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To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. In this study, hospitalized participants will receive a 14-day regimen of high-dose dexamethasone (HD-DXM) plus recombinant human thrombopoietin (rhTPO), followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP patients.
To address the clinical need for improving early response rates, maintaining sustained responses, reducing relapse rates, and minimizing adverse events in the treatment of immune thrombocytopenia (ITP), the investigators developed a comprehensive in-hospital and post-discharge management strategy. This approach is based on the superior early response achieved with recombinant human thrombopoietin (rhTPO) combined with high-dose dexamethasone (HD-DXM), and the favorable efficacy of eltrombopag in maintaining long-term responses and reducing relapse. In this study, hospitalized participants will receive a 14-day regimen of HD-DXM plus rhTPO, followed by a 10-week course of oral eltrombopag olamine dry suspension after discharge. The investigators aim to evaluate the efficacy and safety of this sequential treatment strategy in adult ITP participants, with the goal of enhancing early response, sustaining remission, reducing relapse, and minimizing adverse effects. This study is expected to provide evidence-based support for optimizing ITP treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Eltrombopag Group | Experimental | Participants who achieve a complete response (CR) or response (R) after HD-DXM combined with rhTPO treatment will be randomized in a 2:1 ratio into the eltrombopag group or the placebo group. Observation group: Participants will receive eltrombopag for oral suspension (EPAG-pfos) for ten weeks. |
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| Placebo group | Active Comparator | Participants who achieve a complete response (CR) or response (R) after HD-DXM combined with rhTPO treatment will be randomized in a 2:1 ratio into the eltrombopag group or the placebo group. Control group: Participants did not receive sequential EPAG-pfos treatment. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Participants assigned to the observation group will be treated with eltrombopag oral suspension (EPAG-pfos). |
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| Measure | Description | Time Frame |
|---|---|---|
| Sustained response rate (SRR) at 3 months of treatment. | Sustained response rate (SRR) at 3 months of treatment. | 3 months of the study |
| Measure | Description | Time Frame |
|---|---|---|
| The proportions of patients achieving response (R); | The proportions of patients achieving response (R); | 6 months of the study |
| Platelet count levels at 3 months after treatment | Platelet count levels at 3 months after treatment |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Heng Mei, Ph.D | Contact | +8613886160811 | hmei@hust.edu.cn | |
| Min Xu, Ph.D | Contact | 13212794115 | xu_min1015@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Heng Mei | Union Hospital, Tongji Medical College, Huazhong University of Science and Technology | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Wuhan Union Hospital | Wuhan | Hubei | 430022, | China |
It is not clear whether the data would involve patient privacy or whether patients would agree to the disclosure of the data.
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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 |
|---|---|
| C520809 | eltrombopag |
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| Placebo | Other | Participants in the control group will receive a placebo at the same dose, instead of eltrombopag oral suspension (EPAG-pfos). |
|
| 3 months of the study |
| The proportions of patients achieving complete response (CR); | The proportions of patients achieving complete response (CR); | 6 months of the study |
| The proportions of patients achieving and platelet counts ≥50×10⁹/L; | The proportions of patients achieving and platelet counts ≥50×10⁹/L; | 6 months of the study |
| Sustained response rate (SRR) at 6 months; | Sustained response rate (SRR) at 6 months; | 6 months of the study |
| Time to response (TTR); | Time to response (TTR); | 6 months of the study |
| Time from treatment initiation to first achievement of PLT ≥100×10⁹/L; | Time from treatment initiation to first achievement of PLT ≥100×10⁹/L; | 6 months of the study |
| 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 |