Association of Platelet Parameters With Bleeding Severity in Children With ITP

Immune Thrombocytopenia Clinical Trial

— ITP-APPS  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03810352
• Other study ID #: P00030227
• Status: Completed
• Start date: March 15, 2019
• Est. completion date: November 26, 2023

Study information

• Verified date: March 2024
• Source: Boston Children's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 95
• Est. completion date: November 26, 2023
• Est. primary completion date: November 26, 2023
• Gender: All
• Age group: 6 Months to 20 Years

Eligibility

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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Locations

Country	Name	City	State
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Boston Children's Hospital	Boston	Massachusetts
United States	Duke University Medical Center	Durham	North Carolina
United States	Baylor College of Medicine	Houston	Texas
United States	Columbia University Medical Center	New York	New York
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (7)

Lead Sponsor	Collaborator
Boston Children's Hospital	Baylor College of Medicine, Children's Hospital Colorado, Children's Hospital of Philadelphia, Columbia University, Duke University, Sysmex America, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Association of IPF with concurrent, subsequent and worst-ever bleeding in children with ITP.	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.	February 2019-August 2022
Primary	Association of IPC with concurrent, subsequent, and worst-ever bleeding in children with ITP	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.	February 2019-August 2022
Primary	Association of platelet function markers with concurrent, subsequent, and worst-ever bleeding in children with ITP.	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.	February 2019-August 2022