Objectives and Study

To describe disease course, treatment, and outcome in a multicentre prospective registry of paediatric autoimmune liver disease (p-AILD) patients, developed within ERN Rare-Liver.

Methods

New cases of AILD in children <18 years-old were enrolled in a prospective European database from 2017-2021. Patients were included in the present study if they had ≥ one year of follow-up. Biochemical data and data on medication were collected at diagnosis and after 12 months. Biochemical remission at 1 year was defined as ALT <45 UI/L and IgG <13.5 g/L.

Chi-square test was used to compare frequencies. One-way ANOVA was used to compare continuously variables.

Results

Eighty-seven patients were enrolled from 6 centres. 43 (49%) were females. Diagnosis was 61 (69%) AIH1, 10 (11%) AIH2 and 16 (18%) ASC. AIH2 were younger (p<0.001) at diagnosis. At presentation, neither ALT nor IgG differ between pAILDs. INR was<1.5 in 77 (87,5%) at presentation.

Initial treatment was mainly prednisolone with or without azathioprine. Almost all p-AILD received prednisolone (86 [98%]) and 70 [80%] received azathioprine. One patient had mycophenolate and one had tacrolimus as first choice. During the first year of treatment 15 (17%) changed medication to mycophenolate (8), budesonide (2), tacrolimus (2), everolimus (1), or biological treatment (2).

At one year of follow up, 75% had achieved normal ALT. AIH2 tended to have a higher rate of ALT lower than 45 U/l (64% AIH1, 90% AIH2, 81% ASC p=0.07). IgG below 13.5 g/l was observed in 67%, with the highest rate among AIH2 (90%) compared to the other p-AILDs (p=0.05). Only 52% had normalised both IgG and ALT.

Conclusions

In this large prospective, multicentre study, we describe the distribution of p-AILD. We found a high degree of normal ALT after one year (75%), which is in line with previous studies.