Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Study Overview
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Brief Summary
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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
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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.

Official Title
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Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major

Conditions
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Thalassemia Major

Intervention / Treatment
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* Drug: Busulfan
* Drug: Cyclophosphamide
* Drug: Fludarabine
* Drug: Mycophenolate mofetil
* Drug: Tacrolimus
* Drug: Methotrexate
* Drug: Thymoglobulin
* Drug: Basiliximab


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosed with thalassemia major Lacking of a HLA-identical sibling donor or unrelated donor Indication of haploidentical hematopoietic stem cell transplantation No restrictions for transplantation Exclusion Criteria: Any restriction for transplantation No indication of haploidentical hematopoietic stem cell transplantation

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Haploidentical HSCT<br>Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：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） | Drug: Busulfan<br>* Busulfan（4 mg/kg/day,4 days）<br>* Other names: Bu;Drug: Cyclophosphamide<br>* Cyclophosphamide（50 mg/kg/day,4 days）<br>* Other names: Cy;Drug: Fludarabine<br>* Fludarabine（50 mg/m2/day,3 days）<br>* Other names: Flu;Drug: Mycophenolate mofetil<br>* Mycophenolate mofetil（0.25g/day）<br>* Other names: MMF;Drug: Tacrolimus<br>* Tacrolimus（0.03mg/kg/day）<br>* Other names: FK506;Drug: Methotrexate<br>* Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）<br>* Other names: MTX;Drug: Thymoglobulin<br>* Thymoglobulin（2.5 mg/kg/day,4 days）<br>* Other names: ATG;Drug: Basiliximab<br>* Basiliximab（10mg on day 0 and +4）<br>* Other names: Simulect;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Overall Survival | 2-years overall survival | 2 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease Free Survival | 2-years disease free survival | 2 years | 
| Engraftment | Myeloid engraftment at day +30 | 30 days | 
| Transplant Related Mortality | Transplant-related mortality by 1 year | 1 year | 
| Cumulative Incidence of acute Graft Versus Host Disease | Acute graft versus host disease at day +180 | 180 days | 
| Cumulative Incidence of chronic Graft Versus Host Disease | Chronic graft versus host disease by 2 years | 2 years | 
| Cumulative Incidence of Infectious Complications | Cumulative incidence of bacterial, fungal and viral infections by 2 years | 2 years |