BMJ Paediatrics Open最新文献

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Dexmedetomidine in neonates: utilisation trends and safety profile over time in a neonatal intensive care unit. 右美托咪定在新生儿:在新生儿重症监护病房的使用趋势和安全性概况。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-18 DOI: 10.1136/bmjpo-2024-003004
Gozdem Kayki, Nadir Yalcin, Hasan Tolga Celik, Sule Yigit
{"title":"Dexmedetomidine in neonates: utilisation trends and safety profile over time in a neonatal intensive care unit.","authors":"Gozdem Kayki, Nadir Yalcin, Hasan Tolga Celik, Sule Yigit","doi":"10.1136/bmjpo-2024-003004","DOIUrl":"10.1136/bmjpo-2024-003004","url":null,"abstract":"<p><strong>Background: </strong>Dexmedetomidine is an alpha-2 adrenergic agonist with sedative, anxiolytic and analgesic effects. Its use in neonatal intensive care units (NICUs) has been increasing in the last decade. The aim of this study was to assess the safety profile of dexmedetomidine and to identify specific trends in its use over time.</p><p><strong>Methods: </strong>In this retrospective observational study, data were collected on all patients who received continuous infusion of dexmedetomidine in a level IV NICU in Turkey between 2018 and 2023. Demographic characteristics were compared between preterm and term infants using the Mann-Whitney U test. Differences in adverse effects between term and preterm infants, as well as between lower and higher doses, were analysed using the χ<sup>2</sup> test. Regression analysis was conducted to identify factors influencing adverse effects.</p><p><strong>Results: </strong>A total of 383 patients were included. The participants had a median (IQR) gestational age of 37 (35-38) weeks with a median (IQR) birth weight of 2700 (2140-3270) grams and the median (IQR) postmenstrual age at the time of dexmedetomidine initiation was 38 (36-40) weeks. The most common indication for use was pain control following surgery and/or interventional procedures (81.5%). There was a statistically significant increase in initial doses (p<0.001) and treatment duration (p=0.009). Adverse drug reactions (ADRs) were observed in 5% of cases, mostly bradycardia (50%) and ADRs did not correlate with the dose (0.80), treatment duration (0.96) or gestational age (p=0.93).</p><p><strong>Conclusion: </strong>Our data and experiences demonstrated a significant increase in the dose and duration of dexmedetomidine use in the NICU over the years. Additionally, the findings have suggested that higher doses and treatment duration do not result in an increase in ADRs during the acute period.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11927418/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143655809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
The impact of climate change on child nutrition in Indonesia: a conceptual framework and scoping review of the available evidence. 气候变化对印度尼西亚儿童营养的影响:对现有证据的概念框架和范围审查。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-18 DOI: 10.1136/bmjpo-2024-002980
David Colozza, Isabella Guo, Sri Wahyuni Sukotjo, Astrid Citra Padmita, Rene Gerard Galera, Endang Sulastri, Inti Wikanestri, Mamadou Ndiaye
{"title":"The impact of climate change on child nutrition in Indonesia: a conceptual framework and scoping review of the available evidence.","authors":"David Colozza, Isabella Guo, Sri Wahyuni Sukotjo, Astrid Citra Padmita, Rene Gerard Galera, Endang Sulastri, Inti Wikanestri, Mamadou Ndiaye","doi":"10.1136/bmjpo-2024-002980","DOIUrl":"10.1136/bmjpo-2024-002980","url":null,"abstract":"<p><strong>Background: </strong>Climate change is expected to significantly impact child nutrition, worsening global health inequities. Indonesia, a country highly vulnerable to climate change, also faces substantial child malnutrition challenges. However, comprehensive knowledge on climate change's impacts on child nutrition in Indonesia is limited. This study addresses this gap through a scoping review of the scientific evidence on the effects of climate change on child nutrition in Indonesia.</p><p><strong>Methods: </strong>We developed a conceptual framework based on global literature to guide our systematic search, linking climate change to child nutrition and its determinants in Indonesia. Systematic searches were conducted in English and Indonesian on Scopus, Web of Science and PubMed, supplemented by Google Scholar and citation screening. We included peer-reviewed, Scopus-indexed studies focused on Indonesia, examining either direct or indirect impacts of climate change on child nutrition. A narrative synthesis was performed, structured around outcomes identified in our framework: (1) nutrition-associated conditions, (2) diets and disease, (3) social dynamics and (4) food system shocks.</p><p><strong>Results: </strong>From 3025 records, 134 studies met the inclusion criteria. Studies were either multicountry including Indonesia (23%, n=31), Indonesia-specific across multiple regions (26%, n=35) or region-specific, mainly focused on Java (22%, n=29), Sumatra (11%, n=14), Kalimantan (7%, n=9) and Sulawesi (7%, n=9). Other regions were under-represented (5%, n=7). Most studies used quantitative methods (87%, n=116). Few studies assessed direct links between climate change and nutritional outcomes (n=5), food security or dietary quality (n=7); more focused on indirect pathways such as disease (n=49), social dynamics (n=18) and food system disruptions (n=55).</p><p><strong>Conclusions: </strong>Evidence suggests significant impacts of climate change on child nutrition in Indonesia, highlighting the need for urgent action. Further localised studies that consider contextual factors, and actions focused on strengthening health and nutrition systems, are critical, especially in regions most vulnerable to both climate change and child malnutrition.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11927459/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143655951","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Correction for 'Factors influencing the natural history of non-IgE-mediated gastrointestinal food allergies in paediatric age: a prospective multicentre cohort study'. 儿科非 IgE 介导的胃肠道食物过敏自然史的影响因素:前瞻性多中心队列研究 "的更正。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-15 DOI: 10.1136/bmjpo-2024-003203corr1
{"title":"Correction for '<i>Factors influencing the natural history of non-IgE-mediated gastrointestinal food allergies in paediatric age: a prospective multicentre cohort study'</i>.","authors":"","doi":"10.1136/bmjpo-2024-003203corr1","DOIUrl":"10.1136/bmjpo-2024-003203corr1","url":null,"abstract":"","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11911687/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143639568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparing different administration methods of subanaesthetic propofol to mitigate emergence agitation in preschool children undergoing day surgery: a double-blind, randomised controlled study. 比较亚麻醉异丙酚不同给药方法减轻学龄前儿童日间手术出现躁动:一项双盲,随机对照研究。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-15 DOI: 10.1136/bmjpo-2023-002376
Jiaxiang Chen, Xiaoli Shi, Wei Hu, Rongmu Lin, Ligang Meng, Changsheng Liang, Xinggang Ma, Liang Xu
{"title":"Comparing different administration methods of subanaesthetic propofol to mitigate emergence agitation in preschool children undergoing day surgery: a double-blind, randomised controlled study.","authors":"Jiaxiang Chen, Xiaoli Shi, Wei Hu, Rongmu Lin, Ligang Meng, Changsheng Liang, Xinggang Ma, Liang Xu","doi":"10.1136/bmjpo-2023-002376","DOIUrl":"10.1136/bmjpo-2023-002376","url":null,"abstract":"<p><strong>Background: </strong>Preschool children who received sevoflurane anaesthesia were associated with a high incidence of emergence agitation (EA). Studies have shown that a subanaesthetic dose of propofol (1 mg/kg) at the end of inhalational anaesthesia could reduce EA in paediatric patients, but the optimal administrations are still under investigation.</p><p><strong>Methods: </strong>In a double-blind trial, 160 preschool children (ASA I or II, 2-5 years old) undergoing day surgery of laparoscopic inguinal hernia repair with sevoflurane anaesthesia were randomly assigned into four groups: the control group, single bolus 3 min before the end of the surgery (bolus A), single bolus at the end of the surgery (bolus B) and continuous infusion for 3 min at the end of the surgery (continuous infusion). The dose of propofol in the bolus A group, bolus B group and continued infusion group is 1 mg/kg. The primary outcomes were the incidence and severity of EA assessed by the Paediatric Anaesthesia Emergence Delirium (PAED) scale and Watcha scales. The secondary outcomes included extubation time, emergence time, mean arterial pressure and heart rate.</p><p><strong>Results: </strong>The incidence of EA was as follows: 65.0% in the control group, 30.0% in the bolus A group, 32.5% in the bolus B group and 5.0% in the continuous infusion group (p<0.05). Furthermore, the peak PAED scores in the continuous infusion group were significantly lower than those in the other groups. However, extubation time and emergence time showed no differences among groups.</p><p><strong>Conclusions: </strong>Continuous infusion of subanaesthetic dose propofol (1 mg/kg) for 3 min at the end of sevoflurane anaesthesia seems to be more appropriate than other administration as it reduced EA and did not prolong the time to wake.</p><p><strong>Trail registration number: </strong>NCT05420402.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11911689/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143639460","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Predictors of successful Picture Exchange Communication System training in children with communication impairments: insights from a real-world intervention in a resource-limited setting. 对有沟通障碍的儿童进行成功的图片交换沟通系统训练的预测因素:来自资源有限环境下真实世界干预的见解。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-13 DOI: 10.1136/bmjpo-2024-003282
Prakasit Wannapaschaiyong, Thitaporn Vivattanasinchai, Awassada Wongkwanmuang
{"title":"Predictors of successful Picture Exchange Communication System training in children with communication impairments: insights from a real-world intervention in a resource-limited setting.","authors":"Prakasit Wannapaschaiyong, Thitaporn Vivattanasinchai, Awassada Wongkwanmuang","doi":"10.1136/bmjpo-2024-003282","DOIUrl":"10.1136/bmjpo-2024-003282","url":null,"abstract":"<p><strong>Background: </strong>Children with communication impairments-such as autism spectrum disorder or global developmental delay-face significant challenges affecting their emotional and behavioural development. The Picture Exchange Communication System (PECS) is an augmentative communication tool designed to enhance their skills. However, its effectiveness can vary in resource-limited settings. This study aimed to identify predictors of successful PECS training among children with communication impairments in such environments.</p><p><strong>Methods: </strong>This retrospective study analysed records of 61 children with communication impairments who underwent PECS training at Siriraj Hospital in Bangkok, Thailand, from 2020 to 2023. Success was defined as achieving PECS phase 3 proficiency and a Clinical Global Impression-Improvement score of 1-3 after 1 year. Logistic regression identified predictors of successful outcomes based on demographic, clinical, family and training-related factors.</p><p><strong>Results: </strong>After 1 year, 46% (28 out of 61) of the children achieved successful PECS outcomes. Significant predictors of success were lower severity of communication impairment (Clinical Global Impression-Severity ≤4; adjusted OR= 15.24, p = 0.002), higher frequency of PECS sessions (>6 times per year; OR = 9.11, p = 0.010), higher family income (≥20,000 baht per month; OR = 9.83, p = 0.024) and frequent home practice (≥3 times per week; OR = 7.02, p = 0.066).</p><p><strong>Conclusions: </strong>In resource-limited settings, factors such as severity of impairment, intensity of intervention, socioeconomic status and caregiver involvement significantly influence the success of PECS training. Tailored interventions and strategic resource allocation are crucial to optimise communication outcomes for these children.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11907048/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143630012","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Artificial intelligence for weight estimation in paediatric emergency care. 人工智能在儿科急救中的体重估计。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-12 DOI: 10.1136/bmjpo-2024-002891
Iraia Isasi, Elisabete Aramendi, Erik Alonso, Sendoa Ballesteros-Peña
{"title":"Artificial intelligence for weight estimation in paediatric emergency care.","authors":"Iraia Isasi, Elisabete Aramendi, Erik Alonso, Sendoa Ballesteros-Peña","doi":"10.1136/bmjpo-2024-002891","DOIUrl":"10.1136/bmjpo-2024-002891","url":null,"abstract":"<p><strong>Objective: </strong>To develop and validate a paediatric weight estimation model adapted to the characteristics of the Spanish population as an alternative to currently extended methods.</p><p><strong>Methods: </strong>Anthropometric data in a cohort of 11 287 children were used to develop machine learning models to predict weight using height and the body mass index (BMI) quartile (as surrogate for body habitus (BH)). The models were later validated in an independent cohort of 780 children admitted to paediatric emergencies in two other hospitals. The proportion of patients with a given absolute percent error (APE) was calculated for various APE thresholds and compared with the available weight estimation methods to date. The concordance between the BMI-based BH and the visual assessment was evaluated, and the effect of the visual estimation of the BH was assessed in the performance of the model.</p><p><strong>Results: </strong>The machine learning model with the highest accuracy was selected as the final algorithm. The model estimates weight from the child's height and BH (under-, normal- and overweight) based on a support vector machine with a Gaussian-kernel (SVM-G). The model presented an APE<i><</i>10% and <i><</i>20% for 74.7% and 96.7% of the children, outperforming other available predictive formulas by 3.2-37.5% and 1.3-29.1%, respectively. Low concordance was observed between the theoretical and visually assessed BH in 36.7% of the children, showing larger errors in children under 2 years.</p><p><strong>Conclusions: </strong>The proposed SVM-G is a valid and safe tool to estimate weight in paediatric emergencies, more accurate than other local and global proposals.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11907021/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143613221","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Identification of distinct clinical profiles of sepsis risk in paediatric emergency department patients using Bayesian profile regression. 使用贝叶斯特征回归识别儿科急诊科患者脓毒症风险的不同临床特征
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-12 DOI: 10.1136/bmjpo-2024-003100
Patricia Gilholm, Sainath Raman, Adam Irwin, Paula Lister, Amanda Harley, Luregn J Schlapbach, Kristen S Gibbons
{"title":"Identification of distinct clinical profiles of sepsis risk in paediatric emergency department patients using Bayesian profile regression.","authors":"Patricia Gilholm, Sainath Raman, Adam Irwin, Paula Lister, Amanda Harley, Luregn J Schlapbach, Kristen S Gibbons","doi":"10.1136/bmjpo-2024-003100","DOIUrl":"10.1136/bmjpo-2024-003100","url":null,"abstract":"<p><strong>Background: </strong>Sepsis affects 25 million children and neonates annually, causing significant mortality and morbidity. Early identification and treatment are crucial for improving outcomes. Identifying children at risk is challenging due to clinical heterogeneity and overlap with other conditions. Current evaluations of sepsis criteria adopt a variable-centred approach, evaluating each criterion independently. The objective of this study was to explore associations between patterns of sepsis screening criteria and sepsis risk in children screened in the emergency department (ED) to identify distinct profiles that describe the clinical heterogeneity of suspected sepsis.</p><p><strong>Methods: </strong>This secondary analysis involved 3473 children screened for sepsis across 12 EDs in Queensland, Australia. Bayesian profile regression was used to construct data-driven clinical profiles derived from sepsis screening criteria and their association with suspected sepsis, defined as senior medical officer diagnosis and antibiotic administration in the ED. Posterior risk probabilities (Prs) with 95% credible intervals (CIs) were calculated for each profile. Profiles were internally validated by assessing their association with sepsis, septic shock, organ dysfunction and infection sources, in both adjusted and unadjusted models.</p><p><strong>Results: </strong>Seven distinct clinical profiles were identified. Two profiles were labelled as high risk of suspected sepsis (profile 1, n=22: Pr 0.73, 95% CI 0.55, 0.89; profile 2, n=150: Pr 0.69, 95% CI 0.59, 0.80), four as moderate risk and one as low risk. High-risk profiles were characterised by severe illness indicators and elevated lactate levels. Moderate-risk profiles included criteria such as altered behaviour, young age (<3 months) and respiratory distress. High-risk profiles had strong associations with all clinical outcomes.</p><p><strong>Conclusions: </strong>Seven clinical profiles were identified that varied in their risk of suspected sepsis and associated outcomes. Validation of these profiles in diverse populations and identification of which profiles are likely to benefit from certain interventions is needed.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906992/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143613238","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Evaluation of the Patient-Specific Functional Scale for monitoring paediatric injury patients at a zonal referral hospital in Northern Tanzania. 评估在坦桑尼亚北部地区转诊医院监测儿科损伤患者的患者特异性功能量表。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-12 DOI: 10.1136/bmjpo-2025-003348
Baraka Moshi, Michael J Mahande, Anna Tupetz, Elizabeth M Keating, João Ricardo Nickenig Vissoci, Winifrida C Mwita, William Nkenguye, Kajsa Vlasic, Francis Musa Sakita, Frida Shayo, Emily R Smith, Catherine A Staton, Blandina T Mmbaga, Haleluya Moshi
{"title":"Evaluation of the Patient-Specific Functional Scale for monitoring paediatric injury patients at a zonal referral hospital in Northern Tanzania.","authors":"Baraka Moshi, Michael J Mahande, Anna Tupetz, Elizabeth M Keating, João Ricardo Nickenig Vissoci, Winifrida C Mwita, William Nkenguye, Kajsa Vlasic, Francis Musa Sakita, Frida Shayo, Emily R Smith, Catherine A Staton, Blandina T Mmbaga, Haleluya Moshi","doi":"10.1136/bmjpo-2025-003348","DOIUrl":"10.1136/bmjpo-2025-003348","url":null,"abstract":"<p><strong>Background: </strong>Injuries are a major cause of morbidity and mortality among paediatric populations in low- and middle-income countries (LMICs). The Patient-Specific Functional Scale (PSFS) is a commonly used tool to assess functional recovery. This study aims to evaluate the psychometric properties of the PSFS for monitoring paediatric injury patients at a zonal referral hospital in Northern Tanzania.</p><p><strong>Methods: </strong>This retrospective cohort study used data from the Kilimanjaro Christian Medical Centre paediatric injury registry (November 2020 to June 2024) and included patients under 18 years treated for injuries at a zonal referral hospital in Northern Tanzania. Key outcomes were in-hospital mortality and injury-related morbidity, assessed using the PSFS and Glasgow Outcome Scale Extended Paediatric (GOS-E Ped). The PSFS's reliability was tested with Cronbach's alpha, its smallest meaningful change was calculated and its correlation with GOS-E Ped was analysed using Spearman's rank.</p><p><strong>Results: </strong>Among 1000 paediatric injury patients, the mortality rate was 6.6%. PSFS mean scores improved from 4.3 at discharge to 6.5 at 2 weeks and 9.0 at 3 months post hospital discharge. The PSFS showed good reliability (Cronbach's alpha: 0.90). A moderate negative correlation was found between PSFS and GOS-E Ped at 3 months (Spearman's ρ: -0.74). The minimally clinically important difference was 2.7, with a sensitivity of 0.73, specificity of 0.72 and an area under the curve of 0.83.</p><p><strong>Conclusion: </strong>The PSFS was found to be a valid, reliable and responsive tool for assessing functional changes in paediatric injury patients, demonstrating strong internal consistency. The findings support its use to measure morbidity in this population.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-12","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11906990/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143613234","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Disease burden and health-related quality of life among children with X-linked hypophosphataemia in China: a national cross-sectional survey. 中国x连锁低磷血症儿童的疾病负担和健康相关生活质量:一项全国性横断面调查
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-06 DOI: 10.1136/bmjpo-2024-003165
Lin Zhu, Zhenqing Tang, Jiahao Hu, Dingguo Li, Aihua Zhang, Ying Chen, Li Liu, Feihong Luo, Qi Kang, Chunlin Jin
{"title":"Disease burden and health-related quality of life among children with X-linked hypophosphataemia in China: a national cross-sectional survey.","authors":"Lin Zhu, Zhenqing Tang, Jiahao Hu, Dingguo Li, Aihua Zhang, Ying Chen, Li Liu, Feihong Luo, Qi Kang, Chunlin Jin","doi":"10.1136/bmjpo-2024-003165","DOIUrl":"10.1136/bmjpo-2024-003165","url":null,"abstract":"<p><strong>Background: </strong>X-linked hypophosphataemia (XLH) is a rare inherited disorder often misdiagnosed and lacking sufficient aetiological treatment. Previous studies have shown that XLH is associated with worse health-related quality of life (HRQoL) and greater economic burden for children and their families compared with the general population, but evidence from China is scarce. This study aimed to comprehensively explore the burden and HRQoL of XLH children in China.</p><p><strong>Methods: </strong>An online retrospective survey of paediatric patients with XLH and their caregivers was conducted nationwide during March to June 2021. A self-administered questionnaire was used to collect socio-demographic, clinical and economic data. The EQ-5D-Y-3L instrument was employed to assess HRQoL, and the health utility score was calculated. Direct medical, non-medical and indirect costs were determined. Multivariate regression analysis was performed to explore potential associations between HRQoL and identified influencing factors.</p><p><strong>Results: </strong>The study included 221 subjects with a mean age of 7.25 years, of whom 119 (53.8%) were girls. Most XLH children (63.8%) lived in rural areas, and 39.4% reported a family history. Over 70% experienced misdiagnosis. The average total annual cost per patient was found to be 34 657.85 CNY. Of direct medical costs, patients' out-of-pocket expenses were substantial. The incidence of catastrophic health expenditure was 19.9%. The means (SD) of EQ-5D-Y-3L health utility and EQ-5D VAS scores were 0.83 (0.14) and 56.07 (10.95). Both univariate and multivariate analyses found older age and lower economic affordability were associated with poorer HRQoL.</p><p><strong>Conclusions: </strong>The study underscores the significant burden of XLH on paediatric patients and their families in China, both in terms of HRQoL and economic costs. The findings emphasise the importance of early detection, accurate diagnosis, cost-effective targeted interventions and long-term multidisciplinary management strategies to improve the lives of XLH children and their families.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11887277/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572001","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Influence of time of birth in early neonatal mortality and morbidity: retrospective cohort study. 出生时间对早期新生儿死亡率和发病率的影响:回顾性队列研究。
IF 2 4区 医学
BMJ Paediatrics Open Pub Date : 2025-03-06 DOI: 10.1136/bmjpo-2024-003236
Ginna Cabra-Bautista, Andres F Pérez, Gissel V Ruiz, Daniel Camilo Aguirre-Acevedo, Ivan D Florez, Jose A Calvache
{"title":"Influence of time of birth in early neonatal mortality and morbidity: retrospective cohort study.","authors":"Ginna Cabra-Bautista, Andres F Pérez, Gissel V Ruiz, Daniel Camilo Aguirre-Acevedo, Ivan D Florez, Jose A Calvache","doi":"10.1136/bmjpo-2024-003236","DOIUrl":"10.1136/bmjpo-2024-003236","url":null,"abstract":"<p><strong>Background: </strong>A key target of the 2030 Sustainable Development Goals is to eliminate preventable deaths in newborns and children under 5. This study aimed to estimate the effect of time of birth on early neonatal mortality (ENM) and low Apgar scores at 5 min (LA5) in newborns.</p><p><strong>Methods: </strong>A retrospective cohort study was conducted using vital statistics data on live births, maternal morbidity, congenital defects and perinatal mortality in Cauca-Colombia (2017-2021) excluding out-of-hospital, multiple and major defect cases. A directed acyclic graph was constructed to define the confounder adjustment set. Multivariable logistic, linear and propensity score models evaluated the effect of birth timing on neonatal outcomes, estimating crude and adjusted incidence rate ratios (IRRa).</p><p><strong>Results: </strong>We assessed 65 182 live births, finding similar baseline characteristics for daytime and night-time births. ENM was 0.2% (95% CI 0.19% to 0.26%) at 7 days of follow-up, absolute mortality difference 0.1% (95% CI -0.01% to 0.12%). Night-time births increased the incidence of ENM in the primary analysis IRRa 1.27 (95% CI 0.90 to 1.82), in the secondary IRRa 1.45 (95% CI 0.94 to 2.20), and in the primary and secondary sensitivity analysis, respectively, IRRa 1.48 (95% CI 1.06 to 2.07) and 1.70 (95% CI 1.16 to 2.59). LA5 was present in 0.7% (95% CI 0.60% to 0.72%) of birth, with absolute LA5 difference 0.1% (95% CI -0.02% to 0.22%). Night-time births increased the incidence of LA5 in the primary analysis IRRa 1.31 (95% CI 1.00 to 1.49), in the secondary IRRa 1.44 (95% CI 1.13 to 1.83), and in the primary and secondary sensitivity analysis, respectively, IRRa 1.31 (95% CI 1.08 to 1.59) and IRRa 1.54 (95% CI 1.23 to 1.92).</p><p><strong>Conclusions: </strong>Birth at night-time is associated with worse neonatal outcomes, ENM and low Apgar scores in Colombia's diverse population, highlighting the need for optimised prenatal care, revised work schedules and improved referral systems in maternal health.</p>","PeriodicalId":9069,"journal":{"name":"BMJ Paediatrics Open","volume":"9 1","pages":""},"PeriodicalIF":2.0,"publicationDate":"2025-03-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11887291/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143572002","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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