Table 1 Patient demographics: Response rate, age and SMA-related characteristics
SMA subtype | ||||
|---|---|---|---|---|
Total | I | II | III | |
Response rate | ||||
Total patient number | 265 | 20 | 115 | 130 |
Enrolled respondents | 189 | 12 | 73 | 104 |
Response rate [%] | 71 | 60 | 63 | 80 |
Ratio of patients/parents among respondents [%] | 52/48 | 0/100 | 30/70 | 74/26 |
Age of enrolled patients | ||||
Min. [years] | 0 | 0 | 2 | 2 |
Max. [years] | 73 | 7 | 56 | 73 |
Median [years] | 19 | 1 | 11 | 33 |
Current motor function | ||||
N | 177 | 9 | 69 | 99 |
Walk [%] | 33 | 0 | 0 | 59 |
Sit [%] | 47 | 11 | 65 | 38 |
None [%] | 20 | 89 | 35 | 3 |
Wheelchair use | ||||
N | 154 | 4 | 51 | 99 |
Always [%] | 61 | 50 | 100 | 41 |
Sometimes [%] | 14 | 0 | 0 | 21 |
None [%] | 25 | 50 | 0 | 37 |
Feed tube use | ||||
N | 177 | 9 | 69 | 99 |
Yes [%] | 6 | 22 | 10 | 1 |
No [%] | 94 | 78 | 90 | 99 |
Spine surgery | ||||
N | 176 | 9 | 68 | 99 |
Yes [%] | 23 | 0 | 22 | 8 |
No [%] | 153 | 100 | 78 | 92 |
Non-invasive ventilation | ||||
N | 174 | 9 | 69 | 96 |
Always [%] | 1 | 0 | 1 | 0 |
Sometimes [%] | 12 | 22 | 22 | 4 |
None [%] | 87 | 78 | 77 | 96 |
Invasive ventilation | ||||
N | 174 | 8 | 69 | 97 |
Always [%] | 1 | 13 | 0 | 0 |
Sometimes [%] | 0 | 0 | 0 | 0 |
None [%] | 99 | 88 | 100 | 100 |