Application and research advances in stem cell transplantation for severe aplastic anemia / 中国组织工程研究

ABSTRACT

BACKGROUND: Allogeneic hematopoietic stem cell transplantation is still the only cure method for acquired severe aplastic anemia. How to select patients for treatment has become a research hotspot in recent years. OBJECTIVE: To review the progress of allogeneic hematopoietic stem cell transplantation from three aspects HLA full-phase matched unrelated donor hematopoietic stem cell transplantation (MUD-HSCT), unrelated cord blood transplantation (UCBT) and haploidentical hematopoietic stem cell transplantation (HID-HSCT). METHODS: Literatures on allogeneic hematopoietic stem cell transplantation for severe aplastic anemia collected in PubMed, CNKI full-text database and WanFang database from 2000 to 2018 were retrieved with the keywords “unrelated donor; haploidentical; unrelated cord blood; severe aplastic anemia” in Chinese and English. RESULTS AND CONCLUSION: MSD-HSCT is the first-line treatment for severe aplastic anemia, but in view of China’s special national conditions, HLA matched donor is not easy to find. As an important alternative treatment, MUD-HSCT is close to MSD-HSCT. However, the incidence of graft versus host disease and severe infection after MUD-HSCT is still higher than that after MSD-HSCT. It is still necessary to consider multiple factors when choosing MUD-HSCT treatment. Umbilical cord blood hematopoietic stem cells are widely used because of their abundant sources and high match success rate. The probability of UCBT is very high when the amount of pre-frozen total nucleated cells is more than 3.9×107/kg. However, in view of the delay of UCBT and immune function reconstruction, unless other transplantation methods are not feasible in clinical treatment of severe aplastic anemia and immunosuppressive therapy fails in the first course of treatment, UCBT should not be considered. HID-HSCT has the advantages of easy access and good compliance of donors and is close to full-matched transplantation. It has become an important alternative to transplantation. The use of baliximab and/or antithymocyte globulin is expected to reduce the incidence of graft versus host disease and expand the clinical application of HID-HSCT.