Complement Inhibitors in Rare Diseases
10.12376/j.issn.2097-0501.2022.04.006
- VernacularTitle:补体抑制剂在罕见病的临床应用
- Author:
Cai YUE
1
,
2
;
Yali DU
2
,
3
;
Yangyu HUANG
2
,
4
;
Limeng CHEN
1
,
2
;
Yuzhou GUAN
2
,
4
;
Bing HAN
2
,
3
Author Information
1. Department of Nephrology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
2. Peking Union Medical College, Beijing 100730, China.
3. Department of Hematology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
4. Department of Neurology, Peking Union Medical College Hospital, Chinese Academy of Medical Sciences &
- Publication Type:Journal Article
- Keywords:
complement inhibitors;
paroxysmal nocturnal hemoglobinuria;
atypical hemolytic uremic syndrome;
myasthenia gravis
- From:
JOURNAL OF RARE DISEASES
2022;1(4):391-399
- CountryChina
- Language:Chinese
-
Abstract:
The complement system is a self-protection mechanism of the human body. The abnormal activation of the complement system is involved in the occurrence and development of various diseases. The application of complement inhibitors in many rare diseases was a milestone in leading to the progress of such disease as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and others. Recently, the application of complement inhibitors has gradually expanded to other complement-related diseases. This review summarizes the literature on the current application of complement inhibitors in rare diseases and looks into the prospects of the application in the rare diseases.