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Current first line treatments for immune thrombocytopenia (ITP) usually have transient effects and prolonged platelet response rate off therapy remains low. The aim is to evaluate whether a 12-week course of eltrombopag plus pulsed dexamethasone as first line therapy can increase the proportion of patients with prolonged response. Diagnosis of ITP is established according to the American Society of Hematology guidelines. Eligible ITP subjects have platelet counts <30×109/L or counts <50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above). Subjects must have no prior ITP treatment except platelet transfusions. Treatment consists of eltrombopag 25-75 mg daily according to platelet response for 12 weeks plus pulsed dexamethasone, 40 mg daily for 4 consecutive days every 4 weeks for 1-3 courses. The primary endpoint is prolonged response rate which was defined as the proportion of enrolled subjects maintaining platelet counts >50×109/L for more than 6 months without any ITP therapy after completion of 12-week therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Single Arm | Experimental | Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Eltrombopag Oral Tablet 25-75 mg daily for 12 weeks plus pulsed dexamethasone |
|
| Measure | Description | Time Frame |
|---|---|---|
| The prolonged response rate | Defined as proportion of patients with platelet counts > 50,000/μl at 6 months after completion of therapy and free of ITP rescue therapy | 6 months after completion of therapy |
| Measure | Description | Time Frame |
|---|---|---|
| Time to relapse | Defined as the interval from completion of 3 months treatment to platelet count<30,000/ul and restarting of ITP therapy including but not limited to platelet transfusion, IVIG, corticosteroids, immunosuppressive drugs, rituximab | 6 months after completion of therapy |
| Early response rate |
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Inclusion Criteria:
Subject has signed and dated a written informed consent.
Adults (≥18 years) diagnosed with ITP according to the American Society for Hematology/British Committee for Standards in Haematology (ASH/BCSH) Guidelines. In addition, the peripheral blood smear should support the diagnosis of ITP with no evidence of other disease causative of thrombocytopenia (e.g., pseudo thrombocytopenia, myelofibrosis). The physical examination should be normal or at least not show signs suggestive of any disease likely to be associated with thrombocytopenia.
No prior ITP treatment except platelet transfusions
Subject has no intercurrent medical event, including evidence of any thrombosis.
Normal prothrombin time (PT/INR) and activated partial thromboplastin time (aPTT), no history of hypercoagulable state.
The following clinical chemistries must be within the normal reference range:
creatinine, ALT, AST, total bilirubin, total albumin and alkaline phosphatase.
Subject is practicing an acceptable method of contraception (documented in chart).
Subject is able to understand and comply with protocol requirements and instructions.
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Gregory Cheng, PhD, MD | Humanity & Health Research Centre | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Humanity & Health Research Centre | Hong Kong | Hong Kong SAR | Hong Kong |
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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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Defined as proportion of patients requiring less than 3 courses of pulsed dexamethasone to maintain platelet counts > 150,000/ul |
| 6 months after completion of therapy |
| Health related quality of life | Assessed by SV36 HRQoL questionnaires | 6 months after completion of therapy |
| 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 |