Umbilical Cord-Derived Mesenchymal Stem Cells for Treatment of Steroid-Resistant Severe Acute Graft-Versus-Host Disease

To evaluate the safety profile and efficacy of umbilical cord-derived mesenchymal stem cell infusion in patients with steroid-resistant, severe, acute graft-versus-host disease (aGVHD).

A total of 19 patients with steroid-resistant severe aGVHD received mesenchymal stem cell infusion treatment. We analyzed the treatment response, transplantation-related mortality, events associated with infusion and relapse rate.

Two patients with grade II, 5 patients with grade III and 12 patients with grade ‡W aGVHD received a total of 58 infusions of mesenchymal stem cell. The mean total dose of mesenchymal stem cell was 2.13×10⁶ (range 0.6–7.2×10⁶) cells per kg bodyweight. 7 patients received one infusion, 2 patients received two infusions, and 10 patients received three or more infusions. 11 patients had a complete response and 4 had a partial response and 4 had no response. No patients had side-effects during or immediately after infusions of mesenchymal stem cell and no ectopic tissue was detected to date. 11 patients survived and 8 died, 4 for aGVHD, 1 for infection and 2 for aGVHD with concomitant infection and 1 for underlying leukemia relapse. The cell viability of freshly prepared mesenchymal stem cell is 93% (92%-95%) by trypan blue staining. The cell viability of controlled-rate freezed and thawed cells mesenchymal stem cell is 72% (70%-74%).

Infusion of umbilical cord-derived mesenchymal stem cell expanded in vitro is an effective therapy for patients with steroid-resistant, severe aGVHD without negative impact on relapse. Freshly prepared mesenchymal stem cells are superior to freezed and thawed cells in terms of cell viability.

No relevant conflicts of interest to declare.