C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is treated with complement inhibitors, yet incomplete responses remain a challenge. Terminal inhibition with eculizumab prevents intravascular hemolysis but often leaves residual extravascular hemolysis, while proximal inhibitors such as pegcetacoplan mitiga...
| Autores: | , , , , , , , , |
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| Tipo de recurso: | artículo |
| Estado: | Versión publicada |
| Fecha de publicación: | 2025 |
| País: | España |
| Institución: | Consejo Superior de Investigaciones Científicas (CSIC) |
| Repositorio: | DIGITAL.CSIC. Repositorio Institucional del CSIC |
| OAI Identifier: | oai:digital.csic.es:10261/412352 |
| Acceso en línea: | http://hdl.handle.net/10261/412352 https://api.elsevier.com/content/abstract/scopus_id/105024590466 |
| Access Level: | acceso abierto |
| Palabra clave: | AP50 Complement Eculizumab Genetics Pegcetacoplan |
| Sumario: | Paroxysmal nocturnal hemoglobinuria (PNH) is treated with complement inhibitors, yet incomplete responses remain a challenge. Terminal inhibition with eculizumab prevents intravascular hemolysis but often leaves residual extravascular hemolysis, while proximal inhibitors such as pegcetacoplan mitigate extravascular hemolysis though intravascular hemolysis may persist. Understanding resistance mechanisms is critical to guide therapy. |
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