Primary sclerosing cholangitis (PSC), with no curative intervention, can progress to
end-stage liver disease.
Mizoribine, a
purine antimetabolite, has never been used for the management of PSC. To evaluate the role of
mizoribine with
azathioprine we undertook a preliminary clinical study in children with PSC. Children with PSC and autoimmune features were simultaneously treated with
mizoribine and
azathioprine.
Ursodeoxycholic acid or
mesalazine were not regulated. The primary end-points of our study included improvement of
aspartate aminotransferase,
alanine aminotransferase, and γ-glutamyltransferase. Liver histology, immunostaining studies of the liver, and magnetic resonance cholangiopancreatography (MRCP) were also assessed. We have treated four PSC children: two treatment-naïve patients (cases 1 and 2), and two with established
liver cirrhosis (cases 3 and 4). Both case 1 and 2 showed a normalization of liver
enzymes and case 2 showed an improvement in MRCP findings. Cases 3 and 4 also showed an improvement in
varices, MRCP findings, and liver histology. The combination
therapy may be effective for some children with PSC and autoimmune features. By ameliorating both parenchymal
inflammation and cholangiopathy of PSC, the
therapy might improve the prognosis for patients. It awaits further prospective studies to confirm the efficacy of this
therapy in patients with PSC.