Experience with upadacitinib in refractory inflammatory bowel disease in children: a single-center pilot study
Authors:   National Medical Research Center for Children’s Health, Moscow, Russian Federation, Potapov A.S. ,   I.M.Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russian Federation, Vinokurova A.V. ,   National Medical Research Center for Children’s Health, Moscow, Russian Federation, Pushkareva A.E. ,   I.M.Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russian Federation, Prokhorenkova M.O. ,   I.M.Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russian Federation, Anushenko A.O. ,   National Medical Research Center for Children’s Health, Moscow, Russian Federation, Semikina E.L. ,   National Medical Research Center for Children’s Health, Moscow, Russian Federation,   I.M.Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russian Federation, Krasnovidova A.E. ,   I.M.Sechenov First Moscow State Medical University (Sechenov University), Moscow, Russian Federation, Fisenko A.P. ,   National Medical Research Center for Children’s Health, Moscow, Russian Federation
Published: February 2025

Objective. To evaluate the efficacy and safety of upadacitinib in children with refractory inflammatory bowel disease (IBD).
Patients and methods. Over the period 2023–2024, upadacitinib was administered in 35 patients with IBD (24 with Crohn’s disease (CD), 11 with ulcerative colitis (UC)) aged 2 years to 17 years 10 months (Me – 14.5 years, Q1–Q3: 11.46–16.17 years) due to inefficacy of previous treatment. The efficacy of upadacitinib was analyzed after the end of induction therapy in 18 children with IBD (12 with CD and 6 with UC). Previous treatment in all patients included anti-TNF agents, vedolizumab, tofacitinib (in UC). Clinical and endoscopic indices of disease activity, laboratory parameters at 0–2 months for UC and 0–3 months for CD were recorded.
Results. After induction therapy with upadacitinib, there was a decrease in clinical activity of IBD (remission was achieved in 25% of CD patients and in 4 (66.7%) UC patients). The decrease in the levels of white blood cells (p = 0.03) and platelets (p = 0.022) was noted in dynamics in all CD patients, ESR decreased in 83% (p = 0.052) and CRP in 72% (p = 0.035) of patients; the level of fecal calprotectin normalized in 2 (16.7%) patients. In all UC patients, there was a decrease in leukocytosis, thrombocytosis, ESR and fecal calprotectin level with upadacitinib therapy, but CRP remained unchanged. After induction therapy, 1 (17%) child with CD remained in endoscopic remission, 4 (66%) patients had minimal disease activity, and 1 (17%) patient had moderate disease activity (p = 0.03); all patients with UC (100%) had only minimal endoscopic activity.
Conclusions. High efficacy and good safety profile after induction therapy with upadacitinib in pediatric patients with refractory IBD was shown. The administration of upadacitinib allowed to achieve clinical and laboratory remission and reduction of endoscopic activity of the disease in most children with IBD in the study group. This allows to consider this medication as a promising treatment method for refractory IBD in children in case of inefficacy of standard immunosuppressive and targeted agents and necessity of therapy escalation.
Key words: inflammatory bowel disease, Crohn’s disease, ulcerative colitis, biological therapy, selective immunosuppressants, small molecules, upadacitinib