Evaluation of the effectiveness of treatment of primary sclerosing cholangitis and autoimmune sclerosing cholangitis in children and adolescents

M.B. Dyba; V.S. Berezenko; H.Z. Mykhailiuk; O.M. Tkalyk

doi:10.26641/2307-0404.2026.1.356908

Авторы

M.B. Dyba State institution “Ukrainian Center of Maternity and Childhood of the National Academy of Medical Sciences of Ukraine”, Platona Maіborody str., 8, Kyiv, 04050, Ukraine https://orcid.org/0000-0002-9463-7867
V.S. Berezenko State institution “Ukrainian Center of Maternity and Childhood of the National Academy of Medical Sciences of Ukraine”, Platona Maіborody str., 8, Kyiv, 04050; Bogomolets National Medical University, Taras Shevchenko boulev., 13, Kyiv, 01601, Ukraine https://orcid.org/0000-0002-3777-5251
H.Z. Mykhailiuk State institution “Ukrainian Center of Maternity and Childhood of the National Academy of Medical Sciences of Ukraine”, Platona Maіborody str., 8, Kyiv, 04050, Ukraine https://orcid.org/0000-0002-6720-6674
O.M. Tkalyk State institution “Ukrainian Center of Maternity and Childhood of the National Academy of Medical Sciences of Ukraine”, Platona Maіborody str., 8, Kyiv, 04050, Ukraine https://orcid.org/0000-0002-5449-8399

DOI:

https://doi.org/10.26641/2307-0404.2026.1.356908

Ключевые слова:

primary sclerosing cholangitis, autoimmune sclerosing cholangitis, treatment, biochemical response, SCOPE index, shear wave elastography

Аннотация

Primary sclerosing cholangitis and autoimmune sclerosing cholangitis in children are rare chronic liver diseases characterized by progressive bile duct damage, fibrogenesis, and a risk of developing biliary cirrhosis. Autoimmune sclerosing cholangitis is considered a distinct pediatric phenotype of primary sclerosing cholangitis, combining clinical and morphological features of both autoimmune hepatitis and sclerosing cholangitis. Despite similar immunopathogenetic mechanisms, these diseases differ significantly in clinical course, therapeutic response, and long-term prognosis. The overall effectiveness of treatment remains suboptimal, which may be related to the ongoing progression of bile duct fibrosis even under therapy. Lack of biochemical response within the first 6-12 months is considered an unfavorable prognostic factor and should be regarded as an indication for treatment escalation or transition to alternative therapeutic strategies. Evaluation of treatment efficacy is essential for timely decision-making regarding therapeutic adjustments. The aim of this study was to evaluate the effectiveness of treatment in children with primary sclerosing cholangitis and autoimmune sclerosing cholangitis over a 12-month period based on the dynamics of biochemical response, liver stiffness measurements assessed by elastography, and the SCOPE prognostic index. A total of 56 children aged 2 to 18 years, who underwent evaluation and treatment between 2016 and 2024 at a pediatric hepatology department, were included in the study: 38 with autoimmune sclerosing cholangitis and 18 with primary sclerosing cholangitis. Patients in the first group received combined immunosuppressive therapy, while those in the second group were treated with ursodeoxycholic acid. After 12 months of treatment, complete biochemical response was achieved in 47% and 67% of children, respectively (p=0.27), and normalization of gamma-glutamyltransferase in 63% and 83% (p=0.21). The prognostic index significantly decreased in both groups, while liver stiffness reduction was statistically significant only in children with autoimmune sclerosing cholangitis (p=0.002). These findings indicate partial treatment effectiveness and highlight the importance of comprehensive monitoring to support timely modification of therapeutic strategies.

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