There are no published data on the use of odevixibat, a selective ileal bile acid transporter (IBAT) inhibitor, in children with tight junction protein 2 (TJP2) deficiency (also named as PFIC-4). We describe a case series of five children treated with odevixibat. After treatment, serum bile acids (sBA) decreased compared to baseline [mean value: 244 (+/- 125), vs 38 (+/- 34) mu mol/L; p -- 0.007]; reduction in sBA was >70% from baseline (or <70 mu mol/L) in all. Improvements in pruritus were reported in all patients. The drug was well tolerated. IBAT inhibitors should be considered a valuable treatment option in patients with TJP2 deficiency.

Treatment with an ileal bile acid transporter inhibitor in patients with TJP2 deficiency

Mandato, Claudia;
2023-01-01

Abstract

There are no published data on the use of odevixibat, a selective ileal bile acid transporter (IBAT) inhibitor, in children with tight junction protein 2 (TJP2) deficiency (also named as PFIC-4). We describe a case series of five children treated with odevixibat. After treatment, serum bile acids (sBA) decreased compared to baseline [mean value: 244 (+/- 125), vs 38 (+/- 34) mu mol/L; p -- 0.007]; reduction in sBA was >70% from baseline (or <70 mu mol/L) in all. Improvements in pruritus were reported in all patients. The drug was well tolerated. IBAT inhibitors should be considered a valuable treatment option in patients with TJP2 deficiency.
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Utilizza questo identificativo per citare o creare un link a questo documento: https://hdl.handle.net/11386/4854613
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