This is a prospective, single-arm, open-lable, single-center study and we aimed to determine whether atorvastatin combined with N-acetyl-L-cysteine (NAC) plus romiplostim could induce sustained response off-treatment (SRoT) in adult patients with ITP following CS failure.
This is a prospective, single-arm trial designed to investigate whether atorvastatin combined with N-acetyl-L-cysteine (NAC) plus romiplostim can induce a sustained response off-treatment (SRoT) in adult patients with immune thrombocytopenia (ITP) who experienced failure of first-line corticosteroid therapy. In this study, SRoT is defined as an off-treatment period during which the platelet count remains above 30×10⁹/L in the absence of bleeding events or rescue therapy. The primary endpoint was the proportion of patients who achieved SRoT by Week 24 after the discontinuation of romiplostim. From Week 1 to Week 24, atorvastatin and NAC was administrated as the dose of 20mg qd and 400mg tid,respectively, and were discontinued at the end of Week 24. During the initial 24 weeks, romiplostim was initiated at a starting dose of 3 μg/kg per week. The weekly dose was adjusted based on platelet counts, with a maximum dose of 10 μg/kg per week, to maintain platelet levels within the range of 100-200×10⁹/L. From Week 25 to Week 35, romiplostim was gradually tapered and discontinued, with the goal of maintaining a platelet count ≥30×10⁹/L and no less than twice the baseline level. After all medications (including atorvastatin, NAC, and romiplostim) were discontinued (no later than Week 36), patients were followed up for an additional 24 weeks to evaluate the sustained response rate at 24 weeks post-treatment cessation.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
50
From Week 1 to Week 24, atorvastatin and NAC was administrated as the dose of 20mg qd and 400mg tid,respectively, and were discontinued at the end of Week 24. For romiplostim, the initial dose was 3 μg/kg per week. The weekly dose was adjusted based on platelet counts, with a maximum dose of 10 μg/kg per week, to maintain platelet levels within the range of 100-200×10⁹/L during the initial 24-week period. From Week 25 to Week 35, romiplostim was gradually tapered and discontinued with the goal of maintaining a platelet count ≥30×10⁹/L and no less than twice the baseline level. After all medications (including atorvastatin, NAC, and romiplostim) were discontinued (no later than Week 36), patients were followed up for an additional 24 weeks to evaluate the sustained response rate at 24 weeks post-treatment cessation.
Peking University People's Hospital
Beijing, China
24-week SRoT rate
Sustained response off-treatment (SRoT) rate is defined as the proportion of patients who maintain a platelet count ≥30×10\^9/L and at least a two-fold increase from the baseline count without active bleeding following treatment discontinuation.
Time frame: 24 weeks post-treatment cessation
24-week SCRoT rate
Sustained complete response off-treatment (SCRoT) rate was defined as the proportion of patients who maintain a platelet count of ≥100×10⁹/L without active bleeding after treatment discontinuation.
Time frame: 24 weeks after treatment discontinuation
ORR
Overall response rate (ORR) is defined as the proportion of patients who achieve platelet count ≥30×10\^9/L and more than twice the baseline level, with no signs of active bleeding.
Time frame: Up to the end of week 24
CR rate
Complete response (CR) rate is defined as the proportion of patients who achieve a platelet count ≥100×10⁹/L with no signs of active bleeding.
Time frame: Up to the end of week 24
TTR
Time to response (TTR) is defined as the days from treatment initiation to first platete count reaching ≥30×10\^9/L
Time frame: Up to the end of week 24
Sustained response
Platelet count ≥30×10⁹/L and at least doubled from baseline on at least three of four scheduled visits during the final 8 weeks of the initial 24-week treatment phase without active bleeding.
Time frame: Up to the end of week 24
Bleeding events
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Bleeding incidence and severity per WHO bleeding score
Time frame: Up to the end of week 24; Week 25 to 24 weeks post-treatment cessation
Adverse Events
The proportion of patients with adverse events
Time frame: Up to the end of week 24; Week 25 to 24 weeks post-treatment cessation