Clinical Study of Super Transplantation in the Treatment of Severe β-thalassemia
This is a single-arm, open label, phase 1 study in subjects with beta-thalassemia. This study will evaluate the safety and efficacy of preconditioning-free super-transplantation on beta-thalassemia pediatric patients.
• Diagnosis of severe β -thalassemia
• Age between 7-10 years old, male and female; Weight \< 40kg
• The patient has or does not have an HLA-compatible or semi-compatible donor, but unconditional transplantation or refusal of blood stem cell transplantation; Patients with thalassemia gene therapy without conditions or refusal;
• There are fully compatible or incompatible HLA donors, and the physical examination meets the donor conditions;
• The patient and family members agree to receive hypertransplant therapy and sign a written informed consent prior to the transplant trial.