Table 1.
Baseline characteristics of the study participants, with median values and range shown for continuous variables and group-based differences evaluated by Fisher’s exact test or Mann–Whitney U tests
| Characteristics | Younger cohort | Older cohort | p |
|---|---|---|---|
| N | 9 | 4 | |
| Female | 5 (56%) | 2 (50%) | 1.000 |
| Age at onset (months) | 2.8 (1.1, 6.4) | 5.3 (1.6, 17.2) | 0.414 |
| Feeding tube | 4 (44%) | 4 (100%) | 0.105 |
| BiPAP use | 4 (44%) | 4 (100%) | 0.105 |
| Treatment baseline | |||
| Age (months) | 12.1 (6.3, 21.0) | 137 (44, 213) | 0.003 |
| Weight (kg) | 6.70 (3.6, 8.0) | 15.5 (11.1, 28.3) | 0.003 |
| CHOP INTEND scores | 25 (14, 40) | 11.5 (8, 47) | 0.199 |
| CMAPAPB (mV) | 0.70 (0.30, 1.30) | 0.95 (0.10, 1.80) | 1.000 |
| CMAPADM (mV) | 0.30 (0.20, 0.80) | 0.45 (0.10, 1.12) | 0.940 |
| CSF biomarkers | |||
| NfL (ng/mL) | 7.16 (3.37, 18.20) | 2.10 (2.04, 3.18) | 0.024 |
| Fractalkine (pg/mL) | 34.3 (11.2, 64.3) | 38.0 (33.0, 55.0) | 0.381 |
| IL-8 (pg/mL) | 17.3 (10.4, 43.6) | 18.6 (16.8, 23.9) | 0.548 |
| IP-10 (pg/mL) | 217 (145, 468) | 203 (124, 292) | 0.714 |
| MCP-1 (pg/mL) | 751 (445, 1007) | 666 (460, 705) | 0.714 |
| sAPPα (pg/mL) | 108 (92, 215) | 149 (98, 166) | 0.714 |
| sAPPβ (pg/mL) | 178 (111, 307) | 233 (1, 267) | 0.905 |