Thalassemia Major
Conditions
Keywords
thalassemia major, hematopoietic stem cell transplantation
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.
Interventions
DRUGcyclosporine A
cyclosporine A
DRUGMycophenolate mofetil
Mycophenolate mofetil（0.25g/day）
DRUGTacrolimus
Tacrolimus
DRUGBusulfan
Busulfan（4 mg/kg/day,4 days）
DRUGCyclophosphamide
Cyclophosphamide（50 mg/kg/day,4 days）
DRUGFludarabine
Fludarabine（50 mg/m2/day,3 days）
DRUGThymoglobulin
Thymoglobulin（2.5 mg/kg/day,4 days）
DRUGMethotrexate
Methotrexate
Sponsors
Peking University People's Hospital
Ruijin Hospital
The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army
LiuZhou People's Hospital
The First People's Hospital of Yunnan
Liuzhou Workers' Hospital
Xinqiao Hospital of Chongqing
Affiliated Hospital of Youjiang University of Ethnic Medicine
Hainan People's Hospital
Maoming People's Hospital
Red Cross Hospital of Yulin City
Guilin Medical College
Wuzhou People's Hospital
Guangxi Autonomous Region People's Hospital
The 920th Hospital of Joint Logistics Support Force of People's Liberation Army
First Affiliated Hospital of Guangxi Medical University
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
2 Years to 20 Years
Healthy volunteers
No
Inclusion criteria
1. Diagnosed with thalassemia major. 2. Indication of hematopoietic stem cell transplantation. 3. A cardiac ejection fraction of \>50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.
Exclusion criteria
1. Aspartate aminotransferase levels \> 4-fold the upper limit of the normal range for our institution's lab criteria; 2. Uncontrolled bacterial, viral or fungal infections; 3. Any other restriction for transplantation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Overall survival | 2 years | 2-years overall survival |
| Thalassemia-free survival | 2 years | 2-years thalassemia-free survival |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Cumulative Incidence of acute Graft Versus Host Disease | 100 days | Acute graft versus host disease at day +100 |
| Engraftment | 30 days | Myeloid engraftment at day +30 |
| Cumulative Incidence of Infectious Complications | 2 years | Cumulative incidence of bacterial, fungal and viral infections by 2 years |
| Cumulative Incidence of chronic Graft Versus Host Disease | 2 years | Chronic graft versus host disease by 2 years |
| Transplant Related Mortality | 2 year | Transplant-related mortality by 2 year |
Countries
China
Outcome results
None listed