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

  • End date
    May 31, 2023
  • participants needed
  • sponsor
    First Affiliated Hospital of Guangxi Medical University
Updated on 11 February 2022
stem cell transplantation
mycophenolate mofetil
ejection fraction
cell transplantation
ineffective erythropoiesis


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.

Condition Thalassemia Major
Treatment cyclophosphamide, methotrexate, mycophenolate mofetil, busulfan, Fludarabine, Tacrolimus, Ruxolitinib, Thymoglobulin, Basiliximab, Cyclosporine A
Clinical Study IdentifierNCT04009525
SponsorFirst Affiliated Hospital of Guangxi Medical University
Last Modified on11 February 2022


Yes No Not Sure

Inclusion Criteria

Diagnosed with thalassemia major
Indication of hematopoietic stem cell transplantation
A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function

Exclusion Criteria

Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria
Uncontrolled bacterial, viral or fungal infections
Any other restriction for transplantation
Clear my responses

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