A multicenter, randomized, double-blind placebo-controlled study to report the efficacy and safety of all-trans etinoic acid compared to placebo for the treatment of adults with corticosteriod-resistant/relapsed primary immune thrombocytopenia (ITP).
The investigators are undertaking a parallel-group, multicenter, randomized controlled trial of 192 adults with corticosteriod-resistant/relapsed primary ITP. Patients were randomized to all-trans etinoic acid and placebo group. Platelet count, bleeding, and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
192
10mg twice daily ×24 weeks
10mg twice daily ×24 weeks
Peking University Institute of hematology, People's Hospital
Beijing, China
Durable platelet response
Platelet count of ⩾50 × 10\^9/L or between ⩾30 × 10\^9/L and \<50 × 10\^9/L and at least doubled from baseline on at least four of six scheduled visits between weeks 14 and 24
Time frame: Up to week 24
24-week overall response rate
From enrollment to the end of week 24, the proportion of patients with at least one platelet count of ≥50×10⁹/L or between 30 × 10\^9/L and 50 × 10\^9/L plus at least doubled from baseline
Time frame: Up to week 24
12-week overall response rate
From enrollment to the end of week 12, the proportion of patients with at least one platelet count of ≥50×10⁹/L or between 30 × 10\^9/L and 50 × 10\^9/L plus at least doubled from baseline
Time frame: Up to week 12
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 30×10^9/L)
The proportion of participants who achieved platelet counts ≥ 30×10\^9/L and at least doubled from baseline at two consecutive visits
Time frame: Up to week 24
Consecutive Increased Platelet Counts (≥2 Consecutive PLT ≥ 50×10^9/L)
The proportion of patients with two consecutive platelet counts of 50×10⁹/L or more and doubling from the baseline count
Time frame: Up to week 24
Quality of Life Score
ITP-patient assessment questionnaire (ITP-PAQ) was used to assess the HRQoL before and after treatment.
Time frame: Up to week 24
Adverse Events
The rate of participants with adverse events
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Time frame: Up to week 28
Complete response rate
The proportion of patients with a platelet count of ≥ 100×10\^9/L in absence of bleeding at any time
Time frame: Up to week 24
Duration of response
Number of weeks with platelet count of ≥50 × 10\^9/L or between 30 × 10\^9/L and 50 × 10\^9/L plus at least doubled from baseline
Time frame: Up to 24 weeks
Time to response
Time from treatment initiation to first platelet count reaching ≥30x10\^9/L and doubling from the baseline count in 0-24 weeks
Time frame: Up to week 24
Initial response
Platelet count ≥30×10\^9/L and at least doubling baseline at day 28
Time frame: Up to week 4
Peak platelet count
The peak platelet count without rescue treatment
Time frame: Up to week 24
Bleeding events
Bleeding incidence and severity per WHO bleeding score in 0-12 weeks and 0-24 weeks
Time frame: 0-12 weeks and 0-24 weeks
Rescue treatment
Proportion of patients receiving predefined rescue treatment
Time frame: Up to week 24
Reduced or discontinued concomitant therapy
Proportion of patients with reduced or discontinued baseline concomitant anti-ITP therapy
Time frame: Up to week 24