Single-arm, open-label, single-center study to evaluate the efficacy and safety of teriflunomide for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).
This is a prospective, single-arm, open-label study of teriflunomide in approximately 40 patients with ITP who have relapsed or have an insufficient response to prior therapies. Teriflunomide is administered as 7 mg orally once daily with dose adjustments for 24 weeks. Efficacy and safety outcomes are assessed on scheduled study visits.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
40
Starting dose of 7 mg once daily. Dose adjustments were made throughout the study based on individual platelet counts.
Peking University Insititute of Hematology, Peking University People's Hospital
Beijing, 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
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
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
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.
Time frame: From the start of study treatment (Day 1) to the end of week 24
Health-related quality of life (HRQoL)
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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