Пароксизмална нощна хемоглобинурия – диагноза, клинична картина и нови терапевтични възможности

  • Елина Белева
Keywords: хемоглобинурия, пароксизмална, комплемент инактивиращи агенти

Abstract

Пароксизмалната нощна хемоглобинурия (ПНХ) е хронично мултисистемно, прогресивно и животозастрашаващо заболяване, което се характеризира с вътресъдова хемолиза, тромботични инциденти, тежки инфекции и костно-мозъчна недостатъчност. ПНХ е рядко заболяване с очаквана болестност по данни за Orphanet 2/100 000.

Парадигмата в лечението на ПНХ се променя с въвеждането на комплементна блокада посредством анти-С5 антитела и инхибитори на проксимални компоненти на комплемента. Блокадата на комплемента постига значима редукция на тромботичните усложнения, стабилно ниво на хемоглобин, контрол на хемолизата и подобряване качеството на живот при пациентите с ПНХ. Лечението с комплементни инхибитори подобрява свързаните с ПНХ органни усложнения като бъбречна недостатъчност и пулмонална хипертония.

В настоящия обзор са обобщени и представени актуални насоки за лечение на ПНХ, публикувани от международни и европейски национални дружества по хематология, както и са сравнени индикации за започване и преустановяване на терапия с анти-С5 инхибитори според ПНХ работни групи в редица европейски държави.

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Published
2024-02-09
How to Cite
1.
Белева Е. Пароксизмална нощна хемоглобинурия – диагноза, клинична картина и нови терапевтични възможности. Редки болести и лекарства сираци [Internet]. 2024Feb.9 [cited 2024Feb.29];14(3-4):10-9. Available from: https://journal.raredis.org/index.php/RBLS/article/view/189
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