P0821 Effectiveness and safety of mesenchymal stem cell treatment in fistulizing Crohn’s disease: predictors of treatment success
P Bacsur, D Shaham, Z Serclova, T Resál, B Farkas, P Sarlós, P Miheller, N Maharshak, M Zemel, A Shirit, A Bálint, A Fábián, R Bor, Z Bősze, E Ivány, Z Szepes, K Farkas, I Tóth, G Lázár, K Vlkova, A Tremerova, P Zuskova, S Ábrahám, T MolnárAbstract
Background
Perianal fistulas of Crohn’s disease (CD) create a significant burden on patient lives. However, the efficacy and safety of adipose-derived mesenchymal stem cell treatment are contradicting, and real-world evidence is lacking. We aimed to examine the usability of darvadstrocel therapy in managing perianal CD.
Methods
In this retrospective multicenter study CD patients with perianal fistulas were enrolled and followed. The primary outcome was perianal clinical remission (all treated fistulas have closed) at weeks 26 and 52, while the secondary outcomes were clinical response rates (≥ 1 fistulas have closed), perianal activity (PDAI), patient satisfaction, and adverse events. The data was recorded at the baseline and weeks 12, 26, and 52. Prediction of primary outcomes was performed by logistic regression.
Results
Overall, based on the data of 223 patients (Table 1., male/female ratio: 0.48), perianal clinical remission was achieved in 78.2% and 62.3% until week 26 and 52 (Figure 1.), whereas 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 week 26 and 52. Moreover, the improvement of subjective perianal symptoms was achieved in 77.8% and 78.4% of the patients, respectively. Adverse events occurred in 13.5% of the patients, with perianal abscesses and proctalgia reported the most.
Conclusion
Effectiveness data are higher than the clinical trials. The safety profile is reassuring, and patients’ satisfaction is high. Appropriate patient selection, fistula preparation and expertise may help to achieve treatment success.