Abstract
Background
Data are limited on the safety and efficacy of combining advanced therapies for refractory patients with IBD.
Aim
To evaluate the real-world efficacy and safety of dual advanced therapy (DAT), combining 2 biologics or a biologic with a small molecule, in children and young adults with refractory IBD.
Methods
Primary outcome of this single IBD center cohort was DAT remission (clinical and biomarker remission) at first assessment (T1). Secondary outcomes included remission at T2, if DAT de-intensification (De-I) occurred and T3, if T2 DAT re-intensification (Re-I) occurred. Efficacy and safety outcomes were described.
Results
Of the 30 patients [43% female, 30% CD, median age of 18.3 [15.1–19.8] years], all 11 UST + TOFA achieved T1 remission; 6/10 De-I failed at T2; and 4/4 Re-I achieved T3 remission. Of 9 VDZ + TOFA, 6 achieved T1 remission; 5/6 De-I failed at T2; and 1/1 failed T3 Re-I. Of 4 UST + VDZ, 3 achieved T1 remission; 2/3 De-I failed at T2; and 0 had Re-I. Of 5 UST + UPA, 4 achieved T1 remission; 1/5 De-I failed at T2 but recaptured T3 remission post-Re-I. One VDZ + OZA achieved T1 remission and maintained T2 remission post-De-I to OZA monotherapy. At last follow-up, 43% were on original DAT, 17% on one of original DAT, and 40% neither. One UST + TOFA patient developed mild leukopenia and another developed septic arthritis and venous thromboembolism on VDZ + TOFA and prednisone.
Conclusion
Most children and young adults treated with DAT achieved remission with minimal safety events; however, de-intensification had limited success.
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AK: conducted the study, collected and interpreted the data, and drafted the manuscript; MTD: collected the data and edited the manuscript; EAS: collected the data and edited the manuscript; MCD: conceptualized and planned the study, interpreted the data, and reviewed and edited the manuscript. Each author has approved the final draft of the manuscript submitted.
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Michael T. Dolinger: Consulting fees from Neurologica, a subsidiary of Samsung electronics co., ltd, Pfizer, and BMS. Marla C. Dubinsky: Consulting fees from Abbvie, Abivax, AstraZeneca, Boehringer Ingelheim, Geneoscopy, Janssen, Lilly, Merck, Pfizer, Prometheus Biosciences, Prometheus Labs, Takeda. Amelia Kellar: Nothing to disclose. Elizabeth A. Spencer: Nothing to disclose.
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Kellar, A., Dolinger, M.T., Spencer, E.A. et al. Real-World Outcomes of Dual Advanced Therapy in Children and Young Adults with Inflammatory Bowel Disease. Dig Dis Sci (2024). https://doi.org/10.1007/s10620-024-08379-9
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DOI: https://doi.org/10.1007/s10620-024-08379-9