This registry-based study will collect information from people with Progressive Familial Intrahepatic Cholestasis (PFIC) who take odevixibat (Bylvay) as part of routine clinical care in China. PFIC is a rare genetic liver disease that affects bile secretion and can cause bile acids to build up in the liver, which may lead to symptoms such as severe itching (pruritus). Odevixibat was first allowed to be used for PFIC in babies older than 6 months by the European Medicines Agency (EMA) on 16 July 2021 and by the United States Food and Drug Administration (FDA) on 20 July 2021 for itching in babies older than 3 months. Odevixibat is approved for the treatment of pruritus in PFIC and was approved in China on 01 December 2024 for patients 6 months of age and older with PFIC. The main aim of this registry is to assess long-term real-world safety (based on adverse events) and to describe effectiveness outcomes.
Study Type
OBSERVATIONAL
Enrollment
20
Children's Hospital of Fudan University Endocrinology and Metabolism
Shanghai, China
RECRUITINGPercentage of participants experiencing adverse events (AEs)
An AE is any untoward medical occurrence in a participant administered a medicinal product and does not necessarily have a causal relationship with treatment
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
Percentage of participants experiencing serious adverse events (SAEs)
SAEs are collected as part of safety reporting and include events meeting seriousness criteria (e.g., death, life-threatening, hospitalization, etc.) as defined in the protocol
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection), or 30 days after the last dose of odevixibat (in case of treatment discontinuation), whichever comes first.
Event-free survival (EFS)
EFS is defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion, liver transplant, or death.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection
Surgical biliary diversion-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Liver transplant-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Overall survival
Defined as time from the start of odevixibat treatment to death.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Pruritus improvement
Pruritus improvement described at each patient visit using a (semi-)objective scoring scale to assess level of pruritus from the start of the odevixibat treatment.
Time frame: From first ICF signature and up to end of data collection (approximately 5 years of data collection)
Change from baseline in serum bile acid
Change from baseline assessed by measuring serum bile acid levels at each patient visit.
Time frame: From baseline and up to end of data collection (approximately 5 years of data collection
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