | 19 | No |
| MLD-25 | Adult | 103 | 1 | 3 | 50 (22 y) | 19 | 23 | No |
| MLD-32 | Adult | 70 | 0 | 0 | NA | 16 | 16 | No |
| MLD-56 | Adult | 38 | 1 | 2 | 53 (32 y) | 13 | 17 | No |
GMF and MRI post-HCT are scored at latest follow-up assessment.