BMJ Paediatrics Open最新文献

Background: Management of neuromuscular scoliosis (NMS) is challenging, with both surgical and conservative options involving risks. This study aimed to evaluate multimorbidity in patients with NMS and how this influences multidisciplinary team (MDT) decisions as well as postoperative outcomes.

Methods: A retrospective cohort study of patients referred for assessment by the scoliosis MDT in the 8-year period between 2013 and 2021 from a single tertiary centre.

Results: 84 patients with NMS were referred for assessment to the MDT. The most common underlying cause of NMS was cerebral palsy (51%). The MDT recommended surgery for 60 patients and 24 were conservatively managed. There were no significant differences in age, sex, body mass index or baseline Cobb angle between the two groups. Patients recommended surgery had fewer comorbidities (2.3 vs 3.5, p<0.05) and greater Cobb angle progression in the 18 months prior to MDT decision (22° vs 8°, p<0.05). No single comorbidity significantly influenced the MDT decision. Of the 48 patients that proceeded with surgery, immediate postoperative complications were documented in 54.1%, with no mortality. The most common complications were postoperative anaemia and respiratory infections. Multivariate logistic regression identified the use of non-invasive ventilation, forced vital capacity <70% of predicted and full-time wheelchair use as significant predictors of immediate postoperative complications. Improved posture was the most common long-term outcome (41.7%) and 81.3% of patients reported no complications at 12 months following their surgery.

Conclusions: Multimorbidity in children with NMS influences scoliosis MDT decisions, alongside factors such as scoliosis curve progression. Immediate postoperative complications were common but longer term outcomes were favourable for most patients. Further research aiming to better inform shared decision-making, improve surgical selection and ultimately enhance the quality of life for patients with NMS is required.

Introduction: Achalasia is a rare disease in children. Studies investigating the efficacy of interventions and disease outcomes in paediatric achalasia are predominantly retrospective, consist of small cohorts and report heterogeneous outcomes. The variation in the use and definition of reported outcomes impedes meta-analysis, which is problematic in a rare paediatric condition. Similarly, there is a risk of under-reporting patient-relevant outcomes, such as quality of life. To overcome these issues, a minimum set of important and patient-relevant outcomes should be reported in all studies of paediatric achalasia. Core outcome sets (COS) are a standardised set of outcomes that can guide further research and facilitate data pooling and meta-analysis. The development of a COS in rare paediatric disease is essential, prior to conducting efficacy studies or creating a disease registry, to ensure that the most important outcomes are reported. Currently, no COS exists for children with achalasia. In this study, we aim to define a COS for paediatric achalasia for use in clinical research.

Methods and analysis: This study will consist of three parts. The first will be a systematic review of the literature, evaluating the outcomes and outcome definitions reported in published clinical research studies investigating paediatric achalasia. Second, a three-stage Delphi consensus process will be undertaken to identify and prioritise outcomes. This process will involve healthcare professionals, patients and parent representatives. Third, a consensus meeting will be held, during which the final COS will be defined.

Dissemination: The results of this study will be disseminated to stakeholders via the European Reference Network for Rare Inherited Congenital Anomalies, European Society for Pediatric Gastroenterology Hepatology and Nutrition, European Paediatric Surgeons' Association, and patient groups. The COS will be published in a peer-reviewed journal and uploaded to the Core Outcome Measures in Effectiveness Trials (COMET) initiative website.

Trial registration number: The study was pre-registered with the COMET initiative in July 2024 (https://www.comet-initiative.org/Studies/Details/2568). The systematic review component of the study was pre-registered on PROSPERO (CRD42024509855).

Background: Paediatric sepsis remains a significant contributor to morbidity and mortality, particularly in low- and middle-income countries (LMICs), where healthcare resources are often limited. Paediatric sepsis bundles, which include prompt administration of antibiotics, fluid resuscitation and continuous organ function monitoring, are crucial for improving outcomes, especially when initiated within the first 'golden hour' of sepsis recognition. These bundles, adapted from adult sepsis care protocols through the Surviving Sepsis Campaign, are increasingly emphasised in global sepsis management guidelines. However, the implementation of these protocols in LMICs is challenged by resource limitations and systemic barriers.

Methods: This situational analysis, conducted at two hospitals in Ghana-a tertiary facility and a district (secondary) facility-maps the availability of critical resources for paediatric sepsis care through a structured environmental scan using survey methodology. We assess staffing levels, access to medications, airway support and diagnostic capabilities. Methods were conceptualised through inner and outer settings of the Consolidated Framework for Implementation Research (CFIR) and reported through the Donabedian model for healthcare quality.

Results: This study compared paediatric care at a tertiary hospital (Komfo Anokye Teaching Hosptial (KATH)) and a district hospital (Presbyterian Hospital, Agogo (PreHA)) in Ghana, highlighting KATH's emergency and intensive care unit (ICU) services, specialised staff and broader respiratory support. PreHA, although without a paediatric-specific ICU, leveraged research funding to enhance clinical care capacity. Both hospitals experienced regular power outages but had reliable generators, and while they offered basic medications and treatments, resource limitations, including out-of-pocket costs for families, impacted access to essential medications and laboratory tests.

Conclusion: Concerns around resource availability, compounded by structural determinants such as financial barriers and historical underfunding hypothesised to be rooted in colonialism, highlight the need for context-sensitive adaptations of paediatric sepsis bundles. Our findings underscore the importance of a participatory approach to guideline adaptation and resource distribution, incorporating local expertise and addressing structural inequities to improve paediatric sepsis outcomes in Ghana. Future qualitative research will explore pre- and peri-hospital barriers to care and inform more effective, contextually appropriate interventions.

Introduction: The safest oxygen levels needed for preterm infant respiratory support at birth are uncertain. We aimed to compare the outcomes of infants up to 28⁶ weeks gestation who had respiratory care initiated at birth with fractional inspired oxygen (FiO₂) 0.3 or 0.6, which was adjusted to meet specific oxygen saturations (SpO₂).

Methods: This randomised controlled phase III trial was stratified by (1) site, (2) gestation and (3) multiplicity. Infants between 23+0 to 28+6 weeks gestation were randomised to initial respiratory support with FiO₂ 0.3 or 0.6, adjusted to meet common SpO₂ targets for the first 10 min.

Primary outcome: Survival to 36 weeks gestation without documented brain injury.

Assessments: FiO₂, SpO₂ and heart rate were recorded each minute from delivery for 10 min. Assessments were obtained at baseline, 36 weeks, discharge and at 2 years corrected gestation, along with a parent questionnaire.

Statistical analysis plan: Assuming 32% of infants would die or survive with brain injury by 36 weeks, 735 infants per arm (1470 total) were needed to detect a risk difference of 8% (25% relative risk reduction), with 10% non-adherence to protocol, 85% β and 5% α.

Ethics: Approved by the John Hunter Human Research Ethics Committee (2019/ETH/3837) for waiver of consent for all Australian sites for randomised allocation and primary outcome.

Conclusion: Recruitment started in 2018 and was achieved on 30 September 2024. The Data and Safety Committee review found no major safety concerns at 50% recruitment.

Trial registration number: ACTRN 12618000879268.

Background: The prevalence of type 2 diabetes mellitus (T2DM) in children and young people (CYP) is increasing in the UK and worldwide. Little is known about the experience young people and their families have when attending for their diabetes care. The aim of this study is to analyse the responses to the Parent and Patient Reported Experience Measures (PREM) survey 2021-2022 for patients with T2DM and their families to inform care.

Methods: As part of the National Paediatric Diabetes Audit (NPDA) (2022), the NPDA PREM survey was open online between August 2021 and January 2022. The data have previously been analysed collectively for all diabetes; however, we specifically analysed the data for patients living with T2DM.

Results: 9.2% (105/1144) of young people living with T2DM in England and Wales responded to the NPDA PREM survey, mostly aged 12-16 years (61.9%) and the majority were female (67.6%). 87% of patients and 95% of carers said that they would recommend their diabetes team, and 73% of patients felt happy after appointments. Only just over half of the patients and parents felt well prepared for transferring to adult care. Only 38% of patients felt that their school or college often had the necessary information to support them with their diabetes.

Conclusions: This analysis describes the experience of CYP and their parents/carers of T2DM care and highlights areas for improvement. These findings may help to inform recommendations about the development of better patient-centred care for young people with T2DM.

Background: Despite oral rehydration salt (ORS) solution being a life-saving medication, mothers of affected children often lack proper knowledge and exhibit improper practice of using ORS in sachets. We aimed to assess maternal knowledge, attitude and practice towards commercially available ORS and its use in treating under-5 children with diarrhoea.

Methods: The study was conducted at the Dhaka Hospital, Bangladesh. We included 350 mothers of under-5 children suffering from diarrhoea. Data were collected using a pretested questionnaire and modified Bloom's cut-off was used to determine adequate knowledge (≥80%), positive attitude (≥90%) and proper practice (≥75%). Logistic regression models were developed after adjusting for confounding variables. Adjusted ORs (AORs) and their 95% CIs were reported.

Results: The prevalence of inadequate knowledge, negative attitude and improper practice was 88.0%, 59.1% and 72.5%, respectively. Participants with up to the higher secondary level of education had significantly higher odds of possessing adequate knowledge (AOR 7.47; 95% CI 2.76 to 20.12) and following proper practice (AOR 3.04; 95% CI 1.66 to 5.77). The majority (97.4%) reported being aware of the process of ORS preparation. Only 2.9% correctly knew all recommended steps. Approximately half (51.1%) knew the purpose of ORS use, and 24.6% believed that antibiotics are more helpful than ORS.

Conclusions: The findings of the study emphasise the need for substantial improvements to the existing health education program with a focus on parental education. Emphasis should be given to reading instruction on the sachet and promoting access to information via mass media outlets.

Background: We aimed at identifying the factors influencing the natural history of non-IgE-mediated gastrointestinal food allergies (non-IgE-GIFA), a group of common paediatric conditions including food protein-induced: enteropathy (FPE), allergic proctocolitis (FPIAP), enterocolitis syndrome (FPIES), and motility disorders (FPIMD).

Methods: Prospective multicentre cohort study involving paediatric patients (both sexes, aged ≤14 y) with non-IgE-GIFA diagnosed and followed for 24 months at a Tertiary Centre for Paediatric Allergy, Gastroenterology and Nutrition. Anamnestic and clinical data were collected from all enrolled patients.

Results: 123 non-IgE-GIFA patients were enrolled (56% male, median age (IQR) 150 (60-300) days): FPE (39%), FPIES (17%), FPIAP (16%) and FPIMD (28%). 42% of patients had multiple food allergies (FAs) at baseline, and 64% had a positive family history of allergy. Male sex (OR = 2.24, 95% CI 1.07 to 4.71) and every 1 month of diagnostic delay (OR=1.09, 95% CI 1.01 to 1.18) were positively associated with the occurrence of multiple FAs. At 24-month follow-up, 54% of patients acquired immune tolerance. This rate was higher in FPIAP (75%), when compared with FPIMD (62%), FPE (54%) and FPIES (24%). The odds of 24-month immune tolerance acquisition rate was lower in children with family history of allergy (OR=0.41, 95% CI 0.19 to 0.89) and in those with multiple FAs at baseline (OR=0.24, 95% CI 0.11 to 0.51). At 24-month follow-up, the rate of patients with allergic march was 0.46 (95% CI 0.38 to 0.55, n=57/123), without differences comparing the four phenotypes. The presence of multiple FAs at baseline was associated with an increased risk of developing allergic march (OR=2.22, 95% CI 1.07 to 4.61) at 24-month follow-up.

Conclusions: The results of the study suggest the potential role of modifiable and non-modifiable risk factors influencing the natural history of paediatric patients affected by non-IgE-GIFA.

Introduction: Air pollution is linked with poor neurodevelopment in high-income countries. Comparable data are scant for low-income countries, where exposures are higher. Longitudinal pregnancy cohort studies are optimal for individual exposure assessment during critical windows of brain development and examination of neurodevelopment. This study aims to determine the association between prenatal ambient air pollutant exposure and neurodevelopment in children aged 12, 24 and 36 months through a collaborative, capacity-enriching research partnership.

Methods and analysis: This observational cohort study is based in Nairobi, Kenya. Eligibility criteria are singleton pregnancy, no severe pregnancy complications and maternal age 18 to 40 years. At entry, mothers (n=400) are administered surveys to characterise air pollution exposures reflecting household features and occupational activities and provide blood (for lead analysis) and urine specimens (for polycyclic aromatic hydrocarbon (PAH) metabolites). Mothers attend up to two additional antenatal study visits, with urine collection, and infants are followed through age 36 months for annual neurodevelopment and caregiving behaviour assessment, and child urine and blood collection. Primary outcomes are child motor skills, language and cognition at 12, 24 and 36 months, and executive function at 36 months. The primary exposure is urinary PAH metabolite concentrations. Additional exposure assessment in a subset of the cohort includes residential indoor and outdoor air monitoring for fine particulate matter (PM2.5), carbon monoxide (CO), ultrafine particles (UFP) and black carbon (BC).

Ethics and dissemination: This study was approved by the Kenyatta National Hospital - University of Nairobi Ethics and Research Committee, and the University of Washington Human Subjects Division. Results are shared at annual workshops.

Objective: To assess the relationship between plasma ferritin concentrations and (1) antenatal factors and (2) requirement for red cell transfusion.

Study design: This single-site prospective study recruited infants in the first week of life who were born <32 weeks' corrected gestational age and did not receive a red cell transfusion prior to sampling. Ferritin concentrations were assessed on discard plasma taken as part of routine neonatal investigations in the first week of life (median day of life=3, IQR 2-5 days). Reasons for delivery, placental histology and demographics were recorded.

Results: Plasma ferritin concentrations were not significantly associated with birth weight or gestational age in this cohort of extremely/very preterm neonates (n=114: n=26, <28 weeks' corrected gestational age; n=88, 28-32 weeks' corrected gestational age). Neonates exposed to chorioamnionitis had an increased ferritin concentration versus those who were not. Neonates exposed to pre-eclampsia had a significantly lower ferritin concentration than those who were not. Early ferritin concentration was not associated with time to transfusion in a time to event analysis.

Conclusions: Plasma ferritin concentrations in very/extremely preterm neonates are variable and associated with the intrauterine environment. Ferritin concentration was not predictive of time to transfusion in this cohort and was not significantly different at smaller birth weight or earlier gestation. This is important for considerations of iron storage in very preterm neonates and its developmental consequences.