Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter
(2021)
Files
1-s20-S0049017221000986-main.pdf
Open Access - Adobe PDF
- 374.44 KB
Details
- Authors
- Abstract
- Background: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, potentially life-threatening hematologic disease characterized by various degrees of hemolysis, bone marrow failure, and thrombophilia. Pegcetacoplan (PEG), a C3 complement-inhibitor approved by the FDA for treatment of adults with PNH, has demonstrated improved hemoglobin (Hb) levels for PNH patients with screening Hb levels <10.5 g/dL and prior suboptimal response to eculizumab (ECU; C5-inhibitor) (Hillmen P, et al., N Engl J Med, 2021 384 (11):1028-1037) or complement-inhibitor naïve PNH patients (Wong RS, et al., Blood, 2020 136 [Supplement 1]). While these studies have demonstrated positive results for patients with lower baseline Hb levels, the efficacy and safety of PEG in PNH patients with baseline Hb ≥10.0 g/dL has not been evaluated.
- Affiliations
Citations
Panse, J., Daguindau, N., Okuyama Sasaki, S., Peffault De Latour, R., Schafhausen, P., Straetmans, N., Al-Adhami, M., Ajayi, T., Chen, C., Yeh, M., & Wong, R. S. (2021). Post Hoc Analysis of the Effect of Pegcetacoplan Treatment of Patients with Paroxysmal Nocturnal Hemoglobinuria and Baseline Hemoglobin Levels Greater Than 10 Grams per Deciliter. Blood, 138(Supplement 1), 2194. https://doi.org/10.1182/blood-2021-146962 (Original work published 2021)