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.
Study Type
OBSERVATIONAL
Enrollment
500
The 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.
Chinese Academy of Medical Science and Blood Disease Hospital
Tianjin, China
RECRUITINGOverall 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 frame: 3 years
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
Time frame: 3 years
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
Time frame: 3 years
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
Time frame: 3 years
Emergency treatment
Percentage of subjects who received emergency treatment after initial administration of certain treatment
Time frame: 3 years
Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment
Changes of the subjects' numbers in bleeding score after administration of certain treatment according to the reported bleeding scale for pediatric patients with ITP. The bleeding scale for pediatric patients with ITP is a measure of bleeding severity with the following grades: Grade 1 (minor) Minor bleeding, few petechiae (≤100 total) and/or ≤5 small bruises (≤3 cm in diameter), no mucosal bleeding；Grade 2 (mild) Mild bleeding, many petechiae (\>100 total) and/or \>5 large bruises (\>3 cm in diameter), no mucosal bleeding；Grade 3 (moderate) Moderate bleeding, overt mucosal bleeding, troublesome lifestyle；Grade 4 (severe) Severe bleeding, mucosal bleeding leading to decrease in Hb\>2 g/dL or suspected internal hemorrhage；
Time frame: 3 years
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment
Changes of the subjects' numbers in WHO bleeding score after administration of certain treatment according to the reported World Health Organization's Bleeding Scale. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.
Time frame: 3 years
Recurrence-free survival rate
Time from the start of treatment to the occurrence of a relapse or death event
Time frame: 3 years
Incidence
The incidence of pediatric primary immune thrombocytopenia in China will be described
Time frame: 3 years
Distribution
The population characteristics of pediatric primary immune thrombocytopenia in China will be described
Time frame: 3 years
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
Time frame: 3 years
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
Time frame: 3 years
Prognosis related factors selected from metabolomics data
The prognosis related factors will be selected from metabolomics data and be used to establish prognosis prediction model
Time frame: 3 years
Prognosis related factors selected from microbiome data
The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model
Time frame: 3 years
Incidence, severity, and relationship of treatment emergent adverse events after treatment
Incidence, severity, and relationship of treatment emergent adverse events after treatment will be analyzed
Time frame: 3 years
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