Mesenchymal Stem Cells in the Treatment of Relapsed/Refractory Severe Acquired Aplastic Anemia
Bone Marrow Mesenchymal Stem Cells in the Treatment of Refractory Severe Acquired Aplastic Anemia
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.
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.
Phase 1/Phase 2
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.
Level of cytopenias
Intravenous bone marrow mesenchymal stem cells infusion
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Primary Completion Date
Inclusion Criteria: - Diagnosis of Acquired Aplastic Anemia - Relapse/refractory to at least 1 immunosuppressive first line treatment - Not eligible to allogeneic bone marrow transplantation Exclusion Criteria: - Previous or current malignancy - Active or latent infectious disease - Positive serologic tests for HIV, HCV, HBV, HTLV-1 and 2, Syphilis or Chagas disease - Previous drug reaction for antithymocyte globulin, cyclosporin or corticosteroids - Severe organic impairment (renal, hepatic, cardiac, pulmonary) - Uncontrolled hypertension or diabetes - Pregnancy - Previous history of allergic reaction to penicillin or streptomycin - Severe psychiatric disorder
18 Years - 65 Years
Accepts Healthy Volunteers
Diego V Clé, MD, ,
University of Sao Paulo
Diego V Clé, MD, Principal Investigator, University of São Paulo