Clinical Trial: Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

Official Title: Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia

Brief Summary: Mesenchymal stem cells have been tested in many autoimmune disorders with encouraging results and may be an alternative to the treatment of immune-mediated severe acquired aplastic anemia.

Detailed Summary:

Acquired aplastic anemia is a bone marrow failure syndrome characterized by and empty bone marrow and low blood counts. Most of the cases are immune-mediated. Allogeneic bone marrow transplant is the preferable treatment modality for patients younger than 40 years with a matched sibling donor. Patients not eligible for transplant are treated with intensive immunosuppressive therapy often based on anti-thymocyte globulin and cyclosporine.

However, up to one third of patients treated with immunosuppression are refractory and one third of those who response eventually relapse. Refractory and relapsed cases may be the result of insufficient immunosuppression and these cases may benefit from additional immunosuppression. Mesenchymal stem cells infusion may be a potential treatment option, considering its properties to modulate the immune system.

Refractory or relapsed patients with aplastic anemia will be treated with conventional immunosuppressive regimen (anti-thymocyte globulin plus cyclosporine) combined with intravenous infusion of allogeneic unrelated bone marrow mesenchymal stem cells.


Sponsor: University of Sao Paulo

Current Primary Outcome: Number of Participants with Adverse Events as a Measure of Safety and Tolerability of intravenous allogeneic unrelated mesenchymal stem cells infusion in patients with severe acquired aplastic anemia. [ Time Frame: After the mesenchymal stem cells infusion up to 6 mounth after ]

Adverse events like allergic reactions,infectious diseases,organ dysfunction or other related to mesenchymal stem cells infusion will be assessed.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Level of cytopenias [ Time Frame: weekly until 6 months ]
  • Transfusional requirements [ Time Frame: weekly until 6 months ]
    Units of blood or platelets transfused after the mesenchymal stem cells infusion will be measured and compared to previously.
  • Incidence of infections and febrile neutropenia [ Time Frame: weekly until 6 months ]


Original Secondary Outcome: Same as current

Information By: University of Sao Paulo

Dates:
Date Received: February 16, 2011
Date Started: February 2011
Date Completion:
Last Updated: February 2, 2014
Last Verified: February 2014