Randomized, open-label, multicenter study to compare the efficacy and safety of zanubrutinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were randomized to Zanubrutinib + high-dose dexamethasone and high-dose dexamethasone monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
150
Zanubrutinib 80mg qd po, 6 consecutive weeks
Dexamethasone, iv, 40 mg/d, for 4 days (The 4-day course of dexamethasone was repeated in the case of lack of response by day 10)
Sustained response
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
Time frame: 12 months
Initial complete response
Initial complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding.
Time frame: 1 month
Initial response
Initial response (R) as platelet count more than 30,000 per cubic millimeter and at least 2-fold increase of the baseline count and absence of bleeding.
Time frame: 1 month
Number of patients with bleeding
Number of patients with bleeding complication ( WHO bleeding score).
Time frame: 12 months
Number of patients with adverse events
Number of patients with adverse events
Time frame: 12 months
Time to response (TTR)
The time from starting treatment to time of achievement of CR or R
Time frame: 12 months
Duration of response (DOR)
Duration of response at 12-month follow up
Time frame: 12 months
Loss of response
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)
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Time frame: 12 month
Durable response
Platelet count ≥30 X 10\^9/L and at least doubling of the baseline count at 6 mo
Time frame: 6 months
Early response
Platelet count ≥30 X 10\^9/L and at least doubling baseline at 1 wk
Time frame: 1 week
Remission
Platelet count \>100 X 10\^9/L at 12 month
Time frame: 12 months