Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

NCT06107582 | Recruiting

• 1 other identifier: IIT2023044
• Study Type: observational
• Target: 500
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Primary Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (8)

• 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

• 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

• 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

Secondary Outcomes (7)

• Incidence

  3 years

• Distribution

  3 years

• Prognosis related factors selected from transcriptome data

  3 years

• Prognosis related factors selected from proteomics data

  3 years

• Prognosis related factors selected from metabolomics data

  3 years

Study Arms (1)

Pediatric Primary Immune Thrombocytopenia

Immune thrombocytopenia (ITP) : defined according to the international working group criteria

Other: data collection

Interventions

data collection OTHER

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.

Pediatric Primary Immune Thrombocytopenia

Eligibility Criteria

• Age: 6 Years - 17 Years
• Sex: all
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

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

You may not qualify if:

• 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.

Sponsors & Collaborators

• Beijing Children's Hospital: collaborator
• Tianjin People's Hospital: collaborator
• Henan Cancer Hospital: collaborator
• Tianjin Medical University Second Hospital: collaborator
• The First Affiliated Hospital of Xiamen University: collaborator
• The Second Affiliated Hospital of Kunming Medical University: collaborator
• Institute of Hematology & Blood Diseases Hospital, China: lead

Study Sites (1)

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, 300020, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Data Collection

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Lei Zhang, MD

  Chinese Academy of Medical Science and Blood Disease Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Ting Sun, MD

CONTACT

02223909009; sunting@ihcams.ac.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 26, 2023
• First Posted: October 30, 2023
• Study Start: November 1, 2023
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2036
• Last Updated: February 11, 2026

Record last verified: 2025-12

Locations

CN (1)