Treatment of Crigler-Najjar Syndrome type 1 by hepatic progenitor cell transplantation: a simple procedure for management of hyperbilirubinemia.
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.
Centre for Liver Research and Diagnostics, Deccan College of Medical Sciences, Owaisi Hospital and Research Center, Hyderabad, India.
A A
AA
Khan
N
N
Parveen
V S
VS
Mahaboob
A
A
Rajendraprasad
H R
HR
Ravindraprakash
J
J
Venkateswarlu
P
P
Rao
G
G
Pande
M Lakshmi
ML
Narusu
M N
MN
Khaja
R
R
Pramila
A
A
Habeeb
C M
CM
Habibullah
D000818:Animals; D001663:Bilirubin / Q000097:blood; D002675:Child, Preschool; D003414:Crigler-Najjar Syndrome / Q000097:blood / Q000235:genetics / Q000601:surgery*; D004195:Disease Models, Animal; D005260:Female; D016332:Fetal Tissue Transplantation; D014453:Glucuronosyltransferase / Q000235:genetics; D006499:Hepatic Artery; D022781:Hepatocytes / Q000637:transplantation*; D006801:Humans; D006932:Hyperbilirubinemia / Q000097:blood / Q000235:genetics / Q000601:surgery*; D016133:Polymerase Chain Reaction; D033581:Stem Cell Transplantation / Q000379:methods*
C019787:bilirubin glucuronoside glucuronosyltransferase; D014453:Glucuronosyltransferase; D001663:Bilirubin