The challenges of liver transplantation in children with primary sclerosing cholangitis

Abstract

Primary sclerosing cholangitis (PSC) in children can progress to end-stage liver disease requiring liver transplantation. PSC poses many challenges beginning with evaluation and classification of ductal involvement and overlap syndromes, few options for medical management and unique risks in the post-transplant period. The construct that PSC may be an autoimmune disease is based on positive autoantibodies, association with inflammatory bowel disease, linkage to HLA type and overlap/autoimmune sclerosing cholangitis; however, PSC is not responsive to standard immunosuppression. Study of PSC and post-transplant outcomes in children may provide a unique background in which to study this challenging disease. This is particularly intriguing in the subset of patients diagnosed in the first decade of life, suggesting a strong link to predisposing genetic susceptibility and immune dysregulation. Long-term, multicenter effort is likely to be the only mechanism to study this rare disease in children and to improve outcomes in the future.

Keywords:

• autoimmune liver disease
• children
• liver transplant
• PSC
• transplant outcome

Financial & competing interests disclosure

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

No writing assistance was utilized in the production of this manuscript.

Key issues

• Indications for liver transplant in primary sclerosing cholangitis include decompensated end-stage liver disease and recurrent infection/cholangitis. Malignancy is rare in children.

• Standardized evaluation and nomenclature, documentation of pretransplant diagnosis and classification of primary sclerosing cholangitis (both by histology and cholangiography) is needed for subsequent understanding of post-transplant outcomes.

• Although malignancy related is rare in pediatric populations, targeted screening should be considered in the older teenage population both pre- and post-transplant.

• Post-transplant outcomes related to inflammatory bowel disease /colectomy, recurrence and use of living donors will require long-term follow-up and multicenter collaboration.