[Clinical Characteristics and Prognosis of Children with Myelodysplastic Syndrome].

OBJECTIVE: To analyze the clinical characteristics and prognosis of 40 children with myelodysplastic syndrome (MDS), and provide ideas for clinical diagnosis and treatment. METHODS: The clinical characteristics, risk stratification, and different treatment regimens of 40 cases with MDS admitted in Department of Hematology of Children's Hospital of Soochow University from January 1, 2011 to December 31, 2017 was retrospectively analyzed. Kaplan-Meier survival curve were used to estimate 3-year overall survival (OS) rate and event-free survival (EFS) rate. RESULTS: In 40 cases, the ratio of male to female was 1.4∶1.0, male was more than female, and median age was 6.0 years old. Among them, refractory cytopenia (MDS-RCC) was the most common type, and 11 cases were chromosomal abnormalities, 21 cases genetic abnormalities. Fifteen cases accepted hematopoietic stem cell transplantation (HSCT) treatment, while 25 cases did not but drug therapy alone. The 3-year OS rate of the cases who accepted HSCT or not was (72.2±12.2)% and (35.3±10.2)% (P=0.039), 3-year EFS rate was (65.0±12.9)% and (19.2±8.4)% (P=0.012), respectively. Cox regression analysis showed that age < 7 years old (P=0.0333), initial diagnosed platelet < 50×109/L (P=0.007), presence of complex karyotypes and/or gene mutations (P=0.0002), and treatment without HSCT (P=0.016) were the high-risk factors of prognosis. All the children were classified according to IPSS, WPSS and IPSS-R, while analysis result showed that the above three risk assessment had limitations for risk assessment of MDS in children, they could not comprehensively assess the prognosis of children with MDS. CONCLUSION: MDS-RCC in children is more common. Cox multivariate analysis shows that age < 7 years old, initial diagnosed platelet < 50×109/L, presence of complex karyotypes and/or gene mutation, and treatment without HSCT are the high-risk factors of prognosis in children with MDS. HSCT is the most effective treatment to cure children with MDS at present. The current methods such as IPSS-R commonly used in assessment of prognosis in children with MDS show obvious limitation.

[Clinical Significance of Minimal Residual Disease in Risk Stratification and Prognosis of Childhood B-lineage Acute Lymphoblastic Leukemia].

OBJECTIVE: To explore clinical significance of monitoring the level of minimal residual disease (MRD) at different time point in the risk stratification and prognosis of Childhood B-lineage Acute Lymphoblastic Leukemia. METHODS: Three hundred and eighty cases of children's B-ALL from Augest 2008 to January 2013 in our hospital were enrolled in this study. MRD levels were detected at day 15, day 33 and week 12 after initial chemotherapy. The event-free survival(EFS) and overall survival (OS) were measured on the basis of MRD levels at different stages of chemotherapy and were compared by Kaplan Meier analyses. RESULTS: The patient's age, initial white blood cell count, chromosome, MLL, BCR/ABL, pretreatment reaction, bone marrow MRD at days 33 were closely related with the 5-year EFS rate. Multiparameter flow cytometry showed the marked MRD and unmarked MRD were not significantly different between their 5-year EFS rate(P>0.05), and the every immune phenotype was also no significantly different between the 5-year EFS rate(P>0.05). The children with MRD≥10-2 at day 15(P<0.01), MRD≥10-3 at day 33 (P<0.01) and MRD≥10-3 on week 12(P<0.01) have a decreased 5-year EFS rate and overall survival, which related with poor prognosis obviously. The 5-year EFS rates at the MRD<10-4(negative), 10-4-10-3, 10-3-10-2 and ≥10-2 at day 33 were 86.6±2.7%, 77.5±4.9%, 70.1±8.0%, and 44.8±9.9%(P<0.01) with significant difference respectively; the 5-year OS rate was 89.5±2.7%, 80±4.9%, 76.0±7.8%, and 53.2±10.1% with statistically significant difference(P<0. 01). CONCLUSION: The MRD≥10-2 at day 33 is a high risk factor for significant reduction of the 5-year EFS rate and the 5-year OS rate of children with B-ALL. Thus, dynamic monitoring the MRD level can predict relapse of B-ALL after remission.

[Clinical Features and Prognostic Factors of Children with Acute Lymphoblastic Leukemia in High-Risk Group].

OBJECTIVE: To explore the clinical features and prognostic factors of pediatric acute lymphoblastic leukemia (ALL) in high-risk (HR) group. METHODS: A total of 421 children with ALL in the Children's Hospital of Soochow University from August 2008 to March 2013 were diagnosed and treated according to the Chinese Children Leukemia Group (CCLG)-2008 Protocol. Among different risk-groups, 148 cases were stratified into the low-risk group and 191 cases were included in the moderate-risk group. Eight-two patients of the high-risk group were analyzed retrospectively for their clinical features, 5-year event-free survival (EFS) rate and overall survival (OS) rate. RESULTS: The median follow-up times of 82 patients were 64 months(3.0-76.3 months), 55 patient achieved complete remission(CR) after 1 cycle of induction chemotherapy(CR rate 67.1%), 25 patients relapsed(30.5%) mainly in very early and early relapse phases, significantly different from the low-risk group (P=0.013), 27 pateitns died(32.9%). The 5-year pEFS and pOS were 57.20% and 58.5%, respectively. Ph+ or BCR/ABL+ and MRD>10-2 on the 33rd day in the high-risk group were 2 main factors influencing EFS and OS according to single factor analysis. Ph+ or BCR/ABL+ was an independent prognostic factor, however, the MRD value on the 33rd day was not statistically significant differente by virtue of COX regression analysis. CONCLUSION: The clinical feature of children with ALL in high risk group display low induction CR rate, high recurrence rate and the lower 5-year pEFS. Ph+ or BCR/ABL+ is regarded as an independent factor of poor prognosis.