Table 2

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 onsetn=76; PY=68n=72; PY=650.73
 N (annualised rate; 95% CI)21 (0.31; 0.20 to 0.47)18 (0.28; 0.17 to 0.44)
 Bulbar onsetn=27; PY=24n=25; PY=230.71
 N (annualised rate; 95% CI)4 (0.17; 0.06 to 0.45)5 (0.22; 0.09 to 0.52)
 ALSFRS-R ≥33n=86; PY=79n=84; PY=770.69
 N (annualised rate; 95% CI)17 (0.22;0.13 to 0.35)19 (0.25; 0.16 to 0.39)
 ALSFRS-R <33n=17; PY=13n=13; PY=120.37
 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.