Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia
Condition: Aplastic Anemia
MeSH terms: Anemia; Anemia, Aplastic
Study type: Interventional
Phase: Phase 4

Summary: 
      To the investigator's Knowledge this is the first study that will assess Treatment with
      thrombopoietin Mimetic plus immunosuppressiveTherapy in Egyptian Patients with Aplastic
      Anaemia.

      Aim of the work :

        1. To evaluate the efficacy, tolerability and toxicity of the combination of thrombopoietin
           mimetic and immunosuppressive therapy in Egyptian patients with AA.

        2. To study the influence of this combination on patients' quality of life.

        3. To access evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic
           syndrome , acute leukemia or development of fibrosis
    

Detailed description: 
      Aplastic anemia (AA) is a bone marrow failure syndrome that, although benign in nature, it
      influences patients' quality of life and carries poor prognosis.The pathophysiological basis
      of development of acquired AA include immune-mediated attack, inherent hematopoietic stem
      cell insufficiency and telomere defects. Bone marrow transplantation (BMT) is the only
      curative treatment for AA. Unfortunately it is unavailable for many patients due to lack of
      matched donors furthermore others are ineligible for BMT due to old age or co-morbid
      conditions. Immunosuppressive therapy was the mainstay of treatment for AA for many years,
      however many patients developed resistance or refractoriness. Immunosuppressive therapy was
      in the form of antithymocyte globulin (ATG) which gave hematologic response in nearly 50% of
      patients , adding cyclosporin A increases this response to 70%. Why some patients became
      resistant to immunosuppressive therapy ? The answer is not known.

      Thrombopoietin mimetic (Eltrombopag) was firstly FDA approved for treatment of immune
      thrombocytopenic purpura. Numerous clinical trials proved the efficacy of anthropometric
      mimetic in patients with refractory severe AA, leading to its FDA approval for this group of
      patients. Some researchers proven the efficacy of thrombopoietin mimetic in patients with
      moderate aplastic anemia.

      This study aimed to asses the combination of thrombopoietin mimetic and immunosuppressive
      therapy in patients with AA.
    

Intervention name(s): Combination of thrombopoietin mimetic and cyclosporin A
Intervention type(s): Drug
Intervention description(s): This is a controlled interventional study that will be carried out in the Clinical Hematology Unit, at the Department of Internal Medicine , Assiut University Hospital. Patients will be recruited among those who attending the outpatient clinic or admitted in the unit.
Other intervention name(s): Combination of Eltrombopag plus cyclosporin A
Intervention MeSH terms: Cyclosporine; Cyclosporins
Allocation: Non-Randomized
Intervention model: Parallel Assignment
Intervention model description: Two groups of patients will be included one group will be treated with thrombopoietin mimetic and immunosuppressive therapy , another matched group treated with immunosuppressive therapy alone and enrolled retrospectively
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: thrombopoietin mimetic; Egyptian; Aplastic