About this trial
Bone marrow aplasia, also known as aplastic anemia (AA) is a potentially fatal bone marrow failure syndrome characterized by a paucity of hematopoietic stem cells (HSCs) and progenitor cells with varying degrees of cytopenia and fatty infiltration of the bone marrow space. Underlying mechanisms include immune-mediated attack, telomere defects, and inherent HSC compartment insufficiency. These events may occur individually or in concert, mostly involving effector T cells Historical treatment has included the use of high-dose chemotherapy and allogeneic stem cell transplantation as well as lymphotoxic immunosuppressive therapy (IST) Thrombopoietin (TPO) regulates platelet production, maturation, and release through binding of c-mpl on megakaryocytes.
Eligibility criteria
This trial accepts healthy volunteersQualifiers
None
Disqualifiers
None
Trial design
Single group
Treatments tested in this trial
Eltrombopag
DrugTreatment with eltrombopag in a dose of (50-150mg/d)
Treatment groups
Trial outcomes
Primary outcomes
Determine hematological response after 6 months
Change in CBC elements after 6 months of treatment
Determine overall survival rate.
Number if years estimated to survive after treatment
Secondary outcomes
Rate of relapse in patients deemed responsers at 6 months.
Clonal evolution to myeloid malignancy or new chromosomal abnormality
Change in serum iron and ferritin over time of treatment
Eltrombopag efficacy in increasing platelet count
Locations
Sponsors and contacts
Click on the lead sponsor to view all of their trials.
This trial is not recruiting at the moment. You can still explore other options: