A Case of Progressive Familial Intrahepatic Cholestasis (PFIC)


Dr.Jerin.K.John, Dr.Bhanu Vikraman Pillai (Ped Gastro,AIMS), Dr.C.Jayakumar 

A fifty-day-old ba boy presented with yellowish discoloration of both eyes, dark-colored urine, and pale stools since birth. 
He is the first child of a non-consanguineous marriage. 
Antenatally, an anomaly scan had revealed a single umbilical artery, and the mother had gestational diabetes managed diet control. 

The ba was born at term via emergency LSCS with a birth weight of 3.28 kg. 
On day 5 of life, jaundice was noted, and he has received phototherapy for two days. However, persistent jaundice prompted further evaluation on day 16, which revealed conjugated hyperbilirubinemia. 
An initial ultrasound scan showed no dilatation of the intrahepatic biliary radicles with a normal gallbladder pre and post feed 

Due to ongoing jaundice, he was referred for further assessment. 
There was no history of fever, lethargy, poor feeding, skin bleeds, or mucosal rashes. 
A CT scan of the abdomen revealed hepatomegaly with minimal surface irregularities, mild hypertrophy of the hepatic artery, and splenomegaly. 

. His liver function test (LFT) results showed elevated levels of bilirubin (total bilirubin 13.36 mg/dL and direct bilirubin 9.01 mg/dL), with an increased AST (332.8 IU/L), ALT (236.4 IU/L), and alkaline phosphatase (ALP) (578.0 IU/L). Gamma-glutamyl transferase (GGT) was also elevated at 57.0 U/L. Additionally, his hematological profile indicated leukocytosis with a WBC count of 21.2 K/uL, predominantly lymphocytic (lymphocytes 73.6%), and a low hemoglobin level of 9.8 g/dL.

AHepato biliary Immino diacetic acid (HIDA) scan showed prompt extraction of mebrofenin hepatocytes, indicating preserved hepatocellular function. 
However, there was no progression of tracer into the intestinal loops even on delayed 24-hour imaging.

A liver biopsy done, confirming a diagnosis of Progressive Familial Intrahepatic Cholestasis (PFIC). 
On examination, his liver was palpable. At two years of age, he underwent a living donor liver transplantation. Postoperatively, he was managed with immunosuppressants, including Azathioprine, Advagraf, and Prednisolone. Doppler studies were normal post-surgery, and he recovered well, advancing from an oral liquid diet to a soft diet.
Discussion

Progressive Familial Intrahepatic Cholestasis (PFIC) is a rare autosomal recessive disorder characterized defective bile salt transport, leading to cholestasis, jaundice, and progressive liver disease. 
PFIC typically presents in infancy with symptoms of jaundice, pruritus, and hepatomegaly, progressing to liver failure if untreated. In this case, prolonged jaundice with conjugated hyperbilirubinemia and hepatomegaly raised initial concerns for obstructive liver disease. However, a detailed evaluation, including liver biopsy, confirmed PFIC, underscoring the importance of histological examination in diagnosing this rare condition.
The diagnosis of PFIC often requires a combination of clinical, biochemical, and genetic testing. Imaging can provide clues but may not distinguish PFIC from other causes of neonatal cholestasis, such as biliary atresia. 

Liver biopsy in this case provided the definitive diagnosis, enabling appropriate management. While medical treatment options are limited, liver transplantation remains the primary intervention for those with progressive liver failure, as seen in this patient.
This case highlights the importance of early recognition and comprehensive diagnostic work-up in infants with persistent cholestasis, which allows for timely intervention, especially in conditions like PFIC. Early diagnosis and treatment can help mitigate complications and improve outcomes in affected children.
Carry home message
All cases in which there is no excretion to the intestine need not be due to biliary atresia 
Peropreative cholangiogram is a must to rule out other intrahepatic choleric condition like PFICand Alagillie before one subject the child to Kasai ‘s Procedure