title: Mesenchymal Stem Cell Treatment for Steroid Refractory Graft-versus-Host Disease in Children: A Pilot and First Study from Turkey.
abstract: This study evaluated the efficacy of mesenchymal stem cells (MSCs) from bone marrow of a third-party donor for refractory aGVHD. We report the first experience using MSCs to treat refractory aGVHD in 33 pediatric patients undergoing allogeneic HSCT from Turkey. Totally, 68 doses of bone marrow derived MSCs were infused. The median dose of MSC was 1.18 × 10(6) cells per kg body weight. Overall, complete response (CR) was documented in 18 patients, partial response (PR) was documented in 7 patients, and no response (NR) was documented in 8 patients. The 2-year estimated probability of overall survival (OS) for patients achieving CR and PR/NR was 63.8% and 29.4%, respectively (p = 0.0002). While the cumulative incidence of transplant related mortality (TRM) at day 100 after first MSC infusion was 46.6% in PR/NR patients, there was no any TRM at day 100 after first MSC infusion in CR patients (p = 0.001). Twelve patients developed chronic GVHD (cGVHD); eight of them were alive, with five having extensive disease and three having limited disease. In conclusion, MSCs appear to be safe and effective treatment option for pediatric patients with steroid refractory aGVHD. But the efficacy of MSCs on cGVHD in aGVHD patients treated with MSCs seems to be limited.
authors.affiliation: Department of Pediatric Hematology/Oncology & Bone Marrow Transplantation Unit, Faculty of Medicine, Acıbadem University, Atakent Hospital, 34303 Istanbul, Turkey.
authors.forename: Fatih
authors.initials: F
authors.lastname: Erbey
authors.affiliation: Department of Pediatric Hematology/Oncology & Bone Marrow Transplantation Unit, Faculty of Medicine, Acıbadem University, Atakent Hospital, 34303 Istanbul, Turkey.
authors.forename: Didem
authors.initials: D
authors.lastname: Atay
authors.affiliation: Department of Pediatric Hematology/Oncology & Bone Marrow Transplantation Unit, Faculty of Medicine, Acıbadem University, Atakent Hospital, 34303 Istanbul, Turkey.
authors.forename: Arzu
authors.initials: A
authors.lastname: Akcay
authors.affiliation: Acıbadem University, Acıbadem Labcell, 34303 Istanbul, Turkey.
authors.forename: Ercument
authors.initials: E
authors.lastname: Ovali
authors.affiliation: Department of Pediatric Hematology/Oncology & Bone Marrow Transplantation Unit, Faculty of Medicine, Acıbadem University, Atakent Hospital, 34303 Istanbul, Turkey.
authors.forename: Gulyuz
authors.initials: G
authors.lastname: Ozturk