Advances in the treatment of acute hepatic porphyria
- VernacularTitle:急性肝卟啉病的治疗进展
- Author:
Jiajia LEI
1
;
Shuang LI
1
;
Baixue DONG
1
;
Jing YANG
2
;
Yi REN
2
Author Information
- Publication Type:Review
- Keywords: Porphyrias， Hepatic; Heme; Therapeutics
- From: Journal of Clinical Hepatology 2024;40(4):828-833
- CountryChina
- Language:Chinese
- Abstract: Acute hepatic porphyria （AHP） is a rare disease with abnormal heme metabolism， and breakthroughs have been made in the treatment of this disease in recent years. In addition to conventional treatment methods， this article reviews new therapies for AHP that are in the stage of initial clinical application or are still in the research stage， including RNAi therapy， enzyme replacement therapy， genetic supplementation of DNA or mRNA， drug molecular chaperones， and glycine transporter inhibitors for reducing heme synthesis. Moreover， this article also reviews the treatment of AHP-related comorbidities and complications， such as hyponatremia and posterior reversible encephalopathy syndrome. High glucose infusion is the main treatment method for AHP in China， and the improvement in diagnosis and increased attention to rare diseases in China has promoted the development of the diagnosis and treatment of AHP， and it is expected to explore more suitable treatment methods for AHP in the Chinese population in the future.
