In recent decades,haploidentical stem cell transplantation(haplo-SCT)to treat severe aplastic anemia(SAA)has achieved remarkable progress.However,long-term results are still lacking.We conducted a multicenter prospect...In recent decades,haploidentical stem cell transplantation(haplo-SCT)to treat severe aplastic anemia(SAA)has achieved remarkable progress.However,long-term results are still lacking.We conducted a multicenter prospective study involving SAA patients who underwent haplo-SCT as salvage therapy.Long-term outcomes were assessed,mainly focusing on survival and quality of life(QoL).Longitudinal QoL was prospectively evaluated during pretransplantation and at 3 and 5 years posttransplantation using the SF-36 scale in adults and the PedsQL 4.0 scale in children.A total of 287 SAA patients were enrolled,and the median follow-up was 4.56 years(range,3.01–9.05 years)among surviving patients.During the long-term follow-up,268 of 275 evaluable patients(97.5%)obtained sustained full donor chimerism,and 93.4%had complete hematopoietic recovery.The estimated overall survival and failure-free survival for the whole cohort at 9 years were 85.4%±2.1%and 84.0%±2.2%,respectively.Age(≥18 years)and a poorer performance status(ECOG>1)were identified as risk factors for survival outcomes.For Qo L recovery after haplo-SCT,we found that QoL progressively improved from pretransplantation to the 3-year and 5-year time points with statistical significance.The occurrence of chronic graft versus host disease was a risk factor predicting poorer QoL scores in both the child and adult cohorts.At the last followup,74.0%of children and 72.9%of adults returned to normal school or work.These inspiring long-term outcomes suggest that salvage transplantation with haploidentical donors can be routine practice for SAA patients without human leukocyte antigen(HLA)-matched donors.展开更多
基金supported by the Foundation for Innovative Research Groups of the National Natural Science Foundation of China(81621001)the National Natural Science Foundation of China(82100227)+1 种基金the Key Program of National Natural Science Foundation of China(81930004)the National Key Research and Development Program of China(2017YFA0104500)。
文摘In recent decades,haploidentical stem cell transplantation(haplo-SCT)to treat severe aplastic anemia(SAA)has achieved remarkable progress.However,long-term results are still lacking.We conducted a multicenter prospective study involving SAA patients who underwent haplo-SCT as salvage therapy.Long-term outcomes were assessed,mainly focusing on survival and quality of life(QoL).Longitudinal QoL was prospectively evaluated during pretransplantation and at 3 and 5 years posttransplantation using the SF-36 scale in adults and the PedsQL 4.0 scale in children.A total of 287 SAA patients were enrolled,and the median follow-up was 4.56 years(range,3.01–9.05 years)among surviving patients.During the long-term follow-up,268 of 275 evaluable patients(97.5%)obtained sustained full donor chimerism,and 93.4%had complete hematopoietic recovery.The estimated overall survival and failure-free survival for the whole cohort at 9 years were 85.4%±2.1%and 84.0%±2.2%,respectively.Age(≥18 years)and a poorer performance status(ECOG>1)were identified as risk factors for survival outcomes.For Qo L recovery after haplo-SCT,we found that QoL progressively improved from pretransplantation to the 3-year and 5-year time points with statistical significance.The occurrence of chronic graft versus host disease was a risk factor predicting poorer QoL scores in both the child and adult cohorts.At the last followup,74.0%of children and 72.9%of adults returned to normal school or work.These inspiring long-term outcomes suggest that salvage transplantation with haploidentical donors can be routine practice for SAA patients without human leukocyte antigen(HLA)-matched donors.
