Table 3 Outcome measures, Group 2 (age at treatment initiation >16 years).
ID | 16 | 17 | 18 | 19 | 20 | 21 | 22 | 23 | 24 | |
|---|---|---|---|---|---|---|---|---|---|---|
AGE OF ONSET AGE AT INITIATION OF TREATMENT CURRENT AGE TREATMENT DOSE (mg/kg/d) | >12 y | 50 y | 30 y | 20 y | 60 y | 36 y | 5 y | 30 y | 20 y | |
30 y | 58 y | 59 y | 31 y | 74 y | 60 y | 35 y | 46 y | 57 y | ||
34 y | 62 y | 62 y | 34 y | 75 y | 60 y | 36 y | 46 y | 60 y | ||
320 | 300 | 400 | 400 | 400 | 400 | 350 | 300 | 400 | ||
CK (UI/l) | BASAL | 2435 | 525 | 294 | 303 | 647 | 402 | 548 | 188 | 1142 |
0–6 Months | ND | ND | 123 | 322 | ND | 264 | 107 | ND | 1195 | |
6–12 Months | 393 | ND | 258 | 292 | 105 | ND | 142 | ND | 1348 | |
12–36 Months | 330 | 351 | 108 | 110 | ND | ND | ND | ND | ND | |
>36 Months | 561 | 459 | 185 | 467 | 89 | ND | ND | ND | ND | |
GDF-15 (pg/mL) | BASAL | 2423 | 1529 | 1695 | 2439 | 2483 | 1640 | 2149 | 1261 | 1980 |
0–6 Months | ND | ND | ND | ND | 1364 | 1357 | 473 | 486 | 1052 | |
6–12 Months | ND | ND | ND | ND | 1320 | ND | 413 | ND | 1011 | |
12–36 Months | 464 | 782 | 704 | 1110 | 1099 | ND | ND | ND | ND | |
>36 Months | 480 | 1137 | 753 | 783 | ND | ND | ND | ND | ND | |
FGF-21 (pg/mL) | BASAL | 666 | 252 | 353 | 197 | 897 | 185 | 943 | 190 | 179 |
0–6 Months | ND | ND | ND | ND | 732 | 135 | 47 | ND | 113 | |
6–12 Months | ND | ND | ND | ND | 344 | ND | ND | ND | 155 | |
12–36 Months | 1222 | 944 | 245 | 915 | ND | ND | ND | ND | ND | |
>36 Months | 632 | 1025 | 421 | ND | ND | ND | ND | ND | ND | |
BMI | BASAL | 13.86 | 27.7 | 27.12 | 17.79 | 26.01 | 23.7 | 26.17 | ND | 20,8 |
0–6 Months | ND | ND | ND | 19,06 | 25 | 23.6 | 25.4 | ND | 21 | |
6–12 Months | ND | ND | ND | 21,09 | 27,6 | ND | ND | ND | 20,4 | |
12–36 Months | 16.06 | ND | 26.3 | 21.23 | ND | ND | ND | ND | ND | |
>36 Months | ND | ND | 26.9 | 18,63 | ND | ND | ND | ND | ND | |
6MWT | BASAL | 532 | 475 | 386 | 390 | 345 | ND | 368 | 413 | 225 |
0–6 Months | ND | 500 | 355 | 437 | ND | ND | 435 | 515 | 251 | |
6–12 Months | ND | ND | 400 | 474 | ND | ND | 450 | ND | 228 | |
12–36 Months | 550 | 435 | 354 | 369 | ND | ND | ND | ND | ND | |
>36 Months | 600 | 442 | 450 | 476 | ND | ND | ND | ND | ND | |
NSAA | BASAL | ND | ND | 26 | 21 | 29 | ND | 16 | 30 | ND |
0–6 Months | 30 | 24 | 26 | 21 | ND | ND | 25 | 31 | ND | |
6–12 Months | ND | ND | 29 | 23 | ND | ND | 26 | ND | ND | |
12–36 Months | 29 | 27 | 32 | 28 | ND | ND | ND | ND | ND | |
>36 Months | 30 | ND | 31 | 29 | ND | ND | ND | ND | ND | |
PIM (%) | BASAL | 25 | 40 | 49.9 | 5 | 59 | ND | 37 | 31 | ND |
0–6 Months | ND | ND | 40.2 | ND | 33,9 | ND | 37,8 | ND | ND | |
6–12 Months | 29.5 | ND | 53 | 24 | 47.3 | ND | ND | ND | ND | |
12–36 Months | 21.4 | 38.6 | 49.9 | ND | 39,8 | ND | ND | ND | ND | |
>36 Months | ND | 28 | 53 | 27 | ND | ND | ND | ND | ND | |
FVC (%) | BASAL | 46.5 | 45 | 71.8 | 26 | 69 | 78 | 53.2 | 63.7 | ND |
0–6 Months | ND | 51.4 | 79.5 | 33 | 66,5 | 82.6 | 62 | 64 | 16 | |
6–12 Months | 50.3 | ND | 75 | 23,7 | 78,9 | ND | ND | ND | 17 | |
12–36 Months | 46.7 | 46.7 | 77.7 | 32,2 | 83,4 | ND | ND | ND | ND | |
>36 Months | 50.5 | 43.6 | 72 | 34,8 | ND | ND | ND | ND | ND | |
