Thrombopoietin Agonists in Patients With Idiopathic Thrombocytopenic Purpura

NCT ID: NCT06137105

Last Updated: 2023-11-18

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 100 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2023-12-01
• Study Completion Date: 2026-12-31

Brief Summary

1. Assesement of response to Thrombopoietin receptor agonists (TPO-RAs) as treatment in Idiopathic thrombocytopenia purpura patients in Assiut University hospital.

2. Explore side effects of Thrombopoietin receptor agonists in Idiopathic thrombocytopenia purpura .

3. To study effect of thrombopoietin receptor agonists and Quality life in Idiopathic thrombocytopenia purpura patients.

Detailed Description

Idiopathic thrombocytopenic purpura (ITP) is an autoimmune disorder characterized by reduced platelet counts (<100 × 109/L) and increased bleeding risk in the absence of other causes associated with thrombocytopenia().. It affects 2-4 per 100,000 individuals annually with an overall prevalence of about 10/100,000 individuals().

Manifestations of idiopathic thrombocytopenic purpura( ITP) can be localised haemorrhaging in skin or mucous membranes that are usually of little to no clinical consequence (petechiae, purpura, ecchymoses, epistaxis); more rarely, ITP can be associated with severe bleeding events such as intracranial haemorrhage (ICH). However, most ITP patients are asymptomatic in the presence of platelet counts greater than 50x109/L2.()

Idiopathic thrombocytopenia purpura is diagnosed in absence of secondary causes of immune mediated thrombocytopenia including autoimmune diseases (systemic lupus erthematosus , antiphospholipid antibody syndrome) lymphoproliferative disorders (chronic lymphocytic leukemia ) viral infection (hepatitis c virus , human immunodeficiency virus

The goal of treatment in patients with of idiopathic thrombocytopenic purpura is to maintain the platelet count at a level that reduces the risk of bleeding with minimal treatment-related toxic effects. Observation alone is recommended when no or mild bleeding is present and the platelet count is more than 30,000 per cubic millimeter in adults.23 Treatment in patients with a lower platelet count is indicated only if bleeding occurs or if the platelet count is very low. (For example, most experts would treat a non bleeding patient who had a platelet count of <10,000 per cubic millimeter().

Historically , the treatment of idiopathic thrombocytopenia purpura has been directed at inhibiting the production of anti platelet autoantibodies or opsonization of antibody coated platelets. However, impaired platelet production is increasingly recongnized as a contributor to thrombocytopenia in patients with idiopathic thrombocytopenia purpura.

New treatment guidelines have supported a shift from corticosteroids and splenectomy to newer medical treatments that mitigate the thrombocytopenia and avoid splenectomy. The thrombopoietin receptor agonists (TPO-RA), romiplostim, eltrombopag, and avatrombopag, have markedly altered the treatment of ITP()

Thrombopoietin (TPO) is the main cytokine that stimulates thrombopoiesis, and although platelet counts are low in ITP patients, no compensatory increase in TPO production occurs in these patients().

Thrombopoietin receptor agonists (TPO-RAs) are TPO mimetics that can bind to and activate TPO receptors, leading to megakaryocyte maturation, proliferation and differentiation and resulting in increased platelet production. Two major TPO-RAs, romiplostim and eltrombopag, have been investigated in several randomized controlled trials (RCTs) involving adult and pediatric ITP patients the results of which are encouraging. Currently, romiplostim and eltrombopag are recommended as second-line therapeutic options for adult ITP patients().

Conditions

ITP - Immune Thrombocytopenia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Study Groups

Group 1

Group I : Romiplostim and Eltrombopag

eltrombopag and romiplastim

Intervention Type: DRUG

Group I: eltrombopag and romiplastim

Group II : recived corticosteroid.

Group 2

Group II : recived corticosteroid.

eltrombopag and romiplastim

Intervention Type: DRUG

Group I: eltrombopag and romiplastim

Group II : recived corticosteroid.

Interventions

eltrombopag and romiplastim

Group I: eltrombopag and romiplastim

Group II : recived corticosteroid.

Intervention Type: DRUG

Other Intervention Names

Corticosteroids

Eligibility Criteria

Inclusion Criteria

• Patients presented with Idiopathic thrombocytopenic purpura (ITP)

1. patients above age of 18 years.

Exclusion Criteria

• patients below age of 18 years .

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Association for Training, Education, and Research in Hematology, Immunology, and Transplantation

OTHER

Sponsor Role: collaborator

Assiut University

OTHER

Sponsor Role: lead

Responsible Party

Naira Ibrahem Ahmed Saad

A Study of Treatment with thrombopoietin agonists in patients with Idiopathic thrombocytopenic purpura

Responsibility Role: PRINCIPAL_INVESTIGATOR

Other Identifiers

TPO in ITP

Identifier Type: -

Identifier Source: org_study_id