Paroksismal nokturnal hemoglobinüri

Paroksismal nokturnal hemoglobinüri (PNH) hemolitik anemi ile seyreden edinsel klonal hematopoetik bir kök hücre hastalÕ÷ÕdÕr. Özellikle CD55 ve CD59 gibi yüzey proteinleri, kompleman etkisine karúÕ hücreleri korur ve bu proteinler PNHlÕ mutasyona u÷ramÕú hematopoetik kök hücrelerden kaynaklanan olgun hücrelerde eksiktir. PNH, kronik intravasküler Coombs negative hemolitik anemi, tromboz ve kemik ili÷i yetmezli÷i bulgularÕ ile seyreden bir hastalÕktÕr. AkÕm sitometri, CD55 ve CD59 eksikli÷inin saptanmasÕnda en yaygÕn kullanÕlan tanÕ yöntemidir. PNH tanÕsÕnda FLAER (fluorescent aerolysin) yeni bir yöntemdir. PNHda küratif olan tek tedavi; allojenik kök hücre naklidir (AKHN), fakat mortalite ve morbidite insidansÕ yüksek bir tedavi yöntemidir. AntiC5 monok- lonal antikoru olan eculizumab ile hastalÕ÷Õn en önemli komplikasyonu olan tromboz geliúimi baúta olmak üzere morbidite ve mortalite oranlarÕ azalmÕútÕr. Eculizumab ve yeni geliútirilecek PNH tedavi ajanlarÕ ile PNH da mortalite ve morbiditesi yüksek bir tedavi olan allojenik kök hücre nakline olan gereksinim azalacaktÕr.

Paroxysmal nocturnal hemoglobşnuria

ABSTRACT Paroxysmal nocturnal hemoglobinuria is an acquired clonal hematopoietic stem cell disease undergoing with hemolytic anemia. Surface antigenic proteins, especially like CD55 and CD59, protects cells against the complement effect and these proteins does not exist in PNH mutant hematopoietic stem cell originated mature cells. PNH is a disease presenting with chronic intravascular Coombs negative hemolytic anemia, thrombosis and bone marrow failure. Flow cytometry is the most common diagnosis test in determining CD55 and CD59 deficiency. FLAER (fluorescent aerolysin) is a new method in PNH diagnosis. The only curative treatment is allogenic stem cell transplantation (ASCT) but it has a high incidence of mortality and morbidity. With eculizumab, an anti C5 monoclonal antibody, mortality and morbidity rates and the most important complication; thrombosis, is reduced. Allogenic stem cell transplantation requirement will be reduced with eculizumab and the new medications that are going to be developed for PNH treatment.

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