Platelet Transfusion Refractoriness, Severe Aplastic Anemia
Conditions
Keywords
Severe aplastic anemia, Platelet transfusion refractoriness, Rituximab
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.
Interventions
DRUGRituximab
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.
Sponsors
Institute of Hematology & Blood Diseases Hospital, China
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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. |
| Survival | 60 months | Survival rate within 5 years after diagnosis |
| Clonal evolution to myelodysplasia and acute leukemia. | 60 months | Clonal evolution within 5 years after diagnosis |
Countries
China
Contacts
Primary ContactHuang Jinbo, MD.
Outcome results
None listed