Patient baseline data and demographics
| Patient . | Indication for C5 inhibition . | Time on eculizumab before treatment with pegcetacoplan (mo) . | BTH on eculizumab before treatment with single agent (n) . | Eculizumab dose (mg) before treatment with pegcetacoplan∗ . | Granulocyte PNH population at commencement of C5 inhibition (%) . | PNH red cell population on C5 inhibition (%) . | PNH red cell population once established on pegcetacoplan (%) . | Long-term eculizumab and pegcetacoplan† . |
|---|---|---|---|---|---|---|---|---|
| 1 | Transfusion-dependent classic PNH | 206 | 3 | 1200 | 99 | 54 | 99 | No |
| 2 | Transfusion-dependent classic PNH | 40 | 1 | 1200 | 65 | 35 | 90 | Yes |
| 3 | Classic PNH with thrombosis | 84 | 4 | 900 | 89 | 28 | 97 | Yes |
| 4 | Transfusion-dependent classic PNH | 8 | 0 | 1800 | 99 | 52 | 99 | No |
| 5‡ | Transfusion-dependent classic PNH | 146 | 4 | 1200 | 68 | 25 | NA | Yes |
| Patient . | Indication for C5 inhibition . | Time on eculizumab before treatment with pegcetacoplan (mo) . | BTH on eculizumab before treatment with single agent (n) . | Eculizumab dose (mg) before treatment with pegcetacoplan∗ . | Granulocyte PNH population at commencement of C5 inhibition (%) . | PNH red cell population on C5 inhibition (%) . | PNH red cell population once established on pegcetacoplan (%) . | Long-term eculizumab and pegcetacoplan† . |
|---|---|---|---|---|---|---|---|---|
| 1 | Transfusion-dependent classic PNH | 206 | 3 | 1200 | 99 | 54 | 99 | No |
| 2 | Transfusion-dependent classic PNH | 40 | 1 | 1200 | 65 | 35 | 90 | Yes |
| 3 | Classic PNH with thrombosis | 84 | 4 | 900 | 89 | 28 | 97 | Yes |
| 4 | Transfusion-dependent classic PNH | 8 | 0 | 1800 | 99 | 52 | 99 | No |
| 5‡ | Transfusion-dependent classic PNH | 146 | 4 | 1200 | 68 | 25 | NA | Yes |
BTH, breakthrough hemolysis; NA, not available.
All eculizumab dose increases were due to breakthrough events (because of PK events).
Patients 2, 3, and 5 had pegcetacoplan added to the eculizumab regimen; planned withdrawal of eculizumab for patient 5 was not possible because of death.
Patient 5 died 2 months after starting pegcetacoplan because of noninfectious non–PNH-related causes.