STAT报道：诺华披露脊髓性肌萎缩症基因疗法Zolgensma用药儿童新增7例肿瘤病例，2019年以来累计达9例
据STAT报道，诺华披露7例既往未报告的肿瘤病例，患儿均曾接受其脊髓性肌萎缩症基因疗法Zolgensma治疗，该疗法2019年获批以来肿瘤报告累计达9例，由STAT记者Ed Silverman与Jason Mast报道。本条材料标题同时提及Amgen的Repatha与更少心脏病发作相关，但正文未提供该部分的任何细节。
#儿科学
STAT · 医学与生命科学新闻 STAT · 医学与生命科学新闻 STAT+报道：新发肿瘤病例显示常用基因治疗载体AAV存在致癌风险
STAT新闻报道，今年一系列肿瘤病例为腺相关病毒（AAV）这一广泛使用的基因治疗载体可致癌提供了有力证据，结束了约二十年的争论；AAV载体已被用于向数千名患者递送新基因。罗切斯特大学神经科医生Sam Mackenzie表示，该风险将改变部分脊髓性肌萎缩患者的治疗权衡。全文为STAT+付费内容，具体病例细节未在可见正文中披露。
ClinicalTrials.gov API v2 · 登记更新 Axsome Therapeutics登记solriamfetol治疗儿童青少年ADHD的III期开放标签长期安全性研究（NCT07773883）
Axsome Therapeutics在ClinicalTrials.gov更新登记NCT07773883：一项III期、多中心、开放标签研究，评估solriamfetol在患ADHD（注意缺陷多动障碍）的儿童青少年中的长期安全性。本次材料仅提供方案描述，未报告招募状态、样本量、主要终点或安全性结果，具体状态以登记库后续提交为准。
EMA · What's New 儿科学：Human medicines European public assessment report (EPAR): Yorvipath, palopegteriparatide, Date of authorisation: 17/11/2023, Revision: 6, Status: Authorised
原文摘录：Yorvipath RSS Authorised This medicine is authorised for use in the European Union palopegteriparatide Medicine Human Authorised Application under evaluation CHMP opinion European Commission decision Overview Yorvipath is a hormone replacement medicine fo … 37 KB - PDF) First published: 15/09/2023 View News on Yorvipath Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 September 2023 15/09/2023 More information on Yorvipath EU/3/20/2350 - orphan designation for treatment of hypoparathyroidism EMA/PE/0000241327 - paediatric investigation plan A Study to Assess the Amount of Palopegteriparatide in Breast Milk of Lactating Females Requiring YORVIPATH® (Palopegteriparatide) - post-authorisation study A Global Pregnancy Registry to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to YORVIPATH® (Palopegteriparatide) During Pregnancy and Breastfeeding - post-authorisation study This page was last updated on 06/11/2026
EMA · What's New 儿科学：Human medicines European public assessment report (EPAR): Bylvay, odevixibat, Date of authorisation: 16/07/2021, Revision: 13, Status: Authorised
原文摘录：Bylvay RSS Authorised This medicine is authorised for use in the European Union odevixibat Medicine Human Authorised Application under evaluation CHMP opinion European Commission decision Overview Bylvay is a medicine for treating patients from the age of … More information on Bylvay EU/3/12/1028 - orphan designation for treatment of progressive familial intrahepatic cholestasis EU/3/12/1040 - orphan designation for treatment of Alagille syndrome EMEA-002054-PIP01-16-M03 - paediatric investigation plan EMA/PE/0000240317 - paediatric investigation plan Bylvay - withdrawal of application for variation to marketing authorisation Registry to Document Treatment Effectiveness, Safety, Including Prospective Long-term Outcomes in Participants with Progressive Familial Intrahepatic Cholestasis (PFIC) who take Odevixibat (Bylvay) - post-authorisation study Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS) - post-authorisation study Prospective Registry-Based Study of the Long-Term Safety of Odevixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC) - post-authorisation study This page was last updated on 06/11/2026
PubMed · BMC Public Health 瑞典63,000名青少年潜在画像研究：市政学校卫生服务支出与双因子心理健康画像不均等关联
瑞典斯德哥尔摩地区2006–2024年63,190名九年级学生的潜在画像研究显示，完全心理健康比例从55.6%降至45.4%，Troubled画像从3.4%升至7.2%。
PubMed · Journal of Thoracic and Cardiovascular Surgery 肺动脉闭锁合并室间隔缺损修复中单瓣补片与导管比较的回顾性队列研究
一项回顾性研究纳入1993–2025年130例肺动脉闭锁合并室间隔缺损（PA/VSD）患者，比较0.1-mm膨体聚四氟乙烯单瓣流出道补片（MVOP，n=84）与导管置入（n=46）的右心室流出道相关再手术与长期右心室功能。
PubMed · Psychological Medicine 机器学习分析21,239对中国亲子对预测青少年游戏成瘾的个体、家庭与学校层面因素
一项发表于Psychological Medicine的横断面机器学习研究纳入21,239对中国亲子对（青少年平均年龄13.41岁，45.00%为男性），用Random Forest和XGBoost模型预测游戏成瘾，两者准确率均超过0.80。
PubMed · Journal of Thoracic and Cardiovascular Surgery 先天性心脏病儿童与成人感染性心内膜炎外科治疗结局的单中心回顾分析（2001–2024年96例）
一项回顾性研究纳入2001至2024年间96例因感染性心内膜炎（IE）接受手术的先天性心脏病患者（儿童52例、成人44例），院内死亡率为6.3%，术后5、10、20年生存率分别为90.6%、88.1%和80.5%，20年无复发IE率为96.4%。
PubMed · Pediatric Research 体外研究：BD BACTEC Peds Plus/F 儿童血培养系统对低浓度新生儿败血症常见病原体的检出与阳性时间评估
体外研究将大肠杆菌、无乳链球菌等6种新生儿败血症常见病原体配制成≤1至10-20 CFU/mL的低浓度悬液，接种0.25-2.0 mL至BD BACTEC Peds Plus/F瓶中评估检出率与阳性时间（TTP）。
WHO · Official News OData 儿科学：WHO at COP31: Health Day opens two weeks of activities to deliver on climate change and health
原文摘录：On Monday, 9 November, health takes centre stage as the thirty-first session of the Conference of the Parties (COP31) to the UN Framework Convention on Climate Change (UNFCCC) opens with a dedicated Health Day. … ATACH and AIR give countries the means to do that, during COP31 and beyond
PubMed · Surgical Endoscopy 儿童无肿块/脓肿复杂阑尾炎腹腔镜与开腹阑尾切除术结局比较：CAPP 多中心前瞻性队列亚组分析
荷兰 38 家医院的前瞻性队列纳入 989 例术前疑为复杂阑尾炎（无阑尾炎肿块和/或脓肿）的 18 岁以下儿童，其中 95.3% 行腹腔镜阑尾切除术（LA）、4.7% 行开腹手术（OA），经倾向性加权分析。
PubMed · The Lancet Infectious Diseases Ganaplacide联合lumefantrine固体分散体治疗儿童非复杂性恶性疟的KALUMI开放标签多中心随机对照II期非劣效试验
一项在布基纳法索、马里、科特迪瓦、加蓬和刚果（金）十家机构开展的开放标签、多中心、随机、适应性、II期非劣效试验（NCT04546633，已完成），评估ganaplacide 400 mg联合lumefantrine固体分散体治疗非复杂性恶性疟儿童（6个月至<18岁）的食物效应与非劣效性，对照artemether-lumefantrine。
PubMed · American Journal of Epidemiology 儿科学：Assessing mediation of sexual orientation disparities in miscarriage by age at pregnancy, gravidity, and use of medically assisted reproduction in four national cohorts.
原文摘录：Recent research has documented a higher miscarriage risk among sexual minority individuals than heterosexual individuals. These disparities may be partly mediated by age at pregnancy, gravidity, and use of medically assisted reproduction (MAR). … structural factors in sexual orientation disparities in miscarriage
PubMed · Ophthalmology 小梁切开术与联合术式治疗原发性先天性青光眼三年非劣效随机对照结果
一项前瞻性随机对照非劣效试验纳入248例0-3岁、角膜横径12-14 mm、无既往手术的原发性先天性青光眼患儿，比较小梁切开术与联合术式的36个月结果。意向治疗人群中合格手术成功率为82.3%对84.7%（风险差-2.4%；95%CI，-11.7至6.9；非劣效P=0.004），非劣效界值为-15个百分点，不良事件发生率分别为45.2%与54.0%。
PubMed · The Laryngoscope 儿童与成人胆脂瘤术后复发比较：回顾性队列研究提示儿童年龄为独立复发危险因素
一项回顾性队列研究纳入126例（128耳）初次胆脂瘤手术患者，中位随访5.8年，分析儿童年龄是否独立预测胆脂瘤复发。儿童5年累积复发风险为53.9%（95% CI 34.2%–67.8%），高于成人的19.1%（95% CI 9.5%–27.7%，log-rank p<0.001），差异主要由早期残留病灶驱动（p=0.004）。
PubMed · Journal of the American Academy of Child & Adolescent Psychiatry 随机对照试验：暴露聚焦认知行为治疗增加儿童焦虑中央执行网络与突显网络连接
一项发表于 Journal of the American Academy of Child & Adolescent Psychiatry 的随机对照试验纳入7–18岁焦虑障碍青少年（N=100）及低/无焦虑对照（N=35），比较暴露聚焦CBT（EF-CBT）与放松指导训练（RMT）对静息态fMRI网络连接的影响，112名参与者在治疗后复评。
PubMed · Cerebral Cortex 大麻二酚（CBD）对自闭症儿童额-顶叶静息态EEG功能连接的随机安慰剂对照交叉试验初步分析
一项双盲安慰剂对照交叉试验的静息态EEG分析纳入29名5至12岁自闭症儿童，其中18名接受12周口服大麻二酚（CBD）油与安慰剂（中间8周洗脱）后完成EEG检测。
PubMed · Diseases of the Colon & Rectum 直肠癌根治切除患者低位前切除综合征前瞻性队列研究：术前发生率高于预期且与肠道菌群关联有限
加拿大温哥华St Paul's医院的前瞻性队列纳入71例接受根治性切除并保留肠管连续性的直肠腺癌患者，其中42%（30/71）在治疗前即达到低位前切除综合征（LARS评分≥21）标准，随访结束时64%存在LARS；新发LARS与肿瘤位置更低相关（p=0.04）。
PubMed · Journal of Child Psychology and Psychiatry ABCD队列研究：月经初潮与女性青少年ADHD特质及心理健康的纵向关联
一项基于Adolescent Brain Cognitive Development研究（N=5,318，全部女性，基线平均年龄9.9岁）的纵向分析显示，经前与经后青少年ADHD特质横断面无差异，但初潮后青少年次年ADHD特质更高；乳房发育完全者也预测次年更高ADHD特质。
PubMed · American Journal of Transplantation 2023年OPTN儿童肝分配新规后等待名单失败降低且移植与例外申请未受损：全国中断时间序列研究
一项全国性中断时间序列分析纳入2019年1月至2024年12月登记的12岁以下候者（2,791例登记、368,761人日），评估2023年OPTN儿童医学紧急度更新（修订PELD计算、Status 1A/1B规则及国家肝审查委员会指南）。
medRxiv · 独立预印本 儿科学：Familial factors inflate the estimated effects of genetic liability to ADHD on behavioural outcomes
原文摘录：BackgroundPeople with ADHD have worse health, behavioural and social outcomes, widely interpreted as evidence that ADHD causes these harms, justifying early identification and treatment. … Interventions targeting the individual might not reduce these outcomes as much a
PubMed · NeuroImage 儿科学：Distinct neural patterns of valence and arousal in the developing brain: A representational similarity analysis of MEG.
原文摘录：How children and adolescents encode the affective meaning of visual stimuli remains poorly understood. A key reason is that valence and arousal are inherently correlated across affective stimuli and thus confounded in the categorical contrasts mostly used
EMA · News and Safety Reviews精选 儿科学：AMA and EMA issue joint guidance on Ebola vaccine development
原文摘录：Image credit: © peterschreiber.media | stock.adobe.com AMA and EMA issue joint guidance on Ebola vaccine development 23 October 2026 Clinical trial requirements defined for rapid development of Bundibugyo virus vaccines News Human Clinical trials Research … eu All other enquiries Please submit your request via the online form
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EMA · Withdrawn Applications精选 儿科学：Withdrawn application: Kaftrio, 01/09/2026
原文摘录：Kaftrio - withdrawal of application for variation to marketing authorisation Application withdrawn The application for a change to this medicine's authorisation has been withdrawn ivacaftor / tezacaftor / elexacaftor Post-authorisation Human Overview Vert … News on Kaftrio Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 September 2026 18/09/2026 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 24-27 February 2025 28/02/2025 Combination of cystic fibrosis medicines to treat patients with rare mutations 28/02/2025 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 September 2023 15/09/2023 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 8-11 November 2021 12/11/2021 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 March 2021 26/03/2021 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 June 2020 26/06/2020 New medicine for cystic fibrosis patients 26/06/2020 More information on Kaftrio Kaftrio This page was last updated on 20/10/2026
关注理由：如果关注儿科学，这条临床试验动态可用于了解研究设计，主要终点和试验阶段建议查看原文。
FDA · MedWatch Safety Alerts精选 儿科学：Early Alert: Cardinal Health Issues Correction for Presource Packs Containing BD Alaris Pump Infusion Sets
原文摘录：CDRH is issuing this Early Alert to notify the public of a potentially high-risk device issue. The FDA will keep the public informed and update this web page as significant new information becomes available. … Health care professionals and consumers may report adverse reactions or quality problems they experienced using these devices to MedWatch: The FDA Safety Information and Adverse Event Reporting Program
关注理由：这篇儿科学内容涉及具体研究方向，原文中的样本、结局和限制有助于理解其价值。
FDA · MedWatch Safety Alerts精选 儿科学：Early Alert: Cardinal Health Updates Use Instructions for Presource Packs Containing BD Alaris Pump Infusion Sets
原文摘录：CDRH is issuing this Early Alert to notify the public of a potentially high-risk device issue. The FDA will keep the public informed and update this web page as significant new information becomes available. … Health care professionals and consumers may report adverse reactions or quality problems they experienced using these devices to MedWatch: The FDA Safety Information and Adverse Event Reporting Program
关注理由：这条儿科学资料涉及具体临床问题，研究人群、方法和结局指标可以结合原文进一步查看。
EMA · Withdrawn Applications精选 儿科学：Withdrawn application: Kalydeco, 01/09/2026
原文摘录：Kalydeco - withdrawal of application for variation to marketing authorisation Application withdrawn The application for a change to this medicine's authorisation has been withdrawn ivacaftor Post-authorisation Human Overview Vertex Pharmaceuticals (Irelan … News on Kalydeco Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 September 2026 18/09/2026 Combination of cystic fibrosis medicines to treat patients with rare mutations 28/02/2025 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 19-22 February 2024 23/02/2024 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 11-14 September 2023 15/09/2023 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 8-11 November 2021 12/11/2021 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 22-25 March 2021 26/03/2021 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 September 2020 18/09/2020 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 20-23 July 2020 24/07/2020 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 28-30 April 2020 30/04/2020 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 14-17 October 2019 18/10/2019 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 15-18 October 2018 19/10/2018 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 July 2018 27/07/2018 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 21-24 September 2015 25/09/2015 Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 23-26 June 2014 27/06/2014 More information on Kalydeco Kalydeco This page was last updated on 09/10/2026
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PubMed · Journal of Affective Disorders 多模态机器学习区分青少年抑郁发作住院患者近期自杀行为的回顾性研究
Hao Yu 等在 Journal of Affective Disorders 发表单中心回顾性交叉研究，纳入262名11–17岁抑郁发作住院青少年（其中入院前3个月内实际自杀未遂59例、中断未遂27例），评估多模态机器模型对近期自杀行为的同步区分能力。
PubMed · Food and Chemical Toxicology 发育期双酚A暴露改变雌性小鼠后代的脂肪组织代谢与脂质重塑（动物研究）
C57BL/6J 孕鼠于妊娠第6天至产后第21天经饮水暴露于双酚A（名义目标50 μg/kg/day），断奶后后代分别给予普通或高脂饮食，于PND22、PND36和PND91评估。
PubMed · American Heart Journal 多中心研究：左心发育不良综合征婴儿限制性房间隔伴一期姑息术前间隔干预的结局分析
一项基于 National Pediatric Cardiology Quality Improvement Collaborative 的多中心回顾性队列研究纳入2016–2024年70个中心3,644例接受一期姑息手术（S1P）的婴儿，其中164例（5%）为限制性/完整房间隔（RAS）且行术前间隔干预。
PubMed · Schizophrenia Research ABCD研究9476名青少年：右侧海马体积缩小与持续性痛苦性精神病样体验相关，团体运动参与或减弱该关联（Schizophrenia Research）
基于9476名青少年脑认知发展（ABCD）研究的纵向分析显示，10岁时右侧海马体积缩小与10–13岁持续性痛苦性精神病样体验（PLEs）几率升高相关（每增加1个标准差，aOR=0.91，95% CI 0.85–0.98）。
PubMed · The Journal of Pediatrics 儿科学：School-Based Health and Health Care Utilization Among Rural Youth: Evidence from a School-Based Health Center Opening.
原文摘录：OBJECTIVE: To examine the effects of opening a school-based health center (SBHC) on primary and preventive care utilization among students in a rural setting.
PubMed · Journal of Child Psychology and Psychiatry 青少年自助单次心理健康干预的系统综述与Meta分析：焦虑、抑郁症状呈小效应
一项在PROSPERO注册（CRD42023457030）的系统综述与随机效应Meta分析纳入25项随机对照试验（5,628名参与者，19项进入定量合成），评估青少年自助单次干预（SSIs）对心理健康的效果。