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NCT Number: NCT06107582

Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

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.

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Key information

Conditions

Primary Immune Thrombocytopenia Autoimmune Diseases Blood Coagulation Disorders Blood Platelet Disorders Cytopenia Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhage Hemorrhagic Disorders Immune System Diseases Pathologic Processes Pathological Conditions, Signs and Symptoms Purpura Purpura, Thrombocytopenic Purpura, Thrombocytopenic, Idiopathic Signs and Symptoms Skin Manifestations Thrombocytopenia Thrombotic Microangiopathies

Age range

6 year–17 year

Sex eligibility

All sexes

Study type

Observational

Primary location

Chinese Academy of Medical Science and Blood Disease Hospital

Tianjin, 300020, China

Location status: Recruiting

Location contact

Ting Sun, MD

CONTACT

[email protected]

02223909009

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Data Collection

Other

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 outcomes

  1. Overall response rate

    Time frame: 3 years

    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

  2. Time to onset response

    Time frame: 3 years

    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

  3. Duration of response

    Time frame: 3 years

    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

  4. Sustained response rate

    Time frame: 3 years

    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

  5. Emergency treatment

    Time frame: 3 years

    Percentage of subjects who received emergency treatment after initial administration of certain treatment

  6. 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

    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；

  7. 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

    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.

  8. Recurrence-free survival rate

    Time frame: 3 years

    Time from the start of treatment to the occurrence of a relapse or death event

Secondary outcomes

  1. Incidence

    Time frame: 3 years

    The incidence of pediatric primary immune thrombocytopenia in China will be described

  2. Distribution

    Time frame: 3 years

    The population characteristics of pediatric primary immune thrombocytopenia in China will be described

  3. Prognosis related factors selected from transcriptome data

    Time frame: 3 years

    The prognosis related factors will be selected from transcriptome data and be used to establish prognosis prediction model

  4. Prognosis related factors selected from proteomics data

    Time frame: 3 years

    The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model

  5. Prognosis related factors selected from metabolomics data

    Time frame: 3 years

    The prognosis related factors will be selected from metabolomics data and be used to establish prognosis prediction model

  6. Prognosis related factors selected from microbiome data

    Time frame: 3 years

    The prognosis related factors will be selected from proteomics data and be used to establish prognosis prediction model

  7. Incidence, severity, and relationship of treatment emergent adverse events after treatment

    Time frame: 3 years

    Incidence, severity, and relationship of treatment emergent adverse events after treatment will be analyzed

Study contacts

Contact information is provided by the study sponsor or research team.

Ting Sun, MD

CONTACT

[email protected]

02223909009

Sponsors and collaborators

Lead sponsor

Institute of Hematology & Blood Diseases Hospital, China

Other

Collaborators

  • Beijing Children's Hospital
  • Henan Cancer Hospital
  • The First Affiliated Hospital of Xiamen University
  • The Second Affiliated Hospital of Kunming Medical University
  • Tianjin Medical University Second Hospital
  • Tianjin People's Hospital

Registry information

Official study title

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

Important dates

Study start
2023
Primary completion
2026
Study completion
2036
First posted
Oct 30, 2023
Registry last updated
Feb 11, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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