Abstract

Background:

A-LiNK (Autoimmune Liver disease Network for Kids) is a collaborative, multi-center learning health network uniting patients, parents, clinicians, researchers, and stakeholders to address gaps in care for children with Autoimmune Liver Disease. We seek to define predictors of optimal outcomes and determine current practices in Autoimmune Hepatitis (AIH).

Methods:

Patients aged 1-21 years old with AIH and ≥ 1 year of follow-up were included in a retrospective registry, with clinical data collected at diagnosis and every 3 months for 1 year. The primary endpoints were biochemical remission rate (defined as normal serum AST and ALT based on age/sex) 6 months after diagnosis and predictors of non-remission. In an accompanying prospective cohort, patients with AIH had laboratory values, clinical findings, and patient-reported outcomes documented. Key outcomes were rates of biochemical and steroid-free remission. Statistical analysis for significance utilized Fischer’s p test for dichotomous variables and Wilcoxon rank sum for continuous variables.

Results:

Seven A-LiNK centers included 116 patients with AIH in a retrospective cohort: 64% female, 71% white, and 17% African American. Within 1.5 months of diagnosis, 98% were treated with IV/oral corticosteroids, 14% budesonide, and 54% immunomodulators. Of 72 patients with 6-month biochemistries, 65% were in biochemical remission. Non-remission at 6 months was associated with being female; African American; low serum albumin and hematocrit; and elevated IgG, white blood cell count, and INR (all p<0.05). Type of immunosuppression at induction was not associated with remission at 6 months.

Prospectively, 4 A-LiNK centers enrolled 36 patients with AIH from March 2022-May 2023 with 61 visits. Participants had similar demographics as the retrospective cohort. The median age at enrollment was 16.4 years, with a median 3.8 years since diagnosis. Most patients were on immunosuppression: 42% prednisone, 25% budesonide, 61% azathioprine, 19% mycophenolate mofetil, and 6% tacrolimus. Only 3 patients were off immunosuppression. At initial visit, 11 (31%) endorsed fatigue and/or abdominal pain; 5 had steroid-associated complications, and 2 experienced complications of portal hypertension. Key outcomes of biochemical and steroid-free remission rates were reported monthly, shown in Figure 1.

Conclusion:

In a multi-center retrospective cohort, only 65% of patients with AIH achieved remission 6 months after diagnosis, with female and African American children less likely to enter remission. In a prospective prevalence study, only 61% were in remission and 33% were in steroid-free remission at a median of 3.8 years after diagnosis. These findings highlight the ongoing need for better pharmacological therapy and application of guideline-based care and is foundational for future implementation trials to improve biochemical and steroid-free remission rates in AIH.