Evolution with and without HCT: transplanted patients
| Patient ID . | Transplanted patients . | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| MLD type . | Follow-up since HCT, months . | GMF pre-HCT . | GMF post-HCT* . | Cognition pre-HCT (age in years/months) . | Cognition post-HCT (age in years) . | MRI pre-HCT . | MRI post-HCT* . | Deceased (age in years) . | |
| MLD-50 | Late-infantile | 10 | 2 | 6 | DQ 105 (25 mo) | NA | 2 | 20 | Yes (3 y) |
| MLD-45 | Late-infantile | 60 | 2 | 5 | DQ 101 (35 mo) | 70 (4 y) | 3 | 8 | No |
| MLD-16 | Juvenile | 36 | 0 | 0 | 104 (6 y) | NA | 0 | 0 | No |
| MLD-37 | Juvenile | 127 | 0 | 2 | DQ 116 (26 mo) | 95 (6 y) | 2 | 4 | No |
| MLD-4 | Juvenile | 112 | 1 | 1 | 110 (5 y) | 56 (10 y) | 4 | 8 | No |
| MLD-53 | Juvenile | 11 | 1 | 6 | 74 (7 y) | NA | 20 | 25 | Yes (8 y) |
| MLD-14 | Juvenile | 61 | 1 | 1 | 94 (14 y) | 93 (17 y) | 12 | 11 | No |
| MLD-21 | Adult | 27 | 0 | 0 | 100 (17 y) | 95 (18 y) | 10 | 10 | No |
| MLD-30 | Adult | 94 | 0 | 1 | 104 (19 y) | 107 (21 y) | 7 | 11 | No |
| MLD-41 | Adult | 77 | 0 | 0 | 91 (25 y) | 89 (27 y) | 12 | 13 | No |
| MLD-15 | Adult | 59 | 0 | 0 | 87 (35 y) | 73 (40 y) | 11 | 14 | No |
| MLD-51 | Adult | 2 | 1 | 2 | 61 (22 y) | NA | 16 | 24 | Yes (22 y) |
| MLD-2 | Adult | 62 | 0 | 0 | 72 (28 y) | 76 (31 y) | 13 | 12 | No |
| Patient ID . | Transplanted patients . | ||||||||
|---|---|---|---|---|---|---|---|---|---|
| MLD type . | Follow-up since HCT, months . | GMF pre-HCT . | GMF post-HCT* . | Cognition pre-HCT (age in years/months) . | Cognition post-HCT (age in years) . | MRI pre-HCT . | MRI post-HCT* . | Deceased (age in years) . | |
| MLD-50 | Late-infantile | 10 | 2 | 6 | DQ 105 (25 mo) | NA | 2 | 20 | Yes (3 y) |
| MLD-45 | Late-infantile | 60 | 2 | 5 | DQ 101 (35 mo) | 70 (4 y) | 3 | 8 | No |
| MLD-16 | Juvenile | 36 | 0 | 0 | 104 (6 y) | NA | 0 | 0 | No |
| MLD-37 | Juvenile | 127 | 0 | 2 | DQ 116 (26 mo) | 95 (6 y) | 2 | 4 | No |
| MLD-4 | Juvenile | 112 | 1 | 1 | 110 (5 y) | 56 (10 y) | 4 | 8 | No |
| MLD-53 | Juvenile | 11 | 1 | 6 | 74 (7 y) | NA | 20 | 25 | Yes (8 y) |
| MLD-14 | Juvenile | 61 | 1 | 1 | 94 (14 y) | 93 (17 y) | 12 | 11 | No |
| MLD-21 | Adult | 27 | 0 | 0 | 100 (17 y) | 95 (18 y) | 10 | 10 | No |
| MLD-30 | Adult | 94 | 0 | 1 | 104 (19 y) | 107 (21 y) | 7 | 11 | No |
| MLD-41 | Adult | 77 | 0 | 0 | 91 (25 y) | 89 (27 y) | 12 | 13 | No |
| MLD-15 | Adult | 59 | 0 | 0 | 87 (35 y) | 73 (40 y) | 11 | 14 | No |
| MLD-51 | Adult | 2 | 1 | 2 | 61 (22 y) | NA | 16 | 24 | Yes (22 y) |
| MLD-2 | Adult | 62 | 0 | 0 | 72 (28 y) | 76 (31 y) | 13 | 12 | No |
DQ, developmental quotient; NA, not available.
GMF and MRI post-HCT are scored at latest follow-up assessment.