Efractory Autoimmune hemolytic anemia
Conditions
Interventions
Experimental Group:CAR-T cells are infused through peripheral or central veins by one-time intravenous administration.
Sponsors
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Eligibility
Sex/Gender
All
Age
No minimum to 35 Years
Inclusion criteria
Inclusion criteria: 1.Patients with disease onset at age < 35 years, meeting the diagnostic criteria for refractory/severe AIHA as defined in the Chinese Guidelines for the Diagnosis and Management of Autoimmune Hemolytic Anemia (2023), with confirmed presence of targets such as CD19, CD22, BRAF-R, TAC1, BCMA, etc. 2.Newly diagnosed patients who, after standard treatment, meet at least one of the following criteria:; 3.Resistance to glucocorticoids or requiring a maintenance dose exceeding 15 mg/day (calculated as prednisone). 4.Presence of other contraindications or intolerance to glucocorticoid therapy. 5.Relapsed AIHA. 6.Legal guardians of all enrolled pediatric patients must provide written informed consent. For patients over 8 years old, a child-appropriate assent form must also be signed by the patient themselves. Patients over 18 years old shall sign the adult version of the informed consent form. All patients are enrolled and treated in accordance with ethical requirements.
Exclusion criteria
Exclusion criteria: Those who meet any of the following conditions are not eligible to participate in this study: 1. Any AIHA patient in the acute hemolytic phase or with acute hemolytic symptoms; 2. Expected survival period <12 weeks; 3. Genetic testing shows that there are gene mutations or structural variations at the corresponding targets; 4. Patients with recurrent GVHD after bone marrow transplantation are severe and require immunosuppressant intervention. 5. Recurrence time after bone marrow transplantation is less than 3 months, and no donor can be found; 6. Uncontrollable active infections, such as active hepatitis B and C, HIV, fungal, tuberculosis, EBV, CMV infections, etc. 7. Early loss to follow-up after treatment (<3 months), making it impossible to conduct short-term and long-term prognosis evaluations; 8. There are other systemic diseases that may interfere with this research; 9. Other indicators that the researcher believes can be excluded;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Coombs test results;Complete blood count ?Reticulocyte;Cytokines;Objective Response Rate (ORR);LDH?Bilirubin; | — |
Secondary
| Measure | Time frame |
|---|---|
| CART cell copy number monitoring;B-cell Reconstitution in Bone Marrow and Peripheral Blood; | — |
Countries
China
Contacts
Public ContactLi Benshang
Shanghai Children's Medical Center, Shanghai Jiao Tong University School of Medicine
Outcome results
None listed