Severe beta-thalassemia
Conditions
Sponsors
Dongguan Taixin Hospital
Eligibility
Sex/Gender
All
Age
7 Years to No maximum
Inclusion criteria
Inclusion criteria: 1. Aged >=7 years old, able to understand, agree and sign informed consent form or parent / legal guardian must be able to understand, agree and sign informed consent form; 2. Patients who have been genetically diagnosed with severe beta thalassemia before screening and enrolled in the group need continuous blood transfusion therapy, and have infused at least 100ml / kg of concentrated red blood cells or >=8 times per year within two years before the study; In the case of red blood cell infusion, the hemoglobin content is more than three times =80%, and the Lansky score for children should be >=70%; 5. The expected survival time of the subject is> 6 months; 6. The subject must have participated in treatment in a professional institution and have been followed up for more than two years, and a comprehensive assessment of the disease, including a mental assessment; detailed medical records including data from at least the past two years, for easy comparison; 7. No treatment with hydroxyurea, 5-azacytidine, cytarabine, etc. within three months before enrollment; 8. Subjects previously received standardized blood transfusions, with hemoglobin content >=90g/L within 1 month before peripheral blood mobilization.
Exclusion criteria
Exclusion criteria: 1. A suitable HLA-conforming donor or a person who has previously received an allogeneic hematopoietic stem cell transplant; 2. HIV seropositive, hepatitis B surface antigen positive or hepatitis B core antibody positive with HBV DNA positive, hepatitis C virus antibody positive, and syphilis antibody positive; 3. Those who are contraindicated in taking bone marrow from anesthesia; 4. Severe bacterial, fungal, mycoplasma, virus and other infections; 5. History of malignant tumors; 6. White blood cell counts 3 ULN or subjects with cirrhosis and liver fibrosis; (3) Heart disease with ejection fraction <25% or cardiac MRI T2 * test <10ms; (4) Severe kidney disease and creatinine clearance <30% of normal value; (5) Pulmonary diseases include pulmonary fibrosis, pulmonary hypertension, or significant decline in lung function (eg, partial oxygen pressure <90mmHg and / or CO diffusion <50% of expected value); (5) Endocrine disorders include insulin-dependent diabetes mellitus, hyperthyroidism or insufficiency, and hyperparathyroidism or insufficiency; 13. Participate in another clinical study within 30 days before the screening period; 14. Subjects with severe iron overload judged by researchers; 15. Other circumstances not suitable for hematopoietic stem cell transplantation as determined by the attending physician or researcher; 16. Bone marrow test found chromosomal abnormalities.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Adverse events / adverse events;Blood routine and transfusion volume;Carrier integration analysis; | — |
Countries
China
Contacts
Public ContactChunfu Li ?Jianyun Liao
Dongguan Taixin Hospital
Outcome results
None listed