Immune Thrombocytopenia
Conditions
Keywords
Teriflunomide, Danazol
Brief summary
To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP
Detailed description
This is a prospective, multicenter, randomized, controlled trial of 124 adult patients with steroid-resistant/relapse ITP in China. Patients were randomized to receive either experimental teriflunomide plus danazol or active comparator danazol monotherapy. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patient's request.
Interventions
DRUGTeriflunomide
Starting dose of 7 mg once daily. Dose adjustments are made throughout the study based on individual platelet counts.
DRUGDanazol
200 mg twice daily.
Sponsors
Beijing Luhe Hospital
Chinese PLA General Hospital
Navy General Hospital, Beijing
Beijing Hospital
Beijing Friendship Hospital
Peking University First Hospital
Peking University Third Hospital
China-Japan Friendship Hospital
Beijing Tsinghua Changgeng Hospital
Peking University People's Hospital
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Patients were randomly assigned at a 1:1 ratio to receive teriflunomide plus danazol or danazol. Each group requires 62 patients (considering a 10% drop-off).
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia; 2. 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; 3. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment; 4. Willing and able to sign written informed consent.
Exclusion criteria
1. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection, or patients with confirmed autoimmune disease); 2. Active or a history of malignancy; 3. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than two times the upper limit of normal (ULN); 4. Pregnancy or lactation; 5. Current or recent (\< 4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection; 6. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV); 7. Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB; 8. Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled; 9. Patients who are deemed unsuitable for the study by the investigator.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Sustained response | From the start of study treatment (Day 1) to the end of week 24 | Platelet count over 30,000/μL and at least a 2-fold increase of the baseline count in the absence of bleeding and rescue therapy for at least four of the six visits between weeks 19 and 24. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to response | From the start of study treatment (Day 1) to the end of week 24 | The time from treatment initiation to achieve a CR or a R. |
| Duration of response | From the start of study treatment (Day 1) to the end of week 24 | The time from the achievement of a complete response or a partial response to the loss of response. |
| Initial response | From the start of study treatment (Day 1) up to week 4 of treatment | The number of participants with achievement of CR or R at 4 weeks. |
| Overall response | From the start of study treatment (Day 1) to the end of week 24 | Complete response (CR) was defined as platelet count over 100,000/μL and absence of bleeding. Response (R) was defined as platelet count over 30,000/μL and at least a 2-fold increase of the baseline count and absence of bleeding. |
| Adverse events | From the start of study treatment (Day 1) to the end of week 24 | Adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0. |
| Health-related quality of life (HRQoL) | From the start of study treatment (Day 1) to the end of week 24 | ITP-PAQ was used to assess the Health-Related Quality of Life (HRQoL) before and after treatment. |
| Bleeding events | From the start of study treatment (Day 1) to the end of week 24 | Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale. |
Countries
China
Contacts
Primary ContactXiao-Hui Zhang, MD
Backup ContactLi-Ping Yang, MD
Outcome results
None listed