Crigler-Najjar Syndrome (CNS) is characterized by mild, chronic unconjugated
hyperbilirubinemia resulting from an autosomal-recessive inherited deficiency of hepatic
uridine/diphosphoglucuronate-glucuronosyl
transferase 1Al since birth. Herein we have reported a confirmed case of CNS type 1 in a 2-year-old girl with an unconjugated
hyperbilirubinemia (>30 mg/dL) treated by hepatic progenitor cell infusion through the hepatic artery. No procedure-related complications were encountered. No
kernicterus was observed. The total
bilirubin started falling
at 10 days after cell infusion. Two months after cell infusion the
bilirubin fell from 29.0 to 16 mg/dL, with the conjugated
bilirubin increasing approximately fivefold, the unconjugated
bilirubin decreasing nearly twofold, and the
SGPT also decreasing from 210 U/L to 64 U/L. This study demonstrated the efficacy of hepatic progenitor cells to manage
hyperbilirubinemia in these patients. As the procedure is simple and the patient has tolerated the
cell therapy, infusion can be repeated as required to manage
hyperbilirubinemia, which often causes lethal
kernicterus. This study was developed to assess the safety, feasibility, and efficacy of hepatic progenitor cell
transplantation in a child with CNS type 1.