Mesenchymal Stem Cells (MSC) is a non Hematopoietic Stem Cells (HSC) in adult bone marrow and takes part in the bone marrow microenvironment. The response rate of early treatment on Children's SAA application combined with anti-thymocyte globulin (ATG) (40-50 days after ATG treatment) is associated with long-term effect. The injection of Umbilical Cord Derived Mesenchymal Stem Cells combined with ATG improves the efficacy of children with SAA.
1. Mesenchymal Stem Cells were prepared by in vitro separation, screening, and culture from healthy human umbilical cord tissue; The " injection of mesenchymal stem cells (umbilical) manufacturing and verification regulation " was also formulated. 2. The starting dose of Umbilical Cord Derived MSC was 0.5-1.0 \* 106 cells /kg, based on the previous human studies; And the maximum tolerated dose was 1 \* 107 cells /kg 3. The response and complete remission rate, relapse rate of the injection of Umbilical Cord Derived Mesenchymal Stem Cells (or combined with ATG ) for Child with SAA were determined.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
MSC+ATG:Starting dose was 0.5-1.0 × 106 cells /kg; And the maximum tolerated dose was 1 ×107 cells /kg after ATG application.
Institute of Hematology and Blood Diseases Hospital Chinese Academy of Medical Sciences and Peking Union Medical College
Tianjin, China
RECRUITINGThe response and complete remission rate with different doses of Umbilical Cord Derived MSC to treat child with SAA
Complete response (CR) was defined as achieving normal levels of hemoglobin adjusted for age, platelet count \>100\*109/L, and absolute neutrophil count (ANC) \>1.5\*1.0 9/L. Partial response (PR) was defined as injection independence, reticulocyte count\>30\*109/L, platelet count \>20\*109/L, and ANC\>0.5\*1.0 9/L above the baseline. Persistence of injection requirement or death was evidence of no response (NR).
Time frame: 1 year
The relapse rate with different doses of MSC to treat child with SAA
The relapse was defined as injection dependence again; or progressed or paroxysmal nocturnal hemoglobinuria (PNH)/acute myeloid leukemia/myelodysplasia syndrome(MDS); or cyclosporin A (CsA) dependence.
Time frame: 3-10 year
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