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  • 1 Semmelweis Egyetem, Általános Orvostudományi Kar I. Belgyógyászati Klinika Budapest Korányi Sándor u. 2/A 1083

Az AL-amyloidosis egy olyan szisztémás betegség, ahol kóros sejtek (malignus plazmasejtek) patogén fehérjét termelnek, amelyek lerakódnak a szervezetben. Az amyloidogen fehérjét monoklonális immunglobulin-molekula könnyű lánca alkotja, amely vízben oldhatatlanná válik, az extracelluláris térben kicsapódva a szövetekben lerakódik, szervkárosodás kialakulását eredményezve. A malignus plazmasejtbetegségek közé tartozik, illetve más, monoklonális B-sejtes betegséghez társulhat. A diagnózis – mint minden amyloidosistípusnál – a szövettani eredményre támaszkodik, de sokszor az amyloidogen fehérje azonosításához speciális vizsgálatokra is szükség van. A terápia alapvetően a malignus plazmasejtklón elpusztítására irányul. Az utóbbi évtizedekben a terápiás eszköztár igen jelentősen bővült, több, új hatásmechanizmusú gyógyszer is rendelkezésre áll (thalidomid, lenalidomid, bortezomib). Az arra alkalmas betegeknél ma a standard kezelést a nagy dózisú kemoterápiát követő autológ haemopoeticus őssejtátültetés jelenti, ezt azonban egy nagyon gondos rizikófelmérésnek kell megelőznie. A transzplantációra nem alkalmas betegeket kis dózisú alkilálószerrel, dexametazonnal, a kettő kombinációjával vagy valamelyik új gyógyszerrel lehet kezelni. Igen nagy jelentősége van a korai diagnózisnak és a terápia gyors megkezdésének, mert a parenchymás szervek kialakuló funkciózavara (elsősorban a szívelégtelenség) útját állja a hatékony kezelésnek. Mivel ritka betegségről van szó, a speciális centrumok tevékenységének jelentős szerepe van a klinikai vizsgálatok végzése terén. Orv. Hetil., 2012, 153, 563–573.

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