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Multicenter Study
. 2025 Jan;61(2):335-345.
doi: 10.1111/apt.18359. Epub 2024 Oct 28.

Evaluation of the Effectiveness and Safety of Mesenchymal Stem Cell Treatment in Fistulising Crohn's Disease: An International Real-Life Retrospective Multicentre Cohort Study

Péter Bacsur  1   2 , Daniel Shaham  3 , Zuzana Serclova  4   5 , Tamás Resál  1 , Bernadett Farkas  1 , Patrícia Sarlós  6 , Pál Miheller  7 , Nitsan Maharshak  3 , Meir Zemel  8 , Ariella Bar-Gil Shitrit  9 , Shlomo Yellinek  10 , Anita Bálint  1 , Anna Fábián  1 , Renáta Bor  1 , Zsófia Bősze  1 , Emese Ivány  1 , Zoltán Szepes  1 , Klaudia Farkas  1   2 , Illés Tóth  11 , György Lázár  11 , Katerina Vlkova  4 , Aneta Tremerova  4   5 , Petra Zuskova  4   5 , Szabolcs Ábrahám  11 , Tamás Molnár  1
Affiliations
Multicenter Study

Evaluation of the Effectiveness and Safety of Mesenchymal Stem Cell Treatment in Fistulising Crohn's Disease: An International Real-Life Retrospective Multicentre Cohort Study

Péter Bacsur et al. Aliment Pharmacol Ther. 2025 Jan.

Abstract

Background: Perianal fistulas of Crohn's disease (CD) create a significant burden on patients' lives. However, the efficacy and safety of adipose-derived mesenchymal stem cell treatment are contradicting, and real-world evidence is lacking.

Aims: To examine the usability of darvadstrocel therapy in managing perianal CD.

Methods: We enrolled patients with CD and perianal fistulas in this retrospective multicenter study. The primary outcome was perianal clinical remission (defined as all treated fistulas closed) at weeks 26 and 52. Secondary outcomes were clinical response rates (≥ 1 fistulas closed), perianal activity (PDAI), patient satisfaction, and adverse events. Data were recorded at baseline and weeks 12, 26 and 52. Prediction of primary outcomes was performed by logistic regression.

Results: Overall, among 223 patients (male/female ratio: 0.48), perianal clinical remission was achieved in 78.2% and 62.3% until weeks 26 and 52. Baseline PDAI score (OR 0.75), number of fistulas (OR 0.28) and the number of weeks after preparation for surgery (OR 0.98) were associated with treatment failure. The clinical response rates were 84.8% and 79.8% at weeks 26 and 52. Improvement of subjective perianal symptoms was achieved in 77.8% and 78.4% of patients, respectively. Adverse events occurred in 13.5% of patients; perianal abscesses and proctalgia were the most frequently reported.

Conclusion: Effectiveness data were higher than in clinical trials. The safety profile was reassuring, and patients' satisfaction was high. Appropriate patient selection, fistula preparation and expertise may help to achieve treatment success.

Keywords: Crohn's disease; darvadstrocel; mesenchymal stem cell treatment; perianal disease.

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Conflict of interest statement

T.M. has received speaker's honoraria from MSD, AbbVie, Egis, Goodwill Pharma, Takeda, Pfizer, Janssen, Sandoz, MundiPharma, Phytotec, Roche, Fresenius and Teva. K.F. has received speaker's honoraria from AbbVie, Janssen, Ferring, Takeda and Goodwill Pharma. N.M. has received speaking and/ or consulting fees from Pfizer, Abbvie, Lilly, Takeda, Janssen, Ferring, BiomX, BMS, Nestle, Trobix Innovation and Teva and grant support from Takeda, Janssen, Abbott, Abbvie, Pfizer, BMS and Nestle. P.B., D.S., Z.S., T.R., B.F., P.S., P.M., M.Z., A.B.‐G.S., S.Y., A.B., A.F., R.B., Z.S.B., E.I., Z.S.Z., I.T., G.L., K.V., A.T., P.Z. and S.Á. have declare no conflicts of interest.

Figures

FIGURE 1
FIGURE 1
Flowchart of the patients' enrollment and follow‐ups. Pts, patients.
FIGURE 2
FIGURE 2
The proportion of patients achieving perianal clinical remission and response at weeks 26 and 52. No significant difference was observed between the timepoints and endpoints. P, significance level.
FIGURE 3
FIGURE 3
The proportion of patients with > 4 PDAI score for perianal disease during the follow‐ups, stratified by achieving perianal clinical remission at week 26. Among the patients, perianal disease immediately decreased after treatment. P, significance level.
FIGURE 4
FIGURE 4
The change in patients' symptoms during the follow‐ups, measured on a 3‐point Likert scale. The majority of the treated patients experienced an improvement in perianal symptoms. P, significance level.
FIGURE 5
FIGURE 5
Survival characteristics of adverse events requiring hospitalisation during the follow‐ups. The patients were stratified by achieving perianal clinical remission at week 26. Differences were not observed regarding outcome (X 2 = 0.522, p = 0.47).
See this image and copyright information in PMC

References

    1. Feuerstein J. D. and Cheifetz A. S., “Crohn Disease: Epidemiology, Diagnosis, and Management,” Mayo Clinic Proceedings 92, no. 7 (2017): 1088–1103. - PubMed
    1. Burisch J., Kiudelis G., Kupcinskas L., et al., “Natural Disease Course of Crohn's Disease During the First 5 Years After Diagnosis in a European Population‐Based Inception Cohort: An Epi‐IBD Study,” Gut 68, no. 3 (2019): 423–433. - PubMed
    1. Hellers G., Bergstrand O., Ewerth S., and Holmström B., “Occurrence and Outcome After Primary Treatment of Anal Fistulae in Crohn's Disease,” Gut 21, no. 6 (1980): 525–527. - PMC - PubMed
    1. Solomon M. J., “Fistulae and Abscesses in Symptomatic Perianal Crohn's Disease,” International Journal of Colorectal Disease 11, no. 5 (1996): 222–226. - PubMed
    1. Panés J. and Rimola J., “Perianal Fistulizing Crohn's Disease: Pathogenesis, Diagnosis and Therapy,” Nature Reviews. Gastroenterology & Hepatology 14, no. 11 (2017): 652–664. - PubMed

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