Progressive Familial Intrahepatic Cholestasis Clinical Trial
Official title:
Gastrostomy-Biliary Diversion: Innovative Management for Children With Bile Canalicular Transport Disorders
Progressive familial intrahepatic cholestasis (PFIC) is a group of disorders that can present early in life with cholestasis and intractable pruritus. Their treatment poses a great challenge, with medical treatment is not successful in many cases. Moreover, the available non-transplant surgeries carry many side effects and different degrees of efficacy. Partial external biliary diversion, internal biliary diversion, and ileal exclusion still lack widespread experience with many side effects. Nasobiliary stent placement has little tolerability, especially in younger age. Gastrobiliary tube is a novel modality for external biliary diversion in such patients.
Progressive familial intrahepatic cholestasis (PFIC) is a group of disorders that can present
early in life with cholestasis and intractable pruritus. They result primarily from defective
transporters of different bile constituents on the canalicular membrane. The mildest form of
these defective transporters can present by what is called benign recurrent intrahepatic
cholestasis (BRIC).
More recently both disease categories that represent the severest (PFIC) and mildest (BRIC)
forms of these transporters are better nomenclated as bile canalicular transport disorders,
as some cases can start early as BRIC and later in life progress the PFIC phenotype.
Moreover, a continuous spectrum of severity is present between what were previously known as
PFIC and BRIC.
If the severest forms are not treated effectively they can have devastating outcomes reaching
death. On the other hand, those known as BRIC can have severe attacks with unremitting
pruritus that could be prolonged and sometimes persistent.
Their treatment poses a great challenge, with medical treatment is not successful in many
cases. Moreover, the available non-transplant surgeries carry many side effects and different
degrees of efficacy. Partial external biliary diversion is not always successful and has
disfiguring effects and the stoma can have many side effects. Internal biliary diversion and
ileal exclusion still lack widespread experience and encouraging results due to sometimes
lack of efficacy and others due to side effects. Nasobiliary stent placement was tried in
previous cases with a promising outcome, but with little tolerability, especially in younger
age.
In spite liver transplant is the last resort for failing cases, the availability of
successful non transplant therapy would be the preferable one if it is with adequate efficacy
and little side effects. The innovative intervention proposed in this study is expected to
have a good efficacy due to total biliary diversion rather than the partial ones obtained by
other surgeries. Moreover, it avoids the disfiguring impact of the jeujenal stoma. More
importantly, it can be removed at any time and be used at the times of severe flare of the
disease.
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