TARGET数据库是针对儿童肿瘤的数据库,该数据库力图采用综合基因组方法确定难以治疗的儿童期癌症的发生和发展的分子变化,旨在用数据来协助指导开发有效的、毒性较低的疗法。TARGET数据库由NCI’s Office of Cancer Genomics和Cancer Th...TARGET数据库是针对儿童肿瘤的数据库,该数据库力图采用综合基因组方法确定难以治疗的儿童期癌症的发生和发展的分子变化,旨在用数据来协助指导开发有效的、毒性较低的疗法。TARGET数据库由NCI’s Office of Cancer Genomics和Cancer Therapy Evaluation Program管理,因此颇具权威性且有更强的针对性,并通过数据分析生成对研究者有用的药物作用靶点和预后标志物,以便能够开发和应用新的更有效的治疗方案。本文对TARGET数据库进行了详细的介绍并可通过此文获得TARGET数据的下载方法,供研究者对儿童肿瘤进行更加深入的研究,为儿童肿瘤的诊治工作提供更加具有针对性的循证证据。展开更多
目的分析儿童肿瘤中安德森模型应用现状,为儿童肿瘤防治影响因素研究提供参考。方法以儿童肿瘤、安德森卫生服务利用模型、卫生服务利用为中文关键词,以childhood tumor、Anderson health service utilization model、health service ut...目的分析儿童肿瘤中安德森模型应用现状,为儿童肿瘤防治影响因素研究提供参考。方法以儿童肿瘤、安德森卫生服务利用模型、卫生服务利用为中文关键词,以childhood tumor、Anderson health service utilization model、health service utilization为英文关键词,检索2015-01-01-2024-03-01中国知网及Pubmed相关文献,纳入标准:儿童肿瘤的病因学研究;儿童肿瘤的公共卫生方面研究;安德森模型相关文章。排除标准:儿童肿瘤与其他疾病关系的研究,最终纳入分析文献33篇。结果国外对于儿童肿瘤的研究数量较多,国内研究数量相对较少:从卫生服务利用角度,超过90%的研究以供方的临床诊断和治疗为主,并未从公共卫生角度去探究儿童肿瘤的防治,也没有站在需方患者角度研究影响患儿寻求治疗的各方面因素。应用安德森模型分析儿童恶性肿瘤患者的研究内容包括对首诊医疗机构的选择以及患者就医延迟的影响因素。结论将完整安德森模型应用到对防治儿童肿瘤的影响因素研究中,可较全面的分析患儿的就医现状、儿科医疗团队建设现状、儿童肿瘤防控水平,早日达到降低儿童肿瘤患病率、提高生存率的目标。展开更多
Next-generation sequencing (NGS) is being used in clinical testing.Government authorities in both China and the United States are overseeing the clinical application of NGS instruments and reagents.In addition,the US ...Next-generation sequencing (NGS) is being used in clinical testing.Government authorities in both China and the United States are overseeing the clinical application of NGS instruments and reagents.In addition,the US Association for Molecular Pathology and the College of American Pathologists have jointly released a guidance to standardize the analysis and interpretation of NGS data involved in clinical testing.At present,the analysis strategies and pipelines for NGS data related to the clinical detection of pediatric disease are similar to those used for adult diseases.However,for rare pediatric diseases without linkage to known genetic variants,it is currently difficult to detect the relevant pathogenic genes using NGS technology.Additionally,it is challenging to identify novel pathogenic genes of familial pediatric tumors.Therefore,characterization of the pathogenic genes associated with above diseases is important for the diagnosis and treatment of rare diseases in children.This article introduces the general pipelines for NGS data analyses of diseases and elucidates data analysis strategies for the pathogenic genes of rare pediatric diseases and familial pediatric tumors.展开更多
Nonresectable Low-Grade Astrocytomas (LGA) can compromise function and threaten life. For the majority of patients, the most appropriate strategy is initial chemotherapy followed by Radiation Therapy (RT). Since curat...Nonresectable Low-Grade Astrocytomas (LGA) can compromise function and threaten life. For the majority of patients, the most appropriate strategy is initial chemotherapy followed by Radiation Therapy (RT). Since curative treatment is not available for most of these patients, it is reasonable to conduct clinical studies to evaluate new agents. This Phase II study evaluates efficacy and safety of Antineoplastons A10 and AS2-1 (ANP) in LGA. Sixteen children diagnosed with LGA were treated. They included 12 males and 4 females, ages 1.6 - 17.4 years (median 10.6). Efficacy was evaluated in 16 patients. The majority of patients were previously treated, but 1 patient had stereotactic biopsy only. Out of the remaining 15 patients, 6 patients received chemotherapy, and 7 patients had surgery, and 2 patients received RT and chemotherapy after surgery. The patients received treatment with ANP administered daily every 4 hours (median dose of A10 was 7.71 g/kg/d and AS2-1 was 0.26 g/kg/d) until objective response or stable disease was documented and for 8 months thereafter. The duration of ANP IV ranged from 1.4 to 286 weeks with a median of 83 weeks. A complete response was documented in 25.0%, partial response in 12.5%, and stable disease in 37.5%. Overall survival was 67.7% at 5 years, and 54.2% at 10 and 15 years. Progression-free survival was 48.1%, 34.4% and 34.4% at 5, 10, and 15 years respectively. The treatment was associated with grade 3 or grade 4 Adverse Drug Experiences (ADE) in 6 patients. There were two hypernatremias of grade 4 (12%). Grade 3 ADE included urinary frequency (6%), fatigue (6%) and hypernatremia (6%). There were no chronic toxicities, and there was a high quality of survival. ANP shows efficacy with a very good toxicity profile in this cohort of children with low-grade astrocytoma.展开更多
文摘TARGET数据库是针对儿童肿瘤的数据库,该数据库力图采用综合基因组方法确定难以治疗的儿童期癌症的发生和发展的分子变化,旨在用数据来协助指导开发有效的、毒性较低的疗法。TARGET数据库由NCI’s Office of Cancer Genomics和Cancer Therapy Evaluation Program管理,因此颇具权威性且有更强的针对性,并通过数据分析生成对研究者有用的药物作用靶点和预后标志物,以便能够开发和应用新的更有效的治疗方案。本文对TARGET数据库进行了详细的介绍并可通过此文获得TARGET数据的下载方法,供研究者对儿童肿瘤进行更加深入的研究,为儿童肿瘤的诊治工作提供更加具有针对性的循证证据。
文摘目的分析儿童肿瘤中安德森模型应用现状,为儿童肿瘤防治影响因素研究提供参考。方法以儿童肿瘤、安德森卫生服务利用模型、卫生服务利用为中文关键词,以childhood tumor、Anderson health service utilization model、health service utilization为英文关键词,检索2015-01-01-2024-03-01中国知网及Pubmed相关文献,纳入标准:儿童肿瘤的病因学研究;儿童肿瘤的公共卫生方面研究;安德森模型相关文章。排除标准:儿童肿瘤与其他疾病关系的研究,最终纳入分析文献33篇。结果国外对于儿童肿瘤的研究数量较多,国内研究数量相对较少:从卫生服务利用角度,超过90%的研究以供方的临床诊断和治疗为主,并未从公共卫生角度去探究儿童肿瘤的防治,也没有站在需方患者角度研究影响患儿寻求治疗的各方面因素。应用安德森模型分析儿童恶性肿瘤患者的研究内容包括对首诊医疗机构的选择以及患者就医延迟的影响因素。结论将完整安德森模型应用到对防治儿童肿瘤的影响因素研究中,可较全面的分析患儿的就医现状、儿科医疗团队建设现状、儿童肿瘤防控水平,早日达到降低儿童肿瘤患病率、提高生存率的目标。
基金National Natural Science Foundation of China(81502144,81472369,31671377)%National Key Research and Development Program of China(2015AA020108)%Beijing Health System Top Level Health Technical Personnel Training Plan(20153079)
文摘Next-generation sequencing (NGS) is being used in clinical testing.Government authorities in both China and the United States are overseeing the clinical application of NGS instruments and reagents.In addition,the US Association for Molecular Pathology and the College of American Pathologists have jointly released a guidance to standardize the analysis and interpretation of NGS data involved in clinical testing.At present,the analysis strategies and pipelines for NGS data related to the clinical detection of pediatric disease are similar to those used for adult diseases.However,for rare pediatric diseases without linkage to known genetic variants,it is currently difficult to detect the relevant pathogenic genes using NGS technology.Additionally,it is challenging to identify novel pathogenic genes of familial pediatric tumors.Therefore,characterization of the pathogenic genes associated with above diseases is important for the diagnosis and treatment of rare diseases in children.This article introduces the general pipelines for NGS data analyses of diseases and elucidates data analysis strategies for the pathogenic genes of rare pediatric diseases and familial pediatric tumors.
文摘Nonresectable Low-Grade Astrocytomas (LGA) can compromise function and threaten life. For the majority of patients, the most appropriate strategy is initial chemotherapy followed by Radiation Therapy (RT). Since curative treatment is not available for most of these patients, it is reasonable to conduct clinical studies to evaluate new agents. This Phase II study evaluates efficacy and safety of Antineoplastons A10 and AS2-1 (ANP) in LGA. Sixteen children diagnosed with LGA were treated. They included 12 males and 4 females, ages 1.6 - 17.4 years (median 10.6). Efficacy was evaluated in 16 patients. The majority of patients were previously treated, but 1 patient had stereotactic biopsy only. Out of the remaining 15 patients, 6 patients received chemotherapy, and 7 patients had surgery, and 2 patients received RT and chemotherapy after surgery. The patients received treatment with ANP administered daily every 4 hours (median dose of A10 was 7.71 g/kg/d and AS2-1 was 0.26 g/kg/d) until objective response or stable disease was documented and for 8 months thereafter. The duration of ANP IV ranged from 1.4 to 286 weeks with a median of 83 weeks. A complete response was documented in 25.0%, partial response in 12.5%, and stable disease in 37.5%. Overall survival was 67.7% at 5 years, and 54.2% at 10 and 15 years. Progression-free survival was 48.1%, 34.4% and 34.4% at 5, 10, and 15 years respectively. The treatment was associated with grade 3 or grade 4 Adverse Drug Experiences (ADE) in 6 patients. There were two hypernatremias of grade 4 (12%). Grade 3 ADE included urinary frequency (6%), fatigue (6%) and hypernatremia (6%). There were no chronic toxicities, and there was a high quality of survival. ANP shows efficacy with a very good toxicity profile in this cohort of children with low-grade astrocytoma.
