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Recruiting NCT06107582

Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

— Observational Primary 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
The protocol lists: data collection.
Who it may be relevant to
Registry conditions: Primary Immune Thrombocytopenia. Basic parameters: 6 years — 17 years · 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

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

Overview

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.

Detailed description

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.

Interventions

  • Other data collection
    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.

Primary outcome measures

  • Overall response rate [Time frame: 3 years]
  • Time to onset response [Time frame: 3 years]
  • Duration of response [Time frame: 3 years]
  • Sustained response rate [Time frame: 3 years]
  • Emergency 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 [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 [Time frame: 3 years]
  • Recurrence-free survival rate [Time frame: 3 years]
Secondary outcome measures (7)
  • Incidence [Time frame: 3 years]
  • Distribution [Time frame: 3 years]
  • Prognosis related factors selected from transcriptome data [Time frame: 3 years]
  • Prognosis related factors selected from proteomics data [Time frame: 3 years]
  • Prognosis related factors selected from metabolomics data [Time frame: 3 years]
  • Prognosis related factors selected from microbiome data [Time frame: 3 years]
  • Incidence, severity, and relationship of treatment emergent adverse events after treatment [Time frame: 3 years]

Eligibility criteria

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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Study design

Observational model
Cohort

Study locations

China · 1 center
  • Chinese Academy of Medical Science and Blood Disease Hospital — Tianjin

Identifiers

NCT: NCT06107582 · IIT2023044

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗