Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study

Immune thrombocytopenic purpura (ITP) is a kind of rare childhood disease that involve autoimmune destruction of platelets.The current Pediatric ITP cohorts are mostly based on single-center or multi-center cases, or cohorts with limited sample size in China. There is a lack of comprehensive and large-scale prospective cohort studies in pediatric ITP. The purpose of this study is to analyze the clinical characteristics of Pediatric ITP, the treatment methods, prognosis and prognostic model of these patients in China.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Other: data collection

Detailed Description

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Longitudinal Cohort allows to describe the long-term clinical characteristics of pediatric ITP patients, to study the benefit-risk balance of treatments, including the growing development of targeted therapies and to analyze the prognostic factors and attempts to establish prognostic models.

The study will include pediatric patients diagnosed with primary immune thrombocytopenia in the investigating hospitals, and collect basic information, diagnostic and treatment information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

• Study Type: Observational
• Enrollment (Estimated): 500

Contacts and Locations

• Study Contact: Name: Ting Sun, MD; Phone Number: 02223909009; Email: sunting@ihcams.ac.cn

Study Locations

• China
  • Tianjin, China, 300020
    • Recruiting
    • Chinese Academy of Medical Science and Blood Disease Hospital
    • Contact: Ting Sun, MD; Phone Number: 02223909009; Email: sunting@ihcams.ac.cn

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Pediatric patients who were diagnosed with ITP in the investigating hospitals.

Description

Inclusion Criteria:

• Age 6-17 years old (including both ends), male and female;
• Diagnosis of ITP.

Exclusion Criteria:

• Secondary thrombocytopenia caused by various reasons, such as connective tissue disorders, bone marrow hematopoietic failure disease, myelodysplastic syndrome, malignancy, drugs, inherited thrombocytopenia, common variable immune deficiency, lymphoma, etc.;
• The expected follow-up period is less than 3 months.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Pediatric Primary Immune Thrombocytopenia; Immune thrombocytopenia (ITP) : defined according to the international working group criteria	Other: data collection; The study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate	Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding	3 years
Duration of response	Duration of response defined as the longest duration for which the subject sustained a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding	3 years
Sustained response rate	Sustained response rate defined as proportion of subjects who keep a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding at 6, 12, 24, 36 months after initial administration of certain treatment in absence of rescue therapy	3 years
Emergency treatment	Percentage of subjects who received emergency treatment after initial administration of certain treatment	3 years
Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment	Changes of the subjects' numbers in bleeding score after administration of certain treatment according to the reported bleeding scale for pediatric patients with ITP. The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (>100 total) and/or >5 large bruises (>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb>2 g/dL or suspected internal hemorrhage；	3 years
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment	Changes of the subjects' numbers in WHO bleeding score after administration of certain treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.	3 years
Recurrence-free survival rate	Time from the start of treatment to the occurrence of a relapse or death event	3 years
Time to onset response	Time to onset response defined as the time needed for subjects to have a platelet count ≥ 30 × 10^9/L and at least 2-fold from baseline without bleeding	3 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Incidence	The incidence of pediatric primary immune thrombocytopenia in China will be described	3 years
Distribution	The population characteristics of pediatric primary immune thrombocytopenia in China will be described	3 years
Prognosis related factors selected from transcriptome data	The prognosis related factors will be selected from transcriptome data and be used to establish prognosis prediction model	3 years
Prognosis related factors selected from proteomics data	The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model	3 years
Prognosis related factors selected from metabolomics data	The prognosis related factors will be selected from metabolomics data and be used to establish prognosis prediction model	3 years
Prognosis related factors selected from microbiome data	The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model	3 years
Incidence, severity, and relationship of treatment emergent adverse events after treatment	Incidence, severity, and relationship of treatment emergent adverse events after treatment will be analyzed	3 years

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Collaborators: Henan Cancer Hospital; Beijing Children's Hospital; Tianjin Medical University Second Hospital; The First Affiliated Hospital of Xiamen University; The Second Affiliated Hospital of Kunming Medical University; Tianjin People's Hospital
• Investigators: Principal Investigator: lei zhang, MD, Chinese Academy of Medical Science and Blood Disease Hospital

Study record dates

Study Major Dates

• Study Start (Actual): November 1, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2036

Study Registration Dates

• First Submitted: August 26, 2023
• First Submitted That Met QC Criteria: October 24, 2023
• First Posted (Actual): October 30, 2023

Study Record Updates

• Last Update Posted (Actual): February 11, 2026
• Last Update Submitted That Met QC Criteria: February 9, 2026
• Last Verified: December 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; Health Care Quality, Access, and Evaluation; Investigative Techniques; Epidemiologic Methods; Health Care Evaluation Mechanisms; Quality of Health Care; Public Health; Environment and Public Health; Data Collection
• Other Study ID Numbers: IIT2023044

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No