Low-dose Rituximab Regimens in Chinese Adult Patients With Immune Thrombocytopenia (ITP)

Prospective Multi-center Randomized Controlled Low-dose Rituximab Regimens in Chinese Adult Patients With Immune Thrombocytopenia

A comparative study with rituximab (100 mg weekly for 4 weeks and 375mg/m2 for once) shows low dose rituximab may be a useful alternative therapy in patients with ITP.The aim of this study is to compare the efficacy and tolerability of two different regimens of low doses rituximab for the treatment of adult patients with ITP.

Study Overview

• Status: Completed
• Conditions: Immune Thrombocytopenic Purpura
• Intervention / Treatment: Drug: Rituximab

Detailed Description

This is a multicentre, prospective, open-label, randomised controlled trial. The aim of this study will compare the long-term efficacy and safety of two low-dose rituximab regimens in adult Chinese patients with glucocorticoid-resistant/dependent or relapsed ITP. Group A will receive rituximab100 mg weekly for four weeks, and group B will receive rituximab 375 mg/m2 once. After initiating treatment, if the patient has at least two consecutive evaluations (interval >7 days) without rescue therapy and a platelet count >50×109/L, the concomitant medications such as glucocorticoids can be reduced or stopped.

Within 3 months of the last rituximab dose, rescue therapy is recommended if the patient has an extremely low platelet count and an obvious bleeding tendency. Rescue therapy is limited to IVIG (0.4 g/kg per day for 3-5 days) and glucocorticoid (methylprednisolone 0.8 mg/kg per day for 7-14 days or equivalent dose of other glucocorticoids).

• Study Type: Interventional
• Enrollment (Actual): 100
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Tianjin Municipality
    • Tianjin, Tianjin Municipality, China, 300020
      • Hospital of Blood disease

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 60 years (Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Age 18-60 years old;
• Diagnosis of primary immune thrombocytopenia according to the guidelines of American Society of Hematology for at least 3 months before inclusion;
• Platelet count <30×109/L (measured at least twice during the screening, with at least a 1-week interval);
• Failed or dependent on or relapsed after previous treatment with glucocorticoid;
• If on glucocorticoid maintenance therapy, dose ≤0.5 mg/kg prednisone or equivalent and stabilised for at least 4 weeks;
• Drugs such as azathioprine, danazol, cyclosporine A, tacrolimus, and sirolimus stopped for at least 4 weeks;
• Splenectomy more than 6 months previously;
• Previous rescue therapy of ITP (including methylprednisolone, platelet transfusion and IVIG) completed at least 2 weeks before the first administration;
• Liver and kidney function (including alanine aminotransferase, aspartate aminotransferase, total bilirubin, serum creatine, urea nitrogen, etc.) less than 1.5 times the upper limit of normal value;
• Eastern Cooperative Oncology Group performance status ≤2;
• Cardiac function classification (New York Heart Association) ≤2;
• Understand the research procedure and voluntarily sign a written informed consent form.

Exclusion Criteria:

• Patients with secondary thrombocytopenia (including myelodysplastic syndrome, aplastic anemia, common variant immunodeficiency disease, hereditary thrombocytopenia, drug-induced thrombocytopenia, pseudothrombocytopenia, connective tissue disease secondary thrombocytopenia, thrombocytopenia after liver disease, etc.);
• Previous treatment of RTX or allergic to RTX;
• Uncontrollable primary diseases of important organs (including malignant tumor, liver failure, heart failure, kidney failure and other diseases);
• HIV-positive status;
• Active infection including hepatitis B (HBV), hepatitis C (HCV) and other viral antigens or DNA, RNA positive; cytomegalovirus, Epstein-Barr virus, syphilis chronic or active infection. If HBV core antibodies are positive, HBV-DNA testing is required.
• Extensive and severe bleeding, such as hemoptysis, upper gastrointestinal hemorrhage, intracranial hemorrhage, etc.
• Heart disease or arrhythmia need treatment, or poorly controlled hypertension;
• Thrombotic diseases including pulmonary embolism, thrombosis, atherosclerosis, etc.;
• Previously allogeneic stem cell transplantation or organ transplantation;
• Mental disorders who are unable to obtain informed consent normally and participate in trials and follow-up;
• Symptoms of toxicity from pre-trial treatment have not resolved;
• Other severe conditions that may limit participation in the trial (e.g., diabetes; severe cardiac insufficiency; myocardial infarction or unstable arrhythmia or unstable angina within the last 6 months; gastric ulcer; active autoimmune diseases, etc.);
• Sepsis or other irregular bleeding;
• Taking antiplatelet drugs;
• pregnancy, suspected pregnancy (urine human chorionic gonadotropin positive during screening) or lactation.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Rituximab A group; 375mg/m2 for once	Drug: Rituximab; Other Names: Trade names Rituxan and MabThera
Active Comparator: Rituximab B group; 100mg/week for four weeks	Drug: Rituximab; Other Names: Trade names Rituxan and MabThera

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Overall response rate at week 12	Overall response rate was defined as proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least twice the baseline platelet count without bleeding and subjects with a platelet count ≥ 100 × 10^9/L without bleeding at week 12 after initial administration in absence of rescue therapy, and without having had dose increment of corticosteroids during the study period.	Patients will be followed for 6 months at least after Rituximab treatment.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Sustained response rate over 6 months and 1, 2, 3, 4, and 5 years after RTX initiation	Proportion of subjects with a platelet count ≥ 30 × 10^9/L and at least twice the baseline platelet count without bleeding and subjects with a platelet count ≥ 100 × 10^9/L without bleeding at 6 months and 1, 2, 3, 4, and 5 years after initial administration in absence of rescue therapy	Patients will be followed for 6 months at least after Rituximab treatment.
Time to response (TTR)	Time needed from treatment initiation to platelet count ≥30×10^9/L and at least twice the baseline platelet count.	6 months
Number of subjects with clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale	Changes of the subjects' numbers in WHO bleeding score after RTX treatment according to the reported World Health Organization's Bleeding Scale at month 3. The WHO Bleeding Scale is a measure of bleeding severity with the following grades: grade 0 = no bleeding, grade 1= petechiae, grade 2= mild blood loss, grade 3 = gross blood loss, and grade 4 = debilitating blood loss.	3 months
Adverse events assessment	Incidence, severity, and relationship of treatment emergent adverse events during the study period	12 months

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital, China
• Collaborators: Henan Cancer Hospital; The Second Affiliated Hospital of Kunming Medical University; Langfang Traditional Chinese Medicine Hospital
• Investigators: Principal Investigator: RENCHI YANG, Dr, Hospital of Blood disease

Study record dates

Study Major Dates

• Study Start (Actual): October 1, 2012
• Primary Completion (Actual): October 1, 2015
• Study Completion (Actual): February 1, 2016

Study Registration Dates

• First Submitted: October 26, 2012
• First Submitted That Met QC Criteria: October 30, 2012
• First Posted (Estimated): November 1, 2012

Study Record Updates

• Last Update Posted (Actual): February 11, 2026
• Last Update Submitted That Met QC Criteria: February 9, 2026
• Last Verified: March 1, 2024

More Information

Terms related to this study

• 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; Amino Acids, Peptides, and Proteins; Proteins; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Serum Globulins; Globulins; Antibodies, Monoclonal, Murine-Derived; Rituximab
• Other Study ID Numbers: IIT2012001