Table 5 Comparison of outcomes between LI-MLD and J-MLD sibling pairs
Pair (ID) | MLD Subtype | Age at HSCT (Sx) | GMFC-MLDa | ELFC-MLDa | ADLb | VABSc Age Equivalent | Age at Follow-Up (Deceased) | |||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
M1 | M2 | M3 | M4 | M5 | M6 | E1 | E2 | E3 | E4 | Dress | Feed | Meal | Toilet | Hyg. | Sch. | Work | Ind. | |||||
A (3) | LI | 0,8 (No) | — | — | 1,3 | 1,6 | 2,0 | 2,3 | 2,6 | 2,9 | 3,0 | 5,5 | — | — | — | — | — | — | — | — | 0,1 | 19,11 |
A (3sib) | LI | No HSCT | 1,4 | 1,9 | 2,0 | 2,2 | 2,3 | 2,10 | 1,4 | 1,7 | 1,7 | 1,7 | — | — | — | — | — | — | — | — | N/A | (4,6) |
B (4) | J | 4,10 (Yes) | 3,0 | 12,0 | 12,0 | 12,0 | 12,0 | 24,0 | 3,0 | 5,0 | 18,0 | + | — | 13,0 | — | — | — | 21,0 | 26,0 | — | 1,7 | 34,11 |
B (4sib) | J | No HSCT | 4,0 | 4,0 | 5,0 | 5,0 | 5,0 | 5,0 | 4,0 | 4,0 | 5,0 | 5,0 | — | 4,6 | — | 4,0 | — | 9,0 | — | — | N/A | (9,3) |
C (10) | J | 5,8 (Yes) | 5,0 | 7,2 | 7,9 | 8,0 | 9,0 | + | 6,8 | 8,0 | + | + | 5,10 | 6,6 | — | 6,6 | 7,0 | + | — | — | 1,0 | 24,0 |
C (10sib) | J | No HSCT | 5,0 | 5,6 | 6,8 | 7,0 | 7,4 | 7,10 | 5,6 | 6,0 | 6,8 | 7,2 | 5,3 | 5,10 | — | 5,10 | 5,10 | 9,0 | — | — | N/A | (18,0) |
D (17) | J | 7,8 (Yes) | 6,0 | 7,0 | 9,0 | 9,7 | 12,0 | 13,4 | 6,0 | 9,7 | 12,6 | 15,0 | 9,0 | 9,0 | — | 6,0 | 8,0 | + | — | — | 0,5 | 19,4 |
D (17sib) | J | 4,3 (No) | 13,0 | + | + | + | + | + | 14,0 | + | + | + | + | + | — | + | + | + | — | — | X | 15,2 |
E (27) | J | 16,1 (Yes) | 16,0 | 16,0 | 16,0 | 16,0 | 16,0 | + | 16,0 | + | + | + | 16,1 | 16,1 | 16,1 | 15,0 | + | 21,0 | — | — | 3,0 | 26,6 |
E (22) | J | 11,1 (No) | + | + | + | + | + | + | + | + | + | + | + | + | + | + | + | + | + | + | 21,6 | 21,6 |