Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE4

Total Enrollment

66 participants

Study Classification

INTERVENTIONAL

Study Start Date

2019-11-15

Study Completion Date

2021-06-01

Brief Summary

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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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Detailed Description

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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.

Conditions

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Aplastic Anemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

PARALLEL

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 Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Thrombopoietin mimetic plus immunosuppressive therapy

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 .

Group Type EXPERIMENTAL

Combination of thrombopoietin mimetic and cyclosporin A

Intervention Type 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.

Immunosuppressive therapy

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

Group Type NO_INTERVENTION

No interventions assigned to this group

Interventions

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Combination of thrombopoietin mimetic and cyclosporin A

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.

Intervention Type DRUG

Other Intervention Names

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Combination of Eltrombopag plus cyclosporin A

Eligibility Criteria

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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

Minimum Eligible Age

16 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No