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...

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Authors: Rodríguez de Córdoba, Santiago, Reparaz, Andrea, González Sanz, Silvia, Fernández, Francisco J., Vega, María Cristina, Varela, Enrique Colado, Lada Colunga, Alejandro, Vallejo Llamas, Juan Carlos, Gonzalez-Rodríguez, Ana Pilar
Format: article
Status:Published version
Publication Date:2025
Country:España
Institution:Consejo Superior de Investigaciones Científicas (CSIC)
Repository:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/412352
Online Access:http://hdl.handle.net/10261/412352
https://api.elsevier.com/content/abstract/scopus_id/105024590466
Access Level:Open access
Keyword:AP50
Complement
Eculizumab
Genetics
Pegcetacoplan
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network_acronym_str ES
network_name_str España
repository_id_str
spelling C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuriaRodríguez de Córdoba, SantiagoReparaz, AndreaGonzález Sanz, SilviaFernández, Francisco J.Vega, María CristinaVarela, Enrique ColadoLada Colunga, AlejandroVallejo Llamas, Juan CarlosGonzalez-Rodríguez, Ana PilarAP50ComplementEculizumabGeneticsPegcetacoplanParoxysmal 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.The author(s) declare financial support was received for the research and/or publication of this article. This work was supported by a Spanish grant from the Regional Government of Madrid (S2022/BMD-7278), the Instituto de Salud Carlos III (PI23/01576 and PI24/00925) co-funded by the European Union, the Spanish Ministerio de Ciencia e Innovación-Recovery, Transformation and Resilience Plan (PRTR) grants PDC2022-133713-I00 and CPP2022-009838, Fundación Xti Xtod@s, the Fundación LAIR, the Fundación Alimerka and the Government of the Principality of Asturias through the Agency for Science, Business Competitiveness and Innovation of the Principality of Asturias and co-funded by the European Union, through the Grants for Research Groups of Organizations of the Principality of Asturias for Fiscal Year 2024, with file number IDE/2024/000745. It was additionally supported by the Research Network on Complement in Health and Disease (RED2022-134750-T). We acknowledge support of the publication fee by the CSIC Open Access Publication Support Initiative (PROA) through its Unit of Information Resources for Research (URICI).Peer reviewedFrontiers MediaComunidad de MadridInstituto de Salud Carlos IIIMinisterio de Ciencia e Innovación (España)Fundación Xti Xtod@sFundación LAIRFundación AlimerkaPrincipado de AsturiasCSIC - Unidad de Recursos de Información Científica para la Investigación (URICI)Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]202620262025info:eu-repo/semantics/articlehttp://purl.org/coar/resource_type/c_6501Publisher's versioninfo:eu-repo/semantics/publishedVersionapplication/pdfhttp://hdl.handle.net/10261/412352https://api.elsevier.com/content/abstract/scopus_id/105024590466reponame:DIGITAL.CSIC. Repositorio Institucional del CSICinstname:Consejo Superior de Investigaciones Científicas (CSIC)Inglés#PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE##PLACEHOLDER_PARENT_METADATA_VALUE#S2022/BMD-7278info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2021-2023/PDC2022-133713-I00PI24/00925PI23/01576info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2021-2023/CPP2022-009838IDE/2024/000745RED2022-134750-Thttps://doi.org/10.3389/fimmu.2025.1723596Síinfo:eu-repo/semantics/openAccessoai:digital.csic.es:10261/4123522026-05-22T06:33:51Z
dc.title.none.fl_str_mv C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
title C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
spellingShingle C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
Rodríguez de Córdoba, Santiago
AP50
Complement
Eculizumab
Genetics
Pegcetacoplan
title_short C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
title_full C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
title_fullStr C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
title_full_unstemmed C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
title_sort C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
dc.creator.none.fl_str_mv Rodríguez de Córdoba, Santiago
Reparaz, Andrea
González Sanz, Silvia
Fernández, Francisco J.
Vega, María Cristina
Varela, Enrique Colado
Lada Colunga, Alejandro
Vallejo Llamas, Juan Carlos
Gonzalez-Rodríguez, Ana Pilar
author Rodríguez de Córdoba, Santiago
author_facet Rodríguez de Córdoba, Santiago
Reparaz, Andrea
González Sanz, Silvia
Fernández, Francisco J.
Vega, María Cristina
Varela, Enrique Colado
Lada Colunga, Alejandro
Vallejo Llamas, Juan Carlos
Gonzalez-Rodríguez, Ana Pilar
author_role author
author2 Reparaz, Andrea
González Sanz, Silvia
Fernández, Francisco J.
Vega, María Cristina
Varela, Enrique Colado
Lada Colunga, Alejandro
Vallejo Llamas, Juan Carlos
Gonzalez-Rodríguez, Ana Pilar
author2_role author
author
author
author
author
author
author
author
dc.contributor.none.fl_str_mv Comunidad de Madrid
Instituto de Salud Carlos III
Ministerio de Ciencia e Innovación (España)
Fundación Xti Xtod@s
Fundación LAIR
Fundación Alimerka
Principado de Asturias
CSIC - Unidad de Recursos de Información Científica para la Investigación (URICI)
Consejo Superior de Investigaciones Científicas [https://ror.org/02gfc7t72]
dc.subject.none.fl_str_mv AP50
Complement
Eculizumab
Genetics
Pegcetacoplan
topic AP50
Complement
Eculizumab
Genetics
Pegcetacoplan
description 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.
publishDate 2025
dc.date.none.fl_str_mv 2025
2026
2026
dc.type.none.fl_str_mv info:eu-repo/semantics/article
http://purl.org/coar/resource_type/c_6501
Publisher's version
info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.none.fl_str_mv http://hdl.handle.net/10261/412352
https://api.elsevier.com/content/abstract/scopus_id/105024590466
url http://hdl.handle.net/10261/412352
https://api.elsevier.com/content/abstract/scopus_id/105024590466
dc.language.none.fl_str_mv Inglés
language_invalid_str_mv Inglés
dc.relation.none.fl_str_mv #PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
#PLACEHOLDER_PARENT_METADATA_VALUE#
S2022/BMD-7278
info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2021-2023/PDC2022-133713-I00
PI24/00925
PI23/01576
info:eu-repo/grantAgreement/AEI/Plan Estatal de Investigación Científica y Técnica y de Innovación 2021-2023/CPP2022-009838
IDE/2024/000745
RED2022-134750-T
https://doi.org/10.3389/fimmu.2025.1723596
Sí
dc.rights.none.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv application/pdf
dc.publisher.none.fl_str_mv Frontiers Media
publisher.none.fl_str_mv Frontiers Media
dc.source.none.fl_str_mv reponame:DIGITAL.CSIC. Repositorio Institucional del CSIC
instname:Consejo Superior de Investigaciones Científicas (CSIC)
instname_str Consejo Superior de Investigaciones Científicas (CSIC)
reponame_str DIGITAL.CSIC. Repositorio Institucional del CSIC
collection DIGITAL.CSIC. Repositorio Institucional del CSIC
repository.name.fl_str_mv
repository.mail.fl_str_mv
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