Treatment with an ileal bile acid transporter inhibitor in patients with TJP2 deficiency - 06/10/23
, Marco Sciveres b, Maurizio Fuoti c, Aurelio Sonzogni d, Claudia Mandato e, Lorenzo D'Antiga aHighlights |
• | Children with PFIC-4 may develop cholestasis characterized by high serum bile acids and pruritus. |
• | Odevixibat is effective to reduce serum bile acids levels and treat pruritus in children with PFIC-4. |
• | Odevixibat is safe and well tolerated. |
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.
Le texte complet de cet article est disponible en PDF.Keywords : Progressive familial intrahepatic cholestasis, Pruritus, PFIC-4, TJP2, Odevixibat, IBAT inhibitor
Abbreviations : PFIC, TJP2, MAGUK, GGT, LT, IBAT, ASBT, BA, sBA, AS, EAP, SIGENP, AST, ALT, INR, PGIS, PGIC, PCD, SI, UDCA, PH, TEAEs
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Vol 47 - N° 8
Article 102185- octobre 2023 Retour au numéro
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