Efficacy Of Single And Double Umbilical Cord Blood Stem Cell Transplantation In The Treatment Of Children With Hematological Diseases

Allogeneic hematopoietic stem cell transplantation(allo-HSCT)is an important way to cure refractory hematological malignancies and immunodeficiency disease.Since the success of Cord Blood Transplantation(CBT)in the world in 1988 and the establishment and improvement of umbilical cord blood banks all over the world,umbilical cord blood transplantation has been increasingly used in the treatment of diseases in many countries,especially in Europe and America and Japan.World multi-center cases reported that unrelated HLA 1 to 2 locus mismatched CBT and unrelated HLA matched bone marrow transplantation has the same long-term efficacy and it identifies the status of CBT in child and adult hematopoietic stem cell transplantation.In this study,we retrospectively analyzed the clinical data of 52 children with cord blood transplantation in our hospital.The aim of this study was to compare the effects of single umbilical cord blood and double umbilical cord blood on neutrophil engraftment,platelet engraftment,graft versus host disease(GVHD),infection,transplantation-related mortality(TRM)and overall survival(OS),and provide technical indicators for the selection of cord blood transplantation donor in children.ObjectiveTo analyze the curative effect and prognosis of children with hematologic disease treated by single and double umbilical cord blood stem cell transplantation,and to provide technical indicators for the selection of cord blood transplantation donor in children.MethodFifty-two children with hematological diseases were treated with unrelated umbilical cord blood transplantation,35 males and 17 females were enrolled in the Children's Hospital of Suzhou University from April 2011 to July 2016,including 37 cases of single umbilical cord blood transplantation,15 cases of double umbilical cord blood transplantation.There were 32 cases of malignant hematologic disease,including 19 cases of acute myeloid leukemia(AML),8 cases of acute lymphoblastic leukemia(ALL),1 case of chronic myeloid leukemia(CML),4 cases of myelodysplastic syndrome(MDS)and 20cases of non-maligance hematology disease,including 12 cases of Wiskott-Aldrich syndrome(WAS),3 cases of severe aplastic anemia(SAA),1 cases of Fanconi anemia(FA),severe combined immun deficiency(SCID)1 case,1 case of congenital immunodeficiency disease,1 case of congenital granulocyte deficiency,1 case of high IgM syndrome.Low-resolution HLA-A,B and DRB1 were performed on cord blood and recipients.The number of total nucleated cells in single cord blood transplantation is(1.4-17.76)x10~7/kg,the median of 6.11x10~7/kg;the number of CD34+cells is(0.6-18.4)x10~5/kg,the median of 2.9 x10~5/kg;The number of total nucleated cells in double umbilical cord blood is(1.45-7.61)x10~7/kg,the median of 3.2x10~7/kg;the number of CD34+cells is(0.8-5)x10~5/kg,the median of 2.9 x10~5/kg.The conditioning regimens was mainly modified with Maryland/cyclophosphamide(BU/CY)or total body irradiation/cyclophosphamide(TBI/CY).Allreceptorsreceivedenhancedcombined immunosuppression of cyclosporin A(or tacrolimus)and mycophenolate mofetil(CsA+MMF)to prevent graft versus host disease(GVHD).Active prevention and treatment of other complications after transplantation.The follow-up period was February 28,2017,and the median follow-up was 13 months(2 days to 70 months).The effects of 52 cases of cord blood transplantation were analyzed retrospectively.The number of umbilical cord blood was divided into single umbilical cord blood transplantation group and double umbilical cord blood transplantation group.Comparing the neutrophil engraftment,platelet engraftment,incidence of aGVHD,cytomegalovirus infection,transplant-related mortality(TRM),and overall survival between two groups.Results(1)The engraftment rate of single CBT was 91.2%(31/34),and the engraftment rate of CBT was 93.33%(14/15).There was no significant difference between the two groups(P=1.00).(2)The neutrophil engraftment time for Single CBT is 16(14-21)days,the time for double CBT is 18(16-24)days;The neutrophil engraftment time for double CBT was delayed than single CBT(P<0.05).the platelet engraftment time for single CBT was 20(16-45)days;the time for double CBT is 25(20-84)days,the platelet engraftment time for double CBT was delayed than single CBT(P<0.05).(3)The incidence of acute GVHD at stage II-IV and III-IV after UCBT was 55.6%and 33.3%,respectively.There was no significant difference between the two groups(50%vs 58.1%vs CBT,P>0.05;21.4%vs 38.3%,P>0.05).(4)Successful engraftment and survival of more than 100 days patients in 40 cases,the occurrence of cGVHD were 9 cases;cGVHD incidence for single CBT was 25%(7/28);the incidence of cGVHD for double CBT was 16.7%(2/12);the difference between the two groups was not statistically significant(P>0.05).(5)CMV infection rate was 58.8%(20/34)for single CBT,and the CMV infection rate for double CBT was 73.3%(11/15).There was no significant difference between the two groups(P>0.05)).(6)52 cases of total transplant-related mortality(TRM)was 23.1%,of which 8 cases is single CBT,the TRM for single CBT was 21.6%;4 cases is double CBT,the TRM for double CBT is 26.7%,There was no significant difference between the two groups(P>0.05).(7)At the end of the follow-up period,13 of the 52 children died and the 1-year overall survival rate was 75.5%.The 1-year overall survival rate of single CBT was 76.6%;the 1-year overall survival rate of CBT was 73.3%,which was no statistically significant difference(P>0.05).Conclusion(1)There was no significant difference in the engraftment rate,the incidence and the severity of aGVHD,CMV infection,transplantation-related mortality,overall survival rate between double CBT and single CBT.(2)The neutrophils and platelet engraftment time for double CBT were delayed compared to single CBT.