Blood transfusion-dependent beta-thalassemia
Conditions
Interventions
Cases:ET-01 therapy
Sponsors
Dongguan Taixin Hospital
Eligibility
Sex/Gender
All
Age
8 Years to 25 Years
Inclusion criteria
Inclusion criteria: 1. Aged = 8 years old, able to understand, agree and sign the informed consent form or the parent/legal guardian must be able to understand, agree and sign the informed consent form; 2. Diagnosed as beta thalassemia, patients required continuous blood transfusion therapy, and at least two years of infusion of concentrated red blood cells = 100ml / kg or = 8 times per year before participating in the study; 3. The patient's condition is stable and his condition can tolerate hematopoietic stem cell transplantation; 4. The adult Karsten score needs to be =80%, and the Lansky score for children is =70%; 5. Patient survival is expected to be >6 months; 6. Patients must have been involved in disease diagnosis and treatment in a professional institution, and have been followed for at least two years, with detailed treatment records for the disease, and have undergone a comprehensive assessment including a mental assessment; detailed medical records include information for at least the past two years. In order to carry out self-control; 7. No treatment with hydroxyurea, 5-azacytidine or cytarabine was performed within three months prior to enrollment; 8. According to the needs of patients, services such as sperm cryopreservation, ovarian and testicular biopsy can be provided.
Exclusion criteria
Exclusion criteria: 1. Have a suitable HLA fully compatible donor; 2. HIV seropositive, hepatitis B surface antigen positive, hepatitis C antibody positive; 3. For the anesthesia to take bone marrow contraindications; 4. Severe infections such as bacteria, fungi, mycoplasma, and viruses; 5. History of malignant tumors; 6. Non-spleen hyperactivity factors lead to white blood cell count 3 × normal upper limit or liver cirrhosis, liver fibrosis; (3) Heart disease and ejection fraction <25% or cardiac nuclear magnetic resonance T2* detection <10ms; (4) Severe kidney disease with creatinine clearance <30% normal; - Pulmonary diseases including pulmonary fibrosis, pulmonary hypertension, or decreased lung function such as oxygen partial pressure 90mmHg and / or CO dispersion amount <expected value 50%); - Endocrine disorders include insulin-dependent diabetes mellitus, hyperthyroidism or deficiency, parathyroid Glandular hyperfunction or deficiency. 13. Participate in another clinical study within 30 days of the screening period.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| safety; | — |
Countries
China
Contacts
Public ContactChunfu Li
Dongguan Taixin Hospital
Outcome results
None listed