Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

February 3, 2024 updated by: Institute of Hematology & Blood Diseases Hospital, China

The Study of Rituximab in the Treatment of Severe Aplastic Anemia With Platelet Transfusion Refractoriness

Due to long-term dependence on platelet transfusion, some severe aplastic anemia (SAA) patients suffer platelet transfusion refractoriness (PTR). Unlike immune thrombocytopenia (ITP), glucocorticoids and human immunoglobulin (IVIg) are generally ineffective for PTR. Due to the lack of effective intervention methods, patients with PTR suffer increased platelet transfusions, bleeding events and treatment costs, prolonged hospital stays, and decreased survival rate. SAA with PTR has become a challenge for physicians. The experiment aims to explore the efficacy of rituximab in the treatment of SAA with PTR, and establish a new effective, safe treatment method with relatively low treatment cost.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

During the treatment period, Rituximab is administered at a dose of 100mg per week, a total of 4 times.

Study Type

Interventional

Enrollment (Estimated)

20

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • China
    • Tianjin
      • Tianjin, Tianjin, China, 300020
        • Recruiting
        • Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Initial diagnosed SAA with PTR
  • Age>18 years old, regardless of gender
  • Initial diagnosed SAA with PTR
  • Age>18 years old, regardless of gender

Exclusion Criteria:

  • Allergy to rituximab
  • Severe active infection
  • Hypogammaglobulinemia
  • Pregnant and lactating women
  • Heart failure (NYHA classification IV)
  • Individuals with epilepsy, dementia, and other mental disorders that require medication treatment who cannot understand or follow the research protocol
  • Chronic infections or other chronic diseases that may be risk to the experiment
  • The researchers believe that it is not suitable for participants

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

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

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: rituximab group
Rituximab combined with cyclosporine
Drug: Rituximab
Rituximab is administered at a dose of 100mg per week, a total of 4 times. Cyclosporin is administered at a dose of 3-5mg/kg per day.
Other Names:
  • Mabthera

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
The response and complete remission rate with Rituximab protocol.
Time Frame: 6 months
Response will be evaluated at each clinic visit. Complete response (CR) was defined as achieving all three peripheral blood count criteria: (1) Hb level up to the normal range; (2) ANC≥1.5×109/L; (3) PLT≥100×109/L. Partial response (PR) was defined as transfusion independent, no longer meeting criteria for severe disease. Persistence of transfusion requirement or death was evidence of no response (NR).
6 months

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Relapse rate
Time Frame: 12 months and 60 months
Relapse was defined as a responder who met criteria for SAA again after achieving response and keeping stable blood counts for at least 3 months.
12 months and 60 months
Sustained response (SR)
Time Frame: 12 months and 60 months
SR was defined as Hb > 10 g/dL at 12 months and 60 months, in the absence of any treatment.
12 months and 60 months
Survival
Time Frame: 60 months
Survival rate within 5 years after diagnosis
60 months
Clonal evolution to myelodysplasia and acute leukemia.
Time Frame: 60 months
Clonal evolution within 5 years after diagnosis
60 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Institute of Hematology & Blood Diseases Hospital, China

Investigators

  • Principal Investigator: Huang Jinbo, MD., Institute of Hematology & Blood Diseases Hospital,Chinese Academy of Medical Sciences

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 23, 2023

Primary Completion (Estimated)

December 30, 2026

Study Completion (Estimated)

December 30, 2027

Study Registration Dates

First Submitted

January 19, 2024

First Submitted That Met QC Criteria

February 3, 2024

First Posted (Actual)

February 12, 2024

Study Record Updates

Last Update Posted (Actual)

February 12, 2024

Last Update Submitted That Met QC Criteria

February 3, 2024

Last Verified

February 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023NCRCA0113

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

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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