Umbilical cord-derived MSCs for SR-aGvHD: A real-world study

Results from a single-center, retrospective, real-world study, evaluating umbilical cord-derived mesenchymal stem cells (UC-MSCs) + second-line (2L) standard of care (SoC) in patients with steroid-refractory acute graft-versus-host disease (SR-aGvHD), were published in Leukemia & Lymphoma by Niu et al. Outcomes were compared between patients who received UC-MSCs + SoC (n = 44) or SoC alone (n = 51). The primary outcome was complete response (CR) at Week 4.

Key data: At Week 4, the CR rate was higher with UC-MSCs + SoC vs SoC alone in patients with Grade 2–4 (84.1% vs 58.8%; p = 0.007) or Grade 3–4 SR-aGvHD (54.5% vs 31.4%; p = 0.034). Liver and gastrointestinal CR rates were also higher in the UC-MSC + SoC group (45.5% vs 13.7%; p = 0.009 and 61.4% vs 33.3%; p = 0.003, respectively). The overall response rate (ORR) was higher with UC-MSCs + SoC vs SoC alone in patients with Grade 2–4 (93.2% vs 76.5%; p = 0.026) or Grade 3–4 SR-aGvHD (63.6% vs 41.2%; p = 0.029). After a median follow-up of 27 months, overall survival (OS) was similar between groups (hazard ratio [HR], 1.04; 95% confidence interval [CI], 0.52–2.06; p = 0.911). Age ≥35 years, Grade 4 aGvHD, and hemoglobin ≤90 g/L were independent adverse prognostic factors.

Key learning: Adding UC-MSCs to 2L SoC may improve early disease control in SR-aGvHD, particularly in patients with gut or liver involvement, despite no apparent survival benefit.