Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, seriousblood disease, characterized by uncontrolled activation ofthe complement system that causes hemolysis (destruction ofred blood cells). The complement component 5 (C5) inhibitoreculizumab was the first approved treatment for PNH. ThePEGASUS trial compared eculizumab with pegcetacoplan, a newcomplement component 3 (C3) inhibitor. Because C3 is activatedbefore C5, blocking C3 would also block C5; thus, a C3 inhibitormight prevent hemolysis more completely than a C5 inhibitorin patients with PNH. During the first 16 weeks of PEGASUS,patients received either pegcetacoplan or eculizumab; resultswere published separately. This summary describes results ofthe following 32 weeks of PEGASUS, during which all patientsreceived pegcetacoplan to evaluate if pegcetacoplan continued to be effective and safe for up to 48 weeksThis Plain Language Summary of Publication article (PLSP) from Future Rare Diseases looks at a rare blood disease called Paroxysmal nocturnal hemoglobinuria (PNH). The PEGASUS study, discussed in this summary, describes the results of the 32 weeks study, during which all patients received a medication called pegcetacoplan. Researchers evaluated whether pegcetacoplan continued to be effective and safe for up to 48 weeks.

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This PLSP is based on an article called ‘Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase 3, active-comparator, controlled trial’ that was published in The Lancet Haematology. 

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