Seamless Management of Juvenile Autoimmune Liver Disease: Long-Term Medical and Social Outcome

J Pediatr. 2020 Mar;218:121-129.e3. doi: 10.1016/j.jpeds.2019.11.028. Epub 2020 Jan 16.

Abstract

Objectives: To report baseline features and long-term medical/social outcomes of juvenile autoimmune liver disease, including autoimmune hepatitis (AIH) and autoimmune sclerosing cholangitis (ASC), managed in a single tertiary center.

Study design: Retrospective study of children diagnosed in 2000-2004 with AIH/ASC followed up to date. Patients with abnormal cholangiogram were classified as ASC. Presentation and outcome features were compared.

Results: Eighty-three children were included (42 female, median age 12.1 years [8.5-14.1 years], AIH = 54, ASC = 29). Most (65%) had antinuclear and/or anti-smooth muscle autoantibodies; 6% presented with acute liver failure; 29% had histologic evidence of cirrhosis. The 1999 and simplified International Autoimmune Hepatitis Group criteria failed to diagnose up to 26% of patients with AIH and 48% with ASC, and the proposed the European Society for Pediatric Gastroenterology, Hepatology and Nutrition criteria were accurate. Response to treatment was excellent with 95% achieving normal transaminase levels. During follow-up, 31% had at least 1 relapse episode; 3 patients with AIH developed cholangiopathy and 5 patients with ASC developed progressive bile duct injury. At last follow-up (median of 14.5 years, 10.4-16.8), 99% were alive, 11 underwent transplantation and 1 is listed for transplant. Five-, 10-, and 15-year transplant-free survival rates were 95%, 88%, and 83%; patients with ASC and those relapsing being more likely to require transplant. Social outcome was excellent with 93% in employment/education.

Conclusions: Seamless management of juvenile autoimmune liver disease leads to excellent clinical and social outcomes. Despite good response to immunosuppressive treatment, patients with ASC have a worse prognosis than those with AIH. Diagnostic models developed for adults are unsatisfactory to correctly diagnose juvenile autoimmune liver disease.

Keywords: autoimmune hepatitis; autoimmune sclerosing cholangitis; cholangiopathy; juvenile; relapse.

MeSH terms

  • Adolescent
  • Autoantibodies / immunology
  • Child
  • Cholangitis, Sclerosing / epidemiology
  • Cholangitis, Sclerosing / therapy*
  • Continuity of Patient Care
  • Employment
  • Female
  • Follow-Up Studies
  • Hepatitis, Autoimmune / epidemiology
  • Hepatitis, Autoimmune / therapy*
  • Humans
  • Immunosuppression Therapy
  • Immunosuppressive Agents / therapeutic use
  • Kaplan-Meier Estimate
  • Liver Transplantation
  • Male
  • Patient Care Team
  • Postoperative Period
  • Recurrence
  • Retrospective Studies
  • Tertiary Care Centers
  • Time Factors
  • Treatment Outcome

Substances

  • Autoantibodies
  • Immunosuppressive Agents