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Remarkable Response to Odevixibat in an Adult With Progressive Familial Intrahepatic Cholestasis Type 1 and
Fernando Gil-Lopez1, Lydia A Mercado2, Nicole M Loo3
1Department of Internal Medicine, Mayo Clinic, Jacksonville, FL.
Odevixibat significantly improved a patient with progressive familial intrahepatic cholestasis type 1 (PFIC1) refractory pruritus. The treatment led to biochemical improvements and sustained resolution of itching, enhancing the patient's quality of life.
Area of Science:
- Hepatology
- Gastroenterology
- Pharmacology
Background:
- Progressive familial intrahepatic cholestasis type 1 (PFIC1) is a rare genetic disorder causing severe liver disease and debilitating pruritus.
- Patients with PFIC1 often experience a poor quality of life due to intractable itching and may have limited treatment options.
- Liver transplantation is a potential option but is often deferred in patients with preserved liver function.
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