Analysis of efficacy and prognostic factors in transfusion-dependent thalassemia following allogeneic hematopoietic stem cell transplantation using the ‘gx-07-tm’ regimen
Retrospective analysis shows improved survival in transfusion-dependent thalassemia after allogeneic HSCT using GX-07-TM, highlighting key prognostic factors.
Key Points
To evaluate the efficacy and identify prognostic factors in transfusion-dependent thalassemia patients undergoing allogeneic hematopoietic stem cell transplantation with GX-07-TM.
Retrospective analysis of 1,010 TDT patients undergoing allo-HSCT using GX-07-TM.
Analyzed survival rates and constructed a TFS prediction model using multivariable Cox regression.
Follow-up duration was a median of 65 months.
Cumulative 2-year overall survival was 93.9% and transplant-free survival was 93.3%.
Outcomes improved after 2020, with 2-year OS at 95.7% compared to 92.6% before 2020.
Independent risk factors for inferior transplant-free survival included age over 9 years, pneumonia, and acute graft-versus-host disease.