Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study

First Affiliated Hospital of Guangxi Medical University

Status and phase: Completed; Phase 4

Conditions: Thalassemia Major

Treatments: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Drug: Fludarabine; Drug: Methotrexate; Drug: Thymoglobulin; Drug: cyclosporine A

Study type: Interventional

Funder types: Other

Identifiers

NCT04009525

GX-HSCT-MT 2019

Details and patient eligibility

About

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.

Full 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.

Enrollment: 823 patients

Sex: All

Ages: 2 to 20 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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823 participants in 3 patient groups

MSD-HSCT

Experimental group

Description:

matched sibling donors hematopoietic stem cell transplantation

Treatment:

Drug: cyclosporine A

Drug: Thymoglobulin

Drug: Methotrexate

Drug: Fludarabine

Drug: Mycophenolate mofetil

Drug: Cyclophosphamide

Drug: Busulfan

URD-HSCT

Experimental group

Description:

unrelated donor hematopoietic stem cell transplantation

Treatment:

Drug: Thymoglobulin

Drug: Methotrexate

Drug: Fludarabine

Drug: Mycophenolate mofetil

Drug: Tacrolimus

Drug: Cyclophosphamide

Drug: Busulfan

haplo-HSCT

Experimental group

Description:

haplo-identical hematopoietic stem cell transplantation

Treatment:

Drug: Thymoglobulin

Drug: Methotrexate

Drug: Fludarabine

Drug: Mycophenolate mofetil

Drug: Tacrolimus

Drug: Cyclophosphamide

Drug: Busulfan