Pub Date : 2024-03-21DOI: 10.1016/S2352-4642(23)00345-0
Nadia Hasan MBBS , Prof Clare Nourse MD , Prof H Simon Schaaf MD , Prof Adrie Bekker PhD , Marian Loveday PhD , Betina M Alcântara Gabardo PhD , Christopher Coulter MBBS , Chishala Chabala MMed , Sushil Kabra MD , Eilish Moore BSc , Prof Elizabeth Maleche-Obimbo MMed , Nicole Salazar-Austin MD , Prof Nicole Ritz PhD , Prof Jeffrey R Starke MD , Andrew P Steenhoff MBBCh , Rina Triasih PhD , Steven B Welch FRCPCH , Prof Ben J Marais PhD
Infants born to mothers with tuberculosis disease are at increased risk of developing tuberculosis disease themselves. We reviewed published studies and guidelines on the management of these infants to inform the development of a consensus practice guideline. We searched MEDLINE, CINAHL, and Cochrane Library from database inception to Dec 1, 2022, for original studies reporting the management and outcome of infants born to mothers with tuberculosis. Of the 521 published papers identified, only three met inclusion criteria and no evidence-based conclusions could be drawn from these studies, given their narrow scope, variable aims, descriptive nature, inconsistent data collection, and high attrition rates. We also assessed a collection of national and international guidelines to inform a consensus practice guideline developed by an international panel of experts from different epidemiological contexts. The 16 guidelines reviewed had consistent features to inform the expert consultation process. Two management algorithms were developed—one for infants born to mothers considered potentially infectious at the time of delivery and another for mothers not considered infectious at the time of delivery—with different guidance for high and low tuberculosis incidence settings. This systematic review and consensus practice guideline should facilitate more consistent clinical management, support the collection of better data, and encourage the development of more studies to improve evidence-based care.
{"title":"Management of the infant born to a mother with tuberculosis: a systematic review and consensus practice guideline","authors":"Nadia Hasan MBBS , Prof Clare Nourse MD , Prof H Simon Schaaf MD , Prof Adrie Bekker PhD , Marian Loveday PhD , Betina M Alcântara Gabardo PhD , Christopher Coulter MBBS , Chishala Chabala MMed , Sushil Kabra MD , Eilish Moore BSc , Prof Elizabeth Maleche-Obimbo MMed , Nicole Salazar-Austin MD , Prof Nicole Ritz PhD , Prof Jeffrey R Starke MD , Andrew P Steenhoff MBBCh , Rina Triasih PhD , Steven B Welch FRCPCH , Prof Ben J Marais PhD","doi":"10.1016/S2352-4642(23)00345-0","DOIUrl":"10.1016/S2352-4642(23)00345-0","url":null,"abstract":"<div><p>Infants born to mothers with tuberculosis disease are at increased risk of developing tuberculosis disease themselves. We reviewed published studies and guidelines on the management of these infants to inform the development of a consensus practice guideline. We searched MEDLINE, CINAHL, and Cochrane Library from database inception to Dec 1, 2022, for original studies reporting the management and outcome of infants born to mothers with tuberculosis. Of the 521 published papers identified, only three met inclusion criteria and no evidence-based conclusions could be drawn from these studies, given their narrow scope, variable aims, descriptive nature, inconsistent data collection, and high attrition rates. We also assessed a collection of national and international guidelines to inform a consensus practice guideline developed by an international panel of experts from different epidemiological contexts. The 16 guidelines reviewed had consistent features to inform the expert consultation process. Two management algorithms were developed—one for infants born to mothers considered potentially infectious at the time of delivery and another for mothers not considered infectious at the time of delivery—with different guidance for high and low tuberculosis incidence settings. This systematic review and consensus practice guideline should facilitate more consistent clinical management, support the collection of better data, and encourage the development of more studies to improve evidence-based care.</p></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":null,"pages":null},"PeriodicalIF":36.4,"publicationDate":"2024-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140208765","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-03-19DOI: 10.1016/S2352-4642(24)00017-8
Prof Luregn J Schlapbach PhD , Devika Ganesamoorthy PhD , Clare Wilson BChir , Sainath Raman MBBS PhD , Shane George MPH , Peter J Snelling MPHTM , Natalie Phillips MPhil , Adam Irwin PhD , Natalie Sharp BSc , Renate Le Marsney MPH , Arjun Chavan MD , Allison Hempenstall MPH , Seweryn Bialasiewicz PhD , Anna D MacDonald PhD , Prof Keith Grimwood MD , Jessica C Kling PhD , Stephen J McPherson PhD , Antje Blumenthal Dr rer nat , Myrsini Kaforou PhD , Prof Michael Levin PhD , Lachlan Coin
Background
Sepsis is defined as dysregulated host response to infection that leads to life-threatening organ dysfunction. Biomarkers characterising the dysregulated host response in sepsis are lacking. We aimed to develop host gene expression signatures to predict organ dysfunction in children with bacterial or viral infection.
Methods
This cohort study was done in emergency departments and intensive care units of four hospitals in Queensland, Australia, and recruited children aged 1 month to 17 years who, upon admission, underwent a diagnostic test, including blood cultures, for suspected sepsis. Whole-blood RNA sequencing of blood was performed with Illumina NovaSeq (San Diego, CA, USA). Samples with completed phenotyping, monitoring, and RNA extraction by March 31, 2020, were included in the discovery cohort; samples collected or completed thereafter and by Oct 27, 2021, constituted the Rapid Paediatric Infection Diagnosis in Sepsis (RAPIDS) internal validation cohort. An external validation cohort was assembled from RNA sequencing gene expression count data from the observational European Childhood Life-threatening Infectious Disease Study (EUCLIDS), which recruited children with severe infection in nine European countries between 2012 and 2016. Feature selection approaches were applied to derive novel gene signatures for disease class (bacterial vs viral infection) and disease severity (presence vs absence of organ dysfunction 24 h post-sampling). The primary endpoint was the presence of organ dysfunction 24 h after blood sampling in the presence of confirmed bacterial versus viral infection. Gene signature performance is reported as area under the receiver operating characteristic curves (AUCs) and 95% CI.
Findings
Between Sept 25, 2017, and Oct 27, 2021, 907 patients were enrolled. Blood samples from 595 patients were included in the discovery cohort, and samples from 312 children were included in the RAPIDS validation cohort. We derived a ten-gene disease class signature that achieved an AUC of 94·1% (95% CI 90·6–97·7) in distinguishing bacterial from viral infections in the RAPIDS validation cohort. A ten-gene disease severity signature achieved an AUC of 82·2% (95% CI 76·3–88·1) in predicting organ dysfunction within 24 h of sampling in the RAPIDS validation cohort. Used in tandem, the disease class and disease severity signatures predicted organ dysfunction within 24 h of sampling with an AUC of 90·5% (95% CI 83·3–97·6) for patients with predicted bacterial infection and 94·7% (87·8–100·0) for patients with predicted viral infection. In the external EUCLIDS validation dataset (n=362), the disease class and disease severity predicted organ dysfunction at time of sampling with an AUC of 70·1% (95% CI 44·1–96·2) for patients with predicted bacterial infection and 69·6% (53·1–86·0) for patients with predicted viral infection.
{"title":"Host gene expression signatures to identify infection type and organ dysfunction in children evaluated for sepsis: a multicentre cohort study","authors":"Prof Luregn J Schlapbach PhD , Devika Ganesamoorthy PhD , Clare Wilson BChir , Sainath Raman MBBS PhD , Shane George MPH , Peter J Snelling MPHTM , Natalie Phillips MPhil , Adam Irwin PhD , Natalie Sharp BSc , Renate Le Marsney MPH , Arjun Chavan MD , Allison Hempenstall MPH , Seweryn Bialasiewicz PhD , Anna D MacDonald PhD , Prof Keith Grimwood MD , Jessica C Kling PhD , Stephen J McPherson PhD , Antje Blumenthal Dr rer nat , Myrsini Kaforou PhD , Prof Michael Levin PhD , Lachlan Coin","doi":"10.1016/S2352-4642(24)00017-8","DOIUrl":"10.1016/S2352-4642(24)00017-8","url":null,"abstract":"<div><h3>Background</h3><p>Sepsis is defined as dysregulated host response to infection that leads to life-threatening organ dysfunction. Biomarkers characterising the dysregulated host response in sepsis are lacking. We aimed to develop host gene expression signatures to predict organ dysfunction in children with bacterial or viral infection.</p></div><div><h3>Methods</h3><p>This cohort study was done in emergency departments and intensive care units of four hospitals in Queensland, Australia, and recruited children aged 1 month to 17 years who, upon admission, underwent a diagnostic test, including blood cultures, for suspected sepsis. Whole-blood RNA sequencing of blood was performed with Illumina NovaSeq (San Diego, CA, USA). Samples with completed phenotyping, monitoring, and RNA extraction by March 31, 2020, were included in the discovery cohort; samples collected or completed thereafter and by Oct 27, 2021, constituted the Rapid Paediatric Infection Diagnosis in Sepsis (RAPIDS) internal validation cohort. An external validation cohort was assembled from RNA sequencing gene expression count data from the observational European Childhood Life-threatening Infectious Disease Study (EUCLIDS), which recruited children with severe infection in nine European countries between 2012 and 2016. Feature selection approaches were applied to derive novel gene signatures for disease class (bacterial <em>vs</em> viral infection) and disease severity (presence <em>vs</em> absence of organ dysfunction 24 h post-sampling). The primary endpoint was the presence of organ dysfunction 24 h after blood sampling in the presence of confirmed bacterial versus viral infection. Gene signature performance is reported as area under the receiver operating characteristic curves (AUCs) and 95% CI.</p></div><div><h3>Findings</h3><p>Between Sept 25, 2017, and Oct 27, 2021, 907 patients were enrolled. Blood samples from 595 patients were included in the discovery cohort, and samples from 312 children were included in the RAPIDS validation cohort. We derived a ten-gene disease class signature that achieved an AUC of 94·1% (95% CI 90·6–97·7) in distinguishing bacterial from viral infections in the RAPIDS validation cohort. A ten-gene disease severity signature achieved an AUC of 82·2% (95% CI 76·3–88·1) in predicting organ dysfunction within 24 h of sampling in the RAPIDS validation cohort. Used in tandem, the disease class and disease severity signatures predicted organ dysfunction within 24 h of sampling with an AUC of 90·5% (95% CI 83·3–97·6) for patients with predicted bacterial infection and 94·7% (87·8–100·0) for patients with predicted viral infection. In the external EUCLIDS validation dataset (n=362), the disease class and disease severity predicted organ dysfunction at time of sampling with an AUC of 70·1% (95% CI 44·1–96·2) for patients with predicted bacterial infection and 69·6% (53·1–86·0) for patients with predicted viral infection.</p></div><div><h3>Interpretatio","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":null,"pages":null},"PeriodicalIF":36.4,"publicationDate":"2024-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140186797","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-03-16DOI: 10.1016/S2352-4642(24)00021-X
Hannah Norman-Bruce MBBS , Etimbuk Umana MD , Clare Mills PhD , Hannah Mitchell PhD , Lisa McFetridge PhD , David McCleary BSc , Thomas Waterfield PhD
Background
Febrile infants presenting in the first 90 days of life are at higher risk of invasive and serious bacterial infections than older children. Modern clinical practice guidelines, mostly using procalcitonin as a diagnostic biomarker, can identify infants who are at low risk and therefore suitable for tailored management. C-reactive protein, by comparison, is widely available, but whether C-reactive protein and procalcitonin have similar diagnostic accuracy is unclear. We aimed to compare the test accuracy of procalcitonin and C-reactive protein in the prediction of invasive or serious bacterial infections in febrile infants.
Methods
For this systematic review and meta-analysis, we searched MEDLINE, EMBASE, Web of Science, and The Cochrane Library for diagnostic test accuracy studies up to June 19, 2023, using MeSH terms “procalcitonin”, and “bacterial infection” or “fever” and keywords “invasive bacterial infection*” and “serious bacterial infection*”, without language or date restrictions. Studies were selected by independent authors against eligibility criteria. Eligible studies included participants aged 90 days or younger presenting to hospital with a fever (≥38°C) or history of fever within the preceding 48 h. The primary index test was procalcitonin, and the secondary index test was C-reactive protein. Test kits had to be commercially available, and test samples had to be collected upon presentation to hospital. Invasive bacterial infection was defined as the presence of a bacterial pathogen in blood or cerebrospinal fluid, as detected by culture or quantitative PCR; authors' definitions of serious bacterial infection were used. Data were extracted from selected studies, and the detection of invasive or serious bacterial infections was analysed with two models for each biomarker. Diagnostic accuracy was determined against internationally recognised cutoff values (0·5 ng/mL for procalcitonin, 20 mg/L for C-reactive protein) and pooled to calculate partial area under the curve (pAUC) values for each biomarker. Optimum cutoff values were identified for each biomarker. This study is registered with PROSPERO, CRD42022293284.
Findings
Of 734 studies derived from the literature search, 14 studies (n=7755) were included in the meta-analysis. For the detection of invasive bacterial infections, pAUC values were greater for procalcitonin (0·72, 95% CI 0·56–0·79) than C-reactive protein (0·28, 0·17–0·61; p=0·016). Optimal cutoffs for detecting invasive bacterial infections were 0·49 ng/mL for procalcitonin and 13·12 mg/L for C-reactive protein. For the detection of serious bacterial infections, procalcitonin and C-reactive protein had similar pAUC values (0·55, 0·44–0·69 vs 0·54, 0·40–0·61; p=0·92). For serious bacterial infections, the optimal cutoffs for procalcitonin and C-reactive protein were 0·17 ng/mL and 16·18 mg/L, respectively. Heterogeneity was low for studie
与年龄较大的儿童相比，出生后 90 天内发热的婴儿发生侵袭性和严重细菌感染的风险更高。现代临床实践指南大多使用降钙素原作为诊断生物标志物，可以识别低风险婴儿，因此适合进行有针对性的管理。相比之下，C 反应蛋白可广泛使用，但 C 反应蛋白和降钙素是否具有相似的诊断准确性尚不清楚。我们旨在比较降钙素原和 C 反应蛋白在预测发热婴儿侵入性或严重细菌感染方面的检测准确性。在本系统综述和荟萃分析中，我们使用 MeSH 术语 "降钙素原"、"细菌感染 "或 "发热 "以及关键词 "侵袭性细菌感染*"和 "严重细菌感染*"检索了 MEDLINE、EMBASE、Web of Science 和 Cochrane 图书馆中截至 2023 年 6 月 19 日的诊断测试准确性研究，没有语言或日期限制。研究由独立作者根据资格标准进行筛选。符合条件的研究包括年龄在 90 天或以下、因发烧（≥38°C）或在之前 48 小时内有发烧史而入院的参与者。检测试剂盒必须在市场上有售，检测样本必须在患者入院时采集。通过培养或定量 PCR 检测到血液或脑脊液中存在细菌病原体，即为侵袭性细菌感染；严重细菌感染的定义由作者确定。从选定的研究中提取数据，并针对每种生物标记物使用两种模型对侵袭性或严重细菌感染的检测结果进行分析。根据国际公认的临界值（降钙素原为 0-5 纳克/毫升，C 反应蛋白为 20 毫克/升）确定诊断准确性，并汇总计算每种生物标记物的部分曲线下面积 (pAUC) 值。为每种生物标记物确定了最佳临界值。本研究已在 PROSPERO 注册，编号为 CRD42022293284。在文献检索得出的 734 项研究中，有 14 项研究（n=7755）被纳入荟萃分析。在检测侵袭性细菌感染方面，降钙素原（0-72，95% CI 0-56-0-79）的 pAUC 值高于 C 反应蛋白（0-28，0-17-0-61；p=0-016）。检测侵袭性细菌感染的最佳临界值为：降钙素原 0-49 纳克/毫升，C 反应蛋白 13-12 毫克/升。在检测严重细菌感染时，降钙素原和 C 反应蛋白的 pAUC 值相似（0-55、0-44-0-69 0-54、0-40-0-61；p=0-92）。对于严重细菌感染，降钙素原和 C 反应蛋白的最佳临界值分别为 0-17 纳克/毫升和 16-18 毫克/升。调查降钙素原检测侵袭性细菌感染准确性的研究异质性较低（=23-5%），调查降钙素原检测严重细菌感染准确性的研究异质性较高（=75-5%），调查C反应蛋白检测侵袭性细菌感染准确性的研究异质性中等（=49-5%），调查C反应蛋白检测严重细菌感染准确性的研究异质性中等（=28-3%）。各研究对严重细菌感染缺乏统一的定义是造成研究间差异和潜在偏倚的最大原因。在一个大型发热婴儿队列中，0-5 纳克/毫升的降钙素原临界值比 20 毫克/升的 C 反应蛋白临界值具有更高的 pAUC 值，可用于鉴别侵袭性细菌感染。因此，在无法获得降钙素的情况下，应谨慎使用 C 反应蛋白来鉴别侵袭性细菌感染，并应考虑使用低于 20 毫克/升的临界值。在国际公认的临界值下，C 反应蛋白和降钙素原在鉴别严重细菌感染方面显示出相似的检测准确性。这可能反映了确认严重细菌感染所面临的挑战，以及严重细菌感染缺乏公认的定义。无。
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Pub Date : 2024-03-12DOI: 10.1016/S2352-4642(24)00053-1
Breanna Tory
{"title":"The pain of being a young person experiencing chronic pain","authors":"Breanna Tory","doi":"10.1016/S2352-4642(24)00053-1","DOIUrl":"https://doi.org/10.1016/S2352-4642(24)00053-1","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":null,"pages":null},"PeriodicalIF":36.4,"publicationDate":"2024-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140113977","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-03-12DOI: 10.1016/S2352-4642(23)00310-3
Prof Ravi Savarirayan MD , Prof Julie Hoover-Fong MD , Patrick Yap MD , Svein O Fredwall MD
Achondroplasia is the most common form of dwarfism in humans, caused by a common pathogenic variant in the gene encoding fibroblast growth factor receptor 3, FGFR3, which impairs the process of endochondral ossification of the growing skeleton. In this Review, we outline the clinical and genetic hallmarks of achondroplasia and related FGFR3 conditions, the natural history and impact of achondroplasia over a patient's lifespan, and diagnosis and management options. We then focus on the new and emerging drug therapies that target the underlying pathogenesis of this condition. These new options are changing the natural growth patterns of achondroplasia, with the prospect of better long-term health outcomes for patients.
{"title":"New treatments for children with achondroplasia","authors":"Prof Ravi Savarirayan MD , Prof Julie Hoover-Fong MD , Patrick Yap MD , Svein O Fredwall MD","doi":"10.1016/S2352-4642(23)00310-3","DOIUrl":"https://doi.org/10.1016/S2352-4642(23)00310-3","url":null,"abstract":"<div><p>Achondroplasia is the most common form of dwarfism in humans, caused by a common pathogenic variant in the gene encoding fibroblast growth factor receptor 3, <em>FGFR3</em>, which impairs the process of endochondral ossification of the growing skeleton. In this Review, we outline the clinical and genetic hallmarks of achondroplasia and related <em>FGFR3</em> conditions, the natural history and impact of achondroplasia over a patient's lifespan, and diagnosis and management options. We then focus on the new and emerging drug therapies that target the underlying pathogenesis of this condition. These new options are changing the natural growth patterns of achondroplasia, with the prospect of better long-term health outcomes for patients.</p></div>","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":null,"pages":null},"PeriodicalIF":36.4,"publicationDate":"2024-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140113147","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Pub Date : 2024-02-29DOI: 10.1016/S2352-4642(24)00052-X
The Lancet Child & Adolescent Health
{"title":"Can the 2024 UK election change the child health trajectory?","authors":"The Lancet Child & Adolescent Health","doi":"10.1016/S2352-4642(24)00052-X","DOIUrl":"10.1016/S2352-4642(24)00052-X","url":null,"abstract":"","PeriodicalId":54238,"journal":{"name":"Lancet Child & Adolescent Health","volume":null,"pages":null},"PeriodicalIF":36.4,"publicationDate":"2024-02-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140023810","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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