Longitudinal Observational Study on Immune Thrombocytopenia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- This is an observational study: the protocol does not assign a study treatment.
- Who it may be relevant to
- Registry conditions: Immune Thrombocytopenia (ITP). Basic parameters: No limits · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Retrospective and Prospective Longitudinal Observational Study on Immune Thrombocytopenia
Overview
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.
Primary outcome measures
- Prognosis [Time frame: 5 years]
- Overall response rate [Time frame: 180 days]
- Long-term overall remission rate [Time frame: 1 year]
- Comorbidities [Time frame: 5 years]
Secondary outcome measures (3)
- Incidence [Time frame: 5 years]
- Safety of treatment [Time frame: 1 year]
- Health economic evaluation [Time frame: 5 years]
Eligibility criteria
Inclusion criteria
- Patients with confirmed diagnosis of ITP
- Patients treated at Peking University People's Hospital since January 1, 2005
Exclusion criteria
- 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"
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Observational model
- Cohort
Study locations
China · 1 center
- Peking University Insititute of Hematology, Peking University People's Hospital, Beijing, — Beijing
Publications
- Zhang L, An Z, Zhao P, Huang Q, Wu J, He Y, Zhu X, Fu H, Zhang Y, Kong Y, Jiang Q, Jiang H, Lu J, Chang Y, Xu L, Zhao X, Huang X, Zhang X. Unmasking the link: pulmonary embolism risk and outcomes in adults with immune thrombocytopenia. J Thromb Haemost. 2026 May 9:S1538-7836(26)00281-3. doi: 10.1016/j.jtha.2026.04.030. Online ahead of print. PMID 42114623
Identifiers
NCT: NCT07068425 · PKU-ITP2025