Comparison Of HLA Congruent Relative And Unrelated Thalassemia Transplantation

Research BackgroundThalassemia is an autosomal recessive genetic disease.Currently,there are treatment methods such as blood transfusion,iron removal,hematopoietic stem cell transplantation and gene therapy.Among them,hematopoietic stem cell transplantation is the most mature method to cure the disease.In clinical thalassemia transplantation,the first consideration is to find a HLA-matched donor.In the case of HLA-matched,different stem cell sources and grafts will still lead to different transplantation effects.PurposeTo compare the effects of HLA congruent relative and HLA congruent unrelated thalassemia transplantation;to explore whether different transplants will lead to significant differences in survival rate and event-free survival rate under the premise of HLA congruent relative;to explore whether there is a certain advantage in the treatment of thalassemia with fully matched fresh umbilical cord blood transplantation.MethodCollected the relevant case data of all relative and unrelated HLA congruent thalassaemia transplantation from 2009 to 2020 in Nanfang Hospital Pediatrics,and the relevant case data of all relative HLA congruent thalassaemia transplants from 2009 to 2020 and conducted a retrospective analysis.The deadline for follow-up is December 31,2021.The transplant-related characteristics of patients and donors before transplantation,the differences in pre-treatment and GVHD prevention drugs,and the post-transplant survival rate,event-free survival rate,disease-related mortality rate,engraftment status,post-transplant infection status,the incidence of GVHD and other complications was compared between each transplantation method.ResultA total of 474 patients with thalassemia major were included.In the comparison group of relative and unrelated HLA congruent thalassaemia transplantation,there were 154 relative HLA congruent transplants and 213 unrelated HLA congruent transplants,both of which were derived from peripheral stems,the median follow-up time was 31 months,the median follow-up for implantation was 31 months.In the relative HLA congruent thalassaemia transplantation comparison group,there were 43 cases of fresh cord blood transplantation,18 cases of bone marrow transplantation,154 cases of peripheral stem transplantation,23 cases of frozen cord blood+bone marrow transplantation,and 22 cases frozen cord blood+peripheral stem transplantation,the median follow-up time was 33 months,the median follow-up for implantation was 33 months.(1)The OS and EFS of relative HLA congruent transplantation and unrelated HLA congruent transplantation in our center were 95.9±1.7%and 95.2±1,5%(P=0.71),95.9±1.7%and 93.8±1.7%(P=0.34)respectively,there was no significant difference;the TRM of relative HLA congruent transplantation was 3.9%,and the TRM of unrelated HLA congruent transplantation was 4.7%.(2)There was no significant difference in the engraftment and the three-line engraftment time between the relative HLA congruent transplantation and the unrelated HLA congruent transplantation,but the platelet engraftment situation in the unrelated group was worse than that in the congruent group(P=0.007).(3)There was no significant difference in infection and VOD between the relative HLA congruent transplantation and the unrelated HLA congruent transplantation during transplantation;but after transplantation,the unrelated transplantation group was more prone to complications of urinary system(P=0.005)and ear-nose-throat(P=0.02).After transplantation,the unrelated transplantation group was more prone to CMV(P=0.047)and polyoma virus infection(P=0.003).(4)Gram-positive bacilli and gram-positive cocci were the main types of bacteria that appeared to be infected within 1 year after transplantation.(5)The unrelated transplantation group was more likely to have GVHD than the relative transplantation group(P<0.001),whether it was acute GVHD(P<0.001)or chronic GVHD(P=0.007),the occurrence site was more in the skin(P<0.001 and P=0.02).(6)After transplantation,there was no significant difference in the changes of serum ferritin between relative HLA congruent transplantation and unrelated HLA congruent transplantation.(7)In relative HLA congruence transplantation,compared with other transplantation methods,fresh cord blood transplantation has higher OS and lower EFS,but there is no significant statistical significance.(8)Three-lineage engraftment time for fresh cord blood transplant was longer and statistically significant than other transplant groups,and the three-line engraftment was worse than that of peripheral biood stem cell transplantation(P<0.001,P<0.001,P<0.001).(9)Compared with the other transplant groups,the fresh umbilical cord blood transplant group had no obvious advantage in infection after transplantation,and there was no significant difference in the occurrence of VOD.(10)The incidence of GVHD,the proportion of acute and chronic GVHD,and the severity of acute GVHD in fresh cord blood transplantation were lower than other transplantation methods,but there was no significant statistical significance.ConclusionThere was no significant difference in OS and EFS between relative HLA congruent and unrelated HLA congruent thalassaemia transplants,but the incidence of chronic GVHD was significantly higher in unrelated transplants,and most of them were skin and lung.In relative HLA congruent transplantation,fresh umbilical cord blood transplantation has no obvious advantage over other transplantation methods.