Atorvastatin Combined With NAC Plus Romiplostim for Management of ITP

Atorvastatin Combined With N-Acetyl-L-Cysteine Plus Romiplostim for Management of Steroid-Resistant/Relapsed Immune Thrombocytopenia

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.

Study Overview

• Status: Not yet recruiting
• Conditions: Immune Thrombocytopenic Purpura
• Intervention / Treatment: Drug: atorvastatin, NAC, and romiplostim

Detailed Description

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
• Enrollment (Estimated): 50
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Fu Haixia, Dr.; Phone Number: +861088326002; Email: fuhaixia_210@163.com
• Study Contact Backup: Name: Xiaohui Zhang, Dr.; Phone Number: 861088326001; Email: zhangxh@bjmu.edu.cn

Study Locations

• China
  • Beijing, China
    • Peking University People's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosed with primary ITP;
• Aged ≥18 years;
• Patients with treatment failure or relapse after first-line corticosteriod therapy for ITP;
• Platelet count <30×10⁹/L.

Exclusion Criteria:

• Pregnant or lactating women, and who were possibly pregnant, planning to become pregnant, or who had partners planning to become pregnant;
• Presence of active malignant tumors;
• Active HBV, HCV or HIV infection;
• Active infection requiring systematic treatment;
• Leukemia, myelodysplastic syndrome, aplastic anemia, myelofibrosis or other hematological disorders that may cause thrombocytopenia;
• History or presence of myocardial infarction, unstable ischemic heart disease, stroke, or NYHA Class IV heart failure;
• AST > 2 times the upper limit of normal (ULN), ALT > 2×ULN, or TBIL ≥ 1.5×ULN;
• eGFR < 50 mL/min/1.73m²;
• Any other subjects deemed ineligible for enrollment by the investigator.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: combined therapy; atorvastatin 20mg qd , N-acetyl-L-cysteine (NAC) 400mg tid, and romiplostim

Drug: atorvastatin, NAC, and 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. 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	24 weeks post-treatment cessation

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	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.	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	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.	Up to the end of week 24
Bleeding events	Bleeding incidence and severity per WHO bleeding score	Up to the end of week 24; Week 25 to 24 weeks post-treatment cessation
Adverse Events	The proportion of patients with adverse events	Up to the end of week 24; Week 25 to 24 weeks post-treatment cessation

Collaborators and Investigators

• Sponsor: Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): June 1, 2026
• Primary Completion (Estimated): December 30, 2027
• Study Completion (Estimated): December 30, 2028

Study Registration Dates

• First Submitted: May 16, 2026
• First Submitted That Met QC Criteria: June 19, 2026
• First Posted (Actual): June 23, 2026

Study Record Updates

• Last Update Posted (Actual): June 23, 2026
• Last Update Submitted That Met QC Criteria: June 19, 2026
• Last Verified: June 1, 2026

More Information

Terms related to this study

• Keywords: romiplostim; atorvastatin; immune thrombocytopenia; N-acetyl-L-cysteine; sustained response off-treatment
• Additional Relevant MeSH Terms: Cytopenia; Pathologic Processes; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Thrombocytopenia; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Hemic and Lymphatic Diseases; Purpura, Thrombocytopenic, Idiopathic; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Fatty Acids; Lipids; Azoles; Pyrroles; Heptanoic Acids; Atorvastatin; romiplostim
• Other Study ID Numbers: 2025PHD049-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No