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目的探讨联合降阶梯方案治疗婴幼儿喘息的疗效和安全性。方法以2010年10至12月于上海交通大学医学院附属新华医院哮喘专科门诊及普通儿科呼吸门诊就诊的婴幼儿喘息患儿97例作为研究对象,以规范采用联合降阶梯疗法患儿作为治疗组,以仅采用门诊常规治疗的患儿作为对照组(非联合降阶梯疗法组)。两组均采用相同综合性治疗,包括吸氧、退热等对症处理。治疗组口服泼尼松5mg/d,每天1次,疗程3d;口服阿奇霉素0.1g(/kg·d),疗程3d;每晚用妥洛特罗贴剂1贴(0.5mg/d),疗程7d;口服氯雷他定3mL/d,疗程14d;口服孟鲁司特钠4mg/d,疗程21d。对照组采用目前门诊常用治疗方案,包括采用静脉滴注琥珀氢考或甲强龙、平喘药物、抗生素、祛痰止咳合剂、雾化吸入激素等。结果治疗组患儿咳嗽、喘息、哮鸣音评分及症状体征消失天数显著低于对照组(P<0.05),治疗组1个月内复发次数、治疗费用、家长误工天数显著低于对照组(P<0.05),且治疗组患儿对该方案接受度较高(P<0.05)。结论联合降阶梯疗法应用于婴幼儿喘息疗效肯定,且方便、安全,值得临床应用推广。     

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??Abstract: Objective To assess the efficiency and safety of the flexible bronchoscopic balloon dilatation tracheoplasty in children?? and discuss the optimal indication and combined therapy of central airway stenosis in children. Methods From July 2009 to April 2012?? 34 cases were diagnosed as central airway stenosis by chest CT scan and bronchoscpy in Shanghai Children's Medical Center??and received the treatment of the flexible bronchoscopic balloon dilatation tracheoplasty. The data about the efficiency?? complication and prognosis of the treatment were collected. Results In the 34 cases?? 16 cases were improved after the treatment and the overall response rate was 47.1%. According to the pathology of the stenosis?? these cases were divided into three types: 9 cases were membranous stenosis?? 5 cases were muscular stenosis and 20 cases were osseous stenosis. The response rate was 47.1%?? 100%?? and 20% respectively. According to the anatomy morphology of the stenosis?? 20 cases were short distance stenosis and the response rate was 75%?? 12 cases were long distance stenosis and the response rate was 8.3%?? 2 cases were funnel stenosis and the response rate was 0. Among these cases?? 3 cases accepted the operation of pulmonary artery sling and tracheal sliding angioplasty?? the total response rate being raised to 55.9%. The most common complication in the flexible bronchoscopic balloon dilatation tracheoplasty was transient oxygen desaturation. Conclusion The severity?? length?? pathology?? indication and time of the treatment should be considered completely before the flexible bronchoscopic balloon dilatation tracheoplasty. The balloon dilatation therapy is suitable for membranous with short distance stenosis. The combined interventional treatment is good for muscular stenosis??and the surgery and palliative treatment is an option for osseous with long distance stenosis.     

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??Abstract: Objective To analyze relationship between renal pathology and glomerular podocyte food processes fused in childhood Henoch-Schönlein purpura nephritis. Methods Renal pathological types of 72 children suffering from HSPN were reviewed during the period from January 1st 2008 to January 1st 2011 from Nanjing Command of Jingling Hospital. Pathological grading and immunofluorescence typing were analyzed. The information of podocyte food processes fused was showed by electron microscopy. Results Pathological findings were classified as ISKDC??the International Society of Kidney Disease in Children?? grade ??in 34 ??47.2%?? and grade?? in 38 ??52.8%??. Glomerular podocyte injury was classified by electron microscope as food processes of podocyte fused extensively in 21 cases??food processes of podocyte fused segmentally in 35 cases?? food processes of podocyte without fusion in 11cases.There was no renal glamorous in nephridial tissue by electron microscope in five cases. Conclusion The childhood HSPN is expressed with foot process of podocytes fused more extensively on electron microscope besides glomerular pathologic change. The more serious the degree of podocyte injury is??the more obvious the pathologic changeis.     

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??Abstract??Objective To identify thyroglobulin ?? TG?? gene mutation in patients with congenital hypothyroidism with goiter??in order to provide full evidence for gene diagnosis of congenital hypothyroidism. Methods Totally 11 patients with congenital hypothyroidism with goiter in Qingdao Women and Children hospital from Jan. 2012 to Aug. 2012 were enrolled in this study. The 7??14??22??33 and 38 exons of TG gene were amplified through PCR and the products were sequenced directly. The type and characteristic of TG gene mutation in patients with congenital hypothyroidism with goiter in this region were analyzed. Results Two single nucleotide polymorphism of TG gene were identified??but no gene mutation was observed. TG c.3218-81T??G ??rs 853324??homozygous??in 1 patient and c.3218-81T??G??rs 853324??heterozygosis??in 6 patients were found. Conclusion The incidence of TG gene mutation is very low in patients with congenital hypothyroidism with goiter from Qingdao City. It suggests that TG gene mutation may not serve as the mutation hotspot gene of congenital hypothyroidism with goiter in Qingdao City??     

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??Abstract?? Objective To find out the fractional exhaled nitric oxide?? FENO?? in different control level and the relationship of FENO with childhood asthma control test ??C-ACT?? and bronchial provocation test??BPT??. Methods Totally 48 asthmatic children who underwent routine checkups since March 2011 through September 2011 were enrolled in the study. The patients completed the detection of FENO?? they also underwent pulmonary function test??PFT???? bronchial provocation test ??BPT??and C-ACT. Those indexes and relationships were analyzed in different control groups. Results The geometrical mean FENO of asthmatic children was ??22.5 ± 2.0?? × 10-9 mol/L. Uncontrolled group had higher FENO than controlled group and partly controlled group?? and FENO was slightly related to FEV1% Pred??so did PC20??but no relevance had been found between FENO?? C-ACT or PC20. Conclusion FENO can be an useful tool in managing asthma when used together with C-ACT??PFT and BPT.     

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??Abstract??Objective To study the methylphenidate hydrochloride controlled-release tablets??OROS-MPH??dose titration treatment for children with attention deficit hyperactivity disorder??ADHD??combining conduct disorder??CD??to achieve the best clinical efficacy. Methods According to the DSM-IV diagnostic criteria of attention deficit hyperactivity disorder??ADHD??combining conduct disorder??CD????120 cases of children were randomly divided into the original dose group and dose titration group.Two groups of children accepted the 24-week treatment of OROS-MPH??in which the original dose group were chosen to 18 mg/day?? dose titration group increased to 36 mg/day or 54 mg/day??respectively??in the fifth week and thirteenth week??and after the course of treatment??the following assessments were performed??SNAP-IV scale??Child Behavior Checklist??CBCL??and Treatment Emergent Symptom Scale??TESS??.Results ??1??The best response rate in the dose titration group was significantly higher than original dose group ??P??0.05??.??2??The SNAP-IV hyperactivity / impulsivity?? inattention and confrontation behavior items showed a significant difference between dose titration group and original dose group??P??0.05??.??3??In the CBCL behavior scales??dose titration group and the original dose group showed significant difference in the presence of adverse exchanges??forcedness??hyperactivity??aggression and discipline of the five dimensions??P??0.05??.??4??The incidence of side effects in both groups of children had no significant difference??P??0.05??.Conclusion With different doses of OROS-MPH for the treatment of children with ADHD and CD??effects differ more obviously-the higher dose??36 mg/day-54 mg/day??for the improvement of symptoms is better than lower dose??18 mg/day??.     

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目的探讨氨溴特罗口服液改善毛细支气管炎患儿气道功能的疗效影响。方法将2009年11月至2010年9月广州市妇女儿童医疗中心93例毛细支气管炎患儿随机分为治疗组(氨溴特罗口服液组)和对照组。观察两组患儿治疗前后潮气呼吸流速-容量环(TBFV)的形态改变以及肺功能各项指标的变化。结果两组患儿治疗前TBFV环均变窄,呼气曲线升枝陡峭,高峰提前,降枝呈波谷样凹陷;治疗后明显好转。两组患儿治疗前各项主要参数比较,差异无统计学意义(P>0.05)。两组患儿治疗前后比较:呼吸频率(RR)、呼气峰流速(PEF)、呼吸系统阻力(Rrs)、每千克体重功能残气量(FRC/kg)均降低(P<0.05或P<0.01);每千克体重潮气量(VT/kg)、吸呼比(TI/TE)、呼气达峰时间(TPTEF)、达峰时间比(TPTEF/TE)、呼气达峰容积(VPTEF)、达峰容积比(PFV)、呼出75%潮气量时的呼气流速/呼气峰流速(25/PF)、潮气呼气中期流速/潮气吸气中期流速(ME/MI)、每千克体重顺应性(Crs/kg)均增加(P<0.05或P<0.01)。其中治疗组与对照组比较,反映大小气道阻塞主要指标PFV、25/PF、ME/MI及肺顺...     

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??Abstract??Objective The time-effect relationship in treating postural orthostatic tachycardia syndrome ??POTS?? of children with alpha receptor agonist midodrine hydrochloride was analyzed to explore the optimum course of treatment for POTS children. Methods A total of 104 cases of the POTS children were studied from 2005 to 2011 in Peking University First Hospital. A chi-square test was used to evaluate the relationship between effect and time of medication?? and the time-effect curve was constructed. Results According to the 104 cases?? the time accumulative total efficacy for 1 month?? 2 months?? 3 months?? 4 months?? 5 months and 6 months were 19.23%?? 48.08%?? 69.23%?? 73.08%?? 74.04% and 75.96%?? respectively. The time accumulative total efficacy for 3 months was higher than that for 1 month or 2 months ??P??0.05???? but there was no difference in the efficacy among 4 months?? 5 months and 6 months treatment and with 3 months treatment. Conclusion It has the best treatment efficacy when the course of treatment for POTS children with midodrine hydrochloride is 3 months?? and prolonging the duration of treatment does not significantly improve the therapeutic effect.     

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??Abstract??Objective To investigate the correlation among plasma N-terminal pro-brain natriuretic peptide ??NT-proBNP?? and right ventricular Tei ??RVTei?? and right ventricular fractional area change ??RVFAC?? in children with pulmonary hypertension. Methods From January 2007 to November 2012??echocardiography and serological examination data of 38 patients from Samsung Seoul Hospital in Korea were retrospectively analyzed. Patients were divided into two groups ??16 cases with 30??PASP??70mmHg ??mild to moderate?? and 22 cases with PASP≥70mmHg ??severe??. Results ??1??RVFAC?? PASP and plasma concentration of NT-proBNP had a statistically significant difference??P??0.05?? between mild to moderate PAH group and severe PAH group . RVTei index had no difference between the two groups??P??0.05??. ??2??Correlation analysis??the plasma concentration of NT-proBNP had a positive correlation ??r = 0.544??P??0.05??with PASP??a negative correlation??r = -0.767??P??0.05??with RVFAC??RVFAC had a negative correlation??r = -0.711??-0.767??P??0.05?? with PASP and the plasma concentrations of NT-proBNP?? the RVTei had no correlation with the plasma concentration of NT-proBNP?? PASP or RVFAC??r = -0.041??-0.048??0.016??P??0.05??. Conclusion Whether there are correlations between the plasma concentration of NT-proBNP and RVFAC depends on the degree of PAH. This suggests that the plasma concentration of NT-proBNP and RVFAC can be used as evaluation of right ventricular function in children with pulmonary hypertension.     

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儿童白血病的疗效已取得极大提高。近年来,大量新的分子遗传学异常被发现,使得临床分型更为准确,应用高通量测序技术可识别出极低水平的微小残留病,小分子靶向药物、单克隆抗体治疗、嵌合型抗原受体修饰等新型治疗技术的应用,给攻克难治、复发白血病带来新的希望,以6-巯基嘌呤代谢相关基因多态性为代表的药物遗传学研究为在提高疗效的同时降低毒副反应做出了突出贡献。这些进步无不深刻体现着精准医学的理念与精神,并为今后的研究奠定坚实的基础。     

儿童急性淋巴细胞性白血病ALL-2005方案治疗失败病例分析

目的分析ALL-2005方案治疗儿童急性淋巴细胞性白血病(ALL)失败的原因。方法将初治ALL失败病例分为诱导治疗失败、复发和依从失败以及治疗相关死亡三类,回顾性分析失败的原因。结果 2005年5月1日至2009年4月30日,共388例初治ALL患儿,随访时间中位数48个月(24～72个月)。101例患儿治疗失败,总失败率为26.0%;其中诱导治疗失败6例,复发69例,依从失败17例,治疗相关死亡9例。骨髓复发率为13.2%,单纯中枢神经系统复发率为2.4%,男性患儿睾丸复发率为3.9%。388例ALL患儿中,300例B系ALL患儿在诱导治疗第35天监测微小残留病(MRD),MRD≤0.01%患儿的复发率为15.4%,而MRD>0.01%患儿为28.8%,两者间差异有统计学意义(χ2=5.818,P=0.016)。结论复发是导致ALL-2005方案治疗ALL失败的主要原因,诱导治疗第35天MRD水平可以预测患儿的预后。     

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??Acute myeloid leukemia??AML?? accounts for about 20% of childhood leukemia with the cure rate of more than 65%. Although dramatic improvement in treatment of childhood AML has been achieved based on the optimization of risk classification??risk-based therapeutic regimen??monitoring and supportive care??currently??the prognosis of pediatric AML has reached plateau due to the heterogeneity of AML. Targeted therapy based on the precise genotyping and comprehensive treatment will improve the cure rate and quality of life of children with AML.     

儿童急性淋巴细胞白血病早期治疗反应对预后的预测价值

目的探讨CCCG-2015方案治疗儿童急性淋巴细胞白血病(ALL)早期治疗反应评估对预后的预测价值。方法回顾2015年4月至2019年12月以CCCG-2015方案规范治疗的220例初诊ALL患儿的临床资料,分析诱导治疗第19、46天骨髓细胞学及微小残留病(MRD)监测评估早期治疗反应。结果 220例患儿中,男128例、女92例;中位年龄5.1岁;中位随访时间45个月。诱导缓解率97.7%,长期生存率94.6%,病死率5.4%,复发率6.8%。5年无事件生存(EFS)率(87.5±2.6)%,5年总生存(OS)率(92.7±2.2)%,5年累积复发率(9.4±2.4)%,中位复发时间为32(9～34)个月。低、中、高危组5年EFS差异有统计学意义(P0.05)。诱导第19天,MRD阴性和阳性组的5年EFS率差异有统计学意义(P0.05);MRD阳性组中,MRD 0.01%～0.099%、0.1%～0.99%、≥1%的5年EFS率差异有统计学意义(P0.005)。诱导第46天,MRD阴性和阳性组的5年EFS率差异有统计学意义(P0.001)。COX回归分析发现,第19天 MRD≥1%(HR=7.04,95%CI:2.84～17.46)和第46天MRD≥0.01%(HR=1.67,95%CI:1.17～2.37)是影响EFS的独立危险因素。结论诱导治疗第19天MRD0.01%患儿复发率低,第19天MRD≥1%和第46天MRD≥0.01%患儿预后差。     

ALL-2005方案对儿童急性淋巴细胞白血病髓外白血病防治效果分析

目的总结ALL-2005方案对急性淋巴细胞白血病(ALL)患儿髓外白血病,包括中枢神经系统白血病(CNSL)及睾丸白血病(TL)的防治效果。方法 2005年5月—2008年5月共218例ALL患儿,采用ALL-2005方案进行治疗,总结其临床及治疗情况。结果 218例ALL患儿中,5例初诊时伴CNSL,1例于诱导治疗期间发生CNSL,4例于维持治疗期间发生CNSL;1例初诊时伴TL发生,4例维持治疗期间发生TL。经治疗,2例CNSL患儿骨髓复发,其余8例均缓解至今;TL患儿1例骨髓复发,1例放弃治疗,3例缓解至今。结论 ALL-2005方案疗效良好,有利于提高患儿长期生存质量。[临床儿科杂志,2012,30(5):412-414]     

ALL-2005方案治疗儿童T系急性淋巴细胞白血病35例疗效总结

目的研究ALL-2005方案治疗儿童T系急性淋巴细胞白血病(T-ALL)的疗效。方法对2005年5月-2009年4月初发的35例T-ALL患儿采用ALL-2005方案进行分层治疗及疗效分析。结果 T-ALL患儿35例,男30例(86%),女5例(14%),发病时中位年龄8.3(2.0～15.6)岁。其中>10岁患儿14例,占40%。起病时外周血白细胞≥50×109/L者24例,占69%,起病时6例患儿有纵隔增宽。泼尼松治疗第8天时泼尼松试验反应好者22例(63%)。诱导治疗第35天有29例获完全缓解(83%),骨髓微小残留病(MRD)≤0.01%者共20例(67%)。治疗第55天时MRD≤0.01%共26例(87%)。4例复发,3例失访,6例死亡(包括3例复发和3例第1疗程未缓解而放弃治疗者)。随访至2011年6月31日,中位随访时间39(1～71)个月,5年无事件生存率为73.5%。结论 ALL-2005方案治疗儿童T-ALL疗效较好。     

Children's Oncology Group's 2013 blueprint for research: acute lymphoblastic leukemia

Approximately 90% of the 2,000 children, adolescents, and young adults enrolled each year in Children's Oncology Group acute lymphoblastic leukemia (ALL) trials will be cured. However, high‐risk subsets with significantly inferior survival remain, including infants, newly diagnosed patients with age ≥10 years, white blood count ≥50,000/µl, poor early response or T‐cell ALL, and relapsed ALL patients. Effective strategies to improve survival include better risk stratification, optimizing standard chemotherapy and combining targeted therapies with cytotoxic chemotherapy, the latter of which is dependent upon identification of key driver mutations present in ALL. Pediatr Blood Cancer 2013; 60: 957–963. © 2012 Wiley Periodicals, Inc.     

SCMC-ALL-2005方案治疗儿童B细胞型急性淋巴细胞白血病疗效观察

目的评估上海儿童医学中心-急性淋巴细胞性白血病-2005(SCMC-ALL-2005)治疗方案治疗儿童B细胞型ALL的疗效。方法按照SCMC-ALL-2005方案,5家医院对2005年5月1日至2009年4月30日新发B细胞型ALL患儿进行诊断、治疗和随访。结果研究期间共收治B细胞型ALL患儿601例,539例(89.68%)随访至2011年9月30日。601例患儿中,低危284例、中危231例、高危86例,均按照诊疗建议治疗。诱导期间缓解率为98.84%(7例未缓解),第一次事件发生时的中位时间为35个月(2.94年),至随访终止日的539例随访病例中共完成治疗403例(74.77%);低危组完成治疗223例(86.43%),中危组150例(73.17%),高危组30例(39.47%),三组间的差异有统计学意义(P=0.001)。采用KaplanMeier方法评估患儿随访3年的总生存率为(83.3±1.8)%,3年无事件生存(EFS)率为(79.2±1.9)%;随访5年总生存率为(79.5±3.3)%,5年EFS率为(70.9±3.7)%。低、中、高危三组间3年及5年EFS率差异有统计学意义(P均0.05)。结论SCMC-ALL-2005方案治疗儿童B细胞型ALL的疗效比较满意,多中心协作有助于儿童白血病的规范化治疗。     

Recent progress in the treatment of infant acute lymphoblastic leukemia

Treatment of infants with acute lymphoblastic leukemia (ALL), especially those with mixed lineage leukemia (MLL) rearrangement (MLL‐r), which account for approximately 80% of cases, is still a major challenge for pediatric hematologists and oncologists worldwide. Continuing efforts by collaborative clinical study groups in Europe, North America, and Japan have rescued approximately half of the MLL‐r ALL patients with intensive chemotherapy with or without allogeneic hematopoietic stem cell transplantation. Recent progress has clarified the unique mechanism of MLL‐r ALL: the aberrant methylation and histone modifications via DOT1L and other related molecules by MLL fusion proteins lead to leukemogenetic gene expression, thus to overt leukemia. In order to overcome this dismal subtype of ALL, novel targeted therapy based on leukemia biology is urgently needed. Due to the extreme rarity of the disease, collaboration between the study groups in Europe (Interfant), North America (Children's Oncology Group), and Japan (Japanese Pediatric Leukemia/Lymphoma Study Group) is under way.