Combination of Thrombopoietin Mimetic and Immunosuppressive Therapy in Aplastic Anaemia

NCT03896971 | Completed Phase 4 DMC

• 1 other identifier: SKhaled19
• Study Type: interventional
• Target: 66
• Locations: 1 country
• Sites: 1

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. 1.To evaluate the efficacy, tolerability and toxicity of the combination of thrombopoietin mimetic and immunosuppressive therapy in Egyptian patients with AA.
2. 2.To study the influence of this combination on patients' quality of life.
3. 3.To access evolution to paroxysmal nocturnal hemoglobinuria (PNH), myelodysplastic syndrome , acute leukemia or development of fibrosis

Conditions

Aplastic Anemia

Keywords

thrombopoietin mimetic; Egyptian; Aplastic

Outcome Measures

Primary Outcomes (1)

• Number of patients with hematologjc response

  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 Outcomes (1)

• Number of patients with one of the following

  2-3months

Study Arms (2)

Thrombopoietin mimetic plus immunosuppressive therapy

EXPERIMENTAL

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

Immunosuppressive therapy

NO INTERVENTION

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

Interventions

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.

Also known as: Combination of Eltrombopag plus cyclosporin A

Thrombopoietin mimetic plus immunosuppressive therapy

Eligibility Criteria

• Age: 16 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

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

Sponsors & Collaborators

• Safaa AA Khaled: lead
• Assiut University: collaborator

Study Sites (1)

Assiut university hospital Internal Medicine Department Hematology and BMT Unit

Asyut, 71515, Egypt

Location

Related Publications (6)

• Scheinberg P, Cooper JN, Sloand EM, Wu CO, Calado RT, Young NS. Association of telomere length of peripheral blood leukocytes with hematopoietic relapse, malignant transformation, and survival in severe aplastic anemia. JAMA. 2010 Sep 22;304(12):1358-64. doi: 10.1001/jama.2010.1376.

  PMID: 20858879 BACKGROUND

• Frickhofen N, Kaltwasser JP, Schrezenmeier H, Raghavachar A, Vogt HG, Herrmann F, Freund M, Meusers P, Salama A, Heimpel H. Treatment of aplastic anemia with antilymphocyte globulin and methylprednisolone with or without cyclosporine. The German Aplastic Anemia Study Group. N Engl J Med. 1991 May 9;324(19):1297-304. doi: 10.1056/NEJM199105093241901.

  PMID: 2017225 BACKGROUND

• Frickhofen N, Heimpel H, Kaltwasser JP, Schrezenmeier H; German Aplastic Anemia Study Group. Antithymocyte globulin with or without cyclosporin A: 11-year follow-up of a randomized trial comparing treatments of aplastic anemia. Blood. 2003 Feb 15;101(4):1236-42. doi: 10.1182/blood-2002-04-1134. Epub 2002 Oct 10.

  PMID: 12393680 BACKGROUND

• Rosenfeld S, Follmann D, Nunez O, Young NS. Antithymocyte globulin and cyclosporine for severe aplastic anemia: association between hematologic response and long-term outcome. JAMA. 2003 Mar 5;289(9):1130-5. doi: 10.1001/jama.289.9.1130.

  PMID: 12622583 BACKGROUND

• Kuter DJ. Thrombopoietin and thrombopoietin mimetics in the treatment of thrombocytopenia. Annu Rev Med. 2009;60:193-206. doi: 10.1146/annurev.med.60.042307.181154.

  PMID: 19642221 BACKGROUND

• Desmond R, Townsley DM, Dumitriu B, Olnes MJ, Scheinberg P, Bevans M, Parikh AR, Broder K, Calvo KR, Wu CO, Young NS, Dunbar CE. Eltrombopag restores trilineage hematopoiesis in refractory severe aplastic anemia that can be sustained on discontinuation of drug. Blood. 2014 Mar 20;123(12):1818-25. doi: 10.1182/blood-2013-10-534743. Epub 2013 Dec 17.

  PMID: 24345753 BACKGROUND

MeSH Terms

Conditions

Anemia, Aplastic

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Study Officials

• Safinaz Hussien, MD

  Assiut University

  STUDY DIRECTOR

• Sawsan A Abdelaal, M.B.B.Ch

  Assiut University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Associate professor

Model Details: 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

Study Record Dates

• First Submitted: March 21, 2019
• First Posted: April 1, 2019
• Study Start: November 15, 2019
• Primary Completion: December 1, 2020
• Study Completion: June 1, 2021
• Last Updated: September 29, 2021

Record last verified: 2021-09

Data Sharing

• IPD Sharing: Will not share

Locations

EG (1)