Comparison of efficacy and health-related quality of life of first-line haploidentical hematopoietic stem cell transplantation with unrelated cord blood infusion and first-line immunosuppressive therapy for acquired severe aplastic anemia

A study of human leukocyte antigen-haploidentical hematopoietic stem cells transplantation combined with allogenic mesenchymal stem cell infusion for treatment of severe aplastic anemia in pediatric and adolescent patients

“…In the current study, we report a clinical study of cotransplantation of allogenic MSC and HLA‐haploidentical HSC for juvenile SAA. HSCT from a matched related donor (MRD) has been taken as one of the first‐line treatments for newly diagnosed young patients with SAA, which reduced the heavy blood transfusion and related rejection, avoided the complications caused by IST, and decreased the risk of hematopoietic malignancies resulting from SAA 9,45,46 . The data from the independent transplant units have demonstrated that halploidentical HSCT also exhibited promising outcome for juvenile patients in SAA.…”

“…The previous studies showed that only 25% remain long-term good-quality responders, and 10% to 15% even show clonal evolution to myelodysplastic syndrome or acute myeloid leukemia. [9][10][11][12] HLA-matched hematopoietic stem cell transplantation (HSCT) has become the first-line treatment option for SAA treatment. By removing the clonal autoimmune T cells and restoring normal hematopoiesis, HSCT is a curative therapy for SAA patients.…”

“…However, most patients could not be cured by IST. The previous studies showed that only 25% remain long‐term good‐quality responders, and 10% to 15% even show clonal evolution to myelodysplastic syndrome or acute myeloid leukemia 9‐12 …”

“…avoided the complications caused by IST, and decreased the risk of hematopoietic malignancies resulting from SAA 9,45,46. The data fromthe independent transplant units have demonstrated that halploidentical HSCT also exhibited promising outcome for juvenile patients in SAA.…”

Biological Characteristics of Umbilical Cord Mesenchymal Stem Cells and Its Therapeutic Potential for Hematological Disorders

“…IST with a combination of antithymocyte globulin (ATG) and cyclosporin A (CsA) is the preferred first-line treatment for patients without an MRD and older patients ( Young, 2018 ). However, about 30–40% of the patients will eventually relapse or become refractory to IST; those unresponsive to initial IST will be considered for transplantation using an alternative donor ( Liu et al, 2020a ). However, transplantation in patients with acquired SAA often fail to recover, and graft failure and rejection remain a common fatal risk for patients.…”