ステロイド抵抗性急性移植片対宿主病に対する骨髄由来間葉系間質細胞単回投与対プラセボの無作為化二重盲検試験

Mesenchymal stromal/stem cells (MSCs) may be a useful therapy for severe acute graft-versus-host disease (GVHD).

Patients who developed steroid-refractory grade II-IV acute GVHD were randomly assigned to treatment with one infusion of third-party 2 × 10 E⁶/kg bone marrow (BM)-MSCs (n = 12) or placebo (n = 11). The median cell dose applied was 2.2 × 10 E⁶/kg (range 1.7-4.2). The median age was 37 and 47 years in the two groups (p = 0.24), respectively.

At day 28, the BM-MSC group had six (50%) complete responders and 2 patients (17%) with a partial response. In the placebo group, the corresponding figures were three (27%) complete and three (27%) partial responders (p = 0.68). Transplantation-related mortality (TRM) 100 days after treatment was 17% in the BM-MSC group and 27% in the placebo group. At 1 year, TRM was 33% and 36% in the two groups, respectively (p = 0.59). The 100-day survival rate was 83% in the BM-MSC group and 73% in the placebo group. Five-year survival in all patients was 42% and 45% in the two groups, respectively (p = 0.87).

Although only a few patients were included in the trial, a single dose of BM-MSCs did not seem to be an effective therapy for steroid-refractory acute GVHD.