This study aims to compare the efficacy and safety of HLA-haploidentical hematopoietic stem cell transplantation (HLA-haplo HSCT) versus optimal immunosuppressive therapy (IST) as first-line treatments for severe aplastic anemia (SAA) through a real-world cohort design. The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
116
The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation. Patients in the immunosuppressive therapy group receive a regimen comprising anti-thymocyte globulin (ATG) + cyclosporine A (CSA) + thrombopoietin receptor agonist (TPO-RA).
The selection of treatment regimens for subjects is based on clinical decision-making in real-world practice, comprehensively considering factors including patient age, donor matching status, comorbidities, and treatment preferences, with non-randomized group allocation. Patients in the transplantation group undergo haploidentical hematopoietic stem cell transplantation (haplo-HSCT).
Failure Free Survival, FFS
survival with complete response whereas death, graft failure and relapse are considered treatment failures
Time frame: 2 year
Overall Survival, OS
Time frame: 2 year
Treatment Related Mortality, TRM
Transplantation-related mortality (TRM) was defined as death without graft failure
Time frame: 2 year
hematology remission
hematology recovery, including WBC, Hb and PLT
Time frame: 2 year
Infection incidence
Time frame: 2 year
GVHD incidence
Time frame: 2 year
QoL evaluation
life quality assessment
Time frame: 2 year
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