Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study

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.

Immune thrombocytopenia (ITP) is an organ-specific autoimmune disease, which is characterized by decreased platelet count and skin and mucosal bleeding. ITP is a kind of disease with increased platelet destruction and impaired platelet production caused by autoimmunity. Conventional treatment of adult ITP includes first-line glucocorticoid and immunoglobulin therapy, second line TPO and TPO receptor agonist, splenectomy and other immunosuppressive treatments (such as rituximab, vincristine, azathioprine, etc.). ITP is one of the most common hemorrhagic diseases. At present, the treatment response of ITP is not good, and a considerable number of patients need drug maintenance treatment, which seriously affects the quality of life of patients and increases the economic burden of patients. Longitudinal Cohort allows to describe the long-term clinical characteristics of pediatric ITP patients, to study the benefit-risk balance of treatments, including the growing development of targeted therapies and to analyze the prognostic factors and attempts to establish prognostic models.

The study will include pediatric patients diagnosed with primary immune thrombocytopenia in the investigating hospitals, and collect basic information, diagnostic and treatment information from medical records. The study will use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.

Inclusion Criteria:

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

Exclusion Criteria:

• 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.