Safety and Efficacy of Patient's Own AD-MSC and AD-HSC Transplantation in Patients With Severe Aplastic Anemia

• Status: Unknown status
• Trial end date: 2017-07-01
• Target enrollment: 0
• Participant gender: All

Summary

RATIONALE: It has been shown that about 30% of patients do not respond to immunosuppressive therapy or experience recurrence, and graft rejection and graft-versus-host-disease (GVHD) decrease event-free survival to 30% to 50% in the alternative donor (matched unrelated, partially matched family member) transplantation. Although an overall and disease free survival of 85% to 100%, can be obtained in allogeneic blood or bone marrow stem cell transplantation using an human leukocyte antigen (HLA) matched sibling donor, only about 25% of patients have such a donor.

PURPOSE: In an attempt to avoid GVHD, reduce earlier infection rate and decrease regimen-related toxicity while maintaining better engraftment, this study is to evaluate the effectiveness and safety of patient's own adipose-derived mesenchymal stem cell (AD-MSC) or AD-MSC transdifferentiated HSC (AD-HSC) transplant after an immunosuppressive regimen in treating patients who have severe aplastic anemia.

The patient will be in the study for one year for observation and active monitoring. After treatment and active monitoring are over, the patient's medical condition will be followed indefinitely. The principle measures of safety and efficacy will be :

1. Patient survival probability at 3 months, 6 months and 1 year.

2. Engraftment at 3 months, 6 months and 1 year

3. Incidence of graft versus host disease (GVHD), incidence of acute and chronic GVHD and Incidence of earlier infection rate as well as other complications within 6 months and 1 years.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Navy General Hospital, Beijing
• Collaborators: Chinese Academy of Medical Sciences; General Hospital of Beijing PLA Military Region; Peking Union Medical College Hospital
• Treatments: Antilymphocyte Serum

Criteria

Inclusion Criteria:

Male or female recipients must have histopathologically confirmed diagnosis of SAA-I without or with more than 6 months after less than one treatment with ATG. Diagnostic Criteria for Server Aplastic Anemia will be based on the definitions set forth by the international Aplastic Anemia Study Group.

At least two of the following:

Absolute neutrophil count ≤ 0.5 X 109/l, Platelet count ≤ 20 X 109 /l, Anemia with corrected reticulocyte count ≤ 1%, and Bone marrow cellularity ≤ 25%, or bone marrow cellularity ≤ 50% with fewer than 30% hematopoietic cell, Hepatic: alanine aminotransferase (ALT)/ aspartate aminotransferase (AST) no greater than 4 times normal, Bilirubin: no greater than 2 mg/dl, Renal: Creatinine clearance at least 50 ml/min, Cardiovascular: Shortening fraction or ejection fraction at least 40% of normal for age by echocardiogram or radionuclide scan.

No clinically significant comorbid illnesses (e.g., myocardial infarction or cerebrovascular accident).

Exclusion Criteria:

Active and uncontrolled infection, Active bleeding, Severe allergic history of ATG, HIV-1 infection, Pregnancy or breastfeeding, Carbon monoxide lung diffusion capacity (DLCO) <40% predicted, SAA-II, Patients with severe psychological disorders, Recipients of other clinical trials.