Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia

September 28, 2021 updated by: Safaa AA Khaled

Treatment With Thrombopoietin Mimetic Plus Immunosuppressive Therapy in Egyptian Patients With 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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

66

Phase

  • Phase 4

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Egypt
      • Assiut, Egypt, 71515
        • Assiut university hospital Internal Medicine Department Hematology and BMT Unit

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

16 years to 65 years (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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 .
Drug: 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.
Other Names:
  • Combination of Eltrombopag plus cyclosporin A
No Intervention: Immunosuppressive therapy
A comparative group will be collected retrospectively and treated with cyclosporin A alone as standard.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients with hematologjc response
Time Frame: 2-3months
  1. Platelet number ( time frame(2-3 months) : Increase plt count 20, 000 or more from baseline or independence of platelet transfusion for 2 months
  2. 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
2-3months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of patients with one of the following
Time Frame: 2-3months
  1. Toxicity: development of toxicity leading to cessation of treatment
  2. Evolution: evolution to paroxysmal nocturnal hemoglobinuria , myelodysplastic syndrome or acute leukemia
  3. Bleeding and cytopenias that met the criteria for severe aplastic anemia and require transfusions
2-3months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Safaa AA Khaled

Collaborators

Assiut University

Investigators

  • Study Director: Safinaz Hussien, MD, Assiut University
  • Principal Investigator: Sawsan A Abdelaal, M.B.B.Ch, Assiut University

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

November 15, 2019

Primary Completion (Actual)

December 1, 2020

Study Completion (Actual)

June 1, 2021

Study Registration Dates

First Submitted

March 21, 2019

First Submitted That Met QC Criteria

March 29, 2019

First Posted (Actual)

April 1, 2019

Study Record Updates

Last Update Posted (Actual)

September 29, 2021

Last Update Submitted That Met QC Criteria

September 28, 2021

Last Verified

September 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SKhaled19

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

No

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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