Immune imbalance in pediatric persistent immune thrombocytopenia
| ISRCTN | ISRCTN84894190 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN84894190 |
- Submission date
- 08/09/2025
- Registration date
- 11/09/2025
- Last edited
- 10/09/2025
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Haematological Disorders
Plain English summary of protocol
Background and study aims
Immune thrombocytopenia (ITP) in children is an autoimmune disorder with an incompletely understood pathogenesis. Previous studies have implicated imbalances in T lymphocyte subsets, particularly increased T helper 17 (Th17) cells and decreased regulatory T cells (Tregs), in disease activity. Regulatory B cells (Bregs), which play a critical role in maintaining immune homeostasis, have also been proposed to contribute to ITP pathophysiology. This study aims to explore the dynamic alterations of T helper 17 (Th17) cells, regulatory T (Treg) cells and regulatory B (Breg) cells in children with persistent immune thrombocytopenia (ITP).
Who can participate?
Children with persistent ITP and age- and sex-matched healthy volunteers
What does the study involve?
Children with primary persistent ITP were enrolled in the ITP group, whereas age- and sex-matched healthy children undergoing physical examinations during the same period served as the control group. Patients in the ITP group received the following treatment upon confirmed diagnosis: intravenous immunoglobulin over 1–2 consecutive days; oral prednisone with a treatment course of 4–6 weeks (tapering was conducted gradually based on platelet recovery); additional IVIG doses were administered intermittently if the platelet count remained low or if active bleeding was present.
What are the possible benefits and risks of participating?
Children with persistent ITP have a slight recovery in immune function after treatment.
Where is the study run from?
The First Affiliated Hospital of Xinxiang Medical University (China)
When is the study starting and how long is it expected to run for?
October 2019 to December 2023
Who is funding the study?
The First Affiliated Hospital of Xinxiang Medical University (China)
Who is the main contact?
Shujun Li, ruolin2223@126.com
Contact information
Public, Scientific, Principal Investigator
No. 88 Jiankang Road
Weihui
453100
China
| Phone | +86 (0)13781905766 |
|---|---|
| ruolin2223@126.com |
Study information
| Study design | Prospective cohort study |
|---|---|
| Primary study design | Observational |
| Secondary study design | Cohort study |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a participant information sheet |
| Scientific title | Immune imbalance and dynamic characteristics of Th17, Treg and Breg cells in children with persistent immune thrombocytopenia |
| Study objectives | To explore the dynamic alterations of T helper 17 (Th17) cells, regulatory T (Treg) cells and regulatory B (Breg) cells in children with persistent immune thrombocytopenia (ITP). |
| Ethics approval(s) |
Approved 25/11/2019, Ethics Committee of The First Affiliated Hospital of Xinxiang Medical University (No. 88 Jiankang Road, Weihui, 453100, China; +86 (0)373 4402155; xyyfyxx@163.com), ref: EC-019-133 |
| Health condition(s) or problem(s) studied | Immune thrombocytopenia (ITP) |
| Intervention | 34 children with primary persistent ITP were enrolled in the ITP group, whereas 30 age- and sex-matched healthy children undergoing physical examinations during the same period served as control group. The treatment protocol for the ITP group was as follows. Based on the Chinese Guidelines for the Diagnosis and Treatment of Childhood Primary Immune Thrombocytopenia (2019 Edition), patients in the ITP group received the following first-line therapy upon confirmed diagnosis: 1. Intravenous immunoglobulin – 0.8–1 g/kg/day, administered via intravenous infusion over 1–2 consecutive days 2. Prednisone – oral administration at 1.5–2 mg/kg/day (maximum daily dose: 60 mg), with a treatment course of 4–6 weeks (tapering was conducted gradually based on platelet recovery) 3. Supplemental therapy – additional IVIG doses (0.8 g/kg per administration) were administered intermittently if the platelet count remained <20 × 10⁹/L or if active bleeding manifestations were present |
| Intervention type | Drug |
| Pharmaceutical study type(s) | Pharmacodynamic |
| Phase | Not Applicable |
| Drug / device / biological / vaccine name(s) | Intravenous immunoglobulin; prednisone |
| Primary outcome measure | Immune cell population (Th17 cell level, Treg cells, Breg cells) quantitatively analyzed and measured using BD FACSDx Flex TM flow cytometry at baseline and 3 months of treatment |
| Secondary outcome measures | BD FACSDx FlexTM flow cytometry was used to analyze the proportion of Th17 / Treg and the proportion of Breg cells in CD19 / B lymphocytes at baseline and 3 months before and after treatment |
| Overall study start date | 01/10/2019 |
| Completion date | 31/12/2023 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Upper age limit | 14 Years |
| Sex | Both |
| Target number of participants | 60 |
| Total final enrolment | 60 |
| Key inclusion criteria | ITP group: 1. Patients meeting the diagnostic criteria for primary persistent ITP as outlined in the Chinese Guidelines for the Diagnosis and Treatment of Childhood Primary Immune Thrombocytopenia (2019 Edition), defined as a disease duration exceeding 3 months and a platelet count below 100 × 10⁹/L 2. Age ≤14 years at the time of enrolment 3. No prior treatment with glucocorticoids, intravenous immunoglobulin (IVIG) or immunosuppressive agents within 1 month before initiating study treatment 4. Availability of complete clinical data for analysis. Control group: The control group comprised healthy children undergoing physical examinations during the same period, matched by age and sex to the ITP group. The inclusion criteria were as follows: 1. Age difference within 1 year compared with ITP participants 2. Gender distribution matching that of the ITP group (male-to-female ratio: approximately 1.6:1) 3. Participants had no infections, vaccinations or intake of folic acid, vitamin B12 or vitamin B6 within 4 weeks prior to enrolment |
| Key exclusion criteria | 1. Presence of severe infections, haematologic malignancies or substantial hepatic or renal dysfunction 2. History of vaccination or blood transfusion within 4 weeks prior to enrolment 3. Secondary thrombocytopenia, including but not limited to systemic lupus erythematosus, antiphospholipid syndrome, drug-induced thrombocytopenia or Evans syndrome 4. Known or suspected primary immunodeficiency 5. History of haematopoietic stem cell transplantation 6. Refusal of informed consent by legal guardians |
| Date of first enrolment | 01/12/2019 |
| Date of final enrolment | 30/06/2023 |
Locations
Countries of recruitment
- China
Study participating centre
Weihui
453100
China
Sponsor information
Hospital/treatment centre
No. 88 Jiankang Road
Weihui
453100
China
| Website | http://www.xyyfy.com/ |
|---|---|
"ROR" |
https://ror.org/0278r4c85 |
Funders
Funder type
Government
No information available
Results and Publications
| Intention to publish date | 31/12/2026 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | Planned publication in a high-impact peer-reviewed journal |
| IPD sharing plan | The data sharing plans for the current study are unknown and will be made available at a later date |
Editorial Notes
08/09/2025: Study's existence confirmed by the Ethics Committee of The First Affiliated Hospital of Xinxiang Medical University.
