Table 3.
Parents’ assessment of patients’ condition.
| Parameter | Scores given to patients by parentsa | |||||||
|---|---|---|---|---|---|---|---|---|
| Patient no. | ||||||||
| IIIA-2 | IIIA-4 | IIIA-5 | IIIA-6 | IIIA-7 | IIIB-8 | IIIB-2 | IIIB-5 | |
| Speech performance | 0/+1/0 | 0/+1/0 | −1/0/0 | 0/+1/−1 | −1/0/0 | 0/0/0 | 0/0/0 | −1/+2/+2 |
| Speech understanding | 0/+1/0 | 0/+1/0 | −1/+1/+1 | 0/+2/0 | 0/+1/+1 | 0/+1/+1 | 0/+1/+1 | −1/+2/+1 |
| Activity | 0/+1/+1 | 0/+2/+2 | −1/+2/+2 | 0/+2/+1 | 0/+1/0 | −1/0/+1 | 0/0/+1 | 0/+1/0 |
| General behavior | 0/+1/+1 | 0/+2/0 | −1/+2/+2 | 0/+1/+1 | 0/+1/+1 | 0/+1/+2 | 0/+1/+1 | −1/+2/+2 |
| Sleeping problems | 0/+1/+1 | 0/+1/+2 | −1/+2/+2 | 0/+1/+1 | 0/0/0 | 0/+2/+2 | −1/+1/+1 | −2/+2/+2 |
| Pain | 0/0/+1 | 0/0/0 | 0/0/0 | 0/0/+1 | 0/0/−1 | +1/+2/+2 | 0/0/0 | −2/+2/+2 |
| Walking | 0/+2/−1 | 0/+1/−2 | 0/+2/0 | 0/+2/+2 | 0/0/0 | −1/0/0 | −1/0/+1 | −2/+1/0 |
| Hand mobility | 0/0/+1 | 0/+1/−1 | −1/+2/+2 | 0/0/+2 | 0/0/0 | 0/0/+1 | 0/0/+1 | 0/+1/+2 |
| Joint mobility | 0/0/+1 | 0/+2/−1 | −1/+2/0 | 0/0/+1 | 0/0/0 | 0/0/0 | 0/0/0 | −1/+1/+2 |
| Breathing problems (day) | 0/+1/+2 | +1/+2/0 | 0/0/0 | 0/−1/0 | 0/+2/0 | 0/0/0 | 0/0/0 | 0/0/+2 |
| Breathing problems (night) | 0/+1/+2 | +1/+2/+1 | 0/+2/+2 | 0/−1/+1 | 0/+2/0 | 0/0/0 | 0/0/0 | −1/+2/+2 |
| Infections | −1/+2/+2 | +2/+2/+2 | 0/+1/+2 | 0/−1/0 | 0/0/+2 | 0/0/0 | 0/+2/+2 | −1/+2/+2 |
| Vision | 0/0/0 | 0/0/0 | 0/0/+2 | 0/0/0 | 0/0/+1 | 0/0/0 | −1/0/+2 | 0/0/0 |
| Hearing | 0/0/0 | 0/+2/0 | 0/0/+2 | 0/+2/−1 | 0/0/0 | 0/0/0 | 0/0/0 | 0/0/+2 |
| Skin | 0/+2/+2 | 0/+1/+1 | 0/0/0 | 0/0/0 | 0/0/+2 | 0/+2/+2 | 0/0/0 | −1/+2/+2 |
| Hair | 0/+2/+2 | 0/+1/+1 | −1/+2/+2 | 0/+2/−1 | 0/+1/+1 | 0/+2/+2 | 0/0/+1 | −1/+2/+2 |
| Dyspeptic symptoms | 0/+1/+2 | 0/+1/+1 | −1/+2/+2 | 0/+2/−1 | 0/0/0 | −1/+2/+1 | −1/0/0 | −1/+2/+2 |
| Stools | 0/0/+2 | 0/−1/−1 | −1/+2/+2 | 0/+1/+2 | 0/0/0 | −1/+2/+1 | 0/+1/+2 | −1/+2/+2 |
a
The first number (before the first slash sign) is a score for the comparison of changes in a particular child from a year before the onset of the treatment to the start of the therapy, the number between slashes is a score for the comparison of changes in a particular child from the onset of the treatment to a year after the start of the therapy, and the number after the second slash is a score for the comparison of changes in a particular child from the onset of the treatment to 3 years after the start of the therapy. The following score scale was used: considerably deteriorated (−2), somewhat deteriorated (−1), no changes (0), somewhat improved (+1), considerably improved (+2).