Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Phase 2

Recruiting

• Conditions: Severe Aplastic Anemia; Platelet Transfusion Refractoriness
• Interventions: Drug: Rituximab

• Registration Number: NCT06254560
• Lead Sponsor: Institute of Hematology & Blood Diseases Hospital, China

Brief Summary

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.

Detailed Description

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

Study Timeline

• Status Verified: 2023-02
• Study Start: 2023-02-23
• Study First Submitted: 2024-01-19
• Study First Submitted QC: 2024-02-03
• Last Update Submitted: 2024-02-03
• Study First Posted: 2024-02-12
• Last Update Posted: 2024-02-12
• Primary Completion: 2026-12-30
• Study Completion: 2027-12-30

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 20

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
rituximab group	Rituximab	Rituximab combined with cyclosporine

Primary Outcome Measures

Name	Time	Method
The response and complete remission rate with Rituximab protocol.	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).

Secondary Outcome Measures

Name	Time	Method
Survival	60 months	Survival rate within 5 years after diagnosis
Relapse rate	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.
Sustained response (SR)	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.
Clonal evolution to myelodysplasia and acute leukemia.	60 months	Clonal evolution within 5 years after diagnosis

Trial Locations

Locations (1)

Institute of Hematology & Blood Diseases Hospital Chinese Academy of Medical Sciences; 🇨🇳 Tianjin, Tianjin, China