Table 1.
Patient characteristics
| Cohort | Development cohort (n = 46) | Validation 1 (n = 46) | Validation 2 (n = 33) |
|---|---|---|---|
| Male/Female sex, n (%) | 27 (59)/19 (41) | 32 (65)/14 (35) | 23 (70)/10 (30) |
| Spinal/Bulbar onset, n (%) | 36 (78)/10 (22) | 35 (71)/14 (29) | 26 (79)/7 (21) |
| Age at disease onset, mean (SD), years | 60.1 (11.3) | 61.9 (10.4) | 49.5 (11.7) |
| Age at baseline, mean (SD), years | 61.3 (11.6) | 63.4 (10.2) | 51.3 (11.7) |
| Disease duration at baseline, median (IQR), months | 10.1 (6.51–19.4) | 14.8 (9.4–28.4) | 20 (13.0–29.5) |
| ALSFRS-R Score at baseline, median (IQR), points | 42 (39.8–44.0) | 37 (30.5–42.0) | 39 (34.0–43.0) |
| ΔFRS, median (IQR), -pt/m | 0.56 (0.26–0.98) | 0.60 (0.34–1.15) | 0.44 (0.28–0.76) |
| BMI at baseline, median (IQR), kg/m2 | 25.4 (24.1–28.8) | 24.0 (22.6–26.7) | 23.8 (22.4–26.6) |
| Follow-up time, median (IQR), months | 13.1 (6.8–19.5) | 13.0 (9.0–18.0) | 18.0 (18.0–18.0) |
| ALSFRS-R slope in entire follow-up, median (IQR), -pt/m | 0.73 (0.34–1.16) | 1.02 (0.48–1.55) | 0.61 (0.32–0.86) |
| ALSFRS-R slope in interventional period, median (IQR), -pt/m | 0.83 (0.49–1.49); n = 40 | 0.50 (0.25–0.87); n = 33 | |
| Baseline NfL levels, median (IQR), pg/ml | 115 (64–174) | 94 (54–141) | 54 (33–86) |
| Baseline ln(NfL) levels, mean (SD), pg/ml | 4.63 (0.81) | 4.58 (0.83) | 3.95 (0.66) |
The table shows the patient characteristics of the three cohorts used for model development and validation. No ALSFRS-R slope in interventional period is specified for the development cohort, as this is not an interventional trial cohort