Odevixibat and partial external biliary diversion showed equal improvement of cholestasis in a patient with progressive familial intrahepatic cholestasis

BMJ Case Rep. 2020 Jun 29;13(6):e234185. doi: 10.1136/bcr-2019-234185.


Untreated progressive familial intrahepatic cholestasis (PFIC) type 2, or bile salt exporter protein deficiency, frequently leads to severe pruritus, impaired growth and progressive liver fibrosis with risk of organ failure. We describe a 15-month-old male patient with severe pruritus diagnosed with PFIC type 2 enrolled in an open-label phase 2 study who received 4 weeks of treatment with odevixibat, an ileal bile acid transporter inhibitor under development for cholestatic liver disease treatment. The patient experienced reductions in serum bile acids and improvement in itching and sleep scores, and odevixibat was well tolerated. After the odevixibat study, symptoms returned and the patient underwent partial external biliary diversion (PEBD). Odevixibat treatment and PEBD produced similar normalisation of serum bile acid levels and improvements in pruritus and sleep disruptions. Thus, odevixibat appeared to be as effective as invasive PEBD in treating serum bile acids and cholestatic pruritus in this patient.

Keywords: congenital disorders; gastrointestinal system; liver disease; paediatric surgery; paediatrics.

Publication types

  • Case Reports

MeSH terms

  • Benzodiazepines / therapeutic use*
  • Bile Acids and Salts / blood
  • Biliary Tract Surgical Procedures / methods*
  • Butyrates / therapeutic use*
  • Carrier Proteins / antagonists & inhibitors*
  • Cholestasis, Intrahepatic / blood
  • Cholestasis, Intrahepatic / complications
  • Cholestasis, Intrahepatic / therapy*
  • Combined Modality Therapy
  • Humans
  • Infant
  • Male
  • Membrane Glycoproteins / antagonists & inhibitors*
  • Pruritus / blood
  • Pruritus / etiology
  • Pruritus / therapy*
  • Treatment Outcome


  • Bile Acids and Salts
  • Butyrates
  • Carrier Proteins
  • Membrane Glycoproteins
  • bile acid binding proteins
  • Benzodiazepines
  • odevixibat

Supplementary concepts

  • Cholestasis, progressive familial intrahepatic 2