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1.
Article in Chinese | WPRIM | ID: wpr-880138

ABSTRACT

OBJECTIVE@#To retrospective analyze the reason of death in children with acute lymphoblastic leukemia (ALL) treated with CCLG-ALL 2008 protocol, and the experience was summarized in order to reduce the mortality.@*METHODS@#916 children diagnosed as ALL and accepted CCLG-ALL 2008 protocol from April 2008 to April 2015 in our hospital were enrolled, the dead cases in them were analyzed retrospectively.@*RESULTS@#169 children died, including 111 (65.7%) males and 58 (34.3%) females. Recurrence was the main reason of death. 150 (88.7%) children died due to recurrence, among them, 86 (57.3%) cases gave up directly. The second reason of death was infection. The main clinical sites of infection were concentrated in respiratory system and digestive system. Bacterial infection was most common (Gram-negative was common).@*CONCLUSION@#Enough finance and improving family compliance can decrease the mortality in children with ALL. Early rational use of antibiotics can reduce infection-related mortality in children with ALL.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols , Child , Disease-Free Survival , Female , Humans , Male , Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy , Prognosis , Retrospective Studies
2.
Article in Chinese | WPRIM | ID: wpr-880028

ABSTRACT

OBJECTIVE@#To analyze the outcomes of the children suffered from philadelphia chromosome positive acute lymphoblastic leukemia (Ph@*METHODS@#21 cases of firstly diagnosed Ph@*RESULTS@#Among 21 patients, 17 were male and 4 were female with a median age of 8 years old (range, 4-12 years), the median follow-up time was 30 moths (range, 10-133 months). All the patients were treated with chemotherapy induced by the high-risk project of CCLG-ALL 2008. Among 14 patients treated with TKI plus chemotherapy, nine patients achieved complete remission. During 3 months after treatment, patients without complete molecular response or with the second complete remission and intensity desire of transplantation were treated with allo-HSCT, among 9 patients with allo-HSCT, six patients achieved long term survival.@*CONCLUSION@#At TKI era, TKI combined with strong chemotherapy can make Ph


Subject(s)
Aged , Child , Female , Hematopoietic Stem Cell Transplantation , Humans , Infant , Male , Philadelphia Chromosome , Precursor Cell Lymphoblastic Leukemia-Lymphoma/therapy , Protein Kinase Inhibitors , Retrospective Studies
3.
Article in Chinese | WPRIM | ID: wpr-879845

ABSTRACT

OBJECTIVE@#To study the clinical features and prognosis of childhood acute myeloid leukemia with myelodysplasia-related changes (AML-MRC).@*METHODS@#A retrospective analysis was performed on the medical data of 14 children who were diagnosed with AML-MRC from June 2014 to March 2020, including clinical features, laboratory examination results, and prognosis.@*RESULTS@#Among the 14 children with AML-MRC, there were 9 boys and 5 girls, with a median age of 11 years (range: 1-17 years), a median leukocyte count of 8.3×10@*CONCLUSIONS@#Childhood AML-MRC is often observed in boys, and AML-M5 is the most common type based on FAB classification. Such children tend to have a poor prognosis. HSCT is expected to improve the poor prognosis of children with AML-MRC. However due to the small number of cases, it is necessary to increase the number of cases for further observation.


Subject(s)
Adolescent , Child , Child, Preschool , Female , Hematopoietic Stem Cell Transplantation , Humans , Infant , Leukemia, Myeloid, Acute/therapy , Male , Myelodysplastic Syndromes/therapy , Prognosis , Retrospective Studies
4.
Journal of Experimental Hematology ; (6): 1896-1902, 2021.
Article in Chinese | WPRIM | ID: wpr-922220

ABSTRACT

OBJECTIVE@#To analyze the clinical characteristics and factors affecting prognosis in children with severe aplastic anemia (SAA).@*METHODS@#Two hundred and five children with SAA treated in our department from January 2008 to April 2018 were selected, and the clinical characteristics and factors affecting prognosis were retrospectively analyzed.@*RESULTS@#Among 205 SAA children, the effective rate (CR+PR) at 3, 6 and 12 months after immunosuppressive therapy (IST) treatment was 50.9%, 59.0% and 73.9%, respectively, and 5-year overall survival rate was 93.1%±2.0%. Univariate analysis showed that 5-year overall survival rate of SAA children of spontaneous delivery was higher than that of cesarean section (P=0.039), while multivariate analysis showed that birth way had no significant influence on 5-year overall survival rate (P>0.05). The response rate at 3 months after IST of children with a recent history of decoration before SAA onset was higher than those without history of decoration (P<0.05).@*CONCLUSION@#Most of the SAA children can achieve high response rate and overall survival rate. Patients with recent history of home/school decoration may be the factor affecting hematological response after 3 months of IST, but have no influence on long-term overall survival.


Subject(s)
Anemia, Aplastic , Cesarean Section , Child , Female , Humans , Immunosuppressive Agents , Pregnancy , Prognosis , Retrospective Studies , Treatment Outcome
5.
Journal of Experimental Hematology ; (6): 1831-1836, 2020.
Article in Chinese | WPRIM | ID: wpr-879979

ABSTRACT

OBJECTIVE@#To investigate the consistency between FCM and PCR on the detecting of MRD in TCF3-PBX1@*METHODS@#55 cases of paediatric TCF3-PBX1@*RESULTS@#Among the 55 children with TCF3-PBX1@*CONCLUSION@#The detection result of MRD in TCF3-PBX1 detect by FCM and PCR shows better consistency. MRD positivity detected by FCM at the end of induction therapy (day 33) predicts a high risk of relapse in TCF3-PBX1 ALL patients.


Subject(s)
Adolescent , Bone Marrow , Child , Child, Preschool , Female , Humans , Male , Neoplasm, Residual , Oncogene Proteins, Fusion/genetics , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Prognosis , Recurrence
6.
Article in Chinese | WPRIM | ID: wpr-879790

ABSTRACT

OBJECTIVE@#To study the clinical significance of minimal residual disease (MRD) in B-lineage acute lymphoblastic leukemia (B-ALL) pediatric patients with different fusion gene backgrounds.@*METHODS@#A retrospective analysis was performed on the medical data of 441 B-ALL children who were treated from January 2008 to April 2015. Among the 441 children, 336 had negative fusion gene, 79 had positive @*RESULTS@#In patients with negative fusion gene, the positive MRD group had significantly lower overall survival (OS) rate and event-free survival (EFS) rate (@*CONCLUSIONS@#MRD has the most definite prognostic significance in pediatric B-ALL patients with negative fusion gene, while it has unsatisfactory prognostic significance in those with positive


Subject(s)
Child , Core Binding Factor Alpha 2 Subunit , Disease-Free Survival , Homeodomain Proteins , Humans , Neoplasm, Residual/genetics , Oncogene Proteins, Fusion/genetics , Precursor B-Cell Lymphoblastic Leukemia-Lymphoma/genetics , Prognosis , Retrospective Studies
7.
Article in Chinese | WPRIM | ID: wpr-879771

ABSTRACT

OBJECTIVE@#To study the pharmacokinetic characteristics, clinical effect, and safety of pegylated recombinant human granulocyte colony-stimulating factor (PEG-rhG-CSF) in children with acute lymphoblastic leukemia (ALL).@*METHODS@#A prospective study was performed on children with ALL who cyclophosphamide, cytarabine, and 6-mercaptopurine were used for consolidation therapy. PEG-rhG-CSF (PEG-rhG-CSF group) or rhG-CSF (rhG-CSF group) was injected after chemotherapy. The plasma concentration of PEG-rhG-CSF was measured, and clinical outcome and safety were observed for both groups.@*RESULTS@#A total of 17 children with ALL were enrolled, with 9 children in the PEG-rhG-CSF group and 8 children in the rhG-CSF group. In the PEG-rhG-CSF group, the peak concentration of PEG-rhG-CSF was 348.2 ng/mL (range 114.7-552.0 ng/mL), the time to peak was 48 hours (range 12-72 hours), and the half life was 14.1 hours (range 11.1-18.1 hours). The plasma concentration curve of PEG-rhG-CSF was consistent with the mechanism of neutrophil-mediated clearance. Compared with the rhG-CSF group, the PEG-rhG-CSF group had a significantly shorter median time to absolute neutrophil count (ANC) recovery (P0.05).@*CONCLUSIONS@#The pharmacokinetic characteristics of PEG-rhG-CSF in children with ALL receiving consolidation chemotherapy are consistent with the mechanism of neutrophil-mediated clearance, with a short half life and fast recovery of ANC, and there are no significant differences in safety between PEG-rhG-CSF and rhG-CSF.


Subject(s)
Child , Granulocyte Colony-Stimulating Factor/therapeutic use , Humans , Neutropenia , Polyethylene Glycols , Precursor Cell Lymphoblastic Leukemia-Lymphoma/drug therapy , Prospective Studies , Recombinant Proteins
8.
Article in Chinese | WPRIM | ID: wpr-829052

ABSTRACT

OBJECTIVE@#To study the effect of apoptotic drug Navitoclax (NTX) combined with chemotherapy drug Daunorubicin (DNR) on apoptosis of erythroleukemia cells.@*METHODS@#K562, HEL and TF-1 cells in logarithmic growth phase were treated with NTX, DNR and combination of the two drugs. CCK-8 test, Annexin V-DAPI double-staining flow cytometry, real-time RT-PCR were used to detect cell growth, cell apoptosis and expression of BAX, BAK, BCL-2, BCL-xl and BIM respectively. The effects of NTX, DNR and combination of the two drugs on apoptosis of K562, HEL and TF-1 cells were compared and analyzed.@*RESULTS@#NTX combined with DNR could significantly inhibit the growth of K562, HEL and TF-1 cells; Apoptosis detection results showed that the apoptotic rate of K562, HEL and TF-1 cells in combination group was significantly higher than that in NTX and DNR single group; the expression level of apoptosis-related genes BAK and BAX in K562 cells in combination group was significantly higher than that in two single drug groups, and the expression level of anti-apoptotic protein genes BCL-2 and BCL-xl was significantly lower than that in two single drug groups (P<0.05); the expression level of BAK in HEL cells treated with combined drugs for 24 hours was higher than that in DNR group (P < 0.05); the expression level of BCL-2 in TF-1 cells treated with combined drugs for 24 hours was lower than that in two single drugs groups while the expression level of BAK in 48 hours was the highest in combined drugs group, and the expression level of BCL-2 and BCL-xl in combined drugs group was lower than that in NTX group (P<0.05).@*CONCLUSION@#NTX combined with DNR can significantly promote the apoptosis of erythroleukemia cell lines K562, HEL and TF-1, and induce the expression of apoptosis-related genes. This study provides a new scheme for the clinical treatment of erythroleukemia.


Subject(s)
Aniline Compounds , Apoptosis , Daunorubicin , Humans , K562 Cells , Leukemia, Erythroblastic, Acute , Sulfonamides
9.
Article in Chinese | WPRIM | ID: wpr-828722

ABSTRACT

OBJECTIVE@#To study the clinical features and genetic mutations of children with Shwachman-Diamond syndrome (SDS) and malignant myeloid transformation.@*METHODS@#Next-generation sequencing was used to analyze the gene mutations in 11 SDS children with malignant myeloid transformation, and their clinical features and genetic mutations were analyzed.@*RESULTS@#Of the 11 children with SDS, 9 (82%) presented with refractory cytopenia of childhood (RCC), 1 (9%) had myelodysplastic syndrome with excess blasts (MDS-EB), and 1 (9%) had acute myeloid leukemia with myelodysplasia-related changes (AML-MRC). The median age of onset of malignant myeloid transformation was 48 months (ranged 7 months to 14 years). Of the 11 children, 45% had abnormalities in the hematological system alone. Mutations of the SBDS gene were detected in all 11 children, among whom 5 (45%) had c.258+2T>C homozygous mutation and 3 (27%) had c.184A>T+c.258+2T>C compound heterozygous mutation. The new mutations of the SBDS gene, c.634_635insAACATACCTGT+c.637_638delGA and c.8T>C, were rated as "pathogenic" and "possibly pathogenic" respectively. The 3-year predicted overall survival rates of children transformed to RCC and MDS-EB/AML-MRC were 100% and 0% respectively (P=0.001).@*CONCLUSIONS@#SDS children may have hematological system symptoms as the only manifestation, which needs to be taken seriously in clinical practice. The type of malignant transformation is associated with prognosis.


Subject(s)
Adolescent , Child , Child, Preschool , Exocrine Pancreatic Insufficiency , Humans , Infant , Leukemia, Myeloid, Acute , Mutation , Myelodysplastic Syndromes , Shwachman-Diamond Syndrome
10.
Article in Chinese | WPRIM | ID: wpr-828721

ABSTRACT

OBJECTIVE@#To compare the efficacy of the CAMS-2005 and CAMS-2009 regimens in treating children with non-core binding factor acute myeloid leukemia (non-CBF AML) and to study the prognosis factors.@*METHODS@#A total of 161 children who were initially diagnosed with non-CBF AML from April 2005 to December 2015 were enrolled as study subjects, and were divided into a CAMS-2005 regimen group (n=52) and a CAMS-2009 regimen group (n=109) according to the chemotherapy regimen provided. The efficacy was retrospectively compared between the two groups.@*RESULTS@#The complete remission (CR) rate at the first course of treatment was higher in the CAMS-2009 regimen group than that in the CMAS-2005 regimen group (63.3% vs 46.2%; P0.05). Children who achieved CR at the first course of treatment had significantly higher OS and event-free survival rates than those who did not achieved CR (P<0.01).@*CONCLUSIONS@#The CAMS-2009 regimen is superior to the CAMS-2005 regimen in improving the CR rate in children with non-CBF AML after induction treatment. Whether CR is achieved at the first course of treatment can affect the OS rate of children with non-CBF AML.


Subject(s)
Antineoplastic Combined Chemotherapy Protocols , Child , Humans , Leukemia, Myeloid, Acute , Prognosis , Remission Induction , Retrospective Studies
11.
Article in Chinese | WPRIM | ID: wpr-828676

ABSTRACT

OBJECTIVE@#To study the significance of CD20 combined with white blood cell (WBC) count at diagnosis in the prognosis assessment in children with B-lineage acute lymphoblastic leukemia (ALL).@*METHODS@#A retrospective analysis was performed on the medical data of 821 B-ALL children who were treated with CCLG-ALL2008 regimen from April 2008 to April 2015. Their survival status was followed up.@*RESULTS@#Among the 821 children, 547 (66.6%) were negative, while 274 (33.4%) were positive for CD20 expression. Among 694 children with WBC50×10/L (higher WBC count), the 5-year EFS rates was 64.3%±7.7% and 53.7%±5.5% for CD20 positive and negative patients respectively (P=0.135); the 5-year OS rate was 81.4%±6.4% and 58.6%±5.6% for CD20 positive and negative patients respectively (P=0.022); CD20 positive expression was an independent protective factor for OS (HR=0.367, P=0.016).@*CONCLUSIONS@#In children with B-ALL who are treated with CCLG-ALL2008 regimen, those with CD20 positive expression in lower WBC count at diagnosis have a poor prognosis; however, those with CD20 positive expression in higher WBC count at diagnosis have a better long-time survival.


Subject(s)
Antigens, CD20 , Antineoplastic Combined Chemotherapy Protocols , Child , Disease-Free Survival , Humans , Leukocyte Count , Precursor B-Cell Lymphoblastic Leukemia-Lymphoma , Diagnosis , Prognosis , Retrospective Studies
12.
Article in Chinese | WPRIM | ID: wpr-828674

ABSTRACT

OBJECTIVE@#To study the clinical features and prognosis of core binding factor acute myeloid leukemia (CBF-AML) in children.@*METHODS@#A retrospective analysis was performed from the chart review data of children who were newly diagnosed with CBF-AML in the Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences, from August 2009 to November 2015. According to the type of fusion gene, the children were divided into CBFB-MYH11 and AML1-ETO groups. Clinical features and prognosis were analyzed and compared between the two groups.@*RESULTS@#A total of 91 children with CBF-AML were enrolled in this study, among whom there were 74 (81%) in the AML1-ETO group and 17 (19%) in the CBFB-MYH11 group. Additional chromosomal abnormalities were observed in 38 children (42%), and deletion of sex chromosome was the most common abnormality and was observed in 28 children (31%). After the first course of induction treatment, the complete remission rate was 97% (88/91), the recurrence rate was 29% (26/91), the 5-year event-free survival (EFS) rate was 65%±6%, and the 5-year overall survival (OS) rate was 75%±5%. There were no significant differences between the AML1-ETO and CBFB-MYH11 groups in 5-year EFS rate (62%±7% vs 77%±11%, P>0.05) or 5-year OS rate (72%±6% vs 88%±9%, P>0.05).@*CONCLUSIONS@#AML1-ETO is the main type of fusion gene in children with CBF-AML, and deletion of sex chromosome is the most common type of additional chromosomal abnormalities. Children with CBF-AML often have a good prognosis, and the children with AML1-ETO have a similar prognosis to those with CBFB-MYH11.


Subject(s)
Child , Core Binding Factor Alpha 2 Subunit , Core Binding Factors , Humans , Leukemia, Myeloid, Acute , Oncogene Proteins, Fusion , Prognosis , RUNX1 Translocation Partner 1 Protein , Retrospective Studies
13.
Article in English | WPRIM | ID: wpr-828570

ABSTRACT

Acute lymphoblastic leukemia (ALL) is a common pediatric cancer. The second malignant neoplasms (SMNs) in long-term survivors of pediatric ALL are relatively rare. Herein we report a 10-year-old girl who was diagnosed as primitive neuroectodermal tumor (PNET) 5 years after the initial diagnosis of ALL with radiotherapy-free treatment. PNET is an exceedingly rare neoplasm in SMNs of survivors of childhood ALL. It is predisposed to be misdiagnosed and the pathogenesis is unclear. The outcome is poor. Long-term follow-up is necessary for the survival children of ALL.

14.
Article in English | WPRIM | ID: wpr-828456

ABSTRACT

Pathogenic mutations in 3-keto-dihydrosphingosine reductase (KDSR) gene are associated with keratinization disorders and impaired platelet function. However, no case with both homozygotic mutation of and hepatic hemangioendothelioma has ever been reported due to its low prevalence. Here we report a seven months old Chinese boy with a homozygotic missense mutation in and both of his parents carry a same heterozygous mutation. He was born with thick plate-like scales overlying erythrodermic skin, but the skin symptoms were resolved spontaneously over the first month of his birth. He was also diagnosed with hepatic hemangioendothelioma at birth and accepted a resection surgery at 2 months old. At birth, his platelet count was severely low (10-20×10/L) with recurrent skin and gingival bleeding. Meanwhile, he suffered a mild normocytic, normochromic anemia with normal iron and hematinic levels. The anemia spontaneously recovered over the first 6 months, while the platelet count keeped at a low level (4-20×10/L). Treatment with corticosteroids, immunoglobulin or thrombopoietin was all suboptimal.

15.
Journal of Experimental Hematology ; (6): 1075-1080, 2020.
Article in Chinese | WPRIM | ID: wpr-827158

ABSTRACT

OBJECTIVE@#To study the long-term efficacy of CCLG-ALL2008 protocol used for treatment of childhood acute lymphoblastic leukemia (ALL).@*METHODS@#Nine hundred and forty children with newly diagnosed ALL from January 2008 to April 2015 were treated with CCLG-ALL2008 protocol. Overall survival (OS) and event-free survival (EFS) rates were estimated by the Kaplan-Meier method. Cox proportional hazards model was used for analyses of prognostic factors.@*RESULTS@#Among the 940 newly diagnosed ALL patients, 570 patients were male, and 370 patients were female, the median age of onset was 5 years old (from 1 to 15 years old). The complete reaction rate (CR) was 96.7%. Survival analysis of 916 ALL patients with CR estimated by follow up [ (median follow up period 64 months (from 3 to 123 months) ] showed that, the expected 10 year OS rate was (78.6±1.5)% and the EFS rate was (66.0±1.8)%. The long-term OS rate of standard risk, intermediate risk and high risk patients was (93.0±1.5)%, (77.6±2.7)%, and (59.3±3.7)%, respectively, and the long-term EFS rate in standard risk, intermediate risk and high risk patients was (84.2±2.2)%. (67.8±2.9)%, and (42.1±3.9)% respectively. 10 year OS rate in B-ALL patients (79.8±1.6)% was significantly higher than that in T-ALL patients (53.5±6.3)% (P<0.01). Among of all the patients, patients 201 (21.9%) relapsed, the median relapse time was 19 months (from 2 months to 81 months). The 10 year EFS rate was (81.7±3.7)% in the patients with MRD rate <0.01% after induction therapy, which was significantly higher than that in the patients with MRD rate>0.01% (48.4±9.8)%.@*CONCLUSION@#The therapeutic efficacy of the CCLG-ALL2008 protocol is closed to the level of supior study group in the world. Risk stratification can improve the outcome for childhood ALL. Immunophenotyping shows the outcome of B-ALL is better than that of T-ALL. MRD negative patients after induction therapy shows better prognosis compared with that of MRD positive patients.


Subject(s)
Adolescent , Antineoplastic Combined Chemotherapy Protocols , Child , Child, Preschool , China , Disease-Free Survival , Female , Humans , Infant , Male , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Drug Therapy , Prognosis , Prospective Studies , Treatment Outcome
16.
Article in Chinese | WPRIM | ID: wpr-781460

ABSTRACT

OBJECTIVE@#To analyze the clinical efficacy and side effects of reduced-dose of cyclophosphamide combined cyclosporine A for severe aplastic anemia(SAA) children.@*METHODS@#Ten pediatric patients with SAA from January 2008 to May 2012 were enrolled. All the patients were treated with reduced dose of cyclophosphamide combined cyclosporine A. The dose of cyclophosphamide was 30 mg/(kg·d)×4 d, the dose of cyclosporine A gradually increased >15 mg/L accroding to the blood concentration.@*RESULTS@#The median follow-up time of the 10 pediatric patients was 100 months (6-126 months). Among 10 children with SAA, 4 cases achieved complete response(CR), 3 cases obtained partial response (PR) and the overall response rate was 70%, the remaining 3 cases showed no response (NR). One refractory patient treated by cyclophosphamide was progressed to paroxysmal nocturnal hemoglobinuria(PNH) at 25 months and was dead at 42 months after therapy.@*CONCLUSION@#The results show that reduced-dose cyclophosphamide (30 mg/kg·d for 4 consecutive days) combinated with CsA (initial dose 4 mg/kg·d, and drugvallery concentration >150 ng/ml) can make 7 of 10 children with severe aplastic anemia achieve complete response or partial response, and this regimen may be the second line regimen selected for some SAA children.

17.
Article in Chinese | WPRIM | ID: wpr-775109

ABSTRACT

OBJECTIVE@#To study the association of platelet level at diagnosis with prognosis in children with acute lymphoblastic leukemia (ALL).@*METHODS@#A total of 892 children with ALL who underwent chemotherapy with the CCLG-ALL 2008 regimen were enrolled. According to the platelet count at diagnosis, these children were divided into normal platelet count group (platelet count ≥100×109/L; n=263) and thrombocytopenia group (platelet count 0.05). The normal platelet count group still had a significantly higher 10-year EFS rate than the thrombocytopenia group after the children with MLL gene rearrangement were excluded (P0.05). The <20×10/L subgroup had significantly lower 10-year EFS and OS rates than the normal platelet count group, the (50- <100)×10/L subgroup, and the (20- <50)×10/L subgroup (P<0.05). After the children with MLL gene rearrangement were excluded, the <20×10/L subgroup still had significantly lower 10-year EFS and OS rates than the normal platelet count group, the (50-<100)×10/L subgroup, and the (20- <50)×10/L subgroup (P<0.05).@*CONCLUSIONS@#ALL children with MLL gene rearrangement often have the clinical manifestation of thrombocytopenia. Platelet level at diagnosis is associated with the prognosis of ALL children. The children with normal platelet count have a low recurrence rate and good prognosis, and those with a platelet count of <20×10/L have the worst prognosis.


Subject(s)
Child , Disease-Free Survival , Humans , Immunophenotyping , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Prognosis , Recurrence
18.
Article in Chinese | WPRIM | ID: wpr-775087

ABSTRACT

OBJECTIVE@#To study the long-term clinical effect of the CCLG-ALL2008 regimen in the treatment of children newly diagnosed with acute lymphoblastic leukemia (ALL) with different molecular biological features.@*METHODS@#A total of 940 children who were newly diagnosed with ALL were enrolled in this study. The children were treated with the CCLG-ALL2008 regimen. A retrospective analysis was performed for the long-term outcome of ALL children with different molecular biological features.@*RESULTS@#Among the 940 children with ALL, there were 570 boys and 370 girls, with a median age of onset of 5 years (range 1-15 years) and a median follow-up time of 65 months (range 3-123 months). The complete response (CR) rate was 96.7%, the predicted 10-year overall survival (OS) rate was 76.5%±1.5%, and the event-free survival (EFS) rate was 62.6%±3.0%. After CR was achieved after treatment, the overall recurrence rate was 21.9%. The children with positive ETV6-RUNX1 had the lowest recurrence rate and were prone to late recurrence, and those with positive MLL rearrangement had the highest recurrence rate and were prone to early recurrence. The children with positive ETV6-RUNX1 had a significantly higher predicted 10-year OS rate than those with positive TCF3-PBX1, BCR-ABL, or MLL rearrangement and those without molecular biological features (P<0.05). The children with positive ETV6-RUNX1 had a significantly higher predicted 10-year EFS rate than those with positive BCR-ABL or MLL rearrangement (P<0.05).@*CONCLUSIONS@#Molecular biological features may affect the long-term prognosis of children with ALL, and positive MLL rearrangement and BCR-ABL fusion gene are indicators of poor prognosis. Children with positive ETV6-RUNX1 fusion gene have the highest long-term survival rate.


Subject(s)
Adolescent , Antineoplastic Combined Chemotherapy Protocols , Therapeutic Uses , Child , Child, Preschool , Disease-Free Survival , Female , Fusion Proteins, bcr-abl , Humans , Infant , Male , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Drug Therapy , Prognosis , Retrospective Studies
19.
Article in Chinese | WPRIM | ID: wpr-775064

ABSTRACT

OBJECTIVE@#To study the clinical features and gene mutation spectrum of children with sideroblastic anemia (SA) and the clinical value of targeted next-generation sequencing in the molecular diagnosis of children with SA.@*METHODS@#Clinical data were collected from 36 children with SA. Targeted next-generation sequencing was used to detect mutations in SA-related pathogenic genes and genes associated with heme synthesis and mitochondrial iron metabolism. The association between genotype and clinical phenotype was analyzed.@*RESULTS@#Of the 36 patients, 32 had congenital sideroblastic anemia (CSA) and 4 had myelodysplastic syndrome with ring sideroblasts (MDS-RS). Mutations in CSA-related genes were detected in 19 children (19/36, 53%), among whom 9 (47%) had ALAS2 mutation, 4 (21%) had SLC25A38 mutation, and 6 (32%) had mitochondrial fragment deletion. No pathogenic gene mutation was detected in 4 children with MDS-RS. Among the 19 mutations, 89% (17/19) were known mutations and 11% (2/19) were novel mutations. The novel mutation of the ALAS2 gene c.1153A>T(p.I385F) was rated as "possibly pathogenic" and the novel mutation of the SLC25A38 gene c.175C>T(p.Q59X) was rated as "pathogenic".@*CONCLUSIONS@#ALAS2 and SLC25A38 gene mutations are commonly seen in children with CSA, but mitochondrial gene fragment deletion also accounts for a relatively high proportion. For children with hypoplastic anemia occurring in infancy, mitochondrial disease should be considered.


Subject(s)
5-Aminolevulinate Synthetase , Anemia, Sideroblastic , Genetics , Child , Genetic Diseases, X-Linked , Humans , Mitochondrial Membrane Transport Proteins , Mutation , Myelodysplastic Syndromes , Phenotype
20.
Article in Chinese | WPRIM | ID: wpr-774063

ABSTRACT

OBJECTIVE@#To study the long-term clinical effect of CCLG-ALL2008 regimen in the treatment of children and adolescents, aged >10 years, with newly diagnosed acute lymphoblastic leukemia (ALL).@*METHODS@#A retrospective analysis was performed for the clinical data of 150 ALL children and adolescents aged >10 years who were treated with CCLG-ALL2008 regimen from April 2008 to April 2015. The Kaplan-Meier method was used to analyze overall survival (OS) rate and event-free survival (EFS) rate.@*RESULTS@#Among the 150 children and adolescents, there were 87 (58.0%) boys and 63 (42.0%) girls, with a median age of 11 years (range 10-15 years). Of the 150 children and adolescents, 84 (56.0%) had intermediate risk and 66 (44.0%) had high risk; 122 (81.3%) had B-lineage acute lymphoblastic leukemia (B-ALL) and 28 (18.7%) had T-lineage acute lymphoblastic leukemia (T-ALL). The fusion gene test yielded positive results in 51 children and adolescents (34.0%), among whom 16 (31%) had positive BCR-ABL, 11 (22%) had positive TEL-AML1, 8 (16%) had positive E2A-PBX1, and 16 (31%) were positive for other fusion genes. The complete remission rate was 96.0% (144/150) after one course of treatment with CCLG-ALL2008 regimen. The median follow-up time was 52 months (range 3-122 months). The 5-year OS rate was 79.0%±3.5%, and the 5-year EFS rate was 67.3%±4.1%. There were no significant differences in 5-year OS and EFS rates between the children with intermediate or high risk, as well as between the children with B-ALL or T-ALL (P>0.05). The children and adolescents who achieved complete remission of bone marrow at the end of induction therapy had significantly higher 5-year OS and EFS rates than those who did not achieve complete remission (P10 years, CCLG-ALL2008 regimen can help to achieve high complete remission rate, 5-year OS rate and 5-year EFS rate. The children and adolescents failing to achieve complete remission at the end of induction therapy tend to have a poor prognosis.


Subject(s)
Adolescent , Antineoplastic Combined Chemotherapy Protocols , Child , Disease-Free Survival , Female , Humans , Male , Precursor Cell Lymphoblastic Leukemia-Lymphoma , Drug Therapy , Prognosis , Remission Induction , Retrospective Studies
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