Odevixibat and partial external biliary diversion showed equal improvement of cholestasis in a patient with progressive familial intrahepatic cholestasis
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.
机构:
Northwestern Univ, Div Gastroenterol Hepatol & Nutr, Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USANorthwestern Univ, Div Transplant Surg, Surg Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA
Bass, Lee M.
Superina, Riccardo A.
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Northwestern Univ, Div Transplant Surg, Surg Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USANorthwestern Univ, Div Transplant Surg, Surg Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA
机构:
Univ Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Squires, James E.
Celik, Neslihan
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Univ Pittsburgh, Childrens Hosp Pittsburgh, Thomas E Starzl Transplantat Inst,Med Ctr, Dept Transplant Surg,Hillman Ctr Pediat Transplan, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Celik, Neslihan
Morris, Amy
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Univ Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Morris, Amy
Soltys, Kyle
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Univ Pittsburgh, Childrens Hosp Pittsburgh, Thomas E Starzl Transplantat Inst,Med Ctr, Dept Transplant Surg,Hillman Ctr Pediat Transplan, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Soltys, Kyle
Mazariegos, George
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Univ Pittsburgh, Childrens Hosp Pittsburgh, Thomas E Starzl Transplantat Inst,Med Ctr, Dept Transplant Surg,Hillman Ctr Pediat Transplan, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Mazariegos, George
Shneider, Benjamin
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Baylor Coll Med, Dept Pediat, Div Gastroenterol Hepatol & Nutr, Houston, TX 77030 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
Shneider, Benjamin
Squires, Robert H.
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Univ Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USAUniv Pittsburgh, Childrens Hosp Pittsburgh, Med Ctr, Div Gastroenterol Hepatol & Nutr, Pittsburgh, PA 15213 USA
机构:
Istanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, TurkeyIstanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, Turkey
Erginel, Basak
Soysal, Feryal Gun
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Istanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, TurkeyIstanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, Turkey
Soysal, Feryal Gun
Durmaz, Ozlem
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Istanbul Univ, Istanbul Med Fac, Dept Pediat Gastroenterol, Istanbul, TurkeyIstanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, Turkey
Durmaz, Ozlem
Celik, Alaattin
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Istanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, TurkeyIstanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, Turkey
Celik, Alaattin
Salman, Tansu
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Istanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, TurkeyIstanbul Univ, Istanbul Med Fac, Dept Pediat Surg, TR-34093 Istanbul, Turkey