TY - JOUR
T1 - Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis
AU - Tenca, Andrea
AU - Färkkilä, Martti
AU - Arola, Johanna
AU - Jaakkola, Tytti
AU - Penagini, Roberto
AU - Kolho, Kaija Leena
PY - 2016/8/1
Y1 - 2016/8/1
N2 - Background: The natural history of pediatric-onset primary sclerosing cholangitis (PSC) and overlap with autoimmune hepatitis (PSC/AIH) is poorly known. Objective: The aim of this study was to evaluate the clinical outcome of patients with pediatric-onset disease in a tertiary referral center. Methods: We traced 33 patients (median age at diagnosis 16 years), with PSC or PSC/AIH in cholangiography and liver histology diagnosed between December 1993 and 2011, at Helsinki University Hospital. Diagnostic procedures and long-term follow-up were reassessed until the end of December 2013. Results: PSC was confirmed in all 33 patients; 19 of them had an overlap with AIH. At diagnosis, three of 33 had cirrhosis. Inflammatory bowel disease (IBD) was associated in 76% of the patients, mostly ulcerative colitis (70%); treatment of IBD being a minor determinant of the clinical outcome of liver disease. In the last follow-up (median nine years), all patients were alive, and no malignancy occurred. Most patients (91%) were on ursodeoxycholic acid and 12 PSC/AIH patients on immunosuppression. Endoscopic retrograde cholangiography during follow-up showed a progression of intra-hepatic disease in 12 patients (36%). Four patients (12%) had undergone liver transplantation, and one was listed; no recurrence of the disease in the graft was seen. Conclusion: The clinical course and outcome of pediatric-onset PSC and PSC/AIH seem to be favourable in the majority of patients until early adulthood. In about one-third of patients, however, PSC is progressive, challenging the current treatment guidelines and warranting further studies on disease pathogenesis.
AB - Background: The natural history of pediatric-onset primary sclerosing cholangitis (PSC) and overlap with autoimmune hepatitis (PSC/AIH) is poorly known. Objective: The aim of this study was to evaluate the clinical outcome of patients with pediatric-onset disease in a tertiary referral center. Methods: We traced 33 patients (median age at diagnosis 16 years), with PSC or PSC/AIH in cholangiography and liver histology diagnosed between December 1993 and 2011, at Helsinki University Hospital. Diagnostic procedures and long-term follow-up were reassessed until the end of December 2013. Results: PSC was confirmed in all 33 patients; 19 of them had an overlap with AIH. At diagnosis, three of 33 had cirrhosis. Inflammatory bowel disease (IBD) was associated in 76% of the patients, mostly ulcerative colitis (70%); treatment of IBD being a minor determinant of the clinical outcome of liver disease. In the last follow-up (median nine years), all patients were alive, and no malignancy occurred. Most patients (91%) were on ursodeoxycholic acid and 12 PSC/AIH patients on immunosuppression. Endoscopic retrograde cholangiography during follow-up showed a progression of intra-hepatic disease in 12 patients (36%). Four patients (12%) had undergone liver transplantation, and one was listed; no recurrence of the disease in the graft was seen. Conclusion: The clinical course and outcome of pediatric-onset PSC and PSC/AIH seem to be favourable in the majority of patients until early adulthood. In about one-third of patients, however, PSC is progressive, challenging the current treatment guidelines and warranting further studies on disease pathogenesis.
KW - cirrhosis
KW - inflammatory bowel disease
KW - liver transplantation
KW - Pediatric
KW - primary sclerosing cholangitis
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U2 - 10.1177/2050640615616012
DO - 10.1177/2050640615616012
M3 - Article
AN - SCOPUS:85006213907
VL - 4
SP - 562
EP - 569
JO - United European Gastroenterology Journal
JF - United European Gastroenterology Journal
SN - 2050-6406
IS - 4
ER -