refractory immune thrombocytopenia,RITP
Conditions
Interventions
Intervention group:CAR-T cell therapy
Sponsors
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: According to the definition of refractory/severe ITP in the "Guidelines for the Diagnosis and Treatment of Primary Immune Thrombocytopenia in Children (2019 Edition)", children aged 0-35 years who meet the following criteria are included: 1. Newly diagnosed patients, after standard treatment, are determined to meet one of the following conditions: (1) No response or inability to maintain response to two or more treatments other than first-line therapy; (2) No response to at least two different second-line therapies (i.e., immune modulation therapy targeting B cells or T cells and/or TPO-RA); (3) Recurrence of ITP; 2. Guardians of all enrolled children must sign an informed consent form (if the child is over 8 years old, in addition to the guardian signing the informed consent form, the patient must also sign the pediatric version of the informed consent form; if over 18 years old, the adult version of the informed consent form must be signed), and enrollment and treatment must follow ethical requirements.
Exclusion criteria
Exclusion criteria: 1. Patients with secondary ITP; 2. Expected survival <12 weeks; 3. Gene mutations or structural variations with corresponding targets detected by genetic testing; 4. Patients with a known history or previous diagnosis of arterial thrombosis (such as cerebral thrombosis, myocardial infarction, etc.) or venous thromboembolism complications (such as deep vein thrombosis, pulmonary embolism), or those using anticoagulant/antiplatelet drugs at the start of the trial; 5. Uncontrollable severe infection, organ dysfunction, or any uncontrolled active medical disease; 6. Uncontrollable infectious diseases, such as active hepatitis B or C, HIV, fungal infections, tuberculosis, EBV, CMV infection, etc.; 7. Early post-treatment loss to follow-up (<3 months), making recent and long-term prognosis evaluation impossible; 8. Presence of other systemic diseases that interfere with this study; 9. Other criteria that the investigator deems exclusionary.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Assay for Antibodies Against Platelet Membrane Glycoproteins;Cytokines;Complete Blood Count; | — |
Secondary
| Measure | Time frame |
|---|---|
| CAR-T cell expansion and persistence;B cell aplasia; | — |
Countries
China
Contacts
Public ContactLi Benshang
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed