This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching.
The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.
Diagnosed with ALGS.
On (or starting) active odevixibat treatment.
Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.
Currently participating in a clinical trial with odevixibat.
Currently participating in any interventional clinical trial for ALGS.
Have any contraindication to odevixibat as per the locally approved label.
Had liver transplant before enrolment
Patients diagnosed with ALGS who start treatment with odevixibat (Bylvay) will be enrolled into the registry. Patients who started odevixibat treatment before the implementation of the registry study may also be enrolled.
Other
Prospective
Not listed
Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
Surgical biliary diversion-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Liver transplant-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Overall survival
Defined as time from the start of odevixibat treatment to death.
Change from Baseline in Weight
This endpoint will measure the change in participants' weight from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.
Change from Baseline in Height
This endpoint will measure the change in participants' height from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.
Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
