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

P. Bacsur, D. Shaham, Z. Serclova, T. Resál, B. Farkas, P. Sarlós, P. Miheller, N. Maharshak, M. Zemel, AB. Shitrit, S. Yellinek, A. Bálint, A. Fábián, R. Bor, Z. Bősze, E. Ivány, Z. Szepes, K. Farkas, I. Tóth, G. Lázár, K. Vlkova, A. Tremerova,...

. 2025 ; 61 (2) : 335-345. [pub] 20241028

Jazyk angličtina Země Anglie, Velká Británie

Typ dokumentu časopisecké články, multicentrická studie

Perzistentní odkaz   https://www.medvik.cz/link/bmc25002892

Grantová podpora
125377 Nemzeti Kutatási Fejlesztési és Innovációs Hivatal
129266 Nemzeti Kutatási Fejlesztési és Innovációs Hivatal
134863 Nemzeti Kutatási Fejlesztési és Innovációs Hivatal
143549 Nemzeti Kutatási Fejlesztési és Innovációs Hivatal
BO/00598/19/5 Emberi Eroforrások Minisztériuma
EKÖP-368 Emberi Eroforrások Minisztériuma
UNKP- 23-3 -SZTE-268 Emberi Eroforrások Minisztériuma
UNKP-22-4 -SZTE-293 Emberi Eroforrások Minisztériuma
UNKP-22-4-SZTE-296 Emberi Eroforrások Minisztériuma
UNKP-23-3-SZTE-317 Emberi Eroforrások Minisztériuma
Szent-Györgyi Albert Orvostudományi Kar, Szegedi Tudományegyetem
739593 Horizon 2020 Framework Programme
Szegedi Tudományegyetem

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.

Citace poskytuje Crossref.org

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