In 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), the complement component 5 (C5) inhibitors ravulizumab and 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. … control terminal complement activity and intravascular hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. , 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 anemia: (uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness. 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 reticulocyte: An immature red blood cell. Reticulocytes are normally found in the bone marrow. They are present in the bloodstream only in very low numbers. count ≥120 × 109/L) in adult patients with PNH treated with ravulizumab or eculizumab for 6 months (183 days). The association between csEVH and fatigue (measured using the Functional Assessment of Chronic Illness Chronic Illness: A medical condition that lasts a long time. A chronic illness can affect a person's lifestyle, ability to work, physical abilities and independence. Therapy - Fatigue [FACIT-F] scale) was also evaluated in study 302 patients with stable disease. On day 183 of studies 301 and 302, csEVH prevalence was 23.2% (29/125) and 20.2% (19/94) with ravulizumab and 24.8% (30/121) and 21.3% (20/94) with eculizumab, respectively. All patients in study 302 with csEVH experienced fatigue, which was mostly mild (ravulizumab, 79%; eculizumab, 65%) or moderate (ravulizumab, 21%; eculizumab, 30%). FACIT-F scores remained stable from baseline (ravulizumab, 42.2; eculizumab, 38.3) to day 183 (ravulizumab, 44.3; eculizumab, 38.3), close to general population normative values. This analysis demonstrated that csEVH affected 20% to 25% of patients with PNH, most of whom experienced mild fatigue, with fatigue remaining stable during treatment. Ravulizumab and eculizumab continue to provide benefit to adults with PNH with csEVH.
© 2025 American Society of Hematology. Published by Elsevier Inc. Licensed under Creative Commons Attribution-NonCommercial-NoDerivatives 4.0 International (CC BY-NC-ND 4.0), permitting only noncommercial, nonderivative use with attribution. All other rights reserved.