Open AccessNovel treatment options for acute hepatic porphyrias
Author(s) -
Bruce Wang
Publication year - 2021
Publication title -
current opinion in gastroenterology
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.165
H-Index - 83
eISSN - 1531-7056
pISSN - 0267-1379
DOI - 10.1097/mog.0000000000000734
Subject(s) - medicine , drug , acute pain , porphyria , hemin , intensive care medicine , heme , pharmacology , anesthesia , biochemistry , chemistry , enzyme
Acute hepatic porphyrias (AHP) are a group of rare diseases that are characterized by episodic acute neurovisceral pain episodes caused by abnormal accumulation of the neurotoxic porphyrin precursor delta-aminolevulinic acid (ALA). Patient with frequent recurrent acute attacks have been difficult to treat and these patients sometimes require liver transplantation. Recent developments in small interfering RNA (siRNA)-based therapy led to the development of an effective prophylactic treatment for patients with frequent recurrent attacks. This review will describe treatment options for AHP and highlight management in light of new treatment option.