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.
Download full-text PDF |
Source |
|---|---|
| http://dx.doi.org/10.1016/j.clinre.2023.102185 | DOI Listing |
Publication Analysis
Top Keywords
ileal bile
8
bile acid
8
acid transporter
8
tjp2 deficiency
8
treatment ileal
4
transporter inhibitor
4
inhibitor patients
4
patients tjp2
4
deficiency published
4
published data
4
Similar Publications
Want AI Summaries of new PubMed Abstracts delivered to your In-box?
Enter search terms and have AI summaries delivered each week - change queries or unsubscribe any time!