Table 2 Outcome measures, Group 1 (age at treatment initiation <16 years).
ID | 1 | 2 | 3 | 4 | 5 | 6 | 7 | 8 | 9 | 10 | 11 | 12 | 13 | 14 | 15 | |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
AGE OF ONSET AGE AT INITIATION OF TREATMENT CURRENT AGE TREATMENT DOSE (mg/kg/d) | 23 m | 30 m | 17 m | 15 m | 13 m | 13 m | 13 m | 24 m | 31 m | 36 m | 36 m | 24 m | 24 m | 36 m | 6 y | |
27 m | 10 y | 30 m | 2.5 y | 15 m | 15 m | 14 m | 33 m | 6 y | 14 y | 12 y | 3 y | 3 y | 7 y | 9 y | ||
5 y | 13 y | 7 y | 7 y | 21 m | 21 m | 24 m | 3 y | 7 y | 16 y | 13 y | 5 y | 5 y | 9 y | 10 y | ||
400 | 300 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | 400 | ||
CK | BASAL | 1183 | 538 | 148 | 284 | 872 | 940 | 921 | 285 | 626 | 1789 | 759 | 460 | 945 | 184 | 742 |
0–6 Months | ND | 316 | ND | ND | 649 | 561 | 275 | 133 | 175 | 235 | 243 | 606 | 857 | 164 | 269 | |
6–12 Months | 114 | ND | 68 | 132 | ND | ND | 164 | 130 | 165 | 273 | 290 | 173 | 87 | 111 | 195 | |
12–24 Months | 55 | 712 | ND | 116 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | 87 | 285 | ND | 61 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
GDF-15 (pg/mL) | BASAL | 15000 | 4608 | 14756 | ND | 21641 | 22767 | 27066 | ND | ND | 4797 | 2641 | 3686 | 3841 | 5006 | 2781 |
0–6 Months | 1200 | 762 | ND | ND | 6628 | 2174 | 588 | 4597 | 6825 | 4753 | 3105 | ND | 2635 | 5180 | 2249 | |
6–12 Months | 497 | ND | 5093 | 2021 | ND | ND | ND | 396 | 369 | ND | ND | 341 | 330 | 440 | 345 | |
12–24 Months | 392 | 2084 | 594 | 255 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | 288 | 631 | ND | 284 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
FGF-21 (pg/mL) | BASAL | 1373 | 402 | 3013 | ND | 4088 | 3245 | 7531 | ND | ND | 809 | 398 | 786 | 471 | 1057 | 595 |
0–6 Months | 72 | 228 | ND | ND | 1445 | 776 | 208 | 583 | 743 | 769 | 1090 | ND | 351 | 1077 | 417 | |
6–12 Months | 31 | ND | 2170 | 415 | ND | ND | ND | ND | ND | ND | ND | 42 | <LOQ | <LOQ | 34 | |
12–24 Months | 30 | 689 | 603 | 51 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | 37 | 134 | ND | 100 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
BMI | BASAL | <P3 | <P3 | P3 | P1 | 13,2 | 13,5 | 12,7 | ND | ND | 15,3 | 20,2 | 13,5 | 16 | 11,5 | 10,5 |
0–6 Months | ND | 9.6 | ND | ND | 12,8 | 13,2 | 12,9 | ND | ND | 16,1 | 19,05 | 15,02 | 15,08 | 11 | 10,7 | |
6–12 Months | 17.2 | ND | 16.02 | 12.75 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
12–24 Months | 16.3 | 20 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | 14.9 | 25.3 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
6MWT | BASAL | 0 | ND | ND | ND | ND | ND | ND | ND | 420 | 75 | 342 | 250 | 272 | 0 | 307 |
0–6 Months | 150 | ND | ND | ND | ND | ND | ND | ND | 531 | 350 | 354 | ND | ND | ND | 364 | |
6–12 Months | 250 | ND | ND | ND | ND | ND | ND | ND | 512 | 379 | 375 | 394 | 368 | 15 | ND | |
12–24 Months | 391 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | 477 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
EK2 | BASAL | ND | 26 | 30 | 28 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND |
0–6 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
6–12 Months | ND | 9 | 21 | 14 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
12–24 Months | ND | ND | ND | 12 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | 13 | ND | 11 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
NSAA | BASAL | 4 | ND | ND | ND | ND | ND | ND | 20 | 27 | ND | ND | ND | ND | ND | ND |
0–6 Months | 6 | ND | ND | ND | ND | ND | ND | ND | 33 | ND | ND | ND | ND | ND | ND | |
6–12 Months | 20 | ND | ND | ND | ND | ND | ND | 31 | 33 | ND | ND | ND | ND | ND | ND | |
12–24 Months | 26 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
HMFS | BASAL | ND | ND | ND | ND | ND | ND | ND | ND | ND | 21 | 35 | 38 | 38 | 17 | 38 |
0–6 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | 30 | 33 | ND | ND | ND | 39 | |
6–12 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | 36 | 37 | 39 | 39 | 23 | ND | |
12–24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
HMFSE | BASAL | ND | ND | ND | ND | ND | ND | ND | ND | ND | 26 | 51 | 51 | 53 | 19 | 62 |
0–6 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | 48 | 49 | ND | ND | ND | 65 | |
6–12 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | 57 | 55 | 65 | 65 | 27 | ND | |
12–24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
CHOP INTEND | BASAL | ND | ND | ND | ND | 42 | 45 | 17 | ND | ND | ND | ND | ND | ND | ND | ND |
0–6 Months | ND | ND | ND | ND | 45 | 54 | 64 | ND | ND | ND | ND | ND | ND | ND | ND | |
6–12 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
12–24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
FVC (%) | BASAL | ND | 13 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | 38 |
0–6 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | 19 | 50 | ND | ND | ND | 66 | |
6–12 Months | ND | 22 | ND | ND | ND | ND | ND | ND | ND | 35 | 70 | ND | ND | 71 | ND | |
12–24 Months | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | |
>24 Months | ND | 26 | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND | ND |