Table 7.
Treatment and outcome.
| All patients N = 39 | MPS N = 25 | AFD N = 8 | oLSD N = 6 | p value | |
|---|---|---|---|---|---|
| BMT | 6 (15%) | 6 (24%) | – | – | 0.18 |
| ERT | 13 (33%) | 9 (36%) | 3 (38%) | 1 (17%) | 0.78 |
| No therapy | 22 (56%) | 12 (48%) | 5 (63%) | 5 (83%) | 0.26 |
| Death | 8/38 (21%) | 5/24 (21%) | 0 (0%) | 3 (33%) | 0.28* |
| Mean age death (y) | 12.0 ± 6.1 | 14.2 ± 5.2 | – | 8.3 ± 6.6 | 0.10 |
MPS = mucopolysaccharidosis; AFD = Anderson Fabry disease; oLSD = other lysosomal storage disease; BMT = bone marrow transplantation; ERT = enzyme replacement therapy; * log-rank test.