Immune Markers in Pediatric ITP on Second Line Therapy

November 10, 2023 updated by: Gehad Mahmoud Abdelsalam, Assiut University

Marker Expression in Pediatric Patients With Immune Thrombocytopenic Purpura Receiving Second Line Therapy

Immune thrombocytopenia (ITP) is a common autoimmune disease characterized by low platelet count and increased risk of bleeding. It affects approximately 50 to 100 cases per million people per year, with children accounting for half of the cases.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

Antiplatelet factors in the plasma of ITP patients, specifically IgG, have been attributed to platelet destruction through phagocytosis or complement-mediated lysis. However, these antibodies are only present in 60-70% of ITP patients, suggesting that other mechanisms may be involved in platelet destruction.

B lymphocytes play a critical role in immune responses through antibody production, antigen presentation to T cells, and cytokine secretion. CD4+ T helper cells play a crucial role in supporting B cell development into antibody-secreting plasma cells. Furthermore, evidence of auto reactive CD4+ T cells targeting platelet epitopes has been reported.

It was found that there is clonal expansion of a particular subset of CD8+ T cells, known as terminally differentiated effector memory T cells (TEMRA cells), in refractory ITP. Furthermore, CD8+ T cells induce platelet activation and apoptosis in an antibody-independent mechanism for refractory thrombocytopenia that may be amenable to therapeutic targeting. IFN-γ is an important cytokine involved in host defence and immune regulation. It is primarily produced by T helper, cytotoxic T, and natural killer cells. Dysregulated secretion of IFN-γ has been implicated in the development of autoimmune disorders. Initial studies on ITP focused on the role of autoantibodies. Therefore, drug discovery efforts have focused on suppressing aberrant humoral immunity through B cell depletion, disruption of immunoreceptor, and inhibition of autoantibody activity. By comparing the marker expression in different treatment response groups, the investigator can potentially identify markers that may serve as predictive or prognostic indicators of treatment response. This information could be valuable for guiding treatment decisions and optimizing patient outcomes in pediatric ITP.

Study Type

Observational

Enrollment (Estimated)

42

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

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

Accepts Healthy Volunteers

No

Sampling Method

Probability Sample

Study Population

Pediatric patients with ITP who are receiving second-line therapy who are attending the outpatient pediatric hematology clinic at Assiut University Children Hospital or the clinic at Central Insurance Agency of Assiut.

Description

Inclusion Criteria:

  • Pediatric patients diagnosed with immune thrombocytopenia who are undergoing second line therapy with either eltrombopag or romiplostim.

Exclusion Criteria:

  • Include acute immune thrombocytopenic children receiving first line therapy and those with secondary immune thrombocytopenia.

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

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Respondents to second-line therapy
Immune markers in ITPpatients Respondant to second line therapy
Diagnostic test: complete blood count, CD3+ , CD4+ , CD8+, CD16+, CD56+, IFN-γ.
  1. Full history
  2. Thorough clinical examinations

  3. Laboratory investigations will include:

    1. complete blood count with focus on platelet count, platelet distribution width and mean platelet volume. Platelet count will be confirmed by direct blood film and blood smear.
    2. Measurements of CD3+, CD4+, CD8+ and natural killer cells (CD16+, CD56+) will be conducted using flow cytometry.
    3. Serum IFN-γ levels will be determined using an ELISA kit.
  4. Response to the treatment will be assessed according to The International Working Group criteria which defines Response as platelet count ≥ 30 x 10⁹/L and >2-fold increase in platelet count from baseline and absence of bleeding, measured on 2 occasions greater than 7 days apart. No response is characterized by a platelet count <30 x 10⁹/L or a less than 2-fold increase in platelet count from baseline, or the presence of bleeding.
Non-Respondents to second-line therapy
Immune markers in ITPpatients nonrespondant to second line therapy
Diagnostic test: complete blood count, CD3+ , CD4+ , CD8+, CD16+, CD56+, IFN-γ.
  1. Full history
  2. Thorough clinical examinations

  3. Laboratory investigations will include:

    1. complete blood count with focus on platelet count, platelet distribution width and mean platelet volume. Platelet count will be confirmed by direct blood film and blood smear.
    2. Measurements of CD3+, CD4+, CD8+ and natural killer cells (CD16+, CD56+) will be conducted using flow cytometry.
    3. Serum IFN-γ levels will be determined using an ELISA kit.
  4. Response to the treatment will be assessed according to The International Working Group criteria which defines Response as platelet count ≥ 30 x 10⁹/L and >2-fold increase in platelet count from baseline and absence of bleeding, measured on 2 occasions greater than 7 days apart. No response is characterized by a platelet count <30 x 10⁹/L or a less than 2-fold increase in platelet count from baseline, or the presence of bleeding.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Correlation analysis
Time Frame: One year
Analysis of the correlation between specific markers expression ( CD3,CD4,CD8,Interferon gamma and natural killer cells) and treatment response in pediatric patients with immune thrombocytopenia who are receiving second line therapy.
One year

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assessment of treatment response
Time Frame: One year
Assessment of response to second line therapy (eltrombopag, romiplostim) in pediatric patients with immune thrombocytopenic purpura using clinical and laboratory findings.
One year

Collaborators and Investigators

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

Sponsor

Assiut University

Investigators

  • Study Director: Khaled I El-sayeh, M.D., Assiut University
  • Study Director: Mostafa M Embaby, M.D., Assiut University
  • Study Director: Azhar A Mohammed, M.D., 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 (Estimated)

January 1, 2024

Primary Completion (Estimated)

December 31, 2024

Study Completion (Estimated)

February 1, 2025

Study Registration Dates

First Submitted

October 17, 2023

First Submitted That Met QC Criteria

October 17, 2023

First Posted (Actual)

October 23, 2023

Study Record Updates

Last Update Posted (Actual)

November 14, 2023

Last Update Submitted That Met QC Criteria

November 10, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ITPIMMUNEMARKERS

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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