Favorable Response to Human Adipose Tissue-Derived Mesenchymal Stem Cells in Steroid-Refractory Acute Graft-Versus-Host Disease

There is no consistently effective therapy for patients with steroid-refractory acute graft-versus-host disease (GVHD). Various alternative approaches have been tested, including antithymocyte globulin, mycophenolate mofetil (MMF), pentostatin, and monoclonal antibodies; however, they have only been modestly successful. The purpose of our study was to evaluate the efficacy of human adipose-tissue-derived mesenchymal stem cells (AMSC) as salvage therapy for steroid-refractory acute GVHD. Six patients with steroid-refractory grades III–IV acute GVHD received IV infusions of AMSC. The AMSC dose was 1.0 × 106/kg. No side effects were noted after the AMSC infusions. Five patients were treated once and one patient twice. Two patients received AMSC from haplo-identical family donors and four from unrelated mismatched donors. Acute GVHD disappeared completely in five of six patients, four of whom are alive after a median follow-up of 40 months (range, 18–90 months) after the initiation of AMSC therapy. All four surviving patients are in good clinical condition and in remission of their hematological malignancy. Two patients died—one with no obvious response to AMSC died of multiorgan failure and one a relapse of leukemia. These results suggested that AMSC is a promising treatment for severe steroid-resistant acute GVHD.