Table 1.
Numbers of patients with data available for the analyses.
| Analysis | Number of patients |
|---|---|
| Received eculizumab | 96 |
| Population-PK (eculizumab serum concentration) | 94a,b |
| Serum free C5 | 95a |
| Percent cRBC hemolysis | 95a |
| CSF eculizumab concentration | 4c |
| CSF free C5 | 4c |
| Population-PK: efficacy | 95a,b |
| Population-PK: safety | 95a,b |
C5, complement component 5; cRBC, chicken red blood cell; CSF, cerebrospinal fluid; PK, pharmacokinetic.
Population-PK, serum free C5, and hemolytic-activity data were not available for one patient because of a local protocol requirement; this patient was excluded from the population-PK analysis but was included in the efficacy and safety exposure–response analyses as post-hoc exposure was predicted by the PK model.
Population-PK data were not available for one patient because of an unusual PK profile; no post-hoc PK parameters were obtained for this patient and, therefore, data for this patient were also excluded from the efficacy and safety exposure–response analyses.
Eight eculizumab-treated patients provided informed consent for CSF sampling; however, CSF samples at scheduled visits after the first dose were only available for four eculizumab-treated patients.