Immune Thrombocytopenia
Conditions
Brief summary
An open-label, multicenter study to compare the efficacy and safety of ATRA for the treatment of adults with primary immune thrombocytopenia (ITP)
Detailed description
The investigators are undertaking an open-label, single-arm, multicenter trial of adults patients with ITP in China. Patients with treatmant naive ITP were given ATRA treatment. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Interventions
ATRA is given concomitantly at a daily dose of 10 mg twice daily orally for 12 weeks
Sponsors
Qilu Hospital of Shandong University
Beijing Hospital
Navy General Hospital, Beijing
Peking University People's Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
ATRA is used to treat ITP patients.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
1. Confirmed newly-diagnosed, treatment-naive ITP; 2. Platelet counts \<30×109/L ; 3. Platelet counts \< 50×109/L and significant bleeding symptoms (WHO bleeding scale 2 or above); 4. Willing and able to sign written informed consent.
Exclusion criteria
1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 3 months before the screening visit; 2. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) within 3 months before the screening visit; 3. Received high-dose steroids or IVIG in the 3 weeks prior to the start of the study. 4. Current HIV infection or hepatitis B virus or hepatitis C virus infections; 5. Severe medical condition (lung, hepatic or renal disorder) other than chronic ITP. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia); 6. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; 7. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test; 8. Patients who are deemed unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sustained response | 6 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 6-month follow-up |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Response (R) | day 14 | 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. |
| Number of patients with bleeding | 6 months | Number of patients with bleeding complication ( WHO bleeding score). |
| Number of patients with adverse events | 6 months | Number of patients with adverse events |
| complete response (CR) | day 14 | complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding. |
| Duration of response (DOR) | 6 months | Duration of response at 6-month follow up. |
| loss of response | 6 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) |
| Time to response | 6 months | The time from starting treatment to time of achievement of CR or R |
Countries
China
Contacts
Primary ContactXiaohui Zhang, Doctor
Outcome results
None listed