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P616 Ustekinumab for refractory paediatric Crohn’s disease: experience from two UK tertiary referral centres

R. Rao*1, R. Gadhok1, L. Whitley2, N. Burgess1, P. Amon1, S. Naik1, J. Lindsay1, S. McCartney2, K. Kok1

1The Royal London Hospital, Gastroenterology, London, UK, 2University College London Hospital, Gastroenterology, London, UK

Background

Crohn’s disease (CD) is frequently diagnosed in childhood and follows a more aggressive course when compared with adults. In this population, anti-TNF treatments are well established, but little is known about the efficacy and safety of ustekinumab. We report our experience in its use for paediatric Crohn’s disease across two London hospitals.

Methods

Paediatric patients (<18 years) commenced on ustekinumab were identified from University College London Hospital (UCLH) and The Royal London Hospital (RLH). A retrospective case note review was conducted and data collected on disease phenotype, prior treatment, prior surgery, CRP and weight. Biological response at Week 8 was defined as a 50% reduction in CRP where the baseline CRP was >5 mg/l.

Results

Ten patients with CD were commenced on ustekinumab under the age of 18. The baseline characteristics are summarised in Table 1. All patients had failed at least one anti-TNF and 8 patients had failed two. The mean CRP at baseline was 38 mg/l. Patients received intravenous drug at baseline and 8 weekly subcutaneous dosing thereafter. Two patients discontinued treatment prior to Week 16 owing to primary non-response, both requiring intestinal resection. Four patients had reached Week 16 at the time of analysis. One patient had been followed up for 29 weeks at the time of analysis. No adverse events were reported. Where paired data were available, there was a significant increase in mean weight from baseline (38.9 kg, n = 7) to Week 8 (42.7 kg, n = 7, p = 0.003) and Week 16 (44.0 kg, n = 3, p = 0.001). Where paired data were available, mean CRP (mg/l) improved from 38 at baseline (n = 7) to 22 at Week 8, and 9 at Week 16 (n = 4), although this did not reach significance. The biological response rate was 50% at Week 8. Both patients on steroids at baseline had discontinued these by Week 8.

Table 1. Baseline characteristics of paediatric patients treated with ustekinumab.

Conclusion

We report on the use of ustekinumab to treat anti-TNF refractory Crohn’s disease in 10 paediatric patients. This was well tolerated with no adverse events reported. We found a mean reduction in CRP and significant weight gain at Week 8 which was sustained at Week 16, suggesting clinical benefit. Further studies are needed to establish the safety and efficacy of its use in the paediatric population.