Various Regimens for Autoimmune Hepatitis in Northern European Children Show Equivalent Outcomes at 1 Year: A Retrospective Study

Marianne Hørby Jørgensen; Runar Almaas; Gandom Kharrazi; Vaidotas Urbonas; Helene Kvistgaard; Embjørg Wollen; Bente Utoft Andreassen; Thomas Casswall; Björn Fischler

doi:10.1016/j.jpeds.2025.114635

Various Regimens for Autoimmune Hepatitis in Northern European Children Show Equivalent Outcomes at 1 Year: A Retrospective Study

J Pediatr. 2025 May 8:284:114635. doi: 10.1016/j.jpeds.2025.114635. Online ahead of print.

Authors

Marianne Hørby Jørgensen¹, Runar Almaas², Gandom Kharrazi³, Vaidotas Urbonas⁴, Helene Kvistgaard⁵, Embjørg Wollen⁶, Bente Utoft Andreassen⁷, Thomas Casswall³, Björn Fischler³

Affiliations

¹ Department of Pediatric and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark; European Reference Network (ERN) RARE-LIVER, Hamburg, Germany. Electronic address: marianne.hoerby-joergensen@regionh.dk.
² European Reference Network (ERN) RARE-LIVER, Hamburg, Germany; Division of Paediatric and Adolescent Medicine, Department of Pediatric Research, Oslo University Hospital, Oslo, Norway; Institute of Clinical Medicine, University of Oslo, Oslo, Norway.
³ European Reference Network (ERN) RARE-LIVER, Hamburg, Germany; Pediatric Gastroenterology, Hepatology and Nutrition, Karolinska University Hospital, and CLINTEC, Karolinska Institute, Stockholm, Sweden.
⁴ Clinic of Children's Disease Vilnius University Hospital, Vilnius, Lithuania.
⁵ European Reference Network (ERN) RARE-LIVER, Hamburg, Germany; Department of Paediatrics and Adolescent Medicine, Aarhus University Hospital, Aarhus, Denmark.
⁶ European Reference Network (ERN) RARE-LIVER, Hamburg, Germany; Division of Paediatric and Adolescent Medicine, Department of Pediatric Research, Oslo University Hospital, Oslo, Norway.
⁷ Department of Pediatric and Adolescent Medicine, Rigshospitalet, Copenhagen, Denmark; European Reference Network (ERN) RARE-LIVER, Hamburg, Germany.

PMID: 40345575
DOI: 10.1016/j.jpeds.2025.114635

Abstract

Objective: To compare the remission rates after 1 year of treatment between two regimens: tacrolimus (with or without prednisolone) or prednisolone (with or without azathioprine) in children with autoimmune liver disease.

Study design: Retrospective data from children (n = 157) with biopsy-proven autoimmune liver disease in five tertiary northern European centers were included. Data were collected at presentation and after 3, 6, and 12 months.

Results: Initially, 111 were treated with prednisolone (with or without azathioprine) and 46 received tacrolimus (with or without prednisolone) (median start dose prednisolone: 0.99 mg/kg and 0.43 mg/kg, respectively). At diagnosis, alanine transaminase levels were similar, but the rate of advanced fibrosis was significantly higher in the tacrolimus (with or without prednisolone) group. Lower rates of alanine transaminase normalization in the tacrolimus (with or without prednisolone) group were found at 3 months (26.8 vs 58.0%, P < .001) and 6 months (46.2% vs 68.8, P = .014), but not at 1 year (68.2 vs 76.2%, P > .05). The same pattern was seen for immunoglobulin G.

Conclusion: Despite slower treatment response for patients initially started on tacrolimus (with or without prednisolone) at 3 and 6 months, biochemical and immunological responses to the two different regimens appeared equivalent at 1 year after treatment initiation.

Keywords: autoimmune hepatitis; autoimmune sclerosing cholangitis; calcineurin inhibitors; children; remission; treatment.