Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
- Conditions
- Thalassemia Major
- Interventions
- Registration Number
- NCT04009525
- Lead Sponsor
- First Affiliated Hospital of Guangxi Medical University
- Brief Summary
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
- Detailed Description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 823
- Diagnosed with thalassemia major.
- Indication of hematopoietic stem cell transplantation.
- A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.
- Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
- Uncontrolled bacterial, viral or fungal infections;
- Any other restriction for transplantation.
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description URD-HSCT Thymoglobulin unrelated donor hematopoietic stem cell transplantation MSD-HSCT cyclosporine A matched sibling donors hematopoietic stem cell transplantation MSD-HSCT Thymoglobulin matched sibling donors hematopoietic stem cell transplantation haplo-HSCT Cyclophosphamide haplo-identical hematopoietic stem cell transplantation haplo-HSCT Fludarabine haplo-identical hematopoietic stem cell transplantation MSD-HSCT Mycophenolate mofetil matched sibling donors hematopoietic stem cell transplantation MSD-HSCT Methotrexate matched sibling donors hematopoietic stem cell transplantation MSD-HSCT Busulfan matched sibling donors hematopoietic stem cell transplantation MSD-HSCT Cyclophosphamide matched sibling donors hematopoietic stem cell transplantation MSD-HSCT Fludarabine matched sibling donors hematopoietic stem cell transplantation URD-HSCT Busulfan unrelated donor hematopoietic stem cell transplantation URD-HSCT Cyclophosphamide unrelated donor hematopoietic stem cell transplantation URD-HSCT Fludarabine unrelated donor hematopoietic stem cell transplantation URD-HSCT Methotrexate unrelated donor hematopoietic stem cell transplantation URD-HSCT Tacrolimus unrelated donor hematopoietic stem cell transplantation URD-HSCT Mycophenolate mofetil unrelated donor hematopoietic stem cell transplantation haplo-HSCT Busulfan haplo-identical hematopoietic stem cell transplantation haplo-HSCT Mycophenolate mofetil haplo-identical hematopoietic stem cell transplantation haplo-HSCT Thymoglobulin haplo-identical hematopoietic stem cell transplantation haplo-HSCT Methotrexate haplo-identical hematopoietic stem cell transplantation haplo-HSCT Tacrolimus haplo-identical hematopoietic stem cell transplantation
- Primary Outcome Measures
Name Time Method Overall survival 2 years 2-years overall survival
Thalassemia-free survival 2 years 2-years thalassemia-free survival
- Secondary Outcome Measures
Name Time Method Engraftment 30 days Myeloid engraftment at day +30
Transplant Related Mortality 2 year Transplant-related mortality by 2 year
Cumulative Incidence of acute Graft Versus Host Disease 100 days Acute graft versus host disease at day +100
Cumulative Incidence of chronic Graft Versus Host Disease 2 years Chronic graft versus host disease by 2 years
Cumulative Incidence of Infectious Complications 2 years Cumulative incidence of bacterial, fungal and viral infections by 2 years
Trial Locations
- Locations (1)
First Affiliated Hospital of Guangxi Medical University
🇨🇳Nanning, Guangxi, China