A Study of Eltrombopag and Recombinant Human Thrombopoietin In Primary Immune Thrombocytopenia

NCT04214951 | Unknown Results

• 1 other identifier: ZXH-ITP2019
• Study Type: observational
• Target: 100
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Corticosteroid-resistant or Relapsed ITP

Keywords

Immune Thrombocytopenia; Recombinant human thrombopoietin; Eltrombopag; switching

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (5)

• Treatments Associated Adverse Events

  6 weeks

• Reasons of Switching

  6 weeks

• Number of Participants With Bleeding Events

  6 weeks

• TOR (Time to Response)

  6 weeks

• DOR (Duration of Response)

  6 weeks

Study Arms (2)

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.

Drug: Recombinant human thrombopoietin (rh-TPO)

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.

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

Patients will be given eltrombopag 50mg once daily for 6 weeks.

Eltrombopag group

Recombinant human thrombopoietin (rh-TPO) DRUG

Patients will be given rh-TPO 300 U/kg once daily for 21 days.

Recombinant human thrombopoietin (rh-TPO) group

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

adult ITP patients

You may qualify if:

• years or older
• Primary ITP
• Platelet count ≤ 30 × 109/l
• Normal neutrophils, reticulocyte count, creatinine and liver enzyme values
• Available follow-up of 2 months at least for each period
• Failed initial glucocorticosteroid treatment
• Unwillingness to accept splenectomy or failed splenectomy

You may not qualify if:

• HIV, hepatitis B or C, Helicobacter pylori infection
• Malignancy
• Congenital or acquired immunologic deficit
• History of thrombosis plus two or more risk factors
• Nursing or pregnant women
• Abnormal liver and renal functions: AST/ALT/total bilirubin ≥1.5 × ULN, creatinine ≥1.5 mg/dl
• Severe heart and lung dysfunctions -

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

Peking University Institute of Hematology

Beijing, Beijing Municipality, 100044, China

Location

Related Publications (13)

• 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.

  PMID: 21325604 BACKGROUND

• 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.

  PMID: 19846889 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.

  PMID: 22740443 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.

  PMID: 23594368 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.

  PMID: 23821332 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.

  PMID: 24680422 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.

  PMID: 24273485 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.

  PMID: 22753022 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.

  PMID: 19038790 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.

  PMID: 19631713 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.

  PMID: 31156798 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.

  PMID: 28983953 BACKGROUND

• 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.

  PMID: 36535011 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

eltrombopag; Thrombopoietin

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Colony-Stimulating Factors; Glycoproteins; Glycoconjugates; Carbohydrates; Hematopoietic Cell Growth Factors; Cytokines; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Proteins; Biological Factors

Results Point of Contact

• Title: Dr. Xiaohui Zhang, Director of Clinical Trials
• Organization: Peking University People's Hospital

zhangxh@bjmu.edu.cn

86-10-88324577

Study Officials

• Xiao Hui Zhang, MD

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Clinical Professor

Study Record Dates

• First Submitted: December 28, 2019
• First Posted: January 2, 2020
• Study Start: January 1, 2020
• Primary Completion: August 1, 2022
• Study Completion: December 1, 2022
• Last Updated: October 20, 2020
• Results First Posted: October 20, 2020

Record last verified: 2020-10

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)