The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
30
Busulfan（4 mg/kg/day,4 days）
Cyclophosphamide（50 mg/kg/day,4 days）
Fludarabine（50 mg/m2/day,3 days）
First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, China
RECRUITINGOverall Survival
2-years overall survival
Time frame: 2 years
Disease Free Survival
2-years disease free survival
Time frame: 2 years
Engraftment
Myeloid engraftment at day +30
Time frame: 30 days
Transplant Related Mortality
Transplant-related mortality by 1 year
Time frame: 1 year
Cumulative Incidence of acute Graft Versus Host Disease
Acute graft versus host disease at day +180
Time frame: 180 days
Cumulative Incidence of chronic Graft Versus Host Disease
Chronic graft versus host disease by 2 years
Time frame: 2 years
Cumulative Incidence of Infectious Complications
Cumulative incidence of bacterial, fungal and viral infections by 2 years
Time frame: 2 years
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Mycophenolate mofetil（0.25g/day）
Tacrolimus（0.03mg/kg/day）
Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
Thymoglobulin（2.5 mg/kg/day,4 days）
Basiliximab（10mg on day 0 and +4）