Transfusion dependent thalassemia
Conditions
Interventions
Intervention group:Recombinant anti-CD25 humanized monoclonal antibody Jiannipai combined with standard GVHD prevention regimen
Control group:Standard GVHD prevention regimen
Sponsors
The First Affiliated Hospital of Guangxi Medical University
Eligibility
Sex/Gender
All
Age
3 Years to 20 Years
Inclusion criteria
Inclusion criteria: 1.Age 3-20 years old, gender not limited; 2.Patients with transfusion dependent thalassemia; 3.Individuals planning to receive non matching hematopoietic stem cell transplantation (MUD-HSCT) or HLA haploid hematopoietic stem cell transplantation (HID-HSCT) treatment; 4.Lansky/Karnofsky score >= 70%; 5.The patient (or legal guardian) voluntarily participates in the study and signs an informed consent form;
Exclusion criteria
Exclusion criteria: 1.A donor with HLA gene identical hematopoietic stem cells and willing to accept HLA gene identical hematopoietic stem cells transplant; 2.Known patients with infectious diseases such as hepatitis B, hepatitis C, AIDS, syphilis, and human T-lymphovirus; 3.Serious active bacterial, viral, fungal, malaria or parasitic infections; 4.Autoimmune diseases; 5.History of malignant tumors or current malignant tumor patient; 6.Important organ diseases or laboratory abnormalities, including but not limited to: (1) Suffering from cirrhosis, liver fibrosis, or Active hepatitis and/or abnormal liver function tests (alanine aminotransferase (ALT) and aspartate) Aminotransferase (AST) >= 2.5 × ULN; Alkaline phosphatase >= 2.5 × ULN); (2) Heart disease, or Left ventricular ejection fraction (LVEF)= 1.5 × ULN, creatinine clearance rate<30% normal level; (4) Suffering from endocrine disorders; There are bleeding disorders that have not been corrected; 7.Combined severe mental illness (such as severe depression, schizophrenia, etc.) or cognitive impairment (dementia Delirium, etc.), unable to cooperate in completing the study; 8.Peripheral blood white blood cell (WBC) count<3 × 10^9/L or platelet count<100 × 10^9/L; 9.Received treatment with thalidomide within the past 3 months; 10.Previously received any type of gene and/or cell therapy; 11.Individuals with a history of severe allergies; 12.Pregnant, lactating, and female participants with a pregnancy plan within one year of participating in this trial; 13.Participants in other clinical trials; 14.Other situations assessed by researchers as unsuitable for participation in this clinical trial;
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of grade III-IV acute GVHD (aGVHD) within 100 days after HID-HSCT/MUD-HSCT; | — |
Secondary
| Measure | Time frame |
|---|---|
| Grade II-IV aGVHD within 100 days after HID-HSCT/MUD-HSCT;Incidence of chronic GVHD (cGVHD) after hid-hsct/mud-hsct;Overall survival;Thalassemia-Free Survival (TFS);Non-relapse mortality (NRM);Transplantation related complications;Quality of life situation;Adverse Events and Serious Adverse Events; | — |
Countries
China
Contacts
Public ContactRongrong Liu
The First Affiliated Hospital of Guangxi Medical University
Outcome results
None listed