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| Name | Class |
|---|---|
| Peking University People's Hospital | OTHER |
| Xiyuan Hospital of China Academy of Chinese Medical Sciences | OTHER |
| Second Hospital of Shanxi Medical University | OTHER |
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This study was a single-arm, multicenter, phase Î clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug
This study was a single-arm, multicenter, phase Î clinical study. Patients admitted to the enrollment unit center with a confirmed diagnosis of TDNSAA/VSAA/SAA, treated with IST (p/r-ATG+CSA) in combination with TPO-RA (including eltrombopta or hydtrombopta) for at least 3 months with no hematologic response at 6-month follow-up, and who were not suitable or unwilling to undergo hematopoietic stem cell transplantation (HSCT), were to another novel TPO-RA avatrombopta, 40-60 mg (weight <80 kg), in addition to maintaining the original immunosuppressive therapy ( CSA or equivalent immune potency drugs), switch to another new TPO-RA avatropa 40-60 mg (40 mg daily for weight <80 kg; 60 mg daily for weight >80 kg) orally once daily for at least 3 months and follow up for 3 months to determine the hematologic response and to assess the safety of the drug.Selection of study population Severe aplastic anemia patients with poor efficacy of IST combined with TPO-RA Patients should be judged for inclusion and exclusion criteria. Number of subjects: 35 effective cases, 39 patients should be included according to the dropout rate of 10%.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Avatrombopag in RAA | Experimental | After the patients met the above-mentioned inclusion conditions and signed informed consent, they began to be included in this program. The main research objectives are to take avatrombopag conversion therapy for at least 3 months, to monitor hematological indicators, biochemical indicators and bone marrow related tests, to determine hematological responses, and to evaluate the safety of the drug. In the 6th and 12th months after treatment, comprehensive review of bone marrow and peripheral blood was performed to evaluate the recovery of hematopoiesis, determine the curative effect, evaluate adverse events, and whether there was clonal transformation. After the patients completed the main study observation, they were followed up for at least 3 months, that is, from the time the patients were enrolled, for a total of at least 6 months of follow-up. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| avatrombopag | Drug | Avatrombopag, 40-60 mg (body weight < 80 kg, 40 mg per day; body weight > 80 kg, 60 mg per day) orally once daily for at least 3 months and followed up for 3 months to determine hematological response and evaluate the drug security. |
| Measure | Description | Time Frame |
|---|---|---|
| The rate of HR in patients after switching to avatrombopag. | Percentage of the total number of patients receiving treatment who received APAG | 3 months |
| Incidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading | Incidence of Treatment-Emergent AE by CTCAE | 3 months |
| Percentage of patients with transformation | Rate of patients with transformation to PNH or MDS,AML, or other disease | 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| The rate of HR in patients after switching to avatrombopag. | Percentage of the total number of patients receiving treatment who received APAG | 6months |
| ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading |
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Inclusion Criteria: Subjects eligible for inclusion in this study must meet all of the following criteria:
Exclusion Criteria: Subjects meeting any of the following criteria were excluded from this study:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Fengkui Zhang, Doctor | Contact | 8602223909229 | fkzhang@ihcams.ac.cn | |
| Liping Jing, Doctor | Contact | 8602223909223 | jingliping@ihcams.ac.an |
| Name | Affiliation | Role |
|---|---|---|
| Liping Jing, Doctor | Anemia Treatment Center | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences | Recruiting | Tianjin | Tianjin Municipality | 300020 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 26568159 | Result | Killick SB, Bown N, Cavenagh J, Dokal I, Foukaneli T, Hill A, Hillmen P, Ireland R, Kulasekararaj A, Mufti G, Snowden JA, Samarasinghe S, Wood A, Marsh JC; British Society for Standards in Haematology. Guidelines for the diagnosis and management of adult aplastic anaemia. Br J Haematol. 2016 Jan;172(2):187-207. doi: 10.1111/bjh.13853. Epub 2015 Nov 16. No abstract available. | |
| 30504345 |
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We can share the plan after completing the experiment
Follow-up and publication of the paper are planned for December 2025
After the paper is written and published
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| ID | Term |
|---|---|
| D000741 | Anemia, Aplastic |
| ID | Term |
|---|---|
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D000080983 | Bone Marrow Failure Disorders |
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| ID | Term |
|---|---|
| C533238 | avatrombopag |
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| First Affiliated Hospital of Harbin Medical University |
| OTHER |
| The First Hospital of Hebei Medical University | OTHER |
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|
Incidence of Treatment-Emergent AE by CTCAE |
| 6months |
| Percentage of patients with transformation | Rate of patients with transformation to PNH or MDS,AML, or other disease | 6months |
| The rate of HR in patients after switching to avatrombopag. | Percentage of the total number of patients receiving treatment who received APAG | 12months |
| ncidence of Treatment-Emergent Adverse Events as assessed by information on Common Toxicity Criteria (CTC) AE grading | Incidence of Treatment-Emergent AE by CTCAE | 12months |
| Percentage of patients with transformation | Rate of patients with transformation to PNH or MDS,AML, or other disease | 12months |
| Result |
| Scheinberg P. Activity of eltrombopag in severe aplastic anemia. Hematology Am Soc Hematol Educ Program. 2018 Nov 30;2018(1):450-456. doi: 10.1182/asheducation-2018.1.450. |
| 28423296 | Result | Townsley DM, Scheinberg P, Winkler T, Desmond R, Dumitriu B, Rios O, Weinstein B, Valdez J, Lotter J, Feng X, Desierto M, Leuva H, Bevans M, Wu C, Larochelle A, Calvo KR, Dunbar CE, Young NS. Eltrombopag Added to Standard Immunosuppression for Aplastic Anemia. N Engl J Med. 2017 Apr 20;376(16):1540-1550. doi: 10.1056/NEJMoa1613878. |
| 22762314 | Result | Olnes MJ, Scheinberg P, Calvo KR, Desmond R, Tang Y, Dumitriu B, Parikh AR, Soto S, Biancotto A, Feng X, Lozier J, Wu CO, Young NS, Dunbar CE. Eltrombopag and improved hematopoiesis in refractory aplastic anemia. N Engl J Med. 2012 Jul 5;367(1):11-9. doi: 10.1056/NEJMoa1200931. |
| 35371427 | Result | Peng G, He G, Chang H, Gao S, Liu X, Chen T, Li P, Han B, Miao M, Ge Z, Ge X, Li F, Li Y, Wang S, Wang Y, Shen Y, Zhang T, Zou J, Zhang F. A multicenter phase II study on the efficacy and safety of hetrombopag in patients with severe aplastic anemia refractory to immunosuppressive therapy. Ther Adv Hematol. 2022 Mar 30;13:20406207221085197. doi: 10.1177/20406207221085197. eCollection 2022. |
| 32876852 | Result | Ise M, Iizuka H, Kamoda Y, Hirao M, Kida M, Usuki K. Romiplostim is effective for eltrombopag-refractory aplastic anemia: results of a retrospective study. Int J Hematol. 2020 Dec;112(6):787-794. doi: 10.1007/s12185-020-02971-1. Epub 2020 Sep 2. |
| D001855 | Bone Marrow Diseases |