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Complement C5 inhibitor treatment with ravulizumab or eculizumab for paroxysmal nocturnal hemoglobinuria (PNH) improves outcomes and survival. Some patients remain anemic due to clinically significant extravascular hemolysis (cs-EVH: hemoglobin [Hgb] ≤9.5 g/dL and absolute reticulocyte count [ARC] ≥120×109/L).
View Article and Find Full Text PDFPegcetacoplan: the first and only C3-targeted therapy for the treatment of adults with paroxysmal nocturnal hemoglobinuria.
Expert Rev Hematol
December 2024
Department of Haematology, Singapore General Hospital, Singapore, Singapore.
Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare acquired disorder of hematopoietic stem cells, characterized by somatic mutations of the Phosphatidylinositol Glycan Class A Gene, resulting in increased hemolysis. The advent of complement inhibitors has since changed the way clinicians approach treating PNH. Pegcetacoplan is a C3 inhibitor that has shown promise in this field and improved outcomes for patients who have been diagnosed with PNH.
View Article and Find Full Text PDFAdvancements in PNH treatment: crovalimab's clinical efficacy.
Ann Med Surg (Lond)
December 2024
Faculty of Medicine, Alzaiem Alazhari University, Khartoum, Sudan.
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematologic disease that is characterized by the destruction of red blood cells, leading to a range of severe symptoms and complications. Recent advancements in drug therapies have significantly improved the prognosis for PNH patients. This editorial comprises the impact of PNH drugs, focusing on eculizumab and ravulizumab and comparing them to the recently approved complement inhibitor, crovalimab, which targets the complement system to prevent hemolysis.
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- * Results showed stable health markers (LDH, hemoglobin, reticulocytes) throughout the study, with no significant differences in kidney disease rates or need for blood transfusions among patients.
- * The findings indicate that the biosimilar is both effective and safe for long-term use in PNH patients, with only a low incidence of adverse reactions reported.
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