Treatment Protocol of Child SAA With the Injection of Mesenchymal Stem Cells（Umbilical Cord Derived）

Study Overview
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Brief Summary
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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.

Detailed Description
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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. 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 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.

Official Title
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Child With Severe Aplastic Anemia (SAA) Therapy: the Injection of Umbilical Cord Derived Mesenchymal Stem Cells.

Conditions
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Complications of Organ Transplant Stem Cells Umbilical Cord

Intervention / Treatment
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* Drug: MSC+ATG


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Non-Severe Aplastic Anemia (NSAA)atients Exclusion Criteria: Severe Aplastic Anemia (SAA) patients

Ages Eligible for Study
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Minimum Age: 1 Month
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: MSC+ATG<br>The first agent MSC injection, began two weeks after ATG application; Each patient was injected three times, one time per week; Study on single dose tolerance and efficacy index; Each subjects received three dose groups of treatment. | Drug: MSC+ATG<br>* 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.<br>* Other names: Mesenchymal Stem Cells (Umbilical Cord Derived);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The 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). | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. | 3-10 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Mesenchymal Stem Cells (MSC), Umbilical Cord, thymocyte globulin (ATG), Severe Aplastic Anemia (SAA)