The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids

August 21, 2024 updated by: Shimaa Abdelhameed Mohamed, Sohag University

Immune thrombocytopenic purpura (ITP) is an autoimmune disease characterized by a low platelet count, purpura, and hemorrhagic episodes caused by antiplatelet autoantibodies. The diagnosis is typically made by excluding the known causes of thrombocytopenia. IgG autoantibodies sensitize the circulating platelets. It leads to the accelerated removal of these cells by antigen-presenting cells (macrophages) of the spleen and sometimes the liver or other components of the monocyte-macrophage system. The bone marrow compensates for platelet destruction by increasing platelet production. ITP most often occurs in healthy children and young adults within a few weeks following a viral infection .

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 have markedly altered the treatment of ITP.

The thrombopoietin receptor agonists (TPO-Ras) romiplostim and eltrombopag have shown high therapeutic activity In primary ITP.

Romiplostim, a thrombopoiesis-stimulating peptibody, represents a new therapeutic option in adult refractory chronic immune thrombocytopenia (ITP). This study aimed to assess the short-term efficacy and safety of romiplostim in children withChronic ITP.

The most commonly reported drug-related adverse effects include headache, nausea, and hepatobiliary laboratory abnormalities. Long-term safety data in children are limited, and studies in adults have not revealed a clinically significant increased incidence of thrombosis, marrow fibrosis, or cataract formation.

TPO-RA do not need to be continued forever; about a third of patients In the first year and about another third after two years have a remission. Whether TPO-RA affect the ITP pathophysiology and directly cause remission remains unclear. This review provides a personal overview of the diagnosis and treatment of ITP with a focus on the mechanism of action of TPO-RA, their place in the treatment algorithm, unique aspects of their clinical use, adverse effects.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

100

Phase

  • Not Applicable

Contacts and Locations

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

Study Contact

Study Contact Backup

  • Name: ALZahraa Elsayed Ahmed, Professor
  • Phone Number: 01224340998

Study Locations

  • Egypt
      • Sohag, Egypt
        • Recruiting
        • Sohag University hospitals
        • Contact:
          • Magdy M Ameen, Professor

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

  • Child
  • Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

pediatric patients with persistent or chronic Itp on thrombopoietin receptor agonists unresponsive to steroid.

Exclusion Criteria:

Any patient with other causes of thrombocytopenia. Any patient with chronic disease . Any patient with Acute ITP . Any newly diagnosed patient with ITP.

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: Health Services Research
  • Allocation: N/A
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Persistent or chronic ITP not responsive to steroids
The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
Diagnostic test: CBC
Platelets counts in pediatric patient with perisistent or chronicITP unresponsive to steroids

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Platelets counts in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids on thrombopoietin receptor agonists
Time Frame: 12 months
The Outcome of the Thrombopoietin Receptor Agonists in Pediatric Patient With Persistent or Chronic ITP Unresonsive to Steroids
12 months

Collaborators and Investigators

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

Sponsor

Sohag University

Collaborators

sohag university hospital

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)

July 14, 2024

Primary Completion (Estimated)

July 14, 2025

Study Completion (Estimated)

July 14, 2025

Study Registration Dates

First Submitted

August 21, 2024

First Submitted That Met QC Criteria

August 21, 2024

First Posted (Actual)

August 23, 2024

Study Record Updates

Last Update Posted (Actual)

August 23, 2024

Last Update Submitted That Met QC Criteria

August 21, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • Soh-Med-24-07-12MS

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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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