{
  "abstract": "Upadacitinib (UPA) is a novel orally administered small molecule drug that works as a selective Janus kinase (JAK) inhibitor and is approved for use in adults with ulcerative colitis (UC) and Crohn’s disease (CD). 1 There is increasing off-licence use of UPA in paediatric patients but there remains little data of its safety and efficacy.2–4 We present here early data on use of UPA over 12 months in an adolescent cohort with refractory IBD at a single centre from October 2023 - 2024. We identified patients ≤ 18 years with IBD started on UPA and gathered data on demographics, disease subtype, number of biologic failures, steroid use, adverse events and biomarkers of remission.17 patients with paediatric IBD (median age 16 years, range 12–17 years old, 9 females (53%)) were identified. 4 patients had UC and 13 had CD. 8/17 (47%) commenced UPA following failure of 2 biologics, 6/17 (35%) had failed 3 or more biologics (1 had trialled 4 drugs: adalimumab, infliximab, ustekinumab, vedolizumab). In all patients previous biologics were stopped and UPA was used as monotherapy. All patients had active disease on commencement of UPA; 3 out of 17 were on steroid treatment and all had faecal calprotectin >200µg/g. 5/17 patients (29%) had a high C-reactive Protein (CRP), only 1/17 patients had a low albumin. All patients were started on the recommended 45 mg UPA.By patient and physician global assessment 13/17 patients (82%) had clinical improvement/clinical remission at 4–8 weeks of UPA therapy. For 3 patients on steroids, weaning was successful and no further courses required. Of 13 patients whose results were available at time of data collection, endoscopic remission, suggested by faecal calprotectin <100µg/g, was achieved in 7 out of 13 patients (53%) at 4 weeks of UPA therapy. One patient discontinued UPA at 3 months after induction therapy due to primary non-response. Relapse at 12 weeks following induction therapy was noted in 1 patient after dose decreased to 15 mg (parental choice), then achieved remission on 30 mg maintenance dose. All other patients were prescribed the higher maintenance dose of 30 mg.No serious adverse events occurred; 1 patient with a background of asthma stopped and restarted UPA at a lower dose following increased respiratory sputum burden and emergency department attendance, UPA was subsequently discontinued at 6 months for similar symptoms. 1 patient experienced a pruritic rash on first administration which resolved and did not recur, and 2 patients experienced acne but continued the medication.In summary, this study showed most patients on UPA for refractory IBD had good compliance, tolerance and symptom improvement. Currently, 4.8% of the total paediatric IBD cohort in the study centre is being treated with UPA; its increasing use is in part due to its oral administration and it avoids the use of bridging steroids. It would be beneficial to repeat this audit with increased patient numbers and additional data. In addition, with the expansion of biologic therapies available, there is a mounting case for a nationally held prospective database for paediatric IBD patients.References Honap S, Agorogianni A, Colwill MJ , et al. JAK inhibitors for inflammatory bowel disease: recent advances. Frontline Gastroenterol. 2024;15:59–69.Ashton J, Lee KY, Thangarajah A, et al. Therapeutic options for children and young people with moderate-to-severe ulcerative colitis. Frontline Gastroenterol. 2024;15:387–394.Bergstein S, Spencer E. Single center experience with upadacitinib for refractory pediatric inflammatory bowel disease. Gastroenterology 2023;164(4):79.Spencer E, Bergstein S, Dolinger M, et al. Single-center experience with upadacitinib for adolescents with refractory inflammatory bowel disease. Inflamm Bowel Dis. 2024;30(11):2057–2063.",
  "authors": [
    {
      "affiliations": [
        "John Radcliffe Hospital, Oxford University NHS Foundation Trust"
      ],
      "name": "Tamanna Rahimi"
    },
    {
      "affiliations": [
        "John Radcliffe Hospital, Oxford University NHS Foundation Trust"
      ],
      "name": "Nancy Mew"
    },
    {
      "affiliations": [
        "John Radcliffe Hospital, Oxford University NHS Foundation Trust"
      ],
      "name": "Lucy Howarth"
    },
    {
      "affiliations": [
        "John Radcliffe Hospital, Oxford University NHS Foundation Trust"
      ],
      "name": "Astor Rodrigues"
    }
  ],
  "title": "OC62 Upadacitnib in the treatment of paediatric refractory inflammatory bowel disease; experience at a specialised paediatric UK centre",
  "uid": "62c071f9-916c-5aca-ab67-5553d4c87ce6"
}