http://www.ncbi.nlm.nih.gov/pubmed/27504812

Valentino PL, Wiggins S, Harney S, Raza R, Lee CK, Jonas MM. J Pediatr Gastroenterol Nutr. 2016 Aug 6

Abstract

OBJECTIVES:
Data regarding pediatric PSC natural history are limited. We describe a large pediatric PSC cohort with longitudinal follow-up.

METHODS:
Records review of pediatric PSC patients diagnosed between 1984 and 2014.

RESULTS:
N = 120 (63% M) aged 1-21y (median 14y) at diagnosis. 27% (31/113) had ASC (autoimmune sclerosing cholangitis), 24% had exclusive small duct PSC, METAVIR stage was F3-F4 in 41%. 81% of PSC patients had IBD; most had ulcerative/indeterminate colitis (72/97), typically pancolitis (40/72). PSC-IBD was more common than ASC-IBD (85% vs. 68%, P = 0.03). Median follow-up was 3.7y (IQR 1.5, 6.9). Median GGT decreased from baseline of 221 U/L (IQR 110, 425) to 104 U/L by 1y post-diagnosis ([IQR 18,229], P < 0.0001), and then changed little. Mean fibrosis stage at diagnosis was 2.3 ± 1.4 (N = 91), and at 1-5y was 2.6 ± 1.3 (N = 20). Transplant-free survival at 10y was 89%; there were 6 liver transplants, 2 in patients with small duct PSC and 4 with diffuse large duct PSC. Although the cirrhosis rate was not significantly different in PSC with IBD versus without (22% vs. 41%, P = 0.06), the former had a lower rate of LT (2% vs. 18%, P = 0.01). The rate of cirrhosis was lower in patients diagnosed with IBD before PSC (15% versus 31%, P = 0.05).

CONCLUSIONS:
In this largest reported pediatric PSC cohort, LT rate at 10y was lower than reported in adults. ASC and PSC had similar biochemical abnormalities and degree of fibrosis at diagnosis. PSC that developed after IBD diagnosis had a milder course, possibly reflecting earlier disease detection or milder phenotype.