Odevixibat Treatment for Progressive Familial Intrahepatic Cholestasis
Author Information
Author(s): Marx Milena, Hartleif Steffen, Hilberath Johannes, Berg Christoph P., Tsiflikas Ilias, Singer Stephan, Sturm Ekkehard, Caballero-Mateos Antonio M.
Primary Institution: University Children’s Hospital Tübingen
Hypothesis
The study aims to characterize the real-world effectiveness and safety of odevixibat in patients with progressive familial intrahepatic cholestasis (PFIC).
Conclusion
Odevixibat showed clinical benefits in most patients with PFIC1 and PFIC2, although those with complete loss of BSEP protein did not respond well.
Supporting Evidence
- 5 out of 9 patients showed improvement in serum bile acid levels and pruritus.
- Patients with periodic relapses required at least 3 months of treatment for symptom control.
- Two patients with complete loss of BSEP protein did not respond to treatment.
Takeaway
This study looked at how a medicine called odevixibat helps kids with a liver problem called PFIC. Most kids felt better after taking it, but some didn't.
Methodology
This was a retrospective case series study involving 9 patients treated with odevixibat at a single center in Tübingen, Germany.
Limitations
Standardized assessment tools for pruritus and sleep disturbance were not used consistently, limiting direct comparisons with clinical trial results.
Participant Demographics
Of the 9 patients, 7 were male, 2 were diagnosed with PFIC1, and 7 with PFIC2, with a median age at diagnosis of 8 months.
Digital Object Identifier (DOI)
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