To compare the efficacy and safety of teriflunomide plus danazol versus danazol in patients with steroid-resistant/relapse ITP
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.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
124
Starting dose of 7 mg once daily. Dose adjustments are made throughout the study based on individual platelet counts.
200 mg twice daily.
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, China
RECRUITINGSustained response
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.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Overall response
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.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Time to response
The time from treatment initiation to achieve a CR or a R.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Duration of response
The time from the achievement of a complete response or a partial response to the loss of response.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Initial response
The number of participants with achievement of CR or R at 4 weeks.
Time frame: From the start of study treatment (Day 1) up to week 4 of treatment
Bleeding events
Clinically significant bleeding was assessed using the World Health Organization (WHO) bleeding scale.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Adverse events
Adverse events (AEs) were reported and graded according to the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
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Time frame: From the start of study treatment (Day 1) to the end of week 24
Health-related quality of life (HRQoL)
ITP-PAQ was used to assess the Health-Related Quality of Life (HRQoL) before and after treatment.
Time frame: From the start of study treatment (Day 1) to the end of week 24