Rafael Alejandro Camero Del Vecchio: Paroxysmal Nocturnal Hemoglobinuria Advancing Complement Inhibition with Ravulizumab
Rafael Alejandro Camero Del Vecchio, University professor medicine at Universidad Hispanoamericana (CR), shared a post on LinkedIn:
“Chapter 3.
Rare Diseases. Paroxysmal Nocturnal Hemoglobinuria (PNH) is an acquired clonal hematopoietic stem cell disorder caused by a somatic mutation in the PIGA gene, resulting in loss of GPI anchored complement regulatory proteins (CD55, CD59).
This deficiency leads to uncontrolled terminal complement activation, chronic intravascular hemolysis, anemia, thrombosis, and variable marrow failure.
Free hemoglobin amplifies platelet activation and drives the disease’s marked thrombotic risk. Diagnosis relies on high sensitivity flow cytometry.
Ultomiris is a long acting monoclonal antibody inhibitor of complement C5, engineered to provide sustained terminal complement blockade.
It is approved for adults and pediatric patients higher 10 kg with PNH, including C5 inhibitor naïve and eculizumab experienced patients.
By preventing C5 cleavage, Ultomiris suppresses formation of the membrane attack complex (C5b 9), reducing intravascular hemolysis, improving hemoglobin stability, and lowering thrombotic risk.
Its extended half life allows significantly longer dosing intervals compared with eculizumab while maintaining complete complement inhibition.
As Clinical Immunologist. PNH is a prototypical complement mediated immunologic disease.Ravulizumab provides durable terminal complement inhibition, improving hemolysis control and survival.
Requires meningococcal vaccination and monitoring for breakthrough hemolysis.
References:
Agencia Española de Medicamentos y Productos Sanitarios. Informe de Posicionamiento Terapéutico de ravulizumab (Ultomiris®) en paroxysmal nocturnal hemoglobinuria. IPT 61 2022; 2022.
European Medicines Agency. Ultomiris; INN ravulizumab.
EMA 162681 2023.Gerber GF, Emadi A. Paroxysmal nocturnal hemoglobinuria. MSD Manual Professional Edition; 2026.
Kulasekararaj A, Brodsky R, Schrezenmeier H, Griffin M, Röth A, Piatek C, et al. Ravulizumab demonstrates long term efficacy, safety and favorable patient survival in patients with paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2025;104:81 94″
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