For US Health Care Professionals

Long-Term Outlook for Alagille Syndrome

Current
Therapies

Symptom management of Alagille syndrome (ALGS) only scratches the surface and does not target the source of patients’ suffering. Surgical biliary diversion and liver transplant are viable treatment options, but they present several issues, such as the need for permanent stoma, lifelong immunosuppressive therapy, or surgical complications. These are practical limitations to treatment.1

Current Therapies
Treatment Landscape

Treatment Landscape

For patients with Alagille syndrome (ALGS), there is a need for noninvasive treatment options that target the root cause of cholestasis early in the course of disease.1,2

The goals of these treatments should be to1,2:

Reducethe accumulation of bile acids in the liver and bloodstream

Relievecholestatic pruritus

Reducexanthomas

Limitthe progression of liver disease

Improvelong-term prognosis

Promising
Research

There is promising research underway for new treatment options. These may play a crucial role in reducing the symptoms of Alagille syndrome (ALGS).1

Significant scientific discovery for better treatments of ALGS could mean other children won’t have to suffer like my daughter. –Jazmyn R.
Promising Research

ALGS Resources

  • Children Can't Bear It: A Comprehensive Understanding of ALGS
  • Children’s Liver Disease Foundation’s Yellow Alert
  • The Alagille Syndrome Alliance
  • National Organization for Rare Disorders
  • Johns Hopkins Medicine
ALGS=Alagille syndrome; ASBT=apical sodium-dependent bile acid transporter; IBAT=ileal bile acid transporter.
References: 1. Kamath BM, Stein P, Houwen RHJ, Verkade HJ. Potential of ileal bile acid transporter inhibition as a therapeutic target in Alagille syndrome and progressive familial intrahepatic cholestasis. Liver Int. 2020;40(8):1812-1822. doi:10.1111/liv.14553 2. Spinner NB, Gilbert MA, Loomes KM, Krantz ID. Alagille syndrome. In: Adam MP, Ardinger HH, Pagon RA, et al, eds. GeneReviews. Seattle (WA): University of Washington, Seattle. Published online: May 19, 2000. Updated: December 12, 2019. https://www.ncbi.nlm.nih.gov/books/NBK1273/pdf/Bookshelf_NBK1273.pdf