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) µmol/L; p = 0.007]; reduction in sBA was >70% from baseline (or <70 µmol/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.
Di Giorgio, A., Sciveres, M., Fuoti, M., Sonzogni, A., Mandato, C., D'Antiga, L. (2023). Treatment with an ileal bile acid transporter inhibitor in patients with TJP2 deficiency. CLINICS AND RESEARCH IN HEPATOLOGY AND GASTROENTEROLOGY, 47(8) [10.1016/j.clinre.2023.102185].
Treatment with an ileal bile acid transporter inhibitor in patients with TJP2 deficiency
D'Antiga L.
2023
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) µmol/L; p = 0.007]; reduction in sBA was >70% from baseline (or <70 µmol/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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