Odevixibat in progressive familial intrahepatic cholestasis: a profile of its use
نویسندگان
چکیده
Odevixibat (Bylvay™; Bylvay®) has been approved for progressive familial intrahepatic cholestasis (PFIC) in patients aged ≥ 3 months the USA and those 6 EU UK. In pivotal double-blind placebo-controlled phase PEDFIC 1 study, odevixibat 40 or 120 μg/kg once daily significantly reduced serum bile acids pruritus. also improved growth, sleep quality of life parameters compared with placebo PFIC. The clinical benefits were maintained continued treatment up to 72 weeks, according an interim analysis ongoing open-label 2 study. is generally well tolerated, most adverse events being mild moderate severity. Longer-term safety data (up weeks treatment) revealed no new concerns. Progressive a rare inherited cholestatic liver disease that characterized by disturbed homeostasis, pruritus, accumulation biliary components potentially fatal disease. Recently, ileal acid transporter (IBAT) inhibitors have emerged as novel non-surgical approach Bylvay®), potent IBAT inhibitor, pruritus PFIC UK PFIC, decreased symptoms placebo. Furthermore, improvements growth parameters, measures seen versus Thus, valuable, effective well-tolerated option
منابع مشابه
Progressive familial intrahepatic cholestasis
Progressive familial intrahepatic cholestasis (PFIC) refers to a heterogeneous group of autosomal-recessive disorders of childhood that disrupt bile formation and present with cholestasis of hepatocellular origin. The exact prevalence remains unknown, but the estimated incidence varies between 1/50,000 and 1/100,000 births. Three types of PFIC have been identified and associated with mutations ...
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Introduction Progressive familial intrahepatic cholestasis (PFIC) represents a group of disorders which usually begin in the first months of life and progress to cirrhosis before the end of second decade. The disease occurs due to a defect in bile acid transport leading to cholestasis and resultant hepatocelluler injury1-3. Recent molecular and genetic studies have identified genes responsible ...
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Progressive familial intrahepatic cholestasis (PFIC or Byler disease) is a rare autosomal recessive form of severe and fatal cholestatic liver disease. A locus for PFIC has recently been mapped to chromosome 18q21-q22 in the original Byler pedigree. This region harbours the locus for a related phenotype, benign recurrent intrahepatic cholestasis (BRIC), suggesting that these traits are allelic....
متن کاملGenetic basis of progressive familial intrahepatic cholestasis.
oo~~ssrvn familial intrahepatic cholestasis (PFIC), P originally known as Byler disease, was first described in an Amish kindred (1,2). It is an inherited disorder of childhood in which cholestasis of hepatocellular origin often presents in the neonatal period or the first year of life and leads to death from liver failure at ages ranging from infancy to adolescence. Cholangiograms show normal ...
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ژورنال
عنوان ژورنال: Drugs & Therapy Perspectives
سال: 2022
ISSN: ['1179-1977', '1172-0360']
DOI: https://doi.org/10.1007/s40267-022-00926-6