Szerző szerinti böngészés "Cohen, Shay Shalom"

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    Treatment of paroxysmal nocturnal hemoglobinuria
    Cohen, Shay Shalom; Pórszász, Róbert; Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet; DE--Általános Orvostudományi Kar; Halasi, Barbara Dóra; Szentmiklósi, A. József; Általános Orvostudományi Kar::Igazságügyi Orvostani Intézet; Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet
    Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening hematological disorder characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure. Despite improvements in comprehending the pathophysiology, finding effective pharmacological treatments is still a clinical challenge. This thesis explores the current situation of pharmacological treatments for PNH, with the goal of understanding how they work, their effects on the body, and their safety. By thoroughly examining various sources of literature, this study delves into the importance of complement inhibitors like eculizumab and ravulizumab in treating complications associated with PNH. Furthermore, new treatment approaches such as specific complement inhibitors and innovative complement pathway modifiers are carefully assessed for their ability to enhance patient results and quality of life. Moreover, this thesis emphasizes the pathophysiology, epidemiology, clinical aspects, and diagnostic tools related to PNH. The intention of the insights obtained from this research is to educate healthcare providers and medical students on the various treatment options available for addressing the difficult issues presented by PNH, with the ultimate goal of improving the care and outlook for those affected.