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Early predictors of pruritus response in children with PFIC treated with odevixibat
Angelo Di Giorgio1, Marco Sciveres2, Maurizio Fuoti3
1Paediatric Hepatology, Gastroenterology and Transplantation, Hospital Papa Giovanni XXIII, Bergamo, University of Udine, Italy.
Background:
In our previous study we reported the efficacy of odevixibat treatment (OT) in a real-world cohort of children with progressive familial intrahepatic cholestasis (PFIC). Here we aim to identify the best cut-off of serum bile acid (sBA) early reduction predicting the response of pruritus at 6 months of treatment.
Methods:
We reviewed data on children with PFIC treated with odevixibat. ROC analysis was performed to calculate the best cut-off of sBA reduction at 1 and 3 months associated with reduction in pruritus scale ≥1 point at 6 months.
Results:
24 patients [median age 6.6 years (3.7-12.1), M/F = 11/13] were enrolled; 16 (67%) had classic PFIC types (PFIC-1 = 2; PFIC-2 = 11; PFIC-3 = 3), 8 (33%) had rarer forms (PFIC-4 = 5, PFIC-5 = 1; PFIC-6 = 1; PFIC-9 = 1). At 6 months, 73% were pruritus responders. Reductions of sBA levels ≥48% from baseline at 1 month and ≥52% at 3 months were associated with improvements of pruritus at 6 (1st month: ROC-AUC 0.771; SE=0.75, SP=0.83; 3rd month: ROC-AUC 0.885; SE=0.81, SP=1.00).
Conclusion:
reduction of sBA levels of around 50% from baseline value at 1 and 3 months is associated with a pruritus response to OT at 6 months. This information could be useful to the management of children with PFIC.