This study is a prospective, multicenter, randomized controlled study, planning to enroll 110 ITP patients who failed to respond to conventional-dose rhTPO (300 IU/kg/d) after 14 days of treatment (PLT \< 30×10⁹/L). After a 2-week washout period, they will be randomized to the rhTPO double-dose group (Group A) and EPAG-pfos group (Group B), with blood routine monitored weekly and doses adjusted according to platelet levels, comparing the response rates of the two groups at 6 weeks after switching treatment.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
112
rhTPO (Shenyang Sunshine Pharmaceutical Co., Ltd., National Medical Product Approval No. S20050048, specification 15000U/ml), starting dose 600 IU/kg/d, continuous subcutaneous injection, blood routine monitored weekly, dose adjusted according to platelet levels.
EPAG-PFOS (Shenyang Sunshine Pharmaceutical Co., Ltd.; 25 mg, calculated as C₂₅H₂₂N₄O₄) * Drug-Food Interactions: Administer at least 2 hours before or 4 hours after antacids, dairy products, or cationic mineral supplements. * Dosing: Initiate at 50 mg once daily; adjust the dose in 25 mg increments (not to exceed 75 mg/day) or modify dosing frequency every 2 weeks based on platelet count.
Platelet Response Rate (RR) at 6 weeks after switching treatment: proportion of patients with PLT ≥ 30×10⁹/L, at least 2-fold increase from baseline platelet count, and no bleeding manifestations
This primary endpoint was defined as the proportion of patients who met the "platelet response" criteria at the 6th week (±1 days) after switching from baseline treatment to the intervention plan of this study. "Platelet response" requires the simultaneous satisfaction of the following three conditions: 1) Platelet count ≥ 30 × 10⁹/L; 2) The platelet count has increased by at least twice compared to the baseline value; 3) There were no bleeding events requiring medical intervention or having clinical significance (WHO bleeding grade 0-1). The calculation formula is: (Number of patients achieving response/total number of patients conforming to the protocol analysis set) × 100%.
Time frame: 6th week after treatment conversion (day 42, visitation window allowed: Day 41 to day 43)
Proportion of patients with at least one PLT ≥ 50×10⁹/L at week 6
This indicator assesses the proportion of patients who reach the clinically significant platelet count threshold early after switching to the study intervention protocol. The specific definition is: the percentage of patients with a measured platelet count of ≥ 50 × 10⁹/L in the visit at the 6th week (day 42, with the visiting window being days 41 to 43) after treatment conversion, among the total number of patients in the protocol analysis set. This threshold (50×10⁹/L) is generally regarded as significantly reducing the risk of spontaneous bleeding and is one of the important criteria for evaluating treatment response. Calculation formula: (Number of patients with platelet count ≥ 50×10⁹/L at the 6th week visit/total number of patients conforming to the protocol analysis set) × 100%.
Time frame: Week 6 after treatment conversion (Day 42, visitation window allowed: Day 41 to day 43)
Time to Response (TTR)
The time from the initiation of treatment switching to the first PLT count ≥30×10⁹/L (in days) was recorded. For patients who did not achieve CR or R by the end of the study, the last efficacy assessment date was used as the censored date.
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Time frame: The time from the initiation of treatment switching to the first PLT count ≥30×10⁹/L (in days) was recorded. Visiting window allowed: Days 1 to 42.
Sustained Response Rate (SRR)
Proportion of patients who, after achieving initial response and without other ITP therapeutic interventions or rescue treatments, maintain PLT \> 30×10⁹/L at week 6 or 12 without bleeding symptoms.
Time frame: At the 6th or 12th week after treatment conversion (day 42 or day 85 , visitation window allowed: days 41 to 43 or days 82 to 88 ).
Response Rate (RR), proportion with PLT ≥ 50×10⁹/L, and Complete Response rate (CR, i.e., PLT ≥ 100×10⁹/L without bleeding manifestations) at each visit.
This indicator aims to dynamically evaluate hematological responses of different grades during the treatment period and the follow-up period. The specific definition is as follows: 1) Total response rate: It is defined as the proportion of patients with a platelet count of ≥30×10⁹/L, at least doubling from the baseline, and no bleeding manifestations. 2) Proportion of patients with platelet count ≥50×10⁹/L: Defined as the percentage of such patients. 3) Complete remission rate: Defined as the proportion of patients with a platelet count of ≥100×10⁹/L and no bleeding manifestations. The above three proportions will be calculated separately at each scheduled visit point. The denominators of each proportion are the total number of patients in the protocol analysis set who have valid platelet count and bleeding assessment data at the corresponding visit points.
Time frame: Weeks 1, 2, 3, 4, 5, 6, 8, 10, and 12 after treatment conversion (according to the protocol visit plan).
Duration of response
Duration of response defined as time from first achievement of CR or Response (R) until disease relapse, study discontinuation, or loss to follow-up. For patients discontinuing or lost to follow-up, the last efficacy evaluation date is used as the outcome event; for patients not observed to relapse by study end and dropping out for various reasons, the last efficacy evaluation date is used as censored data.
Time frame: From the date of first achieving remission (CR/R) until the end of the study visit (week 12,Day 85 ±3) or the date of early termination of the study.