Immune Thrombocytopenia
Conditions
Keywords
BTK inhibitor, Thrombopoietin-receptor agonist
Brief summary
Randomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus eltrombopag compared to eltrombopag monotherapy for the first-line treatment of adults with chronic immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a prospective trial of patients with ITP in China. Zanubrutinib is administered as 80 mg po. qd for 6 weeks. Eltrombopag is given with 50 mg once daily for up to 6 weeks. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study. This study assessed the efficacy and safety of eltrombopag plus zanubrutinib.
Interventions
DRUGZanubrutinib
Zanubrutinib 80mg po qd 6 weeks
DRUGEltrombopag
eltrombopag 50 mg qd for up to 6 weeks. The eltrombopag dose could be increased from 50 mg to 75 mg after 3 weeks in patients whose platelet counts were less than 50 000 per μL. Treatment was discontinued in patients who attained a platelet count greater than 200 000 per μL.
Sponsors
Beijing Hospital
Navy General Hospital, Beijing
Beijing Aerospace General Hospital
Qilu Hospital of Shandong University
Beijing Tongren Hospital
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia; 2. Patients with chronic low platelet count (less than 30,000/µL) for 6 months who have failed at least one treatment for chronic low platelet count 2\. Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation 3. Willing and able to sign written informed consent
Exclusion criteria
1. Secondary immune thrombocytopenia (e.g., patients with HIV, HCV, Helicobacter pylori infection or patients with systemic lupus erythematosus) 2. congestive heart failure 3. severe arrhythmia 4. nursing or pregnant women 5. aspartate aminotransferase and alanine transaminase levels ≥ 3× the upper limit of the normal threshold criteria 6. creatinine or serum bilirubin levels each 1•5 times or more than the normal range 7. active or previous malignancy 8. Unable to do blood routine test for the sake of time, distance, economic issues or other reasons 9. History of clotting disorder
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment response | 6 weeks | The proportion of patients who had an increase in platelet counts to 50 000 per μL or more after 6 weeks of treatment |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete response (CR) | 6 weeks | Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding |
| Time to response | 6 weeks | The time from starting treatment to time of achievement of Response |
| Sustained response | 12 month | The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 12-month follow-up |
| Overall response | 6 weeks | The maintenance of platelet count ≥ 30 x 10\^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication after 6 weeks of treatment |
| Number of patients with adverse events | 12 months | Number of patients with adverse events |
| Duration of response (DOR) | 12 months | Duration of response at 12-month follow up |
| Loss of response | 12 months | Platelet counts below 100 x 109/L or bleeding (from CR) or platelet counts below 30 x 109/L, less than 2-fold increase of baseline platelet count or bleeding (from R) |
| Number of patients with bleeding | 12 months | Number of patients with bleeding complication ( WHO bleeding score) |
Countries
China
Contacts
Primary ContactXiaohui Zhang, MD
Backup ContactQiusha Huang, MD
Outcome results
None listed