Table 2.
Patient demographics and baseline characteristics
| Ex-trial patients | Treatment-naïve patients | All patients | |
|---|---|---|---|
| N | 26 | 29 | 55 |
| Female, number (%) | 13 (50.0%) | 15 (51.7%) | 28 (50.9%) |
| Male, number (%) | 13 (50.0%) | 14 (48.3%) | 27 (49.1%) |
| Age at enrolment, years | |||
| N | 26 | 29 | 55 |
| Mean (SD) | 14.4 (11.43) | 15.3 (15.30) | 14.9 (13.50) |
| Median | 9 | 8 | 9 |
| Min, max | 4, 41 | 2, 58 | 2, 58 |
| Weight (kg) | |||
| N | 26 | 25 | 51 |
| Mean (SD) | 28.0 (13.16) | 25.04 (15.72) | 26.6 (14.58) |
| Median | 23.8 | 16.5 | 21.7 |
| Min, max | 14.2, 65.0 | 10.3, 62.6 | 10.3, 65.0 |
| Treatment duration, years* | |||
| N | 26 | 29 | 55 |
| Mean (SD) | 7.46 (1.62) | 2.36 (0.88) | 4.78 (2.86) |
| Median | 6.96 | 2.98 | 3.00 |
| Min, max | 4.08, 9.54 | 0.99, 3.04 | 0.99, 9.54 |
| 6MWT, m | |||
| N | 23 | 24 | 47 |
| Mean (SD) | 220.1 (91.47) | 193.5 (105.80) | 206.7 (98.86) |
| Median | 228 | 184.5 | 215 |
| Min, max | 60, 433 | 20, 420 | 20, 433 |
| FVC, L | |||
| N | 25 | 18 | 43 |
| Mean (SD) | 0.9 (0.66) | 1.2 (0.97) | 1.0 (0.80) |
| Median | 0.6 | 0.8 | 0.7 |
| Min, max | 0.3, 2.9 | 0.3, 3.9 | 0.3, 3.9 |
| FEV1, L | |||
| N | 25 | 18 | 43 |
| Mean (SD) | 0.8 (0.55) | 0.9 (0.69) | 0.8 (0.61) |
| Median | 0.6 | 0.7 | 0.6 |
| Min, max | 0.2, 2.5 | 0.3, 2.4 | 0.2, 2.5 |
| LVEF, % | |||
| N | 21 | 22 | 43 |
| Mean (SD) | 66 (6) | 66 (10) | 65 (8.2) |
| Median | 65 | 69 | 65 |
| Min, max | 55, 80 | 36, 82 | 36, 82 |
| uKS, µg/mg creatinine | |||
| N | 26 | 25 | 51 |
| Mean (SD) | 28.0 (16.01) | 37.5 (23.3) | 32.7 (20.27) |
| Median | 32.4 | 44.3 | 35.1 |
| Min, max | 3.5, 50.3 | 2.6, 74.7 | 2.6, 74.7 |
*Treatment duration at last follow-up
FVC forced vital capacity, FEV1 forced expiratory volume in one second, kg kilograms, LVEF left ventricular ejection fraction, N number of patients, SD standard deviation, uKS urine keratan sulphate