It is a single-center, single-arm, open-labeled clinical trial to evaluate the efficacy and safety of CAR-T cells therapy for Chronic or Refractory Primary Immune Thrombocytopenia (ITP).
Willingness to complete the informed consent process and to comply with study procedures and visit schedule;
Men and women aged 8-75;
Participants diagnosed with chronic (>12 months duration) or refractory (a documented intolerance or insufficient response to the first and second line standard treatment of ITP) ITP;
The results of physical, instrumental, and laboratory examination of patients not suggest any disease which may cause thrombocytopenia other than ITP;
All subjects with diseases which may cause secondary immune thrombocytopenia
Patients with preventive splenectomy;
Hemostatic disorders other than chronic thrombocytopenia;
Subject treated with drugs that affect platelet function (including but not limited to aspirin, clopidogrel and/or NSAIDs) or anti-coagulants for > 3 consecutive days within 2 weeks of the study start and until the end of the study;
Single group
CAR-T cells
Biological/VaccineCAR-T cells will be administered by vein. Before CAR-T infusion，patients will get a 3-5 days lymphodepletion therapy with fludarabine and cyclophosphamide.
Portion of patients with response (R)
platelet count\>30x10\^9/L and at least 2-fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Portion of patients with complete response (CR)
platelet count\>100x10\^9/L, confirmed on at least 2 separate occasions at least 7 days apart, and absence of bleeding
Portion of patients with relapse
platelet count below 30x10\^9/L or less than 2-fold increase of baseline platelet count or bleeding after treating effectively; platelet counts confirmed on at least 2 separate occasions approximately 1 day apart
Time (in days) from treatment start to response (R)
Time calculated from infusion to the day when the response (R) criteria are achieved
Time (in days) from treatment to complete response (CR)
Time calculated from infusion to the day when the complete response (CR) criteria are achieved
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