Contributions
Abstract: EP1249
Type: E-Poster Presentation
Session title: Stem cell transplantation - Clinical
Background
Severe congenital neutropenia(SCN) is a preleukemic bone marrow failure syndrome.Patients are at considerable risk of infections and evolving into leukemia or myelodysplastic syndrome (MDS).According to the reports of international that allogeneic hematopoietic stem cell transplantation (HSCT) is the curative treatment of SCN, but data on outcome about that are scarce in our country.
Aims
The aim of the present study is to clarify the cficacy of HSCT on patients with severe congenital neutropenia(SCN).
Methods
We retrospectively analyzed clinical characteristics about total 11 patients with SCN who underwent HSCT in our hospital from April 2015 to July 2020. ALL patients with EL. ANE-gene mutation.The median age was 3 (1-14) years. Male to female was 7:4.The median discase course from diagnosis to transplant was 37 (8-165) months.Six patients received umbilical cord blood-HSCT,the conditioning regimen was:Fludarabine(40mg/m2/d×5days)+Ara-c(2g/m2/d×5days)+Bu(0.8mg-1.2mg/KgQ6h×4days)+cyclophosphamide(1.8g/m2/d×2days)+ATG(Thymoglobuline,SANOFI,1.25mg/Kg/d×2days).Two patients underwent matched unrelated donor(MUD)-HSCT,two patients underwent Haplo-HSCT,and another one were sibling donor-HSCT,the conditioning regimen was:Ara-c(3g/m2/d×3days)+Bu(0.8mg-1.2mg/Kg Q6h×4days)+Fludarabine(40mg/m2/d×4days)+ATG(ATG-Fresenius S,4mg/Kg/d×4days or p-ALG 20mg/Kg/d×2days for sibling donor-HSCT).For GVHD prophylaxis,CsA/tacrolimus plus MTX were used.
Results
All of the 11 patients tolerated pretreatment successfully.the total implantation rate was 100%.The median time of neutrophil sngraftment was 11 (9-16) days;the median time of platelet engraftment was 12(9-36)days.During the median follow-up period of 25 (range,3-70 months) months:the incidence of CMV was 72.7%;EBV DNA load of all patients who infected with EBV were undetectable after transplant.Grade II-IVaGVHD occurred in 4 cases,the probability was 36.3% .Two cases had cGVHD. Two patients died of GVHD,and the rest of the patients survived, with an overall survival rate (OS) were 81.81%.
Conclusion
Our results have shown that HSCT has higher implantationrate and overall survival rate,and may become a viable therapeutic option. Nevertheless, because of the transplant-related death, a careful selection of HSCT candidates and the time point for HSCT should be undertaken.
Keyword(s): Hematopoietic cell transplantation, Severe congenital neutropenia
Abstract: EP1249
Type: E-Poster Presentation
Session title: Stem cell transplantation - Clinical
Background
Severe congenital neutropenia(SCN) is a preleukemic bone marrow failure syndrome.Patients are at considerable risk of infections and evolving into leukemia or myelodysplastic syndrome (MDS).According to the reports of international that allogeneic hematopoietic stem cell transplantation (HSCT) is the curative treatment of SCN, but data on outcome about that are scarce in our country.
Aims
The aim of the present study is to clarify the cficacy of HSCT on patients with severe congenital neutropenia(SCN).
Methods
We retrospectively analyzed clinical characteristics about total 11 patients with SCN who underwent HSCT in our hospital from April 2015 to July 2020. ALL patients with EL. ANE-gene mutation.The median age was 3 (1-14) years. Male to female was 7:4.The median discase course from diagnosis to transplant was 37 (8-165) months.Six patients received umbilical cord blood-HSCT,the conditioning regimen was:Fludarabine(40mg/m2/d×5days)+Ara-c(2g/m2/d×5days)+Bu(0.8mg-1.2mg/KgQ6h×4days)+cyclophosphamide(1.8g/m2/d×2days)+ATG(Thymoglobuline,SANOFI,1.25mg/Kg/d×2days).Two patients underwent matched unrelated donor(MUD)-HSCT,two patients underwent Haplo-HSCT,and another one were sibling donor-HSCT,the conditioning regimen was:Ara-c(3g/m2/d×3days)+Bu(0.8mg-1.2mg/Kg Q6h×4days)+Fludarabine(40mg/m2/d×4days)+ATG(ATG-Fresenius S,4mg/Kg/d×4days or p-ALG 20mg/Kg/d×2days for sibling donor-HSCT).For GVHD prophylaxis,CsA/tacrolimus plus MTX were used.
Results
All of the 11 patients tolerated pretreatment successfully.the total implantation rate was 100%.The median time of neutrophil sngraftment was 11 (9-16) days;the median time of platelet engraftment was 12(9-36)days.During the median follow-up period of 25 (range,3-70 months) months:the incidence of CMV was 72.7%;EBV DNA load of all patients who infected with EBV were undetectable after transplant.Grade II-IVaGVHD occurred in 4 cases,the probability was 36.3% .Two cases had cGVHD. Two patients died of GVHD,and the rest of the patients survived, with an overall survival rate (OS) were 81.81%.
Conclusion
Our results have shown that HSCT has higher implantationrate and overall survival rate,and may become a viable therapeutic option. Nevertheless, because of the transplant-related death, a careful selection of HSCT candidates and the time point for HSCT should be undertaken.
Keyword(s): Hematopoietic cell transplantation, Severe congenital neutropenia