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| Name | Class |
|---|---|
| Peking University People's Hospital | OTHER |
| Ruijin Hospital | OTHER |
| The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MSD-HSCT | Experimental | matched sibling donors hematopoietic stem cell transplantation |
|
| URD-HSCT | Experimental | unrelated donor hematopoietic stem cell transplantation |
|
| haplo-HSCT | Experimental | haplo-identical hematopoietic stem cell transplantation |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Busulfan | Drug | Busulfan（4 mg/kg/day,4 days） |
|
| Measure | Description | Time Frame |
|---|---|---|
| Overall survival | 2-years overall survival | 2 years |
| Thalassemia-free survival | 2-years thalassemia-free survival | 2 years |
| Measure | Description | Time Frame |
|---|---|---|
| Engraftment | Myeloid engraftment at day +30 | 30 days |
| Transplant Related Mortality | Transplant-related mortality by 2 year | 2 year |
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Inclusion Criteria:
Exclusion Criteria:
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| 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 | Nanning | Guangxi | 510515 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41730859 | Derived | Liu R, Xiao H, Qin C, Hu J, Luo J, Chen H, Zhang X, Yin X, Nai G, Wang G, Li Z, Tang W, Zhang H, Wang X, Lan M, Liao C, Yang T, Wang S, Huang J, He Y, Wang L, Zhang Z, Liu L, Wei Z, Shi L, Wu M, Zhou Q, Andersson BS, Huang X, Lai Y. A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia. Nat Commun. 2026 Feb 23;17(1):3083. doi: 10.1038/s41467-026-69756-8. |
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| LiuZhou People's Hospital |
| OTHER |
| The First People's Hospital of Yunnan | OTHER |
| Liuzhou Workers' Hospital | OTHER_GOV |
| Xinqiao Hospital of Chongqing | OTHER |
| Affiliated Hospital of Youjiang University of Ethnic Medicine | UNKNOWN |
| Hainan People's Hospital | OTHER |
| Maoming People's Hospital | OTHER |
| Red Cross Hospital of Yulin City | OTHER |
| Guilin Medical College | OTHER |
| Wuzhou People's Hospital | OTHER_GOV |
| Guangxi Autonomous Region People's Hospital | UNKNOWN |
| 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China | OTHER |
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| Cyclophosphamide | Drug | Cyclophosphamide（50 mg/kg/day,4 days） |
|
|
| Fludarabine | Drug | Fludarabine（50 mg/m2/day,3 days） |
|
|
| Thymoglobulin | Drug | Thymoglobulin（2.5 mg/kg/day,4 days） |
|
|
| cyclosporine A | Drug | cyclosporine A |
|
|
| Mycophenolate mofetil | Drug | Mycophenolate mofetil（0.25g/day） |
|
|
| Tacrolimus | Drug | Tacrolimus |
|
|
| Methotrexate | Drug | Methotrexate |
|
|
| Cumulative Incidence of acute Graft Versus Host Disease | Acute graft versus host disease at day +100 | 100 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 |
| C512542 | thymoglobulin |
| D016572 | Cyclosporine |
| D009173 | Mycophenolic Acid |
| D016559 | Tacrolimus |
| D008727 | Methotrexate |
| 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 |
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |
| 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 |
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