Table 2.
Caregiver-reported improvements in signs and symptoms and disease impact following participation in the idursulfase-IT triala
| Improvement | Children in the pivotal trial and substudy N = 39 n (%) |
Children in the substudy N = 6 n (%) |
|---|---|---|
| Cognitive improvements | 37 (94.9) | 5 (83.3) |
| Improved speech | 21 (53.8) | 4 (66.7) |
|
Improved ability to communicate needs or wants/engage in conversation |
20 (51.3) | 1 (16.7) |
| Continuing to gain skills | 18 (46.2) | 5 (83.3) |
| Improved recall and memory | 9 (23.1) | – |
| Exhibiting higher-level thinking skills | 9 (23.1) | – |
| Emotional/behavioral improvements | 26 (66.7) | 1 (16.7) |
| Improved ability to focus/longer attention span | 13 (33.3) | 1 (16.7) |
| Decreased aggression | 12 (30.8) | 1 (16.7) |
| Decreased hyperactivity | 8 (20.5) | – |
| Improved mood stability | 2 (5.1) | – |
| Improvements in activities of daily living | 22 (56.4) | 3 (50.0) |
| Improved potty training | 13 (33.3) | 2 (33.3) |
| Improved ability to dress oneself | 8 (20.5) | 3 (50.0) |
| Improved ability to bathe oneself or brush one’s teeth | 5 (12.8) | 1 (16.7) |
| Improved ability to feed oneself | 2 (5.1) | – |
| Social improvements | 17 (43.6) | – |
| Physical improvements | 12 (30.8) | 2 (33.3) |
| Improved balance | 6 (15.4) | 1 (16.7) |
| Improved mobility/decreased stiffness | 5 (12.8) | 1 (16.7) |
| Improved vision | 2 (5.1) | – |
| Improvements in family dynamics | 4 (10.3) | – |
aOverall, improvements were observed in 39/50 children (78.0%). The percentage reported here was calculated based on the 39 children with reported improvements. For 11/50 children (22.0%), improvements were not observed; these children are not represented in this table
IT Intrathecal