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The project was undertaking by Qilu Hospital of Shandong University and other well-known hospitals in China. Aims at evaluating effectiveness and safety of avatrombopag in the treatment of primary immune thrombocytopenia.
The investigators are undertakingSingle-arm, multi-Centre, Observational study of 400 Patients with primary immune thrombocytopenia (ITP) from Qilu Hospital of Shandong University and other well-known hospitals in China. ITP patients will be given one tablet (20 mg) of avatrombopag daily.If the platelet count is higher than 150×10^9/L, the dose should be reduced. Prolonged dosing intervals or a combination of reduced daily dose are preferred.If the drug was taken for ≥1 week and the platelet count was still less than 30×10^9/L, the dosage should be increased. The maximum dose is 40 mg daily. Aplatelet count,AE, laboratory parameters, ECG, vital signs and other symptoms will be evaluated before and after treatment. Adverse events will be recorded throughout the study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Patients with primary immune thrombocytopenia (ITP) | Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)" |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Avatrombopag | Drug | Avatrombopag is an orally bioavailable, small molecule thrombopoietin receptor agonist that has been developed by Dova Pharmaceuticals for the treatment of thrombocytopenic disorders. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of participants achieving platelet response on day 28 of treatment | Percentage of participants achieving platelet response on day 28 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy. | on day 28 of treatment |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of participants achieving platelet response on day 8 of treatment. | Percentage of participants achieving platelet response on day 8 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy. | on day 8 of treatment. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of participants achieving platelet response (R) at the second month of treatment. | Percentage of participants achieving the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy at the second month of treatment. | at the second month of treatment |
Inclusion Criteria:
Aged 18+ years, male or female;
Ever been diagnosed as ITP patients. The diagnostic criteria comply with the "Chinese Guidelines for the Diagnosis and Treatment of Adult Primary Immune Thrombocytopenia (2020 Edition)"
ECOG general status score ≤ 2;
Platelet count < 30×10^9/L;platelet count ≥< 30×10^9/L accompanied by active bleeding; If the platelet count is around 30×10^9/L and no active bleeding, a second examination must be performed to further confirm the platelet count.
Voluntarily signed the informed consent.
Any other circumstances that the investigator considers appropriate for the patient to participate in the study.
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ming Hou, MD PhD | Contact | 86-531-82169114 | 9879 | houming@medmail.com.cn |
| Name | Affiliation | Role |
|---|---|---|
| Ming Hou, MD PhD | Shandong University Qilu Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Qilu Hospital, Shandong University | Recruiting | Jinan | Shandong | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 30799645 | Background | Dlugosz-Danecka M, Zdziarska J, Jurczak W. Avatrombopag for the treatment of immune thrombocytopenia. Expert Rev Clin Immunol. 2019 Apr;15(4):327-339. doi: 10.1080/1744666X.2019.1587294. Epub 2019 Mar 8. | |
| 29995177 | Background | Shirley M. Avatrombopag: First Global Approval. Drugs. 2018 Jul;78(11):1163-1168. doi: 10.1007/s40265-018-0949-8. |
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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 |
|---|---|
| C533238 | avatrombopag |
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|
| Percentage of participants achieving platelet response on day 14 of treatment. | Percentage of participants achieving platelet response on day 14 of treatment. Platelet response rate refers to the platelet count PLT≥30×109/L and increases at least 2 times from the baseline value without bleeding in the absence of remedial therapy. | on day 14 of treatment. |
| Changes in participants' bleeding scores. | Thrombocytopenia bleeding scoring system is used to evaluate the changes in participants' bleeding scores. | through study completion, an average of 6 months |
| Evaluation of adverse effects related to avatrombopag. | Evaluation of adverse effects related to avatrombopag including side effect,toxic reaction,post effect,anaphylactic reaction. | through study completion, an average of 6 months |
| Participants' 6-month continuous remission rate. |
Percentage of participants achieving 6-month continuous remission including symptoms associated with bleeding and mental symptoms |
| 6 month of treatment |
| 31565009 | Background | Cheloff AZ, Al-Samkari H. Avatrombopag for the treatment of immune thrombocytopenia and thrombocytopenia of chronic liver disease. J Blood Med. 2019 Sep 5;10:313-321. doi: 10.2147/JBM.S191790. eCollection 2019. |
| 24802775 | Background | Bussel JB, Kuter DJ, Aledort LM, Kessler CM, Cuker A, Pendergrass KB, Tang S, McIntosh J. A randomized trial of avatrombopag, an investigational thrombopoietin-receptor agonist, in persistent and chronic immune thrombocytopenia. Blood. 2014 Jun 19;123(25):3887-94. doi: 10.1182/blood-2013-07-514398. Epub 2014 May 6. |
| 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 |