Clinical Feames And Prognosis Of Children With Myelodysplastic Syndrome

Purpose:To explore the clinical characteristics,risk stratification and prognosis of children with myelodysplastic syndrome(MDS)in single center and provide evidence for standardized diagnosis and treatment of MDS in childhood.Methods:A retrospective analysis of 53 cases with myelodysplastic syndrome(MDS)admitted in Department of Hematology from January 1,2011 to December 31,2017 in Children’s Hospital Affiliated to Suzhou University was carried out.The clinical characteristics,risk stratification and different treatment regimens were analyzed.Cox regression multivariate analysis and kaplan-meier survival curve were used to predict 1-year and 3-year overall survival(OS)and event-free survival(ESF).Results:(1)For 53 cases,31 patients were male and 22 children were female and the ratio of male to female was 1.4:1.0.Male patients were little more than female patients.Median ages were 4.8 years old(0 years old-15 years old).Among them,24 cases(45%)were diagnosed as refractory hemocytopenia(MDS-RCC),refractory anemia accompanied with increased number of primordial cells(MDS-RAEB)in 9 cases(17%),RAEB to leukemia transformation or transformation(MDS-RAEB-T)in 7 cases(13%),Juvenile granulocytic monocytic leukemia(JMML)in 13 cases(25%).(2)11 cases of chromosomal abnormalities,accounting for 20%of the total number of cases,including 6 cases of 7q+alone、1 cases of Y deletion、2 cases of cen8 trisomy、1 cases of 7q+combined with Y deletion、and 1 cases of 5q+combined with 7q+.(3)51 cases were completed the genetic examination and 12 cases were abnormal(25%,12/51).Main mutation genes included PTPN11(NF1、D61Y、Q272)、ASXL1、TET2F8682(T>G)/U2AF1S34(C>T)、WT1、EVL、MPL。Others include Fanconi gene and WAS gene.(4)In addition to 13 cases of JMML,remaining 40 cases of MDS were classified according to IPSS,WPSs and IPSS-R methods.Charts and analysis showed that the above three types of risk assessment methods had limitations for MDS’s risk assessment in children.They could not comprehensively assess the prognosis of children with MDS.(5)Outcomes:16 patients underwent hematopoietic stem cell transplantation(HSCT),10 survived after transplantation,5 died of transplant failure,and 1 had missed follow-up.10 of remaining 37 children who did not receive HSCT died of this disease(27%,10/37).Over all survival rate(OS)and event free survival rate(ESF)one year in patients with HSCT vs non-HSCT were(75%±10.8%)%vs(66.7%±9.1%)(P=0.565)and(68.8%±11.6%)%vs(18.5%±7.5%)%(P=0.007)respecitively.OS and ESF three years in children with HSCT vs non-HSCT were(72.2%±12.2%)%vs(35.3%±10.2%)(P=0.039)and(68.2%±11.8%)%vs(12.5%±6.6%)%(P=0.001)respecitively.(6)Cox regression analysis showed that no HSCT treatment(P=0.016),age less than 7 years old(P=0.0333),immature localization of precursor cells(ALIP)in bone marrow biopsy(P=0.0168),platelet less than 50 x 10 9/L(P=0.007),and complicated karyotype and/or gene mutation(P=0.0002)were the high-risk factors of prognosis of MDS in children.Hematopoiesis had no effect on prognosis(P>0.05).Conclusions:(1)MDS-RCC in children was the more common type.Cox multivariate analysis showed that age less than 7 years old,ALIP in bone marrow biopsy,platelet less than 50 in initial diagnosis,presence of complex karyotype and/or mutation in gene and treatment without HSCT were the high-risk factors of prognosis.(2)HSCT is only one effective way to cure children with MDS at present.(3)The current method of IPSS-R in assessment of prognosis with MDS in adult has obvious limitation in children.