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The aim of this study was to observe whether maintaining a high level of platelet count after TPO-RA in patients with primary immune thrombocytopenia (ITP) can induce immune tolerance, develop immune balance in ITP patients, and enable patients to achieve a sustained response (SRoT) after TPO-RA discontinuation.
In this study, ITP patients who has reached complete response (PLT ≥100 x 10^9/L)after thrombopoietin receptor agonist (TPO-RA) therapy do not rush to reduce the drug dose, so that a higher level of platelet count can be maintained for a period of time.
The treatment goal is to maintain the patient's platelet count at 300-600 × 10^9/L, and adjust the dosage of hetrombopag (2.5mg/d~7.5mg/d) based on the patient's platelet count. After 24-week TPO-RA treatment, all patients with a platelet count of ≥ 50 × 10^9/L after two consecutive visits will enter an 8-week reduction period. All patients who successfully discontinued the drug and maintained their platelet count at ≥30×109/L entered the efficacy and safety follow-up period.
The aim is to investigate whether this strategy could lead to the development or achievement of immune tolerance, achieving sustained response off treatment (SROT) (PLT≥50×109/L, no other ITP-specific medications, no bleeding) after TPO-RA discontinuation.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| hetrombopag treatment | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Hetrombopag Olamine | Drug | Treatment period: 24-week Hetrombopag (2.5mg/d~7.5mg/d) treatment.
Drug discontinuation period:8-week Hetrombopag (2.5mg/d~7.5mg/d) reduction. Hetrombopag reduces by 2.5mg every 2 weeks, and after reducing to the minimum dose of 2.5mg/d x 2 weeks, it is changed to 2.5mg once every other day (Qod) treatment, with a maximum reduction time of 8 weeks. If the PLT during two consecutive visits is less than 30 × 10^9/L, the patient will be withdrawn. |
| Measure | Description | Time Frame |
|---|---|---|
| Sustained response off treatment (SROT) | The percentage of participants with successful discontinuation of TPO-RAs after tapering and discontinuation | 3 year |
| Measure | Description | Time Frame |
|---|---|---|
| Quality of life (QoL) | The degree to which an individual is healthy, comfortable, and able to participate in or enjoy life events according to the MOS 36-item short-form health survey (SF-36) at week1, week24 and week56 (±5 d). | 3 year |
| Duration of Complete response (DCR) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Tienan Zhu, M.D. | Contact | 01069155027 | zhutn@pumch.cn |
| Name | Affiliation | Role |
|---|---|---|
| Tienan Zhu, M.D. | Peking Union Medical College Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking Union Medical College Hospital | Recruiting | Beijing | Beijing Municipality | China |
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| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
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| ID | Term |
|---|---|
| C000614661 | hetrombopag |
| D001241 | Aspirin |
| ID | Term |
|---|---|
| D012459 | Salicylates |
| D062385 | Hydroxybenzoates |
| D010636 | Phenols |
| D001555 | Benzene Derivatives |
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|
|
| Aspirin | Drug | Aspirin 100mg, qd |
|
The time from the first assessment of complete resonse (platelet count ≥ 100 × 10^9/L, without bleeding) until the date of the first occurrence of progression, or until the date of death.t |
| 3 year |
| Duration of Response (DoR) | The time from the first assessment of response (platelet count ≥ 50 × 10^9/L) until the date of the first occurrence of progression, or until the date of death. | 3 year |
| Adverse events | The incidence and severity of thromboembolism, as well as other commonly used indicators such as abnormal clinical symptoms and vital signs, abnormalities observed in laboratory tests, are recorded.Toxicity will be graded according to NCI-CTC AE 5.0. | 3 years |
| Bleeding events | Bleeding events occurring in patients due to ITP | 3 years |
| D006425 |
| Hemic and Lymphatic Diseases |
| D057049 | Thrombotic Microangiopathies |
| D013921 | Thrombocytopenia |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D006841 |
| Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D009930 | Organic Chemicals |