Objectives: This study investigated outcomes and response to pegcetacoplan pegcetacoplan: EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli injector or with an… in complement inhibitor naïve (CInaïve) and experienced (CIexp) PNH patients.
Methods: Data were drawn from the Adelphi PNH Disease Specific Programme™, a cross-sectional survey of physicians and their patients in Canada, France, Germany, Italy, Spain, and the UK between December 2023 and April 2024. Physicians reported patient characteristics and clinical outcomes, which were used to define pegcetacoplan response. Results were stratified by prior CI prescription. Analyses were descriptive.
Results: Forty-eight physicians reported on 63 patients (CInaïve:11; CIexp:52). Median (IQR) age was 45.0 (33.0-55.0) years, 60.3% were male. At treatment initiation versus at survey for CInaïve and CIexp, respectively, median (IQR) haemoglobin levels (g/dL) were 8.0 (5.0-9.0) versus 12.0 (11.2-12.3), and 9.0 (8.0-9.2) versus 11.0 (10.2-12.0); 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… levels (U/L) were 500 (350-1625) versus 250 (175-525), and 470 (290-588) versus 265 (190-379); 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. counts (x109 /L) were 220 (151-250) versus 110 (95-140), and 212 (134-281) versus 120 (105-228). At pegcetacoplan initiation, 4.4% of patients met criteria of good-to-complete response versus 79.5% at survey.
Conclusions: Pegcetacoplan proved effective for CInaïve and CIexp patients in a real-world setting, with numerically improved clinical outcomes and high response rate.
Keywords: Paroxysmal nocturnal haemoglobinuria; complement inhibitor; pegcetacoplan; treatment outcome.