Thalassemia Major
Conditions
Brief summary
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
Detailed description
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.
Interventions
DRUGCyclophosphamide
Cyclophosphamide（50 mg/kg/day,4 days）
DRUGFludarabine
Fludarabine（50 mg/m2/day,3 days）
DRUGMycophenolate mofetil
Mycophenolate mofetil（0.25g/day）
DRUGTacrolimus
Tacrolimus（0.03mg/kg/day）
DRUGMethotrexate
Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
DRUGBusulfan
Busulfan（4 mg/kg/day,4 days）
DRUGThymoglobulin
Thymoglobulin（2.5 mg/kg/day,4 days）
DRUGBasiliximab
Basiliximab（10mg on day 0 and +4）
Sponsors
First Affiliated Hospital of Guangxi Medical University
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosed with thalassemia major 2. Lacking of a HLA-identical sibling donor or unrelated donor 3. Indication of haploidentical hematopoietic stem cell transplantation 4. No restrictions for transplantation
Exclusion criteria
1. Any restriction for transplantation 2. No indication of haploidentical hematopoietic stem cell transplantation
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall Survival | 2 years | 2-years overall survival |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Engraftment | 30 days | Myeloid engraftment at day +30 |
| Transplant Related Mortality | 1 year | Transplant-related mortality by 1 year |
| Disease Free Survival | 2 years | 2-years disease free survival |
| 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 |
| Cumulative Incidence of acute Graft Versus Host Disease | 180 days | Acute graft versus host disease at day +180 |
Countries
China
Contacts
Primary ContactYongrong Lai, MD
Backup ContactMeiqing Wu, MD
Outcome results
None listed