Longitudinal Cohort of Pediatric Primary Immune Thrombocytopenia (ITP)

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age6-17
SponsorInstitute of Hematology & Blood Diseases Hospital, China

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 volunteers

Qualifiers

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

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • data collection

    Other intervention

    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.

Treatment groups

500 Participants
are divided into 1 treatment group
Group A: Pediatric Primary Immune Thrombocytopenia1 intervention

Trial outcomes

Primary outcomes

1

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 frame
3 years
2

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
3

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
4

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

Secondary outcomes

1

Incidence

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

Time frame
3 years
2

Distribution

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

Time frame
3 years
3

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
4

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

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

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