Longitudinal Observational Study on Immune Thrombocytopenia

NCT07068425 | Not Yet Recruiting DMC

• 1 other identifier: PKU-ITP2025
• Study Type: observational
• Target: 2,000
• Locations: 1 country
• Sites: 1

Brief Summary

Immune Thrombocytopenia (ITP) is a bleeding disorder characterized by immune-mediated destruction and/or impaired production of platelets, leading to clinical manifestations such as petechiae, purpura, mucosal bleeding, and in severe cases, life-threatening visceral or intracranial hemorrhage. Research on the diagnosis, treatment, and prognosis of ITP is critical for improving patient outcomes and quality of life. However, most existing hematologic cohort studies are limited to single-center or small multicenter samples, lacking comprehensive, large-scale prospective investigations. To address this gap, our center plans to conduct a large-sample, combined retrospective and prospective longitudinal observational cohort study of ITP patients. The study will enroll patients to collect baseline demographic and diagnostic data, followed by longitudinal follow-up via questionnaires, telephone interviews, video consultations, online platforms, and in-person visits. Key variables including treatment regimens, comorbidities, and prognostic outcomes will be systematically recorded. Additionally, hospitalization and outpatient expenditure data will be extracted from electronic medical records. This study aims to provide high-quality real-world evidence on the epidemiology, treatment patterns, clinical outcomes, and healthcare costs of ITP patients in China, ultimately informing clinical decision-making and health policy.

Conditions

Immune Thrombocytopenia (ITP)

Keywords

Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (4)

• Prognosis

  To analyze the long-term prognosis of patients with immune thrombocytopenia (ITP), and analyze the risk factors associated with their prognosis.

  5 years

• Overall response rate

  To analyze the therapeutic efficacy of patients with ITP, and analyze the impact factors associated with the efficacy.

  180 days

• Long-term overall remission rate

  Proportion of the participants with an overall remission.

  1 year

• Comorbidities

  The incidence, risk factors, treatments, and prognosis of complications

  5 years

Secondary Outcomes (3)

• Incidence

  5 years

• Safety of treatment

  1 year

• Health economic evaluation

  5 years

Study Arms (3)

Retrospective cohort

Patients whose first visit to our institution and the termination of follow-up both occurred before the opening of this study will contribute to the retrospective cohort.

Prospective cohort

Patients whose first visit to our institution occur after the opening of this study will contribute to the prospective cohort.

Retrospective/Prospective cohort

Patients whose first visit to our institution occurred before the opening of this study and whose follow-up will terminate after the opening of this study will contribute to the ambispective cohort.

Eligibility Criteria

• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with confirmed immune thrombocytopenia (ITP). Diagnostic Criteria for ITP: 1. Sustained platelet count \<100×10⁹/L 2. Normal or increased megakaryocyte count in bone marrow with maturation defects 3. Exclusion of other potential causes of thrombocytopenia including: pseudothrombocytopenia, drug-induced thrombocytopenia, viral infections, helicobacter pylori infection, hematologic disorders, autoimmune diseases, hypersplenism, malignancies

You may qualify if:

• Patients with confirmed diagnosis of ITP
• Patients treated at Peking University People's Hospital since January 1, 2005

You may not qualify if:

• Any condition that may render follow-up data unreliable, including but not limited to severe psychiatric disorders"
• Patients deemed ineligible for the study by investigators"

Sponsors & Collaborators

• Peking University People's Hospital: lead
• Jiangsu Provincial People's Hospital: collaborator
• Wuhan TongJi Hospital: collaborator

Study Sites (1)

Peking University Insititute of Hematology, Peking University People's Hospital, Beijing, Beijing 100010

Beijing, China

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

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

Study Officials

• Xiao-Hui Zhang, MD

  Peking University Institute of Hematology, Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Jin Wu, MD

CONTACT

086-01088326002; wujin1996@126.com

Li-Qin Cao, MD

CONTACT

086-01088326002; caoliqin@pkuph.edu.cn

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice President of Peking University Institute of Hematology Affiliation: Peking University People's Hospital

Study Record Dates

• First Submitted: July 6, 2025
• First Posted: July 16, 2025
• Study Start: July 10, 2025
• Primary Completion (Estimated): December 31, 2035
• Study Completion (Estimated): December 31, 2036
• Last Updated: July 16, 2025

Record last verified: 2025-07

Locations

CN (1)