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

Study Overview
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Brief Summary
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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.

Detailed Description
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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.

Official Title
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A Prospective Observational Study of Switching Eltrombopag and Recombinant Human Thrombopoietin In Primary Immune Thrombocytopenia

Conditions
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Corticosteroid-resistant or Relapsed ITP

Intervention / Treatment
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* Drug: Eltrombopag
* Drug: Recombinant human thrombopoietin (rh-TPO)


Participation Criteria
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Eligibility Criteria
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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: 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 -

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Recombinant human thrombopoietin (rh-TPO) group<br>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)<br>* Patients will be given rh-TPO 300 U/kg once daily for 21 days.<br>|
| Eltrombopag group<br>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<br>* Patients will be given eltrombopag 50mg once daily for 6 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | 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 | 

Secondary Outcome Measures

| Outcome Measure | 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 | 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Eltrombopag, Immune Thrombocytopenia, Recombinant human thrombopoietin, switching