The management of children with chronic
liver disease (CLD) mandates a multidisciplinary approach.
CLDs can be classified into 'potentially' curable, treatable non-curable, and end-stage diseases. Goals pertaining to the management of
CLDs can be divided into prevention or minimization of progressive liver damage in curable CLD by treating the primary cause; prevention or control of complications in treatable CLD; and prediction of the outcome in end-stage CLD in order to deliver definitive
therapy by
surgical procedures, including
liver transplantation. Curative, specific
therapies aimed at the primary causes of
CLDs are, if possible, best considered by a pediatric hepatologist. Medical management of
CLDs in children will be reviewed in two parts, with part I (this article) specifically focusing on 'potentially' curable
CLDs.
Dietary modification is the cornerstone of management for
galactosemia,
hereditary fructose intolerance, and certain
glycogen storage diseases, as well as non-
alcoholic steatohepatitis. It is also essential in
tyrosinemia, in addition to
nitisinone [2-(nitro-4-trifluoromethylbenzoyl)-1,3-cyclohexanedione]
therapy, as well as in
Wilson disease along with
copper-
chelating agents such as
D-penicillamine,
triethylenetetramine dihydrochloride, and
ammonium tetrathiomolybdate.
Zinc and
antioxidants are adjuvant drugs in
Wilson disease. New advances in chronic viral
hepatitis have been made with the advent of oral
antivirals. In children, currently available drugs for the treatment of
chronic hepatitis B virus infection are standard
interferon (IFN)-α-2, pegylated IFN-α-2 (PG-IFN), and
lamivudine. In adults,
adefovir and
entecavir have also been licensed, whereas
telbivudine,
emtricitabine,
tenofovir disoproxil fumarate,
clevudine, and
thymosin α-1 are currently undergoing clinical testing. For
chronic hepatitis C virus infection, the most accepted treatment is PG-IFN plus
ribavirin.
Corticosteroids, with or without
azathioprine, remain the basic strategy for inducing remission in
autoimmune hepatitis.
Ciclosporin (
cyclosporine) and other immune suppressants may be used for patients who do not achieve remission, or who have significant side effects, with
corticosteroid/
azathioprine therapy. The above
therapies can prevent, or at least minimize, progression of liver damage, particularly if started early, leading to an almost normal quality of life in affected children.