A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag 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
96
ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.
The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
Beijing Tongren hospital
Beijing, Beijing Municipality, China
Department of Hematology, Beijing Friendship Hospital, Capital Medical University
Beijing, China
Department of Hematology, Beijing Hospital
Beijing, China
Sustained Response Rate (SR) at 18 months
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 18-month follow-up
Time frame: 18 months
Complete response rate (CR)
The complete response (CR) was defined as platelet count more than 100×10\^9/L and absence of bleeding.
Time frame: 18 months
Response rate (R)
The Response rate (R) was defined as platelet count more than 30×10\^9/L and more than 2 times higher than baseline, without bleeding
Time frame: 18 months
Inefficiency (NR)
Platelet count \<30×10\^9/L, or less than 2-fold increase from baseline or associated with bleeding
Time frame: 18 months
Recurrence rate (relapse)
After the treatment is effective, the platelet count drops below 30×10\^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur
Time frame: 18 months
Early response
Platelet count ≥30×10\^9/L and at least doubling baseline at 1 wk.
Time frame: 1 week
Initial response
Initial response as platelet count more than 30×10\^9/L and at least 2-fold increase of the baseline count and absence of bleeding.
Time frame: 1 month
Time to response (TTR)
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Department of Hematology, Senior Department of Hematology, the Fifth Medical Center of PLA General Hospital
Beijing, China
The time from starting treatment to time of achievement of CR or R
Time frame: 18 months
Time to relapse (duration of efficacy)
The time from achievement of CR or R to time of relapse
Time frame: 18 months