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Myelodysplastic syndrome (MDS) is a kind of clonal myeloid tumor. The major manifestation is decrease of tri-lineages of blood due to ineffective and abnormal hematopoiesis, some of which can progress to acute myeloid leukemia. According to the international prognosis scoring system (IPSS) of MDS, about 10% low/intermediate risk-1 MDS patients have severe thrombocytopenia (PLT < 30 × 109/ L). These patients have both decreased platelet count and platelet dysfunction, resulting in a high risk of bleeding. In the new prognostic score, such as IPSS-r, the degree of thrombocytopenia is regarded as a poor prognostic factor. Platelet transfusion is mainly used in the treatment of this kind of patients. The indications of transfusion include bleeding events or severe platelet count reduction (< 10 × 109 / L). However, platelet transfusion can only lead to short-term platelet elevation, while repeated transfusion increases the possibility of infection and ineffective platelet transfusion. TPO is a newly discovered hematopoietic promoting factor, which can specifically bind to the TPO receptor on the cell and participate in the regulation of proliferation, differentiation, maturation and division of megakaryocyte to form functional platelet. The efficacy and safety of the TPO receptor agonists eltrombopag and romiplostim in the treatment of thrombocytopenia in low/intermediate risk-1 MDS patients have been successfully confirmed in foreign studies. Recombinant human thrombopoietin (rhTPO) is also a kind of a TPO receptor agonists which is highly specific platelet stimulating factor. At present, there is no large report on the application of rhTPO in such patients. The purpose of this study is to explore the short-term and long-term therapeutic effect and safety of rhTPO on low/intermediate risk-1 MDS patients.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| TPO treatment group | Active Comparator | Danazol 0.2g tid po+rhTPO (recombinant human thrombopoietin injection) 300U/kg/d×14d si every month (stop when PLT≥100×10e9/L or increased more than 50×10e9/L), total course 6 months |
|
| control | Placebo Comparator | Danazol 0.2g tid po+ control (sodium chloride)×14d si every month, total course 6 months |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Danazol + rhTPO (recombinant human thrombopoietin injection) | Drug | subcutaneous injection, 300U/kg/d×14d every month,stop if the PLT≥100×10e9/L or increased >50×10e9/L, total course is 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| overall response rate | overall response rate of platelet | 1 year |
| rate of side effects | rate of all kinds of side effects | 1 year |
| Measure | Description | Time Frame |
|---|---|---|
| WHO bleeding score | to evaluate frequency and severity of bleeding | 1 year |
| change of platelet transfusion | the frequency and amount of platelet transfusion |
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Inclusion Criteria:
Exclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking union medical college hospital | Beijing | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34778037 | Derived | Yang Y, Tang Z, Ji J, Yang C, Chen M, Han B. Recombinant Human Thrombopoietin Accelerates the Recovery of Platelet in Patients With Lower-Risk Myelodysplastic Syndrome: A Proof-of-Concept Study. Front Oncol. 2021 Oct 28;11:721764. doi: 10.3389/fonc.2021.721764. eCollection 2021. |
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protocol, consent form, clinical data would be available by personal contact
10 years
personal contact including emails
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| ID | Term |
|---|---|
| D003613 | Danazol |
| D013926 | Thrombopoietin |
| D012965 | Sodium Chloride |
| ID | Term |
|---|---|
| D011245 | Pregnadienes |
| D011278 | Pregnanes |
| D013256 | Steroids |
| D000072473 | Fused-Ring Compounds |
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| Danazol + sodium chloride | Drug | Danazol 0.2g tid po+ control (sodium chloride) |
|
| 1 year |
| onset time for overall response | onset time for complete and partial response | through study completion, an average of 1 year |
| duration of overall response | during time for complete and partial response | through study completion, an average of 1 year |
| incidence of TPO antibody | rate of presence of TPO antibody | 1 year |
| life quality for MDS patients | life quality for MDS patients by QoL-E questionaire | 1 year |
| the increased number of myeloblasts in bone marrow and peripheral blood | the increased number of myeloblasts in bone marrow and peripheral blood | 1 year |
| incidence of progression to high-risk MDS or leukemia | incidence of progression to high-risk MDS or leukemia | 1 year |
| D011083 |
| Polycyclic Compounds |
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
| D016298 | Hematopoietic Cell Growth Factors |
| D016207 | Cytokines |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| D002712 | Chlorides |
| D006851 | Hydrochloric Acid |
| D017606 | Chlorine Compounds |
| D007287 | Inorganic Chemicals |
| D017670 | Sodium Compounds |