Chapter 10 - Cotransplantation of Autologous Perinatal Hematopoietic and Mesenchymal Stem Cells for Pediatric Patients With Very Severe Aplastic Anemia

Abstract

Childhood acquired aplastic anemia (AA), characterized by failure of hematopoiesis, is rare and potentially life-threatening. Since mesenchymal stem cells (MSCs) enhance hematopoietic stem cells (HSCs) engraftment, cotransplantation of MSCs and HSCs could be a potential strategy to treat AA patients. In the present study, the transplantation of autologous perinatal stem cells was conducted in three children with severe aplastic anemia (SAA). There was no severe side effect happened during cell transplantation, although fever was observed during the period of pancytopenia in the patients. All patients showed significant improvement within 1 month and complete recovery of hematopoiesis within 3 months after cell transplantation. Among the three patients, two patients receiving cotransplantation of cord blood HSCs and umbilical cord MSCs (UC-MSCs) achieved a long-term stable recovery of hematopoiesis, even after cyclosporine withdrawal. By contrast, the patient treated only by cord blood HSC transplantation (HSCT) showed an unstable blood cell accounts and became cyclosporine-dependent. All three patients achieved complete response (CR) at year 1 after transplantation. Therefore, autologous perinatal HSCs and MSCs might provide another opportunity for the patients who have stored autologous perinatal stem cells.