Efficacy and Safety of Mesenchymal Stem Cells in Treating Steroid-Refractory Acute Graft-Versus-Host Disease
Background
Many patients with acute graft-versus-host disease (aGVHD) do not respond well to corticosteroids. Mesenchymal stem cells (MSCs) may offer an effective second-line treatment for these patients, but more clinical studies are needed.
Study Overview
This study involved 78 patients with steroid-refractory aGVHD. They were randomly assigned to receive either umbilical cord-derived MSCs or a placebo, alongside standard treatment. MSCs were given twice a week.
Key Findings
- The overall response rate (ORR) at 28 days was 60% for those receiving MSCs and 50% for the placebo group.
- Patients receiving MSCs had a lower incidence of moderate to severe chronic graft-versus-host disease (cGVHD) over two years (13.8% vs. 39.8%).
- For patients with gut involvement, the ORR was significantly higher in the MSC group (66.7% vs. 33.3%).
- Overall, the treatment was well tolerated with similar adverse event rates in both groups.
Conclusion
This exploratory study indicates that while MSCs did not show a significant overall response advantage at 28 days, they may be beneficial for those completing treatment, especially patients with gut involvement. MSCs were well tolerated in treating steroid-refractory aGVHD.
Clinical Implications
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