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hemolysis

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 weeks

26th Jun 2024

PEGASUS study: A plain language summary looking treatment of paroxysmal nocturnal hemoglobinuria with pegcetacoplan for 48 weeks

This plain language summary is about a phase 3 clinical trial called PEGASUS. The PEGASUS trial studied adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder usually acquired in adulthood without a known cause. Patients with PNH have defects in the complement system, which is part of the immune defense system. This complement defect results in the destruction of red blood cells; this is called hemolysis. Hemolysis then causes anemia. People with anemia do not have enough red blood cells to carry oxygen around the body, which can cause fatigue (extreme tiredness), shortness of breath, or headache. Anemia can be measured by the level or amount of hemoglobin in the blood. Hemolysis can be measured by the amount of hemoglobin, lactate dehydrogenase (LDH), and reticulocytes in the blood. Hemoglobin and LDH are proteins inside red blood cells. In patients with PNH, hemolysis causes hemoglobin levels to go down and LDH levels go up. Reticulocytes are immature red blood cells; their level goes up during hemolysis to replace destroyed red blood cells. Eculizumab is the current standard of care for patients with PNH. Eculizumab is a medicine that blocks or inhibits the complement component 5 (C5). Pegcetacoplan is a new medicine, the first that inhibits the complement component C3. The PEGASUS study compared the new medicine pegcetacoplan with eculizumab in adults with PNH who remained anemic even after being treated with eculizumab for at least 3 months. In PEGASUS, 41 participants received pegcetacoplan and 39 people received eculizumab for 16 weeks.

25th Jul 2023

hemolysis

PEGASUS study: A plain language summary looking treatment of paroxysmal nocturnal hemoglobinuria with pegcetacoplan for 48 weeks

Plain Language Summary: the PEGASUS study comparing pegcetacoplan with eculizumab for 16 weeks in people with paroxysmal nocturnal hemoglobinuria