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:
Participant gender:
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
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