Table 1.
Patient 1 | Patient 2 | Patient 3 | |
---|---|---|---|
Age at diagnosis |
4 y 9 mo |
1 y 8 mo |
7 y 8 mo |
Phenotype |
severe |
severe |
mild |
Age at start of ERT |
4 y 11 mo |
2 y 5 mo |
11 y 3 mo |
ERT at hospital (number of infusions) |
69 |
157 |
128 |
ADRs at hospital |
0 |
2 |
1 |
Age at start of ERT at home |
6 y 4 mo |
6 y 2 mo |
16 y 2 mo |
ERT at home (number of infusions) |
87 |
73 |
74 |
ADRs at home | 0 | 0 | 0 |
ADR adverse drug reaction, ERT enzyme replacement therapy.