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Pathological demand avoidance: further research is required 病理性需求回避:需要进一步研究
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00192-5
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引用次数: 0
Kawasaki disease: contemporary perspectives 川崎病:当代视角
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00169-X

Kawasaki disease is a paediatric vasculitis that presents with fever, rash, conjunctivitis, mucositis, lymphadenopathy, and extremity changes, and primarily affects children younger than 5 years. Coronary artery aneurysms are observed in approximately 20% of patients without treatment. Giant coronary artery aneurysms are rare but can result in substantial morbidity and mortality due to the risk of thrombosis, stenosis, and myocardial infarction. Infants younger than 6 months and children with coronary artery abnormalities are at highest risk for the development of large or giant coronary artery aneurysms, necessitating swift identification and aggressive treatment. The children at high risk for coronary artery aneurysms warrant primary intensification therapy; however, what the most optimal adjunct therapy might be to reduce their risk is unclear and large-scale international trials are needed. Kawasaki disease is a clinical diagnosis that shares many features with other common febrile illnesses, including multisystem inflammatory syndrome in children. Identifying biomarkers that can distinguish Kawasaki disease from similar conditions and predict coronary artery aneurysm risk are needed to aid timely diagnosis, guide management, and improve patient outcomes.

川崎病是一种儿科血管炎,表现为发热、皮疹、结膜炎、粘膜炎、淋巴结病和四肢病变,主要影响 5 岁以下的儿童。约有 20% 的患者在未经治疗的情况下会出现冠状动脉瘤。巨大的冠状动脉瘤很少见,但由于存在血栓形成、狭窄和心肌梗死的风险,可导致严重的发病率和死亡率。6 个月以下的婴儿和冠状动脉畸形的儿童发生巨大冠状动脉瘤的风险最高,必须迅速识别并积极治疗。冠状动脉瘤高风险患儿需要进行初级强化治疗;然而,降低其风险的最佳辅助疗法是什么尚不清楚,需要进行大规模的国际试验。川崎病的临床诊断与其他常见发热性疾病(包括儿童多系统炎症综合征)有许多共同之处。我们需要确定能将川崎病与类似疾病区分开来并预测冠状动脉瘤风险的生物标志物,以帮助及时诊断、指导治疗并改善患者预后。
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引用次数: 0
Childhood-onset systemic lupus erythematosus in China, 2016–21: a nationwide study 2016-21年中国儿童期系统性红斑狼疮:一项全国性研究
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00172-X
<div><h3>Background</h3><p>Childhood-onset systemic lupus erythematosus (SLE) is a complex autoimmune disorder that can lead to serious organ damage. Despite the prevalence of SLE among children in Asian countries, treatment guidelines, prognosis, and clinical decision making for children with SLE are limited by gaps in region-specific epidemiological data. The aim of this study was to analyse epidemiological characteristics of childhood-onset SLE and associated organ involvement and in-hospital mortality in China.</p></div><div><h3>Methods</h3><p>In this nationwide study, we searched standardised hospital discharge records submitted to the Hospital Quality Monitoring System (HQMS) between Jan 1, 2016, and Dec 31, 2021, for patients with a diagnosis of childhood-onset SLE based on the 2019 American College of Rheumatology or 2012 Systemic Lupus International Collaborating Clinics classification criteria. We selected records for patients aged 18 years and younger containing relevant ICD 10th revision diagnostic codes (specifically M32) among discharge diagnostic codes. We excluded records for patients younger than 5 years, whose SLE diagnosis was presumed to be monogenic lupus, and for patients with overlap syndromes or unidentified sex. Date of diagnosis (equal to the first hospital discharge date), age, organ involvement, intensive care unit (ICU) treatment, and in-hospital mortality were extracted from the records. Incidence rates for 2017, 2018, 2019, 2020, and 2021 were identified with five washout periods ranging from 12 months (Jan 1–Dec 31, 2016) to 60 months (Jan 1, 2016–Dec 31, 2020). Data were stratified by sex, age relative to puberty onset, organ involvement and concurrent infection at time of diagnosis, human development index of region of residence, hospital level, and hospital type. Incidence trends by sex, age relative to puberty onset, and year were derived by joinpoint regression analysis, with 95% CIs calculated by the Poisson exact method. Major organ involvement was assessed according to definitions in the British Isles Lupus Assessment Group 2004 disease activity index. Outcomes of ICU admission after first diagnosis and in-hospital death after ICU admission were analysed in Cox proportional hazards models, with p values and 95% CIs calculated with the parametric method.</p></div><div><h3>Findings</h3><p>Between Jan 1, 2016, and Dec 31, 2021, the HQMS received 134 956 hospital discharge records containing the M32 discharge diagnostic code for patients aged 18 years or younger. 6286 records were excluded, leaving 128 670 records representing 54 338 patients aged 5–18 years; of these, 43 756 patients (36 153 girls and 7603 boys) received their childhood-onset SLE diagnosis on or after Jan 1, 2017. Between Jan 1, 2017, and Dec 31, 2021, the SLE incidence rate was 3·97 (95% CI 3·93–4·01) per 100 000 person-years, with a declining trend during the 5-year period. Joinpoint analysis showed sex-dependent and age-dependent incidence
他们强调了儿童期系统性红斑狼疮发病机制的复杂性,并强调了对儿童期系统性红斑狼疮进行分层精准治疗、知情干预和医疗保健规划的必要性。
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引用次数: 0
Definitions of adverse events associated with extracorporeal membrane oxygenation in children: results of an international Delphi process from the ECMO-CENTRAL ARC 与儿童体外膜氧合相关的不良事件的定义:ECMO-CENTRAL ARC 国际德尔菲进程的结果
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00132-9

Extracorporeal membrane oxygenation (ECMO) is a high-risk and low-volume life support with increasing clinical study. However, heterogenous outcome definitions impede data assimilation into evidence to guide practice. The Academic Research Consortium (ARC), an international collaborative forum committed to facilitating the creation of stakeholder-driven consensus nomenclature and outcomes for clinical trials of medical devices, supported the ECMO Core Elements Needed for Trials Regulation And quality of Life (ECMO-CENTRAL) ARC. The ECMO-CENTRAL ARC was assembled to develop definitions of paediatric ECMO adverse events for use in clinical trials and regulatory device evaluation. An initial candidate list of ECMO adverse events derived from the mechanical circulatory support ARC was supplemented with a review of ECMO-relevant adverse event definitions collated from literature published between Jan 1, 1988, and Feb 20, 2023. Distinct teams of international topic experts drafted separate adverse event definitions that were harmonised to existing literature when appropriate. Draft definitions were revised for paediatric ECMO relevance with input from patients, families, and an international expert panel of trialists, clinicians, statisticians, biomedical engineers, device developers, and regulatory agencies. ECMO-CENTRAL ARC was revised and disseminated across research societies and professional organisations. Up to three rounds of internet-based anonymous surveys were planned as a modified Delphi process. The expert panel defined 13 adverse event definitions: neurological, bleeding, device malfunction, acute kidney injury, haemolysis, infection, vascular access-associated injury, non-CNS thrombosis, hepatic dysfunction, right heart failure, left ventricular overload, lactic acidaemia, and hypoxaemia. Definitional structure varied. Among 165 expert panel members, 114 were eligible to vote and 111 voted. Consensus was achieved for all proposed definitions. Agreement ranged from 82% to 95%. ECMO-CENTRAL ARC paired rigorous development with methodical stakeholder involvement and dissemination to define paediatric ECMO adverse events. These definitions will facilitate new research and the assimilation of data across clinical trials and ECMO device evaluation in children.

体外膜肺氧合(ECMO)是一种高风险、低容量的生命支持系统,临床研究越来越多。然而,不同的结果定义阻碍了将数据吸收为指导实践的证据。学术研究联盟(ARC)是一个国际合作论坛,致力于促进创建利益相关者驱动的医疗设备临床试验术语和结果共识,并支持 ECMO 试验监管和生活质量所需的核心要素(ECMO-CENTRAL)ARC。ECMO-CENTRAL ARC 的目的是制定儿科 ECMO 不良事件的定义,以用于临床试验和监管设备评估。在对 1988 年 1 月 1 日至 2023 年 2 月 20 日期间发表的文献中整理的 ECMO 相关不良事件定义进行审查后,对机械循环支持 ARC 得出的 ECMO 不良事件初步候选清单进行了补充。不同的国际专题专家小组分别起草了不良事件定义,并酌情与现有文献进行了统一。根据患者、家属以及由试验专家、临床医生、统计学家、生物医学工程师、设备开发人员和监管机构组成的国际专家小组的意见,修订了与儿科 ECMO 相关的定义草案。对 ECMO-CENTRAL ARC 进行了修订,并在研究协会和专业组织中进行了传播。计划采用改良德尔菲流程,在互联网上进行多达三轮的匿名调查。专家组定义了 13 个不良事件定义:神经系统、出血、设备故障、急性肾损伤、溶血、感染、血管通路相关损伤、非中枢神经系统血栓形成、肝功能异常、右心衰竭、左心室负荷过重、乳酸血症和低氧血症。定义结构各不相同。在 165 名专家组成员中,114 人有资格投票,111 人投了票。所有提议的定义都达成了共识。一致性从 82% 到 95% 不等。ECMO-CENTRAL ARC 将严格的开发与有条不紊的利益相关者参与和传播相结合,对儿科 ECMO 不良事件进行定义。这些定义将有助于开展新的研究,并在儿童临床试验和 ECMO 设备评估中吸收数据。
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引用次数: 0
Course-correcting youth mental health care 纠正青少年心理健康护理的方向
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00238-4
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引用次数: 0
Pathological demand avoidance: further research is required 病理性需求回避:需要进一步研究
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00189-5
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引用次数: 0
Correction to Lancet Child Adolesc Health 2024; 8: 713–15 柳叶刀儿童青少年健康》2024; 8: 713-15 更正
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00239-6
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引用次数: 0
Pathological demand avoidance: further research is required – Authors' reply 病理性需求回避:需要进一步研究 - 作者回复
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-17 DOI: 10.1016/S2352-4642(24)00203-7
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引用次数: 0
Asking difficult questions about fetal alcohol spectrum disorder in the context of the child, the mother, and the systems in which they live 结合孩子、母亲和他们所处的环境,提出有关胎儿酒精中毒谱系障碍的难题。
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-09-16 DOI: 10.1016/S2352-4642(24)00188-3
Alcohol is a known teratogen and prenatal alcohol exposure remains a major ongoing public health concern. Fetal alcohol spectrum disorder has become the diagnosis for describing individuals who have been affected by prenatal alcohol exposure. In this Viewpoint, we raise major concerns about its continued use as a diagnostic term in how it perpetuates a misleading and outdated narrative about child development and maternal health. We argue that the term fetal alcohol spectrum disorder has contributed to a culture of racism and discrimination for many who are diagnosed with it. The term fetal alcohol spectrum disorder fails to capture the progress made in our collective understanding of neurodevelopment through advancements in the field of genetics and in understanding the effects of trauma and adversity. We call for urgent international collaborative action to review the use of it as a diagnostic term and, more broadly, to reconsider the practice of diagnosing disabilities as medical illnesses. We suggest that this practice fails to recognise that outcomes of functioning and participation in individuals are not only the results of health conditions, but are also the products of complex interactions and experiences of individuals within the families and societies in which they live.
酒精是一种已知的致畸剂,产前接触酒精仍然是一个重大的公共卫生问题。胎儿酒精谱系障碍已成为描述受产前酒精暴露影响的个体的诊断方法。在本 "观点 "中,我们对胎儿酒精谱系障碍作为诊断术语的持续使用提出了极大的担忧,因为它延续了对儿童发育和孕产妇健康的误导和过时的说法。我们认为,胎儿酒精谱系障碍这一术语助长了种族主义文化,并歧视了许多被诊断出患有该疾病的人。胎儿酒精中毒谱系障碍这一术语未能反映我们通过遗传学领域的进步以及对创伤和逆境影响的理解,在对神经发育的集体认识方面所取得的进展。我们呼吁采取紧急国际合作行动,审查将其作为诊断术语的使用情况,并从更广泛的角度重新考虑将残疾诊断为医学疾病的做法。我们认为,这种做法没有认识到,个人功能和参与的结果不仅是健康状况的结果,也是个人在其生活的家庭和社会中复杂互动和经历的产物。
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引用次数: 0
Late effects after allogeneic haematopoietic cell transplantation in children and adolescents with non-malignant disorders: a retrospective cohort study 患有非恶性疾病的儿童和青少年接受异体造血细胞移植后的后期影响:一项回顾性队列研究。
IF 19.9 1区 医学 Q1 PEDIATRICS Pub Date : 2024-08-30 DOI: 10.1016/S2352-4642(24)00167-6
<div><h3>Background</h3><p>Continued advances in haematopoietic cell transplantation (HCT) for children with non-malignant diseases (NMDs) have led to a growing population of survivors in whom late occurring toxic effects remain a challenge. We investigated the incidence of and risk factors for post-transplant toxicities in a contemporary cohort of children and adolescents undergoing HCT for NMDs.</p></div><div><h3>Methods</h3><p>In this retrospective cohort study, we extracted data from the Center for International Blood and Marrow Transplantation Research (CIBMTR) database to analyse timing and incidence of effects and risk factors associated with late effects of HCT for treatment of NMDs at age 21 years or younger. Late effects of interest were avascular necrosis, cataracts, congestive heart failure, myocardial infarction, diabetes, gonadal dysfunction, growth hormone deficiency, hypothyroidism, renal failure requiring dialysis, and neurological events (stroke and seizure). Cumulative incidence of each late effect was calculated at 5 years and 7 years after HCT. Risk factors were evaluated in Cox proportional hazards regression analyses. Main exposures were primary NMD, age, sex, ethnicity and race, insurance, donor and graft type, myoablative conditioning, total-body irradiation exposure, graft-versus-host disease (GVHD), and transplant year. Primary outcomes were rates, cumulative incidence probability (95% CI), and risk-factors for organ-specific late effects.</p></div><div><h3>Findings</h3><p>Between Jan 1, 2000, and Dec 31, 2017, 7785 patients aged 21 years or younger underwent HCT. 1995 patients were ineligible or did not consent to be included. 5790 patients from 171 centres were included in the analysis. 3505 (60·5%) of 5790 patients were male and 2285 (39·5%) were female. 2106 (36·4%) patients were White, 771 (13·3%) were Hispanic, and 773 (12·7%) were Black. 1790 (30·9%) patients were non-USA residents. Median age at HCT was 5·5 years (range 0·0–21·0). 1127 (19%) of 5790 patients had one late effect, and 381 (7%) had at least two. At 7 years post-HCT, the cumulative incidence probability was 1·9 (95% CI 1·5–2·3) for cataracts, 4·9 (4·3–5·6) for diabetes, 2·6 (2·1–3·1) for gonadal dysfunction, 3·2 (2·7–3·8) for hypothyroidism, 5·0 (4·4–5·7) for growth disturbance, 8·1 (7·4–8·9) for renal failure, 1·6 (1·3–2·0) for avascular necrosis, 0·6 (0·4–0·8) for congestive heart failure, 0·2 (0·1–0·3) for myocardial infarction, and 9·4 (8·6–10·2) for neurological effects. Age 10 years or older at HCT, unrelated donor source, total-body irradiation, and GVHD were identified as risk factors for long-term effects.</p></div><div><h3>Interpretation</h3><p>The findings highlight the need for, and access to, multidisciplinary and lifelong follow-up for children undergoing HCT for NMDs. As more children undergo treatment with cellular therapies for non-malignant conditions, further analyses of post-transplant data could increasingly guide treatment deci
背景:针对非恶性疾病(NMDs)儿童的造血细胞移植(HCT)技术不断进步,导致幸存者人数不断增加,而后期出现的毒性反应仍是一项挑战。我们对当代接受非恶性疾病 HCT 治疗的儿童和青少年队列中移植后毒性反应的发生率和风险因素进行了调查:在这项回顾性队列研究中,我们从国际血液和骨髓移植研究中心(CIBMTR)的数据库中提取数据,分析了 21 岁或 21 岁以下接受 HCT 治疗 NMDs 的时间、影响发生率以及与后期影响相关的风险因素。晚期效应包括血管性坏死、白内障、充血性心力衰竭、心肌梗塞、糖尿病、性腺功能障碍、生长激素缺乏、甲状腺功能减退、需要透析的肾功能衰竭和神经系统事件(中风和癫痫发作)。每种晚期效应的累积发生率在 HCT 后 5 年和 7 年进行计算。通过 Cox 比例危险回归分析评估了风险因素。主要暴露因素包括原发性 NMD、年龄、性别、民族和种族、保险、供体和移植物类型、肌烧蚀调理、全身照射暴露、移植物抗宿主病(GVHD)和移植年份。主要结果是器官特异性晚期效应的发生率、累积发生概率(95% CI)和风险因素:2000年1月1日至2017年12月31日期间,7785名年龄在21岁或以下的患者接受了造血干细胞移植。1995名患者不符合条件或不同意纳入。来自 171 个中心的 5790 名患者被纳入分析。5790 名患者中有 3505 名(60-5%)男性,2285 名(39-5%)女性。2106名(36-4%)患者为白人,771名(13-3%)为西班牙裔,773名(12-7%)为黑人。1790名(30-9%)患者为非美国居民。接受 HCT 时的中位年龄为 5-5 岁(范围为 0-0-21-0)。5790 名患者中有 1127 人(19%)出现过一次晚期效应,381 人(7%)至少出现过两次。白内障、糖尿病、性腺功能障碍、甲状腺功能减退、生长障碍的累积发病率分别为 1-9 (95% CI 1-5-2-3)、4-9 (4-3-5-6)、2-6 (2-1-3-1)、3-2 (2-7-3-8)、5-0 (4-4-5-7)、肾功能衰竭为 8-1(7-4-8-9),血管性坏死为 1-6(1-3-2-0),充血性心力衰竭为 0-6(0-4-0-8),心肌梗死为 0-2(0-1-0-3),神经系统影响为 9-4(8-6-10-2)。造血干细胞移植时的年龄为 10 岁或以上、非亲缘供体来源、全身照射和 GVHD 被确定为长期影响的风险因素:研究结果凸显了对接受 HCT 治疗 NMD 儿童进行多学科和终身随访的必要性。随着越来越多的儿童接受细胞疗法治疗非恶性疾病,对移植后数据的进一步分析将越来越多地指导治疗决策和后续的长期监测:国家癌症研究所、国家心肺和血液研究所、国家过敏和传染病研究所、卫生资源和服务管理局以及海军研究办公室。
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