Umbilical Cord Derived Mesenchymal Stem Cells Therapy in Aplastic Anemia

Study Overview
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Brief Summary
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This study is to evaluate the safety and efficacy of Umbilical Cord Derived Mesenchymal Stem Cells transplantation in aplastic anemia.

Detailed Description
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Aplastic anemia (AA) is a disorder thought to be caused by an immune-mediated bone marrow failure. Not all people with AA are eligible for today's standard treatments. One new treatment approach uses umbilical cord derived mesenchymal stem cells-specialized cells capable of developing into other types of cells-to provide the basis for clinical application. The aim of the present study is to investigate the safety and efficacy of vein infusion of allogeneic mesenchymal stem cells in patients with AA .

Official Title
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Evaluation of Safety and Efficacy in Patients With Aplastic Anemia by Transplantation of Umbilical Cord Derived Mesenchymal Stem Cells

Conditions
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Aplastic Anemia

Intervention / Treatment
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* Biological: mesenchymal stem cells


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of AA according to established criteria in 2010 Age from 14 to 60 years Suffering from AA within six months No serious infection or acute hemorrhage. Left ventricular ejection fraction (LVEF) ≥ 50% No acute infectious diseases. Understanding and willingness to sign a written informed consent document. Eastern Cooperative Oncology Group(ECOG) score of 0-2pionts. Exclusion Criteria: - Patients with AA have to be disqualified from this study if any of the following is applicable. Severe aplastic anemia(SAA) with severe infection. Severe aplastic anemia(SAA) with active hemorrhage. Severe heart attack, liver and kidney disease following serious complications Patients with allergic constitution. Pregnancy and lactation. Accompanied by malignant tumors and other clonal disease. Patients with active tuberculosis, acute sever hepatitis or infectious period of diseases.

Ages Eligible for Study
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Minimum Age: 14 Years
Maximum Age: 60 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

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: mesenchymal stem cells<br>According to the inclusion and exclusion criteria, selected patients were divided into a cell therapy group and a control group. Umbilical cord derived mesenchymal stem cells at a dose of 100-300 million by intravenous infusion. | Biological: mesenchymal stem cells<br>* Patients in the cell treated group were given umbilical cord derived mesenchymal stem cells by intravenous infusion besides conventional therapy. There was only conventional treatment in the control group. All patients after treatment for 1months, 3months, 6months and 9months were evaluated respectively the therapeutic effect.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemoglobin levels in peripheral blood | The recovery of bone marrow erythrocyte is evaluated by hemoglobin levels (g/L) in peripheral blood. | Post cell transplantation: 9months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The number of red blood cell in peripheral blood | The improvement of bone marrow erythrocyte is evaluated by red blood cell count (×10^12/L) in peripheral blood. | Post cell transplantation: 1, 3, 6, 9months | 
| The number of granulocyte in peripheral blood | The recovery of bone marrow granulocyte is evaluated by granulocyte count (×10^9/L) in peripheral blood. | Post cell transplantation: 1, 3, 6, 9months | 
| The number of white blood cell in peripheral blood | The improvement of bone marrow granulocyte is evaluated by white blood cell count (×10^9/L) in peripheral blood. | Post cell transplantation: 1, 3, 6, 9months | 
| The evaluation of bone marrow megakaryocytic series | The status of megakaryocytic series is evaluated by platelet count (×10^9/L) in peripheral blood. | Post cell transplantation: 1, 3, 6, 9months | 
| adverse reaction | Adverse reaction includes temperature changes(℃), the change of blood pressure(mmHg) and allergic reaction. | Post cell transplantation: 1, 3, 6, 9months |