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NCT Number: NCT03896971

Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia

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

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Key information

Conditions

Aplastic Anemia Anemia Anemia, Aplastic Bone Marrow Diseases Bone Marrow Failure Disorders Hematologic Diseases Hemic and Lymphatic Diseases

Age range

16 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Assiut university hospital Internal Medicine Department Hematology and BMT Unit

Asyut, 71515, Egypt

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Eligible for treatment with thrombopoietin mimetic.
  • Willing to participate in the study and signed informed consent.
  • Unavailability of BMT -

Exclusion criteria

  • Patient with inherited aplastic anemia
  • Underlying immune deficiency
  • Contra indication to cyclosporin A
  • Endstage hepatic or renal disease
  • Pregnancy and lactation

Treatment and study plan

Combination of thrombopoietin mimetic and cyclosporin A

Drug

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 names: Combination of Eltrombopag plus cyclosporin A

Primary outcomes

  1. Number of patients with hematologjc response

    Time frame: 2-3months

    • Platelet number ( time frame(2-3 months) : Increase plt count 20, 000 or more from baseline or independence of platelet transfusion for 2 months
    • Hemoglobin percent (time frame (2-3 months): increase Hb by 1.5 g/dl from baseline or reduction 4 units of packed red blood cell transfusions for 2- months C) Absolute neutrophilic count response (time frame (2-3 months): increase absolute neutrophil count by 500 per microliter from baseline.

    d) Bone marrow cellularity (time frame (2-3 months): increase bone marrow cellularity from baseline

Secondary outcomes

  1. Number of patients with one of the following

    Time frame: 2-3months

    • Toxicity: development of toxicity leading to cessation of treatment
    • Evolution: evolution to paroxysmal nocturnal hemoglobinuria , myelodysplastic syndrome or acute leukemia
    • Bleeding and cytopenias that met the criteria for severe aplastic anemia and require transfusions

Sponsors and collaborators

Lead sponsor

Safaa AA Khaled

Other

Collaborators

  • Assiut University

Registry information

Official study title

Treatment With Thrombopoietin Mimetic Plus Immunosuppressive Therapy in Egyptian Patients With Aplastic Anaemia

Important dates

Study start
2019
Primary completion
2020
Study completion
2021
First posted
Apr 1, 2019
Registry last updated
Sep 29, 2021

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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