Longitudinal Observational Study on Immune Thrombocytopenia

NCT ID: NCT07068425

Last Updated: 2025-07-16

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Total Enrollment: 2000 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2025-07-10
• Study Completion Date: 2036-12-31

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.

Detailed Description

Immune Thrombocytopenia (ITP) is an immune-mediated acquired bleeding disorder characterized by thrombocytopenia with normal or increased megakaryocytes in the bone marrow accompanied by maturation defects. Epidemiological studies estimate its incidence at approximately 2-5 cases per 100,000 individuals in the general population.

The primary clinical manifestation of ITP is bleeding, ranging from minor cutaneous/mucosal hemorrhage to life-threatening organ bleeding. Notably, some patients also exhibit increased risk of thrombotic/embolic events. Embolic complications in critical organs (e.g., heart, brain) may significantly impair quality of life and lead to life-threatening conditions.

Currently, most ITP cohort studies in China are limited to single-center designs or small multicenter studies with insufficient sample sizes. There remains a notable lack of large-scale, longitudinal prospective cohort studies.

To address these limitations, this multicenter, retrospective-prospective longitudinal observational study will enroll diagnosed ITP patients to systematically collect:

Baseline demographic and diagnostic data Prospective follow-up data via questionnaires, telemedicine, and clinical visits Treatment regimens, comorbidities, and prognostic outcomes Healthcare utilization costs through electronic medical records

By establishing this comprehensive real-world database, the study aims to:

Elucidate the epidemiology, treatment patterns, and economic burden of ITP in China Provide evidence-based support for developing novel clinical strategies Improve long-term hemorrhage-free survival rates This investigation will serve as a foundational platform for optimizing ITP management and enhancing patient outcomes across China.

Conditions

Immune Thrombocytopenia (ITP)

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: OTHER

Study Groups

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.

No interventions assigned to this group

Prospective cohort

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

No interventions assigned to this group

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.

No interventions assigned to this group

Eligibility Criteria

Inclusion Criteria

1. Patients with confirmed diagnosis of ITP

2. Patients treated at Peking University People's Hospital since January 1, 2005

Exclusion Criteria

1. Any condition that may render follow-up data unreliable, including but not limited to severe psychiatric disorders"

2. Patients deemed ineligible for the study by investigators"

• Eligible Sex: ALL
• Accepts Healthy Volunteers: Yes

Sponsors

Jiangsu Provincial People's Hospital

OTHER

Sponsor Role: collaborator

Wuhan TongJi Hospital

OTHER

Sponsor Role: collaborator

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao Hui Zhang

Vice President of Peking University Institute of Hematology Affiliation: Peking University People's Hospital

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xiao-Hui Zhang, MD

Role: PRINCIPAL_INVESTIGATOR

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

Locations

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

Beijing, , China

Countries

China

Central Contacts

Jin Wu, MD

Role: CONTACT

wujin1996@126.com

086-01088326002

Li-Qin Cao, MD

Role: CONTACT

caoliqin@pkuph.edu.cn

086-01088326002

Other Identifiers

PKU-ITP2025

Identifier Type: -

Identifier Source: org_study_id