Background
It has been suggested that the transition of patients from paediatric to adult care units may be key in the outcomes of inflammatory bowel disease (IBD). However, the impact of transition in real clinical practice has been barely studied. Aims: Principal: to evaluate the impact of transition on clinical outcomes in IBD. Secondary: to describe the prevalence of transition programs in Spain; to identify predictive factors of poor clinical outcomes; and to evaluate the percentage of patients with loss to follow-up.
Methods
Multicenter, retrospective, and observational study of IBD patients transferred between 2017-2020. Two groups (transition/no-transition) were compared retrospectively. Transition was defined as a structured process with at least 1 join visit involving the gastroenterologist, paediatrician, and a program coordinator, as well as the parents and the patient. Outcomes within the first 12 months after transfer were analysed. The main variable was the presence of “poor clinical outcome” defined as an IBD flare, hospitalisation, surgery or any change of the treatment due to an IBD flare. Predictive factors of poor clinical outcome were identified in multivariate analysis.
Results
A total of 278 patients from 34 Spanish hospitals were included: 185 patients (67%) from 22 hospitals (65%) performed a structured transition. In hospitals without transition, 91% of the patients were transferred to an IBD-specialist. In 66% of the patients in the transition group, 1 joint visit was performed. The median age of transfer was 16 years [interquartile range (IQR)=12-20]. Baseline characteristics of both groups are detailed in Figure 1.
At 1-year after transfer, hospitalisations and corticosteroid treatment were more frequent in the no-transition group (10 vs. 3%; p=0.025; 16 vs. 5%; p=0.002). At 1-year after transfer, 89 patients (27% transition vs. 43% no-transition; p=0.005) had poor clinical outcome [median time: 9.3 months; 95% confidence interval (CI)=8.4-10.1 in no-transition; 10.4 months (95%CI 9.9-10.9) in the transition group]. In the multivariate analysis, the lack of transition [Hazard Ratio (HR)=2.1; 95%CI=1.4-3.3], IBD activity at transfer (HR=4.9; 95%CI=3.1-7.9), BMI <18.5 (HR=1.9; 95%CI=1.1-3.2) and corticosteroid treatment at transfer (HR=4.8; 95%CI=2.1-10.9) were associated with a poor clinical outcome. Twelve patients (4%) were lost to follow-up [1.1% in the no-transition vs. 5.9% in the transition group (p=0.06)].
Conclusion
In the present study, to our knowledge the largest performed so far, the benefit of paediatric to adult transition program on patients’ outcomes has been demonstrated. The importance of achieving remission before transfer has also been highlighted.
Background
Tofacitinib, a Janus kinase (JAK) inhibitor, has recently been approved for treatment of moderate to severe active ulcerative colitis (UC) in adults. Data on efficacy and safety in pediatrics are limited. In this multicenter study from the Paediatric IBD Porto group of ESPGHAN, we describe the short-term effectiveness and safety of tofacitinib in an international pediatric IBD cohort.
Methods
Retrospective review of children (2-18 years) diagnosed with UC treated with tofacitinib from 15 pediatric centers internationally. Primary outcome was corticosteroid-free clinical remission (PUCAI<10) at week 8, with secondary outcomes including clinical response (≥20 point decrease in PUCAI), colectomy rate and safety. Primary outcome was calculated utilizing non-response imputation (NRI), whereby drug cessation for any reason was considered treatment failure.
Results
78 patients (43 (55%) female, mean age at diagnosis 12.5 (±2.7) years, median disease duration 20 months (IQR 10.3-38.8)), all with previous biologic failure, including 20/78 (26%) with previous failure of three biologic classes.
15/78 (19%) patients achieved corticosteroid-free clinical remission at week 8 with a further 18/78 (23%) demonstrating clinical response. 9/78 (12%) underwent colectomy by week 8, and 21/78 (27%) by week 24. Twelve adverse events were reported including five infective (three of which deemed possibly related to treatment – zoster, HSV-2 cheilitis and septic arthritis), one case of pancreatitis, and abnormal blood test results in 5 children (anemia, lymphopenia, elevated hepatic transaminases and hypercholesterolemia).
Conclusion
In this largest real-life cohort of tofacitinib in pediatric UC to date, tofacitinib seemed effective in at least 19% of highly refractory patients by week 8. Adverse reactions and safety were largely consistent with adult data.
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