Immune Thrombocytopenia
Conditions
Keywords
Teriflunomide, Dexamethasone
Brief summary
A randomized, open-label, multicenter study to compare the efficacy and safety of teriflunomide plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with newly diagnosed primary immune thrombocytopenia (ITP).
Detailed description
This is a parallel-group, multicenter, randomized controlled trial of 132 adults with ITP in China. Patients were randomized to teriflunomide plus high-dose dexamethasone and high-dose dexamethasone monotherapy group. Patients who do not respond to dexamethasone may receive another cycle of high-dose dexamethasone therapy within 2 weeks. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Interventions
DRUGTeriflunomide
Teriflunomide 7 mg orally once daily for 24 weeks. Dose adjustments were made throughout the study based on individual platelet counts.
DRUGDexamethasone
Dexamethasone 40 mg orally once daily for four consecutive days (the 4-day course of dexamethasone was repeated in the case of lack of response by day 14).
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
Eligibility
Sex/Gender
ALL
Age
18 Years to 80 Years
Healthy volunteers
No
Inclusion criteria
1. Newly diagnosed, treatment naïve ITP patients 2. Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with bleeding manifestations at the enrollment; 3. Willing and able to sign written informed consent.
Exclusion criteria
1. Received first-line and second-line ITP-modifying therapy (any previous dose of corticosteroids or other immune-suppressive agents); 2. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit; 3. Active or a history of malignancy; 4. Positive test result for hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV); 5. Pregnancy or lactation; 6. Pre-existing acute or chronic liver disease, or serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN); 7. Current or recent (\<4 weeks before screening) clinically serious viral, bacterial, fungal, or parasitic infection; 8. 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 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 follow-up | 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-patient assessment questionnaire (ITP-PAQ) was used to assess the 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
Outcome results
None listed