Real-World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International PNH Registry

Ravulizumab, a second-generation complement component 5 inhibitor (C5i) derived from eculizumab eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … , with improved pharmacokinetics, is the current standard-of-care treatment for patients with paroxysmal nocturnal hemoglobinuria paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH), where available. Pivotal trials have demonstrated durable long-term efficacy, safety, and improved survival, and increasing real-world evidence is required. This analysis utilized data from the International PNH Registry (NCT01374360) to evaluate the real-world effectiveness and safety of ravulizumab, and to assess baseline characteristics at treatment initiation. Adults (aged 18-65 years) enrolled in the registry as of July 1, 2024, who were treated with ravulizumab for ≥ 6 months were included and stratified by prior eculizumab use (eculizumab-experienced and C5i-naive). Ravulizumab treatment outcomes were assessed for up to 24 months and included change in lactate dehydrogenase lactate dehydrogenase: (LAK-tate dee-high-DROJ-uh-nase) An enzyme found in the blood and in many of the body's organs. High levels of LDH in the blood can mean that red blood cells are breaking apart (hemolysis) or that there is tissue damage in the body. It is important for patients with paroxysmal nocturnal… (LDH) ratio from baseline, transfusion avoidance, and adverse event rates. Data for 203 eculizumab-experienced and 23 C5i-naive patients were analyzed. During treatment, LDH ratio was maintained near normal (< 1.5 × the upper limit of normal in both groups); 86.8% of eculizumab-experienced and 76.5% of C5i-naive patients were transfusion-independent, and a low rate of major adverse vascular events was reported (eculizumab-experienced, 0.4 per 100 person-years; C5i-naive, no events). These findings reinforce the pivotal trial outcomes and demonstrate the effectiveness and safety of ravulizumab in the real-world setting, further supporting ravulizumab as the first-line treatment of choice for patients with PNH, where available. Trial Registration: International PNH Registry: NCT01374360.