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Clinical course and prognosis of pediatric-onset primary sclerosing cholangitis
Andrea Tenca1, Martti Färkkilä2, Johanna Arola3
1Clinic of Gastroenterology, University of Helsinki and Helsinki University Hospital, Helsinki, Finland; Gastroenterology and Endoscopy Unit, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico; Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Italy.
Insights
Pediatric primary sclerosing cholangitis (PSC) and PSC with autoimmune hepatitis (AIH) often have favorable outcomes. However, about one-third of pediatric PSC patients experience progressive disease, necessitating further research into its causes.
Area of Science:
- Hepatology
- Pediatric Gastroenterology
- Autoimmune Diseases
Background:
- The natural history of pediatric-onset primary sclerosing cholangitis (PSC) and its overlap with autoimmune hepatitis (PSC/AIH) is not well understood.
- Understanding disease progression is crucial for effective management strategies.
Purpose of the Study:
- To evaluate the clinical outcomes of pediatric-onset PSC and PSC/AIH patients.
- To assess disease progression and treatment responses in a long-term cohort.
Main Methods:
- Retrospective analysis of 33 patients diagnosed with PSC or PSC/AIH between 1993-2011 at a tertiary referral center.
- Inclusion criteria included cholangiography and liver histology confirmation.
- Long-term follow-up data were collected until December 2013.
Main Results:
- All 33 patients survived the follow-up period (median 9 years); 19 had PSC/AIH overlap. Cirrhosis was present at diagnosis in 3 patients.
- Inflammatory bowel disease (IBD), primarily ulcerative colitis, was associated in 76% of patients.
- Disease progression occurred in 36% of patients, with 12% undergoing liver transplantation without recurrence.
Conclusions:
- Pediatric-onset PSC and PSC/AIH generally have favorable outcomes into early adulthood.
- A significant subset (one-third) experiences progressive disease, challenging current treatment approaches.
- Further research into PSC pathogenesis is warranted to improve treatment guidelines.
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.
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