[Translation] A registry study documenting the efficacy and safety (including prospective long-term outcomes) of treatment in participants with progressive familial intrahepatic cholestasis (PFIC) taking ODEVIXIBAT (BYLVAY).
主要目的:根据不良事件 (AE) 评价odevixibat的长期安全性。
次要目的:评价odevixibat在以下方面的有效性:
1.PFIC参与者整体人群以及不同PFIC类型参与者亚群的首次临床事件(手术胆道分流、肝移植、死亡)。
2.PFIC参与者整体人群以及不同PFIC类型参与者亚群的无手术胆道分流生存期。
3.PFIC参与者整体人群以及不同PFIC类型参与者人群的无肝移植生存期。
4.PFIC参与者整体人群以及不同PFIC类型参与者亚群的总生存期。
5.PFIC参与者整体人群以及不同PFIC类型参与者亚群的瘙痒改善。
6.PFIC参与者整体人群以及不同PFIC类型参与者亚群的血清胆汁酸较基线的变化。
[Translation] Primary objective: To evaluate the long-term safety of odevixibat based on adverse events (AEs).
Secondary objective: To evaluate the efficacy of odevixibat in the following areas:
1. First clinical event (surgical biliary shunt, liver transplant, death) in the overall PFIC participant population and different PFIC subgroups.
2. Surgical biliary shunt-free survival in the overall PFIC participant population and different PFIC subgroups.
3. Liver transplant-free survival in the overall PFIC participant population and different PFIC subgroups.
4. Overall survival in the overall PFIC participant population and different PFIC subgroups.
5. Improvement in pruritus in the overall PFIC participant population and different PFIC subgroups.
6. Change in serum bile acids from baseline in the overall PFIC participant population and different PFIC subgroups.