Primary sclerosing cholangitis (PSC) is an idiopathic, inflammatory, and fibrotic disease characterized by strictures and dilatations of both the intra- and extrahepatic bile ducts.
1
An effective medical therapy remains undetermined. PSC may occur in children; however, its etiology in children is unknown. Several studies proposed that genetic inheritance (e.g., HLAA1, B8, and DR3 genotypes) and autoimmune, infectious, and environmental factors (diet and gastrointestinal microbiome) are related to the disease. A total of 60%–80% of PSC patients have concomitant inflammatory bowel disease (IBD), with ulcerative colitis (UC) being the most common form and less frequently Crohn's disease (CD). However, pediatric IBD with PSC is uncommon, affecting 9.9% of children with UC and 0.6% of patients with CD.2
Regarding the clinical course of the disease, pediatric patients having UC with PSC (UC-PSC) have less severe bowel disease and receive less treatment with steroids or infliximab and experience fewer IBD adverse events, including hospital admissions and colectomy, than patients having colitis without PSC.
3
However, several studies do not support the result. Although the clinical activity of pediatric UC-PSC patients is mild, the absence of symptoms does not always indicate lack of mucosal inflammation. Children with UC-PSC are at an increased risk of active endoscopic and histologic disease in the absence of symptoms and present significant pancolitis with a right-sided predominance, macroscopic rectal-sparing, and a higher risk of colorectal cancer.4
The European Society of Pediatric Gastroenterology, Hepatology, and Nutrition (ESPGHAN) has suggested that ileocolonoscopy should be conducted in IBD patients with concurrent PSC annually or at least biannually, starting from the time of PSC diagnosis.Pediatric UC-PSC patients have less severe colitis with respect to clinical activity index and endoscopic severity index; however, they are more likely to need an immunomodulator and less likely to be steroid-free in the long term for control of liver disease.
5
Moreover, children with PSC-IBD have more serious growth impairments than children with UC or unclassified IBD.6
- Ricciuto A.
- Hansen B.E.
- Ngo B.
- Aloi M.
- Walters T.D.
- Church P.C.
- et al.
Primary sclerosing cholangitis in children with inflammatory bowel diseases is associated with milder clinical activity but more frequent subclinical inflammation and growth impairment.
Clin Gastroenterol Hepatol. 2020; 18: 1509-1517
In pediatric PSC-IBD, 28% of patients present a unique phenotype characterized by an overlap with autoimmune hepatitis (AIH).
7
The overlap syndrome of PSC and AIH is more common in children than in adults and induces milder colitis activity and may be treated with steroids and immunomodulators.In conclusion, pediatric UC-PSC has a distinct phenotype, including milder bowel disease, less treatment with steroids or infliximab, lower rate of IBD-related admission and surgery, more common pancolitis and rectal-sparing, higher risk of colorectal cancer, and greater growth impairment. Annual or at least biannual ileocolonoscopy is recommended. PSC and AIH overlap syndrome is more common in children and can be treated with steroids and immunomodulators.
References
- Primary sclerosing cholangitis.N Engl J Med. 2016; 375: 1161-1170
- Primary sclerosing cholangitis, autoimmune hepatitis, and overlap in Utah children: epidemiology and natural history.Hepatology. 2013; 58: 1392-1400
- Unique inflammatory bowel disease phenotype of pediatric primary sclerosing cholangitis: a single-center study.J Pediatr Gastroenterol Nutr. 2017; 65: 404-409
- The IBD and PSC phenotypes of PSC-IBD.Curr Gastroenterol Rep. 2018; 20: 16
- The characteristics of pediatric ulcerative colitis with primary sclerosing cholangitis: a single-center study in Taiwan.Pediatr Neonatol. 2021; 62: 483-490
- Primary sclerosing cholangitis in children with inflammatory bowel diseases is associated with milder clinical activity but more frequent subclinical inflammation and growth impairment.Clin Gastroenterol Hepatol. 2020; 18: 1509-1517
- The natural history of primary sclerosing cholangitis in 781 children: a multicenter, international collaboration.Hepatology. 2017; 66: 518-527
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