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

University of Sao Paulo9 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Aplastic Anemia

Interventions

Intravenous bone marrow mesenchymal stem cells infusionBIOLOGICAL

After standard immunosuppressive therapy with rabbit antithymocyte globulin 3,5 mg/Kg/day during 5 days, allogeneic unrelated bone marrow derived mesenchymal stem cells will be infused intravenously. Oral cyclosporine 5 mg/Kg/day (with dose correction weekly to keep serum cyclosporine level between 150-250 mg/dl) up to 6 months will be added.

Eligibility

Sex: ALLMin age: 18 YearsMax age: 65 Years

Medical Language ↔ Plain English

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

Locations (1)

Hospital das Clínicas da Faculdade de Medicina de Ribeirão Preto da Universidade de São Paulo

Ribeirão Preto, São Paulo, Brazil

Outcomes

Primary Outcomes

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.

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

Time frame: After the mesenchymal stem cells infusion up to 6 mounth after

Secondary Outcomes

Level of cytopenias

Time frame: weekly until 6 months

Transfusional requirements

Units of blood or platelets transfused after the mesenchymal stem cells infusion will be measured and compared to previously.

Time frame: weekly until 6 months

Incidence of infections and febrile neutropenia

Time frame: weekly until 6 months

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.