Bone Marrow Failure Disorders

In this review, comparing study 301 and study 302, the authors found weight-based dosing of ravulizumab (Q 8 wk) was associated with fewer episodes of breakthrough hemolysis (BTH) versus eculizumab (900 mg Q 2 wk) over 26 weeks of complement inhibitor therapy in patients with paroxysmal nocturnal hemoglobinuria and high disease activity. They conclude that “[t]he observed differences in BTH rates for ravulizumab versus eculizumab may be attributable to the ability of ravulizumab to completely inhibit free C5 [complement protein 5] over the entire 8-week dosing interval.”