C3 mutations and poor pegcetacoplan response in paroxysmal nocturnal hemoglobinuria
These findings show that C3 MG-ring mutations can undermine both terminal and proximal complement inhibitors, directly impacting treatment efficacy in PNH. Complement genetic testing, combined with functional readouts, may help identify patients at risk of suboptimal responses and support personalized therapeutic strategies.
Authors
- Juan Carlos Vallejo Llamas
- Francisco J. Fernández (ORCID: https://orcid.org/0000-0002-5015-1849)
- M. Cristina Vega (ORCID: https://orcid.org/0000-0003-0628-8378)
- Santiago Rodrı́guez de Córdoba (ORCID: https://orcid.org/0000-0001-6401-1874)
- Silvia Sanz
- Enrique Colado (ORCID: https://orcid.org/0000-0001-8675-8207)
- Ana Pilar González (ORCID: https://orcid.org/0000-0002-0565-9133)
- Alejandro Lada Colunga
- Andrea Reparaz Suevos
Institutions
- Complejo Hospitalario de Salamanca (ES)
- Hospital Universitario Central de Asturias (ES)
- Centro de Investigaciones Biológicas Margarita Salas (ES)
Publication Details
- Journal
- Frontiers in Immunology
- Published
- 2025-11-26
- DOI
- https://doi.org/10.3389/fimmu.2025.1723596
- Citations
- 1
- Primary Topic
- Complement system in diseases
- Type
- article
- Field-Weighted Citation Impact
- 0.80
Funders
- Gobierno del Principado de Asturias
- European Commission
- Instituto de Salud Carlos III