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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Haploidentical HSCT | Experimental | 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） |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfan | Drug | Busulfan（4 mg/kg/day,4 days） |
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| Measure | Description | Time Frame |
|---|---|---|
| Overall Survival | 2-years overall survival | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Disease Free Survival | 2-years disease free survival | 2 years |
| Engraftment | Myeloid engraftment at day +30 | 30 days |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yongrong Lai, MD | Contact | +86（0771）5356510 | laiyongrong@hotmail.com | |
| Meiqing Wu, MD | Contact | +86（0771）5356510 | wumeiqing1309@163.com |
| Name | Affiliation | Role |
|---|---|---|
| Yongrong Lai, MD | First Affiliated Hospital of Guangxi Medical University | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| First Affiliated Hospital of Guangxi Medical University | Recruiting | Nanning | Guangxi | 510515 | China |
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| Cyclophosphamide | Drug | Cyclophosphamide（50 mg/kg/day,4 days） |
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| Fludarabine | Drug | Fludarabine（50 mg/m2/day,3 days） |
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| Mycophenolate mofetil | Drug | Mycophenolate mofetil（0.25g/day） |
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| Tacrolimus | Drug | Tacrolimus（0.03mg/kg/day） |
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| Methotrexate | Drug | Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11） |
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| Thymoglobulin | Drug | Thymoglobulin（2.5 mg/kg/day,4 days） |
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| Basiliximab | Drug | Basiliximab（10mg on day 0 and +4） |
|
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| 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 |
| ID | Term |
|---|---|
| D017086 | beta-Thalassemia |
| ID | Term |
|---|---|
| D013789 | Thalassemia |
| D000745 | Anemia, Hemolytic, Congenital |
| D000743 | Anemia, Hemolytic |
| D000740 | Anemia |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006453 | Hemoglobinopathies |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
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| ID | Term |
|---|---|
| D002066 | Busulfan |
| D003520 | Cyclophosphamide |
| C024352 | fludarabine |
| D007252 | Influenza Vaccines |
| D009173 | Mycophenolic Acid |
| D016559 | Tacrolimus |
| D008727 | Methotrexate |
| C512542 | thymoglobulin |
| D000077552 | Basiliximab |
| ID | Term |
|---|---|
| D002072 | Butylene Glycols |
| D006018 | Glycols |
| D000438 | Alcohols |
| D009930 | Organic Chemicals |
| D008698 | Mesylates |
| D000476 | Alkanesulfonates |
| D017738 | Alkanesulfonic Acids |
| D000473 | Alkanes |
| D006839 | Hydrocarbons, Acyclic |
| D006838 | Hydrocarbons |
| D013451 | Sulfonic Acids |
| D013456 | Sulfur Acids |
| D013457 | Sulfur Compounds |
| D010752 | Phosphoramide Mustards |
| D009588 | Nitrogen Mustard Compounds |
| D009150 | Mustard Compounds |
| D006846 | Hydrocarbons, Halogenated |
| D063088 | Phosphoramides |
| D009943 | Organophosphorus Compounds |
| D014765 | Viral Vaccines |
| D014612 | Vaccines |
| D001688 | Biological Products |
| D045424 | Complex Mixtures |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D005227 | Fatty Acids |
| D008055 | Lipids |
| D018942 | Macrolides |
| D007783 | Lactones |
| D000630 | Aminopterin |
| D011622 | Pterins |
| D011621 | Pteridines |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| D006571 | Heterocyclic Compounds |
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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