What are the limitations of the evidence on long‑term safety and efficacy of darvadstrocel in perianal CD

What is known

- The ADMIRE-CD 104-week extension included only 40 patients (25 treated, 15 control), and remission may be sustained to 104 weeks with good tolerability in this small cohort. ¹ - A systematic review concludes short-term efficacy can be sustained long-term for most patients, but long-term efficacy has not been proven significantly superior to placebo — only a favorable trend. ² - Real-world data are limited and evidence on long-term effectiveness and economic implications in routine practice is scarce; ongoing registries (INSPIRE, 36 months) are still maturing. ^2,3 - Head-to-head comparisons with surgical options such as advancement-flap surgery are lacking, and the Japanese phase 3 study broadly reproduced European efficacy/tolerability. ^2,4

What is unknown / caveats

- Small long-term follow-up cohort (n=40 at week 104) - No head-to-head trials vs surgical alternatives - Safety data lacking in breastfeeding mothers; registries still maturing - Whether long-term benefit truly exceeds placebo remains unresolved because the extension cohort was small and the separation was only a trend.

## References

1. Garcia-Olmo D, Gilaberte I, Binek M, D Hoore AJL, Lindner D, Selvaggi F, Spinelli A, Panés J. Follow-up Study to Evaluate the Long-term Safety and Efficacy of Darvadstrocel (Mesenchymal Stem Cell Treatment) in Patients With Perianal Fistulizing Crohn's Disease: ADMIRE-CD Phase 3 Randomized Controlled Trial. Dis Colon Rectum. 2022;65(5):713-720. PMID: 34890373.

2. Fousekis FS, Mpakogiannis K, Lianos GD, Koukoudis A, Christodoulou DK, Papaconstantinou I, Katsanos KH. Effectiveness and safety of darvadstrocel in patients with complex perianal fistulizing Crohn's disease: a systematic review. Ann Gastroenterol. 2024;37(1):46-53. PMID: 38223244.

3. Casanova-Martínez C, Espino-Paisán E, Lema-Oreiro M, Álvarez-Sánchez MJ, Buján-de-Gonzalo L. Darvadstrocel: real-world clinical outcomes and economic impact in the Spanish national health system. Ann Gastroenterol. 2025;38(6):1-6. PMID: 41079272.

4. Furukawa S, Mizushima T, Nakaya R, Shibata M, Yamaguchi T, Watanabe K, Futami K. Darvadstrocel for Complex Perianal Fistulas in Japanese Adults with Crohn's Disease: A Phase 3 Study. J Crohns Colitis. 2023;17(3):369-378. PMID: 36149832.

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_Draft — generated by the IBDology RAG and not yet clinician-reviewed. Answers are grounded in the retrieved literature listed above; a high faithfulness score means the answer matches its sources, not that the sources are correct._