Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Thrombopoietin Receptor Agonists (TPO-ra) are novel treatments for patients with refractory Primary Immune Thrombocytopenia (ITP). Rh-TPO and eltrombopag increase the number of platelets through different mechanism. If there is cross-resistance between 2 drugs for the treatment of adult ITP is still no answer. The purpose of this study is to investigate the efficacy and safety of switching eltrombopag and Rh-TPO in adults with ITP.
Non-interventional study. Patients who fail previous steroids and receive rh-TPO and then switch to EPAG or vice versa will be enrolled. The reason for switch will be recorded. Patients in the rh-TPO group were given rh-TPO 300 U/kg once daily for 21 days, and those in the eltrombopag group were given eltrombopag 50mg once daily for 6 weeks. Rh-TPO and eltrombopag were terminated any time the platelet counts increased above 100 × 10^9/L in the rh-TPO group and 300 × 10^9/L in the eltrombopag group. The efficacy, safety, and patient/physician preference will be assessed and compared between the two agents.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Recombinant human thrombopoietin (rh-TPO) group | Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. |
| |
| Eltrombopag group | Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Eltrombopag | Drug | Patients will be given eltrombopag 50mg once daily for 6 weeks. |
| |
| Measure | Description | Time Frame |
|---|---|---|
| Response Rate at 6 Weeks After Switching | The percentage of patients who have reached platelet count ≥ 50×10^9/L at 6 weeks after switching. | 6 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Treatments Associated Adverse Events | Adverse event/serious adverse event associated with study drugs during 6 weeks after switching | 6 weeks |
| Reasons of Switching | Reasons of switching eltrombopag and rh-TPO will be recorded, including lack of efficacy, patient preference, side effects, platelet count fluctuation |
Not provided
Inclusion Criteria:
1.18 years or older
2.Primary ITP
3.Platelet count ≤ 30 × 109/l
4.Normal neutrophils, reticulocyte count, creatinine and liver enzyme values
5.Available follow-up of 2 months at least for each period
6.Failed initial glucocorticosteroid treatment
7.Unwillingness to accept splenectomy or failed splenectomy
-
Exclusion Criteria:
Not provided
Not provided
adult ITP patients
Not provided
| Name | Affiliation | Role |
|---|---|---|
| Xiao Hui Zhang, MD | Peking University People's Hospital | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Peking University Institute of Hematology | Beijing | Beijing Municipality | 100044 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 21325604 | Background | Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA; American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011 Apr 21;117(16):4190-207. doi: 10.1182/blood-2010-08-302984. Epub 2011 Feb 16. | |
| 19846889 | Background |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | Eltrombopag Group | Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. Eltrombopag: Patients will be given eltrombopag 50mg once daily for 6 weeks. |
| FG001 | Recombinant Human Thrombopoietin (Rh-TPO) Group | Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. Recombinant human thrombopoietin (rh-TPO): Patients will be given rh-TPO 300 U/kg once daily for 21 days. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | Recombinant Human Thrombopoietin (Rh-TPO) Group | Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. Recombinant human thrombopoietin (rh-TPO): Patients will be given rh-TPO 300 U/kg once daily for 21 days. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Median |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Response Rate at 6 Weeks After Switching | The percentage of patients who have reached platelet count ≥ 50×10^9/L at 6 weeks after switching. | Posted | Count of Participants | Participants | 6 weeks |
|
6 weeks
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Eltrombopag Group | Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. Eltrombopag: Patients will be given eltrombopag 50mg once daily for 6 weeks. |
Not provided
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Elevated liver enzymes | Hepatobiliary disorders | Systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Xiaohui Zhang, Director of Clinical Trials | Peking University People's Hospital | 86-10-88324577 | zhangxh@bjmu.edu.cn |
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP_ICF | Yes | Yes | Yes | Study Protocol, Statistical Analysis Plan, and Informed Consent Form | Aug 13, 2020 | Aug 13, 2020 | Prot_SAP_ICF_000.pdf |
Not provided
| ID | Term |
|---|---|
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C520809 | eltrombopag |
| D013926 | Thrombopoietin |
| ID | Term |
|---|---|
| D003115 | Colony-Stimulating Factors |
| D006023 | Glycoproteins |
| D006001 | Glycoconjugates |
| D002241 | Carbohydrates |
Not provided
Not provided
Not provided
Not provided
Not provided
| Recombinant human thrombopoietin (rh-TPO) |
| Drug |
Patients will be given rh-TPO 300 U/kg once daily for 21 days. |
|
| 6 weeks |
| Number of Participants With Bleeding Events | Number of participants with bleeding events of the two groups during 6 weeks after switching | 6 weeks |
| TOR (Time to Response) | The time to achieve platelet count ≥ 50×10^9/L after switching. | 6 weeks |
| DOR (Duration of Response) | The duration of achieving platelet count ≥ 50×10^9/L after switching. | 6 weeks |
| Provan D, Stasi R, Newland AC, Blanchette VS, Bolton-Maggs P, Bussel JB, Chong BH, Cines DB, Gernsheimer TB, Godeau B, Grainger J, Greer I, Hunt BJ, Imbach PA, Lyons G, McMillan R, Rodeghiero F, Sanz MA, Tarantino M, Watson S, Young J, Kuter DJ. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010 Jan 14;115(2):168-86. doi: 10.1182/blood-2009-06-225565. Epub 2009 Oct 21. |
| 22740443 | Background | Ghanima W, Godeau B, Cines DB, Bussel JB. How I treat immune thrombocytopenia: the choice between splenectomy or a medical therapy as a second-line treatment. Blood. 2012 Aug 2;120(5):960-9. doi: 10.1182/blood-2011-12-309153. Epub 2012 Jun 26. |
| 23594368 | Background | Deutsch VR, Tomer A. Advances in megakaryocytopoiesis and thrombopoiesis: from bench to bedside. Br J Haematol. 2013 Jun;161(6):778-93. doi: 10.1111/bjh.12328. Epub 2013 Apr 18. |
| 23821332 | Background | Kuter DJ. The biology of thrombopoietin and thrombopoietin receptor agonists. Int J Hematol. 2013 Jul;98(1):10-23. doi: 10.1007/s12185-013-1382-0. Epub 2013 Jul 3. |
| 24680422 | Background | Bussel JB, Lakkaraja M. Thrombopoietic agents: there is still much to learn. Presse Med. 2014 Apr;43(4 Pt 2):e69-78. doi: 10.1016/j.lpm.2014.02.008. Epub 2014 Mar 27. |
| 24273485 | Background | Wormann B. Clinical indications for thrombopoietin and thrombopoietin-receptor agonists. Transfus Med Hemother. 2013 Oct;40(5):319-25. doi: 10.1159/000355006. Epub 2013 Sep 11. |
| 22753022 | Background | Wang S, Yang R, Zou P, Hou M, Wu D, Shen Z, Lu X, Li Y, Chen X, Niu T, Sun H, Yu L, Wang Z, Zhang Y, Chang N, Zhang G, Zhao Y. A multicenter randomized controlled trial of recombinant human thrombopoietin treatment in patients with primary immune thrombocytopenia. Int J Hematol. 2012 Aug;96(2):222-8. doi: 10.1007/s12185-012-1124-8. Epub 2012 Jun 30. |
| 19038790 | Background | Erickson-Miller CL, Delorme E, Tian SS, Hopson CB, Landis AJ, Valoret EI, Sellers TS, Rosen J, Miller SG, Luengo JI, Duffy KJ, Jenkins JM. Preclinical activity of eltrombopag (SB-497115), an oral, nonpeptide thrombopoietin receptor agonist. Stem Cells. 2009 Feb;27(2):424-30. doi: 10.1634/stemcells.2008-0366. |
| 19631713 | Background | Erhardt JA, Erickson-Miller CL, Aivado M, Abboud M, Pillarisetti K, Toomey JR. Comparative analyses of the small molecule thrombopoietin receptor agonist eltrombopag and thrombopoietin on in vitro platelet function. Exp Hematol. 2009 Sep;37(9):1030-7. doi: 10.1016/j.exphem.2009.06.011. Epub 2009 Jul 24. |
| 31156798 | Background | Gonzalez-Porras JR, Godeau B, Carpenedo M. Switching thrombopoietin receptor agonist treatments in patients with primary immune thrombocytopenia. Ther Adv Hematol. 2019 May 9;10:2040620719837906. doi: 10.1177/2040620719837906. eCollection 2019. |
| 28983953 | Background | Cantoni S, Carpenedo M, Mazzucconi MG, De Stefano V, Carrai V, Ruggeri M, Specchia G, Vianelli N, Pane F, Consoli U, Artoni A, Zaja F, D'adda M, Visentin A, Ferrara F, Barcellini W, Caramazza D, Baldacci E, Rossi E, Ricco A, Ciminello A, Rodeghiero F, Nichelatti M, Cairoli R. Alternate use of thrombopoietin receptor agonists in adult primary immune thrombocytopenia patients: A retrospective collaborative survey from Italian hematology centers. Am J Hematol. 2018 Jan;93(1):58-64. doi: 10.1002/ajh.24935. Epub 2017 Nov 9. |
| 36535011 | Derived | Cai X, Fu H, Zhao X, Lu J, Jiang Q, Chang Y, Huang X, Zhang X. Switching between eltrombopag and recombinant human thrombopoietin in patients with immune thrombocytopenia: an observational study. Chin Med J (Engl). 2022 Oct 5;135(19):2344-2350. doi: 10.1097/CM9.0000000000002346. |
| BG001 | Eltrombopag Group | Patients who fail previous steroids and rh-TPO and then switch to eltrombopag will be enrolled. The reason for switch will be recorded. Patients will be given eltrombopag 50mg once daily for 6 weeks. Eltrombopag will be terminated any time the platelet counts increased above 300× 10^9/L.The efficacy, safety, and patient/physician preference will be assessed. Eltrombopag: Patients will be given eltrombopag 50mg once daily for 6 weeks. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed.
Recombinant human thrombopoietin (rh-TPO): Patients will be given rh-TPO 300 U/kg once daily for 21 days.
|
|
| Secondary | Treatments Associated Adverse Events | Adverse event/serious adverse event associated with study drugs during 6 weeks after switching | Posted | Number | participants | 6 weeks |
|
|
|
| Secondary | Reasons of Switching | Reasons of switching eltrombopag and rh-TPO will be recorded, including lack of efficacy, patient preference, side effects, platelet count fluctuation | Posted | Count of Participants | Participants | 6 weeks |
|
|
|
| Secondary | Number of Participants With Bleeding Events | Number of participants with bleeding events of the two groups during 6 weeks after switching | Posted | Count of Participants | Participants | 6 weeks |
|
|
|
| Secondary | TOR (Time to Response) | The time to achieve platelet count ≥ 50×10^9/L after switching. | Posted | Mean | Full Range | weeks | 6 weeks |
|
|
|
| Secondary | DOR (Duration of Response) | The duration of achieving platelet count ≥ 50×10^9/L after switching. | Posted | Mean | Full Range | weeks | 6 weeks |
|
|
|
| 0 |
| 10 |
| 0 |
| 10 |
| 1 |
| 10 |
| EG001 | Recombinant Human Thrombopoietin (Rh-TPO) Group | Patients who fail previous steroids and eltrombopag and then switch to Rh-TPO will be enrolled. The reason for switch will be recorded. Patients will be given rh-TPO 300 U/kg once daily for 21 days. Rh-TPO will be terminated any time the platelet counts increased above 100 × 10^9/L. The efficacy, safety, and patient/physician preference will be assessed. Recombinant human thrombopoietin (rh-TPO): Patients will be given rh-TPO 300 U/kg once daily for 21 days. | 0 | 10 | 0 | 10 | 1 | 10 |
| fever | General disorders | Systematic Assessment |
|
Not provided
Not provided
| 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 |
| 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 |
| side effects |
|
| platelet count fluctuation |
|