Immune Thrombocytopenia, ITP
Conditions
Brief summary
Randomized, open-label, multicenter study to compare the efficacy and safety of Combination of High-dose Dexamethasone and Tacrolimus versus High-dose Dexamethasone for the first-line treatment of adults with primary immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a parallel group, multicenter, randomized controlled trial of 100 adults with ITP in China. Patients were randomized to tacrolimuis plus 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.
Interventions
DRUGDexamethasone
Dexamethasone 40 mg per day, 4 consecutive days (the 4-day course of dexamethasone is repeated in the case of lack of response by day 14).
DRUGTacrolimus
Tacrolimus is given at a dose of 0.03mg/kg·d, and the dose is adjusted to maintain the trough concentration of tacrolimus at approximately 3-5 ng/mL for 12 weeks.
Sponsors
Beijing Hospital
Beijing Friendship Hospital
China-Japan Friendship Hospital
The First Affiliated Hospital of Zhengzhou University
First Affiliated Hospital of Chongqing Medical University
Shanxi Bethune 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. 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 6 months before the screening visit; 2. Received first-line and second-line ITP-specific treatments (eg, steriods, cyclophosphamide, 6-mercaptopurine, vincristine, vinblastine, etc) ; 3. Current HIV infection or hepatitis B virus or hepatitis C virus infections; 4. Active infection; 5. Maligancy; 6. 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); 7. Female patients who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period; a history of clinically significant adverse reactions to previous corticosteroid therapy 8. 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; 9. Patients who are deemed unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sustained（Durable） 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. |
| Time to response | 6 months | The time from starting treatment to time of achievement of CR or R. |
| 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) |
| Complete response (CR) | Day 14 | Complete response (CR) was defined as platelet count more than 100,000 per cubic millimeter and absence of bleeding. |
| Immune Thrombocytopenia Patient Assessment Questionnaire (ITP-PAQ) | Time Frame: From the start of study treatment (Day 1) up to the end of week 24. | In all participants ,use ITP-PAQ to assess the Health Related Quality of Life(HRQoL) before and after treatment. |
| Functional Assessment of Chronic Illness Therapy fatigue subscale (FACIT-F) | Time Frame: From the start of study treatment (Day 1) up to the end of week 24. | In all participants ,use FACIT-F to assess the Health Related Quality of Life(HRQoL) before and after treatment. |
| Number of Participants with side effects of the drugs | Time Frame: From the start of study treatment (Day 1) up to the end of week 24. | Side effects of the drugs included fever, headache, serum disease, infection, hypotension, rashes, infection liver injury, hypokalaemia, etc. |
| Number of participants with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale | From the start of study treatment (Day 1) up to the end of week 24. | The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss. |
Countries
China
Outcome results
None listed