Supergraft: This study is a prospective, single-center, single-arm clinical study, and it is planned to include 3-5 patients with Eastern Mediterranean major who have no HSCT indication and cannot undergo Eastern Mediterranean gene therapy. The diagnosis of Eastern Mediterranean major was determined by the genotype of Eastern Mediterranean and the clinical manifestations of patients, and patients who met the inclusion criteria were screened and enrolled and received super transplant therapy.
Study summary
Yunnan is a high-incidence area of Eastern Mediterranean (thalassemia) in China, and the treatment cost of thalassemia patients is high, hematopoietic stem cell transplantation (HSCT) is the only means to cure thalassemia, but there are problems in donor screening and the risk of complications. Professor Ai Huisheng's team proposed a new concept of hypertransplantation, which does not require pretreatment and has no risk of GVHD, and animal experiments have shown good efficacy. Under the guidance of Professor Ai, the center plans to carry out clinical research on hypertransplantation and explore safe and effective new therapies for thalassemia.
Eligibility
Sex
ALL
Min age
7 Years
Max age
12 Years
Healthy volunteers
No
Patient inclusion criteria :
* Diagnosed with severe Mediterranean anemia, with no restrictions on alpha and beta types;
* Age between 7-12 years old, male or female not limited; Weight\<40kg
* The patient has or does not have HLA matched or semi matched donors, but unconditionally transplants or refuses to undergo blood stem cell transplantation treatment; And patients who unconditionally or refuse to undergo gene therapy for thalassemia;
* There are HLA matched or mismatched donors who meet the donor criteria through physical examination;
* The patient and their family agree to receive super transplantation treatment and sign a written informed consent form before the transplantation trial.
（2） Exclusion criteria for patients:
* Psychiatric patients;
* Participants in clinical trials of other drugs within the past month;
* There are no suitable HLA mismatched donors available.
* Other researchers have determined that they are not suitable to participate in this study.
（3） Supplier screening criteria:
* HLA typing matches the patient's haplotype
* KIR configuration
* NIMA
* DSA negative
* Routine physical examination
* Genetic screening for carriers of thalassemia, mild or no carriers of thalassemia
* Sign the informed consent form.
Primary outcome measure(s)
Donor cell implantation rate — 3 months after transplantation Using polymerase chain reaction (PCR) to amplify short tandem repeat sequences Monitor the chimerism status of the donor and recipient to quantitatively evaluate the implantation rate of donor cells. Specifically, we will determine the percentage of donor cells and evaluate the implantation rate by comparing the DNA sequence differences between donor and recipient cells before and after transplantation.
Trial sites (1)
Facility
City
Region
Status
Yunnan First People's Hospital
Kunming
Yunnan
Recruiting
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
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