Pediatric Paroxysmal Nocturnal Hemoglobinuria in Egypt: Ravulizumab

A study published in Pediatric Research is shedding light on pediatric paroxysmal nocturnal hemoglobinuria (PNH) in Egypt, focusing on real-world outcomes for children treated in resource-limited settings. The multicenter cohort study describes PNH as an acquired clonal blood disorder involving intravascular hemolysis, bone marrow failure, and thrombosis, driven by somatic mutations in the PIGA gene that cause deficiency of GPI-anchored proteins. Researchers reported that poor outcomes in Egypt were driven primarily by diagnostic delays, meaning children were often identified after the disease had progressed. The study also highlighted the real-world effectiveness of ravulizumab, a long-acting C5 complement inhibitor, which was associated with notable clinical improvement despite constrained healthcare conditions. It contrasts ravulizumab’s dosing every eight weeks with eculizumab’s need for infusions every two weeks, which can be logistically difficult. The article notes that treatment success depends on access, and that both transplantation options and complement inhibition therapies remain limited by cost, infrastructure, and regulatory hurdles.