This is a prospective, single-center, open-label, single-arm study, to evaluate the efficacy and safety of Anti-BCMA chimeric antigen receptor T cell therapy(BCMA CAR-T)for patients with relapse/refractory Immune thrombocytopenia(R/R ITP).
Immune thrombocytopenia (ITP) is a disorder that can lead to easy or excessive bruising and bleeding. Approximately two-thirds of patients achieve remission after/during first-line therapies. However, the other part of patients could not achieve durable remission or even refractory to initial treatments. Those cases, known as relapse/refractory Immune thrombocytopenia (R/R ITP), undergo the heavy burden of disease which decreases the quality of life. Lots of pathogeneses take part in the occurrence of R/R ITP, and the most important one of them is antibody-mediated immune platelet destruction. As far as it is known,human platelet autoantibodies are mainly secreted by plasma cells, especially long-lived plasma cells. Researchers want to explore that can BCMA CAR-T help R/R ITP patients increase platelet count, reduce bleeding episodes and the dose of concomitant medications.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
5
Lymphoadenodepletion chemotherapy with FC (fludarabine 30mg/ m2 for 3 consecutive days and cyclophosphamide 300mg/m2 for 3 consecutive days) will be given at day -5, -4 and -3 before CAR T-cells infusion. A total of 1.0-2.0×10e7/Kg autologous anti-BCMA CAR T-cells will be infused by dose-escalation after the lymphoadenodepletion chemotherapy. Dose of CAR T-cells are allowed to be adjusted according to the severity of cytokine release syndrome.
The First Affiliated Hospital of Soochow University
Suzhou, Jiangsu, China
RECRUITINGoverall response
The number of participants who achieved CR (defined as platelet count≥100x10e9/L) and PR (defined as platelet count ≥30x10e9/L and at least a 2-fold increase the baseline count and absence of bleeding) at follow-up.
Time frame: 6-months
Time to response
Time since infusion of CAR T-cells to the time to achieve the response.
Time frame: 6-months
Duration of response
Period from the achievement of response to the loss of response.
Time frame: 6-months
Incidence of adverse events
Adverse events will be assessed daily during the first 2 weeks after the BCMA CAR-T treatment, and monthly thereafter. Adverse events will be evaluated according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time frame: 6-months
Evaluation of bleeding events
Bleeding events will be evaluated according to Bleeding rating system of ITP('Chinese guideline on the diagnosis and management of adult primary immune thrombocytopenia (version 2020)').
Time frame: 6-months
Evaluation of concomitant therapy
Duration of discontinuation or dose reduction of concomitant treatment,and the degree of decrease of combined treatment from the baseline in patients.
Time frame: 6-months
Evaluation of health-related quality of life
Health-related quality of life will be evaluated according to ITP-PAQ (Primary Immune Thrombocytopenia Patient Assessment Questionnaire).
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Time frame: 6-months