About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age 6-17 years old (including both ends), male and female;
Diagnosis of ITP.
Disqualifiers
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.
Trial population
Pediatric patients who were diagnosed with ITP in the investigating hospitals.
Trial design
Cohort
Prospective
Treatments tested in this trial
data collection
Other interventionThe study will collect basic information, diagnostic and treatment information from medical records and use questionnaire to measure the exposure of patients, and prospectively follow-up to collect the prognosis information.
Treatment groups
Trial outcomes
Primary outcomes
Overall response rate
Overall response rate defined as proportion of subjects with a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
Time to onset response
Time to onset response defined as the time needed for subjects to have a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
Duration of response
Duration of response defined as the longest duration for which the subject sustained a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding
Sustained response rate
Sustained response rate defined as proportion of subjects who keep a platelet count ≥ 30 × 10\^9/L and at least 2-fold from baseline without bleeding at 6, 12, 24, 36 months after initial administration of certain treatment in absence of rescue therapy
Secondary outcomes
Incidence
The incidence of pediatric primary immune thrombocytopenia in China will be described
Distribution
The population characteristics of pediatric primary immune thrombocytopenia in China will be described
Prognosis related factors selected from transcriptome data
The prognosis related factors will be selected from transcriptome data and be used to establish prognosis prediction model
Prognosis related factors selected from proteomics data
The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model
Sponsors and contacts
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Institute of Hematology & Blood Diseases Hospital, China
Lead sponsor
Beijing Children's Hospital
Collaborator
Tianjin People's Hospital
Collaborator
Henan Cancer Hospital
Collaborator
Tianjin Medical University Second Hospital
Collaborator
The First Affiliated Hospital of Xiamen University
Collaborator
The Second Affiliated Hospital of Kunming Medical University
Collaborator