Pediatric autoimmune liver disease (AILD) includes autoimmune hepatitis (AIH) and autoimmune sclerosing cholangitis (ASC). Children with AILD not responding to standard immunosuppression (IS) may progress to end-stage liver disease and require liver transplantation.

Despite the absence of strong evidences the empirical use of candidate therapies has significantly increased in the last decades. Oral vancomycin has an immunomodulatory effect and it has been used in patients with primary sclerosing cholangitis. In pediatrics, the experience with oral vancomycin treatment (OVT) in patients with AIH or ASC is very limited.

In this study we evaluated: (1) The response to standard IS in a large cohort of pediatric patients with AILD; and (2) The efficacy of OVT to normalize transaminases (biochemical remission) and to achieve immunological remission in patients not responding to standard IS.

Retrospective study of children diagnosed with AILD (AIH or ASC) at Hospital Papa Giovanni XXIII, Bergamo, Italy, in the last decade. Response to IS treatment and need for OVT was reported in all patients and compared between the two groups (AIH vs ASC).

Seventy-five patients diagnosed with AILD were included in this study (median age 10.5 years, range 5.6-13.4; F = 69%); 12 patients (16%, 10 with ASC) required OVT. Response to OVT was observed in 75% of patients and the percentage of those who achieved biochemical remission increased overall from 81% to 92%. Decrease in Sclerosing Cholangitis Outcomes in Pediatrics (SCOPE) index was reported in 42% of patients.

This study shows that OVT may be considered as a valuable treatment option to achieve biochemical remission in children with AILD not responding to standard IS. Decrease in SCOPE index after OVT may suggest improvements in the long-term outcome.

These promising preliminary results suggest that further prospective studies are needed to better define the efficacy of OVT in AILD.