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

Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia


Lead sponsor: Safaa AA Khaled

Collaborator: Assiut University

Source Assiut University
Brief 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.

Overall Status Recruiting
Start Date May 15, 2019
Completion Date March 1, 2021
Primary Completion Date May 1, 2020
Phase Phase 4
Study Type Interventional
Primary Outcome
Measure Time Frame
Number of patients with hematologjc response 2-3months
Secondary Outcome
Measure Time Frame
Number of patients with one of the following 2-3months
Enrollment 60

Intervention type: Drug

Intervention name: Combination of thrombopoietin mimetic and cyclosporin A

Description: 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.

Arm group label: Thrombopoietin mimetic plus immunosuppressive therapy

Other name: Combination of Eltrombopag plus cyclosporin A



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

Gender: All

Minimum age: 16 Years

Maximum age: 65 Years

Healthy volunteers: No

Overall Official
Overall Contact

Last name: Safaa AA Khaled, Prof.

Phone: 01064170058

Phone ext: 002

Email: [email protected]

facility status contact
Assiut university hospital Internal Medicine Department Hematology and BMT Unit Recruiting Ahmed MK El Menshawy, Professor 08822080150 002 [email protected]
Location Countries


Verification Date

June 2019

Responsible Party

Responsible party type: Sponsor-Investigator

Investigator affiliation: Assiut University

Investigator full name: Safaa AA Khaled

Investigator title: Associate professor

Has Expanded Access No
Condition Browse
Number Of Arms 2
Arm Group

Arm group label: Thrombopoietin mimetic plus immunosuppressive therapy

Arm group type: Experimental

Description: The intervention group will be given cyclosporin A plus thrombopoietin (TPO) mimetic starting with 50 mg orally daily that would be increased or decreased according to response, for 3-months. Patients will be kept on weekly follow up visits and assessed by peripheral hemogram .

Arm group label: Immunosuppressive therapy

Arm group type: No Intervention

Description: A comparative group will be collected retrospectively and treated with cyclosporin A alone as standard.

Patient Data No
Study Design Info

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

Primary purpose: Treatment

Masking: None (Open Label)