Clinical trial NCT06107582: Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)
Official title: Clinical Characteristics, Prognosis and Prognostic Model of Pediatric Immune Thrombocytopenia: a Prospective, Multicenter, Observational Cohort Study
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Primary Immune Thrombocytopenia
Lead sponsor: Institute of Hematology & Blood Diseases Hospital, China
Sponsor class: OTHER
Collaborators: name: Beijing Children's Hospital
name: Tianjin People's Hospital
name: Henan Cancer Hospital
name: Tianjin Medical University Second Hospital
name: The First Affiliated Hospital of Xiamen University
name: The Second Affiliated Hospital of Kunming Medical University
First posted: 2023-10-30
Enrollment: 500
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Pediatric patients who were diagnosed with ITP in the investigating hospitals.

Brief summary: 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.

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.

Primary outcomes:
measure: Overall response rate
timeFrame: 3 years
measure: Time to onset response
timeFrame: 3 years
measure: Duration of response
timeFrame: 3 years
measure: Sustained response rate
timeFrame: 3 years
measure: Emergency treatment
timeFrame: 3 years
measure: Number of subjects with clinically significant bleeding as assessed using the bleeding scale for pediatric patients with ITP after initial administration of certain treatment
timeFrame: 3 years
measure: Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale after initial administration of certain treatment
timeFrame: 3 years
measure: Recurrence-free survival rate
timeFrame: 3 years
Secondary outcomes:
measure: Incidence
timeFrame: 3 years
measure: Distribution
timeFrame: 3 years
measure: Prognosis related factors selected from transcriptome data
timeFrame: 3 years
measure: Prognosis related factors selected from proteomics data
timeFrame: 3 years
measure: Prognosis related factors selected from metabolomics data
timeFrame: 3 years
measure: Prognosis related factors selected from microbiome data
timeFrame: 3 years
measure: Incidence, severity, and relationship of treatment emergent adverse events after treatment
timeFrame: 3 years
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Chinese Academy of Medical Science and Blood Disease Hospital
Study officials:
affiliation: Chinese Academy of Medical Science and Blood Disease Hospital
name: Lei Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: