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临床试验/NCT07068425
NCT07068425尚未招募不适用

Retrospective and Prospective Longitudinal Observational Study on Immune Thrombocytopenia

Peking University People's Hospital1 个研究点 分布在 1 个国家目标入组 2,000 人开始时间: 2025年7月10日最近更新:

试验速览

阶段
不适用
状态
尚未招募
入组人数
2,000
试验地点
1
主要终点
Prognosis

研究概览

简要总结

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.

详细描述

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

研究设计

研究类型
Observational
观察模型
Cohort
时间视角
Other

入排标准

性别
All
接受健康志愿者

入选标准

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

排除标准

  • 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"

结局指标

主要结局

Prognosis

时间窗: 5 years

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

Comorbidities

时间窗: 5 years

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

Overall response rate

时间窗: 180 days

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

Long-term overall remission rate

时间窗: 1 year

Proportion of the participants with an overall remission.

次要结局

  • Incidence(5 years)
  • Safety of treatment(1 year)
  • Health economic evaluation(5 years)

研究者

申办方类型
Other
责任方
Principal Investigator
主要研究者

Xiao Hui Zhang

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

Peking University People's Hospital

研究点 (1)

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