Novel Insights into Factor D Inhibition |
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Authors: | Eleni Gavriilaki Anna Papakonstantinou Konstantinos A. Agrios |
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Affiliation: | 1.Hematology Department, G Papanicolaou Hospital, 57010 Thessaloniki, Greece;2.Department of Urology, Aristotle University of Thessaloniki, 54124 Thessaloniki, Greece;3.Department of Chemistry, Villanova University, 800 Lancaster Ave., Villanova, PA 19085, USA |
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Abstract: | Complement-mediated diseases or complementopathies, such as Paroxysmal nocturnal hemoglobinuria (PNH), cold agglutinin disease (CAD), and transplant-associated thrombotic microangiopathy (TA-TMA), demand advanced complement diagnostics and therapeutics be adopted in a vast field of medical specialties, such as hematology, transplantation, rheumatology, and nephrology. The miracle of complement inhibitors as “orphan drugs” has dramatically improved morbidity and mortality in patients with otherwise life-threatening complementopathies. Efficacy has been significantly improved by upstream inhibition in patients with PNH. Different molecules may exert diverse characteristics in vitro and in vivo. Further studies remain to show safety and efficacy of upstream inhibition in other complementopathies. In addition, cost and availability issues are major drawbacks of current treatments. Therefore, further developments are warranted to address the unmet clinical needs in the field of complementopathies. This state-of-the-art narrative review aims to delineate novel insights into factor D inhibition as a promising target for complementopathies. |
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Keywords: | complement factor D paroxysmal nocturnal hemoglobinuria |
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