Treatment of paroxysmal nocturnal hemoglobinuria

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dc.contributor.advisorPórszász, Róbert
dc.contributor.advisordeptÁltalános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet
dc.contributor.authorCohen, Shay Shalom
dc.contributor.departmentDE--Általános Orvostudományi Kar
dc.contributor.opponentHalasi, Barbara Dóra
dc.contributor.opponentSzentmiklósi, A. József
dc.contributor.opponentdeptÁltalános Orvostudományi Kar::Igazságügyi Orvostani Intézet
dc.contributor.opponentdeptÁltalános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet
dc.date.accessioned2024-07-01T11:06:52Z
dc.date.available2024-07-01T11:06:52Z
dc.date.created2024-04-19
dc.description.abstractParoxysmal 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.
dc.description.courseáltalános orvos
dc.description.courselangangol
dc.description.degreeegységes, osztatlan
dc.format.extent42
dc.identifier.urihttps://hdl.handle.net/2437/375141
dc.language.isoen
dc.rights.accessHozzáférhető a 2022 decemberi felsőoktatási törvénymódosítás értelmében.
dc.subjectPNH
dc.subjectParoxysmal nocturnal Hemoglobinuria
dc.subject.dspaceMedicine
dc.titleTreatment of paroxysmal nocturnal hemoglobinuria
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