High-dose Dexamethasone Plus Hetrombopag vs High-dose Dexamethasone Alone as Frontline Treatment for Newly Diagnosed Adult Primary Immune Thrombocytopenia: A Prospective, Multicenter, Randomized Trial
Study Details
Study Description
Brief Summary
The project was undertaking by Qilu Hospital of Shandong University in China. In order to report the efficacy and safety of Hetrombopag plus high-dose dexamethasone for the treatment of adults with newly-diagnosed primary immune thrombocytopenia (ITP).
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Detailed Description
The investigators anticipate to undertaking a parallel group, randomised controlled trial of 100 ITP patients. One part of the participants are randomly selected to receive hetrombopag with starting dose 5mg po qd for 8 weeks（increase daily dose to a maximum of 7.5 mg/day if platelet count<50000 per μL following at least 2 weeks of treatment) combining with dexamethasone (given at a dose of 40 mg qd for 4 consecutive days). The others are selected to receive high-dose of dexamethasone alone. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study. The purpose of this study is to report the efficacy and safety of Hetrombopag combining with high-dose dexamethasone therapy for the treatment of newly diagnosed ITP.
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Experimental: Hetrombopag plus High-dose Dexamethasone Hetrombopag 5mg po qd; HD-DEX 40mg qd for 4 days |
Drug: hetrombopag 5mg po qd
hetrombopag 5mg po qd for 8 weeks, combining with dexamethasone 40 mg qd for 4 days
Drug: High-dose Dexamethasone
dexamethasone 40 mg qd for 4 days
|
| Active Comparator: High-dose Dexamethasone HD-DEX 40mg qd for 4 days |
Drug: High-dose Dexamethasone
dexamethasone 40 mg qd for 4 days
|
Outcome Measures
Primary Outcome Measures
- 26 week sustained overall response to ITP treatments [26-week after treatment started]
Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.
Secondary Outcome Measures
- 28-day initial complete response to ITP treatment [28 days after treatment started]]
Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.
- 28-day initial overall response to ITP treatment [28 days after treatment started]
Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.
- 8-week complete response to ITP treatment [8 weeks after treatment started]
Complete response was defined as a platelet count of 100 000 per μL or higher and an absence of bleeding. Partial response was defined as a platelet count of 30000 per μL or higher,and at least a doubling of the baseline platelet count and an absence of bleeding. No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.
- 8-week overall response to ITP treatment [8 weeks after treatment started]
No response was defined as a platelet count of less than 30000 per μL, or less than two-times increase from baseline platelet count, or bleeding.
- time to response [an average of 6 months]
the time from treatment initiation to achieve a complete response or a partial response
- duration of response [through study completion, an average of one year]
the time from achievement of a complete response or a partial response to the loss of response
- therapy associated adverse events [through study completion, an average of one year]
Eligibility Criteria
Criteria
Inclusion Criteria:
-
Older than 18 years
-
Meet the diagnostic criteria for newly diagnosed immune thrombocytopenia (diagnosed within 3 month);
-
platelet count <3010^9/L, or < 5010^9/L with bleeding manifestations, both;
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Willing and able to sign written informed consent
Exclusion Criteria:
-
secondary thrombocytopenia or graded MF≥2 myelofbrosis based on the European Consensus Scale
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Previous history of treatment for ITP, except Platelet transfusion, ITP-directed Prednisone therapy no more than 2 weeks or TPO therapy no more than 1 week and stopped ≥1 week before randomization
-
No response to TPO-RA or rhTPO
-
HIV, hepatitis C or B virus infection
-
pregnancy or lactation;
-
arterial or venous thromboembolism within the 6 months before screening
-
total bilirubinalanine, aminotransferase or aspartate transaminase>3×upper limit of normal (ULN), serum creatinine>1.5×ULN
-
congestive heart failure (New York Heart Association [NYHA] class III/IV);
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neoplastic disease within the past 5 years;
-
liver cirrhosis
-
people who could not adhere to the protocol or were planning to have a surgical procedure in 6 months.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- Shandong University
Investigators
None specified.Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- ITP-Hetrombopag plus HD-DEX