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2. 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

Author

Panse, Jens, primary, Daguindau, Nicolas, additional, Okuyama Sasaki, Sonia, additional, Peffault De Latour, Regis, additional, Schafhausen, Philippe, additional, Straetmans, Nicole, additional, Al-Adhami, Mohammed, additional, Ajayi, Temitayo, additional, Chen, Crystal, additional, Yeh, Michael, additional, and Wong, Raymond S, additional

Published
2021
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3. 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

Author

UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service d'hématologie, Panse, Jens, Daguindau, Nicolas, Okuyama Sasaki, Sonia, Peffault De Latour, Regis, Schafhausen, Philippe, Straetmans, Nicole, Al-Adhami, Mohammed, Ajayi, Temitayo, Chen, Crystal, Yeh, Michael, Wong, Raymond S, UCL - SSS/IREC/SLUC - Pôle St.-Luc, UCL - (SLuc) Service d'hématologie, Panse, Jens, Daguindau, Nicolas, Okuyama Sasaki, Sonia, Peffault De Latour, Regis, Schafhausen, Philippe, Straetmans, Nicole, Al-Adhami, Mohammed, Ajayi, Temitayo, Chen, Crystal, Yeh, Michael, and Wong, Raymond S

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.

Published
2021

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