paroxysmal nocturnal hemoglobinuria definition

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA DEFINITION DRIVER

Um klinisch relevant zu werden muss der PNH-Klon einen relevanten Beitrag zur Blutbildung leisten. In den letzten Jahren haben wir viel über den Zusammenhang zwischen komplementvermittelter Hämolyse und ihrer klinischen Konsequenzen wie Thrombose, Niereninsuffizienz oder Pulmonale Hypertension und möglicher Behandlungsstrategien gelernt. Die klassische hämolytische PNH zeigt Granulozytenklongrößen von 90% und mehr. Solche Zellen sind anfällig gegenüber komplementvermittelter Hämolyse. Ursache für die PNH ist eine klonale Dominanz von hämatopoietischen Progenitorzellen aus welchen Blutzellen entstehen, bei denen komplementinhibitorischen Proteine wie CD55 oder CD59 nicht exprimiert werden können.

This review will focus on the most important findings in pathophysiology, clinical course and treatments during the last years.ĭie paroxysmale nächtliche Hämoglobinurie (PNH) ist eine seltene gutartige klonale Erkrankung der Blutbildung. The success of eculizumab in the treatment of PNH patients has stimulated the development of a variety of new strategies for complement inhibition. Recently, data about coexisting mutations in cancer genes have been published and new mechanisms for autoimmunity have been presented.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA DEFINITION DRIVER

The driver for the clonal dominance of the PIGA-mutated hematopoiesis is still not clear. Such symptoms can be seen in a relevant proportion of patients with clone size smaller than 10%. To gain clinical relevance, the PNH clone has to make a relevant contribution to blood cell generation however, a minimal clone size as a threshold for the occurrence of symptoms such as thrombosis, dyspnea, chest or abdominal pain cannot be given. Much progress has been made during the last years in understanding the interrelations of complement-dependent hemolysis its consequences like thrombosis, renal failure or pulmonal hypertension and possible treatment strategies. In classic hemolytic PNH, granulocyte clone sizes can be up to 90% or more. These cells are prone to complement-induced hemolysis. Its etiologic basis is the clonal dominance of a PIGA-mutated hematopoietic progenitor that gives rise to GPI-deficient blood cells that are unable to express complement inhibitors like CD55 or CD59. Paroxysmal nocturnal hemoglobinuria (PNH) is a rare benign clonal disorder of hematopoiesis.

Paroxysmal nocturnal hemoglobinuria definition