Rituximab for Serious Aplastic Anemia With Platelet Transfusion Refractoriness

Phase 2RecruitingNCT06254560

Institute of Hematology & Blood Diseases Hospital, China20 enrolled

Overview

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.

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Severe Aplastic Anemia Platelet Transfusion Refractoriness

Interventions

Eligibility

Sex: ALLMin age: 18 YearsMax age: 70 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

The response and complete remission rate with Rituximab protocol.

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).

Time frame: 6 months

Secondary Outcomes

Relapse rate

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.

Time frame: 12 months and 60 months

Sustained response (SR)

SR was defined as Hb \> 10 g/dL at 12 months and 60 months, in the absence of any treatment.

Time frame: 12 months and 60 months

Survival

Survival rate within 5 years after diagnosis

Time frame: 60 months

Clonal evolution to myelodysplasia and acute leukemia.

Clonal evolution within 5 years after diagnosis