Table 1.
Literature review regarding pegcetacoplan use in paroxysmal nocturnal hemoglobinuria.
| Reference | Trial name | N° of patients | Patient population | Phase of study | Comments |
|---|---|---|---|---|---|
| Grossi FV et al., Blood 2016 (5) | – | 51 | Healthy volunteers | Phase I | Single and multiple doses of Peg were safe; serum concentrations close to steady state reached after 28 days of daily dosing. A reduction in AP50 was seen following a single dose of 1440mg and multiple daily doses of 180 and 270 mg. |
| Wong RSM, et al, Annals of Hematology 2022 (6) | PADDOCK | 26 | PNH naive | Phase Ib | Hemolytic markers and Hb values normalization with Peg, 65% achieved transfusion independence. One hypersensitivity event related to Peg. No thrombotic events or infectious complications. |
| Wong RSM, et al, Annals of Hematology 2022 (6) | PALOMINO | 4 | PNH naive | Phase IIa | 100% transfusion independence. No SAE related to Pegcetacoplan. |
| De Castro C et al, American J. of Hematology 2020 (7) | PHAROAH | 6 | PNH on C5-inhibitor | Phase Ib | Pegcetacoplan improved hematological response and enabled eculizumab discontinuation and transfusion independence in 4 patients. |
| Wong RSM et al, Ther Adv Hematology 2022 (2) | PRINCE | 53 | PNH naive | Phase III | Pegcetacoplan vs SoC excluding complement inhibitors. Hb and LDH normalization in 45.7% and 65.7% of patients respectively with Peg, 0% with SoC. No SAE registered. |
| Hillmen P et al, NEJM 2021 (3) | PEGASUS | 80 | PNH on C5-inhibitor | Phase III | Pegcetacoplan proved superior to eculizumab in Hb increase and transfusion independence. Safety profile comparable to eculizumab, and lower rate of BTH with pegcetacoplan. Overall, in patients with suboptimal response to eculizumab, pegcetacoplan treatment is effective and well tolerated. |
| Bhak RY et al, Blood 2022 (8) | PEGASUS and 302 | 69 and 195 | PNH on C5-inhibitor | Matching-adjusted indirect comparison | MAIC allowed examination of the comparative effectiveness of pegcetacoplan vs ravulizumab in the absence of a head-to-head trial. Pegcetacoplan superior to ravulizumab in terms of transfusion avoidance. |
| Tamura S et al, British J. of Hematology 2022 (9) | – | 1 | PNH on C5-inhibitor | Case Report | Pegcetacoplan effective in one patient with PNH and C5 genetic variant, non-responding to eculizumab. |
Peg, pegcetacoplan; PNH, paroxysmal nocturnal hemoglobinuria; SoC, standard of care; SAE, serious adverse events; MAIC, matching adjusted indirect comparison; BTH, breakthrough hemolysis.