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

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional




Official Title: A Multicenter, Randomized, Controlled Study of the Efficacy and Safety of the Combination of Adipose Tissue-derived Hematopoietic Stem Cells (AD-HSCs) and ATG in the Treatment of Severe 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.