Characterizing clinically significant extravascular hemolysis in adults with PNH on ravulizumab or eculizumab treatment.

ABSTRACT:

Abstract

In patients with paroxysmal nocturnal hemoglobinuria (PNH), the complement component 5 (C5) inhibitors ravulizumab and eculizumab control terminal complement activity and intravascular hemolysis, which are drivers of morbidity and mortality. During C5 inhibitor treatment, ongoing C3 fragment deposition on surviving PNH red blood cells may cause clinically significant extravascular hemolysis (csEVH) in some patients. csEVH is not thought to be life threatening but may cause symptomatic anemia and need for transfusion. This post hoc analysis of studies 301 (NCT02946463) and 302 (NCT03056040) evaluated the prevalence of csEVH (symptomatic anemia [hemoglobin levels <9.5 g/dL] with absolute reticulocyte count ≥120 × 109/L) in adult patients with PNH treated with ravulizumab or

SUBMITTER: Kulasekararaj AG 

PROVIDER: S-EPMC12513279 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature