PSC in children presents as ‘chronic, progressive and relentless’

BOSTON — Primary sclerosing cholangitis affects children quickly and harshly, with 30% of patients requiring a liver transplant by 10 years after diagnosis, according to a presenter at The Liver Meeting 2016.

“PSC has a chronic, progressive and relentless course in children,” Mark R. Deneau, MD, MS, from the University of Utah, said during his presentation. “Half of all patients will develop an adverse liver outcome within 10 years of diagnosis, despite over 80% being treated with ursodeoxycholic acid.”

Deneau and colleagues presented data on all available cases of pediatric PSC in patients aged younger than 18 years diagnosed with PSC or PSC and autoimmune hepatitis (n = 781) from 36 centers. Of these cases, 61% were boys, median age was 12 years and median follow-up time was 5.5 years. Ulcerative colitis was diagnosed in 83% and Crohn’s in 17%. Autoimmune hepatitis was diagnosed in 33% while 83% were treated with ursodiol therapy.

Among these patients, there was a steady progression to portal hypertension complications after PSC diagnosis, he showed. While 5% of patients had portal hypertension at diagnosis, 23% developed within 5 years and 38% within 10 years.

Additionally, many pediatric patients with PSC eventually required a liver transplant, with the median age at transplant being 16.5 years. At 5 years, 88% of the patients still had their native liver, but at 10 years, that number fell to 70%.

Deneau reported 11 liver-related deaths at a median of 9 years after diagnosis; two from end-stage liver disease, three from cholangiocarcinoma and six from post-transplant complications.

“Cholangiocarcinoma is rare, but does occur preferentially in boys, over age 15, without AIH overlap,” Deneau said.

By year 10, nearly half of patients presented with a liver-related event. Event-free survival was at 70% at 5 years and 53% at 10 years.

“One-third of all patients will need a liver transplant by 10 years,” Deneau said. “The identification of portal hypertensive or biliary complications represents a tipping point in the natural history of the disease. Two-thirds of these patients require a liver transplant within 5 years.” – by Katrina Altersitz

Reference:

Deneau MR. Abstract 133. Presented at: The Liver Meeting; Nov. 11-15, 2016; Boston.

Disclosures: Deneau reports no relevant financial disclosures.