Analysis of efficacy for the primary outcome at 12-month follow-up
| rhEPO (n=103; PY=92) | Placebo (n=97; PY=88) | p Value | |
|---|---|---|---|
| Overall events (death, tracheotomy, >23 h NIV) | |||
| N (annualised rate; 95% CI) | 25 (0.27; 0.18 to 0.40) | 23 (0.26; 0.17 to 0.39) | 0.88 |
| Death | |||
| N (annualised rate; 95% CI) | 10 (0.11; 0.06 to 0.20) | 7 (0.08; 0.04 to 0.17) | 0.52 |
| Tracheotomy or >23 h NIV | |||
| N (annualised rate; 95% CI) | 15 (0.16; 0.10 to 0.27) | 16 (0.18; 0.11 to 0.30) | 0.77 |
| Overall events stratified by disease onset and severity | |||
| Spinal onset | n=76; PY=68 | n=72; PY=65 | 0.73 |
| N (annualised rate; 95% CI) | 21 (0.31; 0.20 to 0.47) | 18 (0.28; 0.17 to 0.44) | |
| Bulbar onset | n=27; PY=24 | n=25; PY=23 | 0.71 |
| N (annualised rate; 95% CI) | 4 (0.17; 0.06 to 0.45) | 5 (0.22; 0.09 to 0.52) | |
| ALSFRS-R ≥33 | n=86; PY=79 | n=84; PY=77 | 0.69 |
| N (annualised rate; 95% CI) | 17 (0.22;0.13 to 0.35) | 19 (0.25; 0.16 to 0.39) | |
| ALSFRS-R <33 | n=17; PY=13 | n=13; PY=12 | 0.37 |
| N (annualised rate; 95% CI) | 8 (0.60; 0.30 to 1.20) | 4 (0.35; 0.13 to 0.93) | |
The analysis was performed in terms of the annualised rate with the corresponding 95% CI and p value using a χ2 test with one degree of freedom for rate comparison (based on Poisson regression).
NIV, non-invasive ventilation; PY, total patient-years of follow-up; rhEPO, recombinant human erythropoietin.