Off-label pharmacologic agents are often used to manage symptoms of Alagille syndrome with limited success. As a result, surgical biliary diversion and liver transplant represented the only effective treatment option for most liver symptoms of Alagille syndrome, but they present several issues. In the case of a surgical biliary diversion, individuals typically require a stoma, which many view as stigmatizing. In the case of liver transplants, issues include lifelong immunosuppressive therapy or surgical complications. These are practical limitations to treatment.1
Recently, a class of medicines called IBAT inhibitors have been approved by the FDA to treat the itch in certain liver disorders.1-3
For patients with Alagille syndrome, there is a need for more noninvasive treatment options.1,4
The goals of these treatments should be to1,4:
Reducethe accumulation of bile acids in the liver and bloodstream
Limitthe progression of liver disease
There is promising research underway for additional treatment options. These may play a crucial role in reducing the symptoms of Alagille syndrome.1
Significant scientific discovery for better treatments of Alagille syndrome could mean other children won’t have to suffer like my daughter. –Jazmyn R.