Immune Thrombocytopenia
Conditions
Keywords
Immune Thrombocytopenia, tacrolimus, danazol
Brief summary
Randomized, open-label, multicenter study to compare the efficacy and safety of combination of tacrolimus and danazol versus danazol for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
Detailed description
The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Interventions
DRUGTAC
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.
DRUGDanazol
Danazol is given at 200mg bid for 12 weeks.
Sponsors
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
The investigators are undertaking a randomized controlled trial of 120 adults with steroid-resistant/ relapse ITP in China. Patients were randomized to tacrolimus plus danazol and danazol monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
Inclusion criteria
* Primary immune thrombocytopenia (ITP); * 18 years older; * Platelet count of less than 30×10\^9/L at enrollment; * 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; * Patients who were receiving other maintenance regimens, primarily corticosteroids, ciclosporin, or mycophenolate mofetil, were also eligible if the dose of treatment had been stable in the past month and the dose was expected to be stable after enrolment and remained unchanged at least for the first 4 weeks of study until initial response was assessed, unless severe adverse events were suspected.
Exclusion criteria
* Secondary ITP (e.g., patients with HIV, HBV, HCV, Helicobacter pylori infection or SLE); * Congestive heart failure, severe arrhythmia; * Nursing or pregnant women; * ALT or AST levels ≥ 3× the upper limit of the normal threshold; * Creatinine or serum bilirubin levels ≥ 1.5× the upper limit; * Active or previous malignancy ; * Patients who had received danazol treatment or did not respond to danazol; * Patients unable to have routine blood tests because of reasons such as insufficient time.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sustained response | 6 months | Sustained response was defines as the maintenance of platelet count ≥ 30 x 10\^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication at the 6-month follow-up. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Complete response | 6 months | Complete response was defines as the maintenance of platelet count ≥ 100 x 10\^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication. |
| Response | 6 months | Response was defines as the maintenance of platelet count ≥ 30 x 10\^9/L, and at least 2-fold increase of the baseline count, and the absence of bleeding, and no need for rescue medication. |
| Time to response | 6 months | Time to response was defined as the time from starting treatment to the time to achieve the response. |
| Duration of response | 6 months | Duration of response was measured from the achievement of response to the loss of response. |
| Adverse events | 6 months | Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0. |
Countries
China
Contacts
Primary ContactXiao-Hui Zhang, MD
Backup ContactXuan Cai, MD
Outcome results
None listed