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Association of Platelet Parameters With Bleeding Severity in Children With ITP (ITP-APPS)

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ClinicalTrials.gov Identifier: NCT03810352
Recruitment Status : Recruiting
First Posted : January 18, 2019
Last Update Posted : March 20, 2019
Sponsor:
Collaborators:
Sysmex America, Inc.
Children's Hospital Colorado
Children's Hospital of Philadelphia
Texas Children's Hospital
Columbia University
Duke University
Information provided by (Responsible Party):
Andrew Frelinger, Boston Children’s Hospital

Brief Summary:
Patients with severe immune thrombocytopenia (ITP) present with similarly low platelet counts but varying bleeding symptoms, making it difficult to predict the disease course and to decide on an appropriate treatment plan. In a single-center study, platelet parameters including the immature platelet fraction, the absolute immature platelet count , and functional response markers were found to be significantly associated with patient bleeding severity, independent of platelet count. This study aims to confirm and replicate these findings in a multi-center patient population and to investigate the use of these parameters to better predict disease severity and bleeding events.

Condition or disease
Immune Thrombocytopenia

Detailed Description:

Many children with severe immune thrombocytopenia (ITP) present with mild symptoms and their disease spontaneously resolves within 3 to 6 months. However, a subset of pediatric ITP patients experience severe bleeds and their symptoms persist for more than 6 to 12 months. Both patient populations present with similarly low platelet counts, making it difficult to predict the disease course and to decide on a treatment plan. The current American Society of Hematology treatment guidelines advise that most cases of ITP may be managed through close observation, while pharmacological interventions that may result in treatment-related toxicities may be used in patients with more severe bleeding symptoms. In order to improve the care and management of pediatric patients with ITP, it is necessary to develop a better predictor of bleeding events and disease severity than the patient's platelet count.

In a previous single-center study, investigators studied the association of different platelet parameters with patient bleeding severity. Using whole blood from patients diagnosed with severe ITP, investigators measured the immature platelet fraction (IPF) and absolute immature platelet count (IPC) through a hematology analyzer (Sysmex XN-1000). Investigators performed functional tests on the platelets and analyzed them through flow cytometry. In this study, the investigators found that the IPF and IPC is associated with patient bleeding severity, independent of platelet count. It was also determined that functional activation markers such as P-selectin and glycoprotein (GP) IIb-IIIa are significantly associated with subsequent bleeding severity in children, independent of platelet count. The results of these proposed studies in ITP patients may suggest clinically relevant uses of these assays.

To confirm these findings, this trial will repeat the previous study in a multi-center patient population, including a greater number of patients with severe bleeding and low platelet counts.


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Study Type : Observational
Estimated Enrollment : 150 participants
Observational Model: Cohort
Time Perspective: Cross-Sectional
Official Title: Association of Platelet Parameters Identified by the Sysmex XN-1000 and Flow Cytometry With Concurrent and Subsequent Bleeding Severity in Children With Immune Thrombocytopenia (ITP): A Multicenter Study
Actual Study Start Date : March 15, 2019
Estimated Primary Completion Date : February 1, 2021
Estimated Study Completion Date : August 1, 2021


Group/Cohort
Patients with ITP
Patients with primary or secondary immune thrombocytopenia



Primary Outcome Measures :
  1. Association of IPF with concurrent, subsequent and worst-ever bleeding in children with ITP. [ Time Frame: February 2019-August 2022 ]
    Evaluate in a multi-center study the association, independent of platelet count, of IPF measured by the Sysmex XN-1000 with concurrent, subsequent, and worst-ever bleeding in children with ITP.

  2. Association of IPC with concurrent, subsequent, and worst-ever bleeding in children with ITP [ Time Frame: February 2019-August 2022 ]
    Evaluate in a multi-center study the association, independent of platelet count, of immature platelet parameters measured by the Sysmex XN-1000 (IPC, Plt-F, and FSC and other research parameters as applicable) with concurrent, subsequent, and worst-ever bleeding in children with ITP.

  3. Association of platelet function markers with concurrent, subsequent, and worst-ever bleeding in children with ITP. [ Time Frame: February 2019-August 2022 ]
    Evaluate in a multi-center study the association, independent of platelet count, off circulating andd agonist-stimulated platelet surface P-selectin and activated GPIIb-IIIa with concurrent, subsequent, and worst-ever bleeding in children with ITP.


Biospecimen Retention:   Samples Without DNA
The investigators are using blood samples to measure platelet parameters and biomarkers for platelet function.


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Ages Eligible for Study:   6 Months to 20 Years   (Child, Adult)
Sexes Eligible for Study:   All
Sampling Method:   Non-Probability Sample
Study Population
Pediatric patients diagnosed with primary or secondary immune thrombocytopenia.
Criteria

Inclusion Criteria:

  • Diagnosed with primary or secondary immune thrombocytopenia.
  • Platelet count of < 50 x 10^9/L

Exclusion Criteria:

  • May not have received aspirin 10 days prior to study entry.
  • May not have received nonsteroidal anti-inflammatory drugs (NSAIDs) 3 days prior to study entry.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03810352


Contacts
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Contact: Andrew L. Frelinger, PhD 617-919-2537 andrew.frelinger@childrens.harvard.edu
Contact: Latoya Lashley 617-355-7407 latoya.lashley@childrens.harvard.edu

Locations
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United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Latoya Lashley    617-355-7407    latoya.lashley@childrens.harvard.edu   
Principal Investigator: Andrew L. Frelinger, PhD         
Sub-Investigator: Rachael Grace, MD         
Sub-Investigator: Alan Michelson, MD         
Sponsors and Collaborators
Boston Children’s Hospital
Sysmex America, Inc.
Children's Hospital Colorado
Children's Hospital of Philadelphia
Texas Children's Hospital
Columbia University
Duke University

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Responsible Party: Andrew Frelinger, Assistant Professor of Pediatrics, Harvard Medical School, Boston Children’s Hospital
ClinicalTrials.gov Identifier: NCT03810352     History of Changes
Other Study ID Numbers: P00030227
First Posted: January 18, 2019    Key Record Dates
Last Update Posted: March 20, 2019
Last Verified: March 2019

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Andrew Frelinger, Boston Children’s Hospital:
ITP
platelet function
Sysmex

Additional relevant MeSH terms:
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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms