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??Abstract??Objective To evaluate the therapeutic effectiveness and prognostic factors of ALL in infants treated with ALL-2005 protoco1. Methods Eighty-eight patients with ALL under 3 years of age were retrospectively studied ??who were newly diagnosed by morphologic and immunologic genetics and molecular biology ??MICM???? and were enrolled in the ALL-2005 treatment protocol from May 2005 to April 2009 in Shanghai Children’s Medical Center SCMC. Results Data were retrospectively collected since 2005 using the pediatric oncology networked database ??POND??. And altogether 88 children under 3 years of age with ALL received treatment in SCMC in the following four years. Among the 88 cases of ALL?? 61 ??69.32%?? were males and 27 ??30.68%?? were females. The median age at diagnosis was 2.08 years ??0??2.92 years??. Eighy-four were B lineage-ALL and 4 were T lineage-ALL. Thirty-six ??40.91%?? children were classified as low-risk?? 50 ??56.82%?? were standard-risk and 2 ??2.27%?? were high-risk. Eighty-one??92.05%?? children completely remitted in induction while 17 ??19.32%?? children relapsed. After a median follow-up of 33 months ??1.0??68.2 months???? the estimated 5-year event free survival ??EFS??rate and overall survival rate were 69.51% and 82.95%?? respectively. Conclusion The estimated 4-year EFS of ALL in infants has been close to 70%?? the therapeutic effectiveness of the ALL-2005 protocol for ALL in infants is satisfactory.     

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目的了解早期B细胞因子1(EBF1)基因异常在急性B淋巴细胞白血病(B-ALL)患儿中的发生情况,并进一步分析EBF1基因异常与B-ALL患儿预后的相关性。方法应用多重连接探针扩增(MLPA)技术检测2008年4月至2013年4月中国医学科学院北京协和医学院血液病医院血液学研究所儿童血液病诊疗中心初诊的B-ALL195例患儿EBF1基因异常情况。根据有无EBF1基因缺失将所有B-ALL患儿分为EBF1缺失组和非EBF1缺失组。结果 195例中15例(7.7%)发生EBF1缺失。两组初诊各项临床特征差异无统计学意义(P>0.05)。Kaplan-Meier法分析显示EBF1缺失组无病生存率(DFS)及无事件生存率(EFS)明显低于非EBF1缺失组[(59.5±14.8)%vs.(85.5±3.2)%;(55.6±14.3)%vs.(84.2±2.9)%;P均<0.05]。但两组总生存率(OS)差异无统计学意义[(86.7±8.8)%vs.(91.9±2.1)%,P>0.05]。Cox法分析显示在排除多项影响因素后,EBF1缺失仍为影响患儿DFS及EFS的不利因素(P<0.05)。结论部分B-ALL患儿初诊时伴EBF1基因缺失,EBF1缺失为B-ALL患儿DFS及EFS的独立危险因素。     

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??Objective??To study the clinical features of childhood acute lymphoblastic leukemia??ALL?? with secondary myelofibrosis. Methods??The clinical data of 208 cases of newly diagnosed childhood ALL were analyzed retrospectively??who had difficulty in sucking the bone marrow fluid and underwent bone marrow biopsy from November 2001 to September 2012 in Hematology Hospital of Chinese Academy of Medical Sciences. The clinical characteristics and prognosis of childhood ALL patients with and without myelofibrosis were compared. Results??All the cases were separated into two groups??group A included 33 cases??who were diagnosed with ALL combined with bone marrow fibrosis confirmed by pathology??175 patients diagnosed with ALL without bone marrow fibrosis were enrolled into group B. From November 2001 to December 2007??we treated patients using CAMSBDH-ALL regimen??and from January 2008 to September 2012 we used CCLG-ALL 2008 regimen. In group A??42.4% of patients showed pancytopenia of peripheral blood. The 5-year overall survival rate??OS?? was 64.3%. The 5-year OS was 58.3% in the patients treated with CAMSBDH-ALL regimen. In group B??15.6% of patients showed pancytopenia of peripheral blood??lower than that of group A. The 5-year OS was 85.0%??which was significantly higher than that of group A ??P??0.010??. The 5-year OS was 68.8% in the patients treated with CAMSBDH-ALL regimen??which was significantly lower than that of patients treated with CCLG-ALL2008 regimen ??5-year OS being 87.2%?? in group B ??P??0.044??. Conclusion??Pancytopenia in childhood ALL with myelofibrosis are more common and with poorer prognosis than those without myelofibrosis. The overall survival of ALL patients without bone marrow fibrosis can be improved by modifying the treatment strategies??but the prognosis can’t be improved for those ALL patients with myelofibrosis.     

伊马替尼治疗儿童Ph阳性急性淋巴细胞白血病的疗效和安全性

目的 研究GGLG-08方案联合络氨酸激酶抑制剂(TKI)-伊马替尼治疗儿童Ph阳性急性淋巴细胞白血病(Ph+ ALL)的疗效及安全性。方法 回顾性分析2008年10月至2013年12月初诊年龄<15岁的53例Ph+ ALL患儿的临床资料,给予患儿CCLG-ALL2008(高危组 HR)方案化疗(HR组,26例)或伊马替尼联合CCLG-ALL2008(高危组 HR)方案化疗(TKI+HR组,27例),比较两组的疗效及不良反应。结果 TKI+HR组诱导治疗后完全缓解(CR)率为100%,诱导期相关病死率为0;HR组CR率为75%,诱导相关病死率为15%;HR组3年无事件生存率(EFS)为(6±5)%;TKI+HR组5年EFS为(52±11)%。与HR组比较,TKI+HR组未增加化疗相关毒性,诱导期感染发生率反而下降。结论 伊马替尼的应用使儿童Ph+ ALL的临床疗效获得明显改善,同时具有良好的安全性。     

提高化疗强度对伴IKZF1基因缺失儿童急性淋巴细胞白血病预后的影响

目的 总结伴IKZF1基因缺失儿童急性淋巴细胞白血病（ALL）的临床特征并观察提高化疗强度对其预后的影响。方法 2015年12月至2018年2月间确诊并按照中国儿童白血病协作组-ALL 2008（CCLG-ALL 2008）方案规范治疗的ALL患儿共278例,根据有无IKZF1基因缺失将其分为IKZF1基因缺失组和IKZF1基因正常组,IKZF1基因缺失组均接受CCLG-ALL 2008高危（HR）方案治疗,IKZF1基因正常组则按临床危险度分型接受不同强度化疗,比较两组的临床特征及无事件生存（EFS）率。结果 278例患儿中共24例（8.6%）检出IKZF1基因外显子大片段缺失。IKZF1基因缺失组初诊时WBC ≥ 50×10⁹/L、BCR-ABL1融合基因阳性、诱导缓解治疗第15天微小残留病≥ 10%、微小残留病-HR、临床危险度-HR所占比例均高于IKZF1基因正常组（P < 0.05）。IKZF1基因缺失组3年EFS率（76%±10%）低于IKZF1基因正常组（84%±4%）,但差异无统计学意义（P=0.282）;其中,IKZF1基因缺失组-非HR（实际按CCLG-ALL 2008 HR方案化疗）的预计3年EFS率为82%±12%,低于IKZF1基因正常组-非HR（86%±5%）,但差异无统计学意义（P=0.436）。结论 伴IKZF1基因缺失的儿童ALL早期治疗反应更差,提高化疗强度可能改善其预后。     

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目的探讨儿童紫癜性肾炎(HSPN)的临床、病理分型分级与预后的关系。方法回顾性分析2000年1月至2008年10月在新疆维吾尔自治区人民医院儿科住院的55例HSPN患儿的临床、病理特点,分析对远期预后影响的因素。结果临床表现为血尿和蛋白尿者22例(40.0%),肾病综合征16例(29.1%),单纯性血尿或蛋白尿、急性肾炎和急进性肾炎者分别为12例(21.8%)、4例(7.3%)和1例(1.8%)。肾脏病理表现为系膜增生、肾小球硬化、新月体形成,国际小儿肾脏病研究组分级多见Ⅱ级和Ⅲ级,分别为27例(49.1%)和16例(29.1%)。临床分型与病理分级有相关性(P<0.05),临床分型越严重,肾脏病理损害越重,预后不佳。病理分级与预后有相关性(P<0.05),病理分级越重,预后越不佳。肾小管-间质病理分型与病理分级有相关性(P<0.01),随着肾小管-间质病变程度加重,肾脏病理损伤的程度越重,患儿预后不良。结论儿童紫癜性肾炎的临床表现、病理分级与远期预后密切相关,临床表现重,病理分级高,预后差。     

不同诊断标准下儿童急性不明谱系白血病的临床特点和预后分析

目的 探索不同诊断标准下儿童急性不明谱系白血病(acute leukemias of ambiguous lineage,ALAL)的临床特点和预后.方法 回顾性收集2015年12月至2019年12月诊治的39例儿童ALAL的临床资料,其中34例接受治疗.根据世界卫生组织和欧洲白血病免疫学分型协作组ALAL的诊断标准,...     

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??Objective??To analyze the clinical characteristics and outcomes of children with stage I testicular germ cell tumors??TGCT????and to discuss the treatment strategy for this disease after radical inguinal orchiectomy. Methods??Clinical data of 41 patients with stage I TGCT at Shanghai Children’s Medical Center??Shanghai Jiaotong University School of Medicine between June 2003 to December 2013 were retrospectively analyzed. Their clinical characteristics??therapy and outcomes were analyzed. Results????1??The median age at diagnosis was 18??3 to 43??months old. Among them??38 children??92.7%?? were younger than 3 years old.The pathological subtype included 35 cases of yolk sac??85.4%????4 immature teratoma??9.7%?? and 2 mixed germ cell tumors??4.9%??.??2??Serum AFP levels were elevated in 38 patients??92.7%??. AFP reduced to normal in 27 cases??71.1%??4 weeks after surgery and in 35 cases??92.1%?? after 2 courses of chemotherapy respectively??but 2 cases of them relapsed with AFP increasing again.??3??Forty out of 41 patients underwent surgery and 3??4 courses of chemotherapy with cyclophosvnamide??vincristine and dactinomycin D??VAC??. One patient received surgery alone. The median follow-up period was 64 months. One of the 32 patients who received adjuvant chemotherapy relapsed and then lost follow-up without treatment. The recurrence rate was 3.1% and the 5-year overall survival was 100%. No complication related to chemotherapy occurred during the follow-up. The only one patient treated with surgery alone relapsed at 5 months and achieved complete response after salvage treatment. Conclusion????1??The relationship between the decrease of serum AFP and the prognosis is uncertain and needs to be further studied.??2??Short course chemotherapy with mild toxic side effects can reduce the recurrence rate effectively?? so it can be used as a necessary treatment for patients with high risk of relapse??and it can also be used as a selective treatment for patients with low risk of recurrence.     

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??Objective To detect the expression of UBE2C in neuroblastoma??NB?? tissue and analyze its association with the clinical features of NB patients. Methods Paraffin-embedded surgical tissue specimens from 51 NB patients kept by the Department of Pathology?? Xinhua Hospital??from January 2012 to January 2015??were collected. The expression of UBE2C protein was detected by immunohistochemistry??and the clinicopathological characteristics of these patients were analyzed retrospectively. The survival curve was established with Kaplan-Merier analysis. Results Of 51 cases of NB?? the male-to-female ratio was 1.8??1. The median age at diagnosis was 36 months??ranging 3.3-156 months?? and median duration of follow-up was 25.6 months??ranging 5.5-42.7 months??. The results of immunohistochemistry showed that the UBE2C protein positive expression rate in stage ?? and ?? group ??90.0%?? was significantly higher than that in stage ???? and ??s group ??47.6%????P??0.001?? ?? the UBE2C protein positive expression rate in recurrence group??91.3%?? was also higher than that in the non-recurrence group??57.1%????P??0.001??. The survival curve based on Kaplan-Merier analysis revealed that patients with UBE2C positive expression had poor prognosis??P??0.006??. Conclusion The expression of UBE2C protein is closely related to the stage and the relapse of NB patients. Positive expression of UBE2C protein implies the poor prognosis for NB. UBE2C may play an important role in the invasion??metastasis and relapse of NB. It might become a new biomarker and potential drug target to NB.     

MRD监测下88例儿童急性B淋巴细胞白血病长期疗效的分析

目的对88例儿童急性B淋巴细胞白血病（B—ALL）治疗的长期随访结果进行分析,探讨微小残留病（MRD）监测下儿童B—ALL的无事件生存率（EFS）。方法回顾性分析2005年1月．2008年5月接受儿童ALL诊疗建议（第三次修订草案）治疗的88例B．ALL患儿,应用流式细胞术（FCM）检测MRD,采用Kaplan—Meier方法评估患儿EFS,各临床危险度分组间患儿EFS差异用Logrank检验。结果88例患儿骨髓完全缓解（CR）率为97．7％,2年、3年、4年、5年EFS率分别为87．5％、86．4％、81．8％、77．2％,标危、中危、高危5年EFS率分别为86．2％、84．6％、63．1％。5例死亡,9例复发（10．5％）,复发中位时间为23（3—59）个月。结论采用儿童ALL诊疗建议（第三次修订草案）治疗CR率较高,在MRD监测下指导治疗,总体上B—ALL患儿的5年EFS提高。