Research Appraisals
Evidence-based critical appraisals of the latest medical research, systematically evaluated using Oxford CEBM methodology.
Showing 13 appraisals
Journal of pediatric endocrinology & metabolism : JPEM
Beyond A1c: a narrative review of mental health integration in pediatric type 1 diabetes care
Background Youth with type 1 diabetes (T1D) experience substantially higher rates of depression, anxiety, diabetes distress, attention-deficit/hyperactivity disorder (ADHD), and disordered eating behaviors (DEB) than peers. These conditions may impair self-management and contribute to worsened glycemic outcomes.ContentWe synthesize prevalence and consequences of common comorbidities; summarize International Society for Pediatric and Adolescent Diabetes (ISPAD) and American Diabetes Association (ADA) guidance on psychosocial assessment; and appraise integrated models: co-located behavioral health, stepped care, and telehealth integration. We map barriers (workforce, workflow, stigma, fragmented communication, digital inequities) to concrete actions.SummaryPsychological morbidity and glycemic outcomes may be connected through behavioral and neuroendocrine pathways and family dynamics. Programs embedding behavioral health in pediatric diabetes care facilitate routine psychosocial screening and are associated with improved engagement and quality of life, and in some models, improved glycemic outcomes.OutlookTo close the implementation gap, teams should standardize routine psychosocial screening, embed trained mental-health professionals, use stepped-care models matching interventions to symptom severity, track referral follow-through, and leverage hybrid telehealth. Policy levers include reimbursement for integrated visits and investment in the behavioral-health workforce. The consistent associations between psychological and glycemic outcomes in youth with T1D support integrating psychosocial care as a core component of comprehensive diabetes management.
25 July 2026
Read appraisal →Child's nervous system : ChNS : official journal of the International Society for Pediatric Neurosurgery
A systematic review of long-term outcomes (>10-year follow-up) of paediatric craniopharyngioma
PURPOSE: Adamantinomatous craniopharyngiomas (ACP) in children are rare benign tumours. Relatively few publications have addressed the long-term impact of this tumour and its treatment. We undertake a systemic review of the existing literature aiming to define the outcome of ACP beyond 10 years' follow-up. METHODS: Ovid MEDLINE, Embase and Google Scholar were used to search the literature. The search criteria were children (aged under 18 at diagnosis), any intervention for and outcomes of ACP. Publications with less than 10 years' follow-up and single case reports were excluded. RESULTS: Twenty-one studies were included reporting on 1152 children, with a mean age of 8.8 years at diagnosis. Mean follow-up was 14 years. The most common presenting symptom was headache. The mean overall survival at 5, 10, 15 and 20 years post-diagnosis was 91.6%, 84.4%, 77.5% and 68.0%, respectively. In children undergoing gross total resection only or radiotherapy only, survival was higher than with combined treatments. Seventy-five percent of children had endocrine dysfunction after intervention; the most prevalent was hypothyroidism (81.9%). The prevalence of endocrinopathies was similar across all treatment groups. Lower QoL was reported than the general population, predominantly related to the effects of hypothalamic injury. One in ten patients suffered a late complication related to radiotherapy with vasculopathy being the most prevalent. CONCLUSIONS: The long-term morbidity and mortality for paediatric ACP remain high. The impact from hypothalamic injury cannot be understated and is likely the cause for this finding. As surgical treatment advances, less hypothalamic injury may arise and improved outcomes may follow. Gross total resection, if felt achievable without hypothalamic injury, should form the mainstay of treatment due to the late radiotherapy-related complications that arise.
10 July 2026
Read appraisal →The Journal of clinical endocrinology and metabolism
Diabetes technology: an update.
Diabetes is one of the most prevalent chronic diseases worldwide, with rates that continue to increase. More than 30 years ago, the Diabetes Control and Complications Trial demonstrated that intensive glucose management decreased long-term vascular complications. Unfortunately, many people with diabetes still struggle to meet glycemic goals. In this mini-review, we highlight advances in diabetes technology that are associated with improvements in glycemic management. Continuous glucose monitoring (CGM) and automated insulin delivery systems are associated with significant improvements in glycated hemoglobin (HbA1c), time in range (70-180 mg/dL), and decreases in severe hypoglycemia and diabetic ketoacidosis in people with both type 1 and type 2 diabetes. Recent data show that these technologies improve outcomes early in the course of type 1 diabetes. CGM is also being explored as a tool to monitor progression through early-stage type 1 diabetes (2 antibodies positive) to identify individuals who may benefit from disease-modifying therapies as well as to prevent the onset of diabetic ketoacidosis at onset of stage 3 (insulin-requiring) type 1 diabetes. Adjunctive pharmacologic therapies and artificial intelligence may further expand and improve therapies, offering potential synergistic benefits. However, there continue to be significant disparities in access to diabetes technologies and access to insulin worldwide. This mini-review summarizes recently published data, highlights emerging applications, and underscores the need to pair technological innovation with strategies that promote equitable access and support for diabetes care to improve outcomes for all people with diabetes.
19 June 2026
Read appraisal →Neurology
Vamorolone Safety, Pharmacokinetics, and Exploratory Efficacy in Duchenne Muscular Dystrophy: A Phase II, Nonrandomized, Multiple-Dose Study in 2-<4-Year-Old Boys
BACKGROUND AND OBJECTIVES: Vamorolone is a dissociative corticosteroid (CS) approved by the US Food and Drug Administration in 2023 for treating Duchenne muscular dystrophy (DMD). This study evaluated the safety, tolerability, and pharmacokinetics (PK) of vamorolone in young boys with DMD; exploratory objectives included efficacy and patient-reported outcomes. METHODS: A 12-week, phase II, open-label, multiple-dose study (VBP15-006) enrolled CS-naive boys with DMD aged 2-<4 years in Canada. Participants received 2 or 6 mg/kg/d oral vamorolone. An ongoing expanded access protocol (EAP) provided longer-term data. Primary end points were the occurrence of treatment-emergent adverse events (TEAEs) and changes from baseline to week 12 in height, weight, and body mass index (BMI); other end points included PK and effects of vamorolone on muscle function. RESULTS: Twenty boys (mean age [SD] 3.4 [0.39] years) with similar baseline characteristics were enrolled; all completed VBP15-006 and 19 continued to receive vamorolone through EAP Canada. TEAEs were mostly mild, with no deaths, serious TEAEs, or TEAEs leading to drug discontinuation during VBP15-006. TEAEs were more frequent with 6 mg/kg/d vamorolone than with 2 mg/kg/d, with gastrointestinal disorders and infections/infestations, respectively, most reported. Vamorolone use was associated with morning serum cortisol reductions in all patients, more pronounced with 6 mg/kg/d. Stable growth trajectories were observed at both doses throughout VBP15-006 and the EAP Canada follow-up period, with a total median (Q1; Q3) exposure to vamorolone of 2 years (1.7; 2.3). By EAP Canada last visit, 8/19 patients had weight and BMI z-scores changes >0.5, suggestive of excessive weight gain over time. Vamorolone displayed a dose-dependent PK profile with rapid absorption and no accumulation. Dose-dependent improvements in motor function (measured by Bayley-III gross motor scaled score) were observed. Glucose metabolism and bone turnover biomarkers remained stable. Ease of administration was rated favorably. DISCUSSION: Vamorolone was well tolerated in 2-<4-year-old boys with DMD, with no new safety concerns identified. A dose-dependent PK profile was observed, consistent with previous studies. Exploratory evidence suggested dose-dependent improvements in gross motor function. These findings are consistent with potential therapeutic benefit of vamorolone for young boys with DMD. TRIAL REGISTRATION INFORMATION: ClinicalTrials.gov: NCT05185622 (VBP15-006; clinicaltrials.gov/study/NCT05185622), NCT03863119 (EAP; clinicaltrials.gov/study/NCT03863119). First submitted November 9, 2021. First patient enrolled: March 21, 2022. CLASSIFICATION OF EVIDENCE: The VBP15-006 study provides Class IV evidence that in boys with DMD aged 2-<4 years, 12-week treatment with vamorolone was not associated with serious adverse events or changes in weight, height, or BMI. Gastrointestinal events and infections were the most reported TEAEs.
9 June 2026
Read appraisal →BMJ paediatrics open
Impact of flash glucose monitoring versus capillary blood glucose monitoring on glycaemia in Indian youth with type 1 diabetes: a randomised crossover study
BACKGROUND: Data relating to flash glucose monitoring (FGM) use in low-resource settings are limited. Our study investigated the impact of FGM versus self-monitoring of blood glucose (SMBG) on glycaemia in Ahmedabad, India. METHODS: A randomised crossover study of FGM versus SMBG was undertaken. Participants aged 10-25 years (type 1 diabetes (T1D)>1 year) were randomised 1:1 to either Freestyle Libre 1 FGM use (intervention) for 6 months, followed by SMBG≥4 times/day (local care standard) for 6 months; or SMBG≥4 times/day, followed by FGM. After 12 months, everyone entered a 6-month observation period using the monitoring method choice. Insulin and dietary adjustments were undertaken as per local care standards. Glycated haemoglobin (HbA1c) was measured at 0, 3 and 6 months (period 1) and 9 and 12 months (period 2). Analyses included use of linear mixed-effects analysis of covariance, a paired Wilcoxon signed-rank test, and cumulative link mixed tests and model. RESULTS: Of 106 participants, 91 (86%) completed 12 months follow-up (47.3% female). At baseline, mean±SD age was 17.8±3.8 years, diabetes duration 9.9±5.0 years and HbA1c 8.6±1.9% (70±21 mmol/mol).HbA1c decreased when using FGM versus SMBG (difference -0.44% (4.8 mmol/mol); p=0.006), with greater reduction in those with higher baseline HbA1c (p=0.003). In the FGM→SMBG group, HbA1c initially decreased from 8.6±2.2% (70±24 mmol/mol) to 7.9±1.1% (62±12 mmol/mol) at 3 and 6 months (both p<0.05). After SMBG crossover, HbA1c was unchanged (7.9±1.1% (63±12 mmol/mol) at 9 months and 8.1±1.3% (65±14 mmol/mol) at 12 months). In the SMBG→FGM group, HbA1c was initially stable (8.6±1.4% (70±15 mmol/mol) at baseline; 8.8±1.7% (73±19 mmol/mol) at 6 months), but decreased after crossover to FGM (8.4±1.4% (68±15 mmol/mol) at 9 months; 8.3±0.9% (67±10 mmol/mol) at 12 months; p=0.023). CONCLUSION: This study provides evidence of improvement in glycaemic outcomes while using FGM in youth living with T1D in India. TRIAL REGISTRATION NUMBER: Clinical Trial Registry of India (CTRI/2022/02/020175).
1 June 2026
Read appraisal →Neurology
Vamorolone for Duchenne Muscular Dystrophy: A Cross-Trial Efficacy Comparison With Classic Corticosteroids From the FOR-DMD Trial
BACKGROUND AND OBJECTIVES: Vamorolone demonstrated similar efficacy for Duchenne muscular dystrophy (DMD) compared with prednisone in a 24-week exploratory analysis and may reduce key side effects compared with classic corticosteroids. In this study, we compare the efficacy and anthropometric effect of vamorolone 6 mg/kg/d with prednisone 0.75 mg/kg/d and deflazacort 0.9 mg/kg/d in steroid-naïve boys aged 4 to <7 years using data from 2 trials. METHODS: VISION-DMD was a phase 2b, 48-week randomized, double-blind trial assessing vamorolone 2 and 6 mg/kg/d vs placebo and prednisone 0.75 mg/kg/d to 24 weeks. Finding the Optimum Steroid Regimen for DMD (FOR-DMD) was a double-blind, parallel-group randomized trial comparing daily prednisone 0.75 mg/kg/d, daily deflazacort 0.9 mg/kg/d, and intermittent prednisone 0.75 mg/kg (10/10 days on/off). Entropy balancing generated weighted data for mixed model for repeated measures analyses that compared VISION-DMD vamorolone 6 mg/kg/d efficacy and anthropometric outcomes with FOR-DMD prednisone and deflazacort outcomes at 6 and 12 months. Inclusion criteria were boys with genetically confirmed DMD, age 4 to <7 years, ambulatory, and able to complete a time-to-stand (TTSTAND) assessment in <10 seconds at baseline. RESULTS: All interventions showed motor improvements relative to baseline at 6 and 12 months. Using the weighted cohorts and at 12 months, vamorolone 6 mg/kg/d (n = 28) had similar changes in TTSTAND velocity, the primary motor endpoint component in both trials, vs daily prednisone (n = 50) or deflazacort (n = 55; mean baseline age 5.42 years all groups), but with CIs overlapping minimal clinically important thresholds of 0.023 rises per second (TTSTAND velocity least squares mean [LSM] difference [95% CI]: vamorolone 6 mg/kg/d vs prednisone 0.75 mg/kg/d, 0.004 rises per second [-0.025 to 0.032] and vs deflazacort 0.9 mg/kg/d, 0.001 rises per second [-0.027 to 0.028]). Body mass index (BMI) Z-scores increased in all groups, greatest with vamorolone (12-month LSM difference [95% CI]: vamorolone vs prednisone 0.57 [0.24-0.90]; vamorolone vs deflazacort 0.29 [0.03-0.56]), whereas growth slowdown occurred in prednisone- and deflazacort-treated boys but not vamorolone (12-month LSM difference in height Z-scores [95% CI]: vamorolone vs prednisone 0.57 [0.24-0.90]; vamorolone vs deflazacort 0.72 [0.53-0.91]). DISCUSSION: Vamorolone demonstrated numerically similar TTSTAND velocity changes to prednisone and deflazacort at 1 year; however, interpretations of differences are limited by 95% CIs crossing minimally important difference thresholds. Further evidence of the growth-protective effect of vamorolone was observed; however, all treatments increased BMI. Vamorolone provides a linear growth-protective classic corticosteroid alternative. TRIAL REGISTRATION INFORMATION: NCT03439670; NCT01603407. CLASSIFICATION OF EVIDENCE: This Class III study did not definitively identify differences in efficacy between vamorolone and classic corticosteroids but found that vamorolone protects linear growth in boys with DMD.
30 May 2026
Read appraisal →European journal of pediatrics
Gut microbiota-modulating interventions in paediatric type 1 diabetes: a systematic review and meta-analysis
To evaluate the efficacy and safety of gut microbiota-modulating interventions in children and adolescents with type 1 diabetes. A systematic review and meta-analysis was conducted according to PRISMA 2020. PubMed/MEDLINE, Scopus, Web of Science, and CENTRAL were searched from inception to 28 February 2026 for randomised controlled trials of oral probiotics, prebiotics, or synbiotics in participants aged ≤ 19 years with type 1 diabetes. Primary outcomes were glycated haemoglobin, fasting blood glucose, total daily insulin dose, and C-peptide. Immune-inflammatory, microbiota-related, intestinal permeability, and safety outcomes were narratively synthesised. Twelve randomised controlled trials involving 808 paediatric participants were included. Interventions varied in formulation, dose, and duration. Pooled analyses showed significant reductions in glycated haemoglobin and fasting blood glucose, whereas no significant effects were found for total daily insulin dose or C-peptide. Immune-inflammatory, microbiota-related, and intestinal permeability outcomes suggested possible benefits, but reporting was inconsistent and quantitative pooling was not feasible. Overall, the evidence was limited by substantial clinical and methodological heterogeneity. Conclusion: Gut microbiota-modulating interventions may improve glycaemic control in children and adolescents with type 1 diabetes, particularly glycated haemoglobin and fasting blood glucose. However, current evidence is insufficient to support any specific formulation, dose, or duration, and larger well-designed paediatric trials are still needed. What is Known: • Gut microbiota-modulating interventions are biologically plausible adjunctive strategies in paediatric type 1 diabetes. • Earlier paediatric reviews included few randomised trials and reported inconsistent glycaemic findings. What is New: • This updated review included 12 randomised controlled trials involving 808 children and adolescents. • It adds a structured synthesis of immune-inflammatory, microbiota/barrier, metabolite-related and safety outcomes beyond glycaemic endpoints.
26 May 2026
Read appraisal →JMIR formative research
Complication Risk Classification in Children and Adolescents With Type 1 Diabetes: Interpretable Machine Learning Study Based on Saudi Clinical Guidelines
BACKGROUND: Complication risks in children and adolescents with type 1 diabetes (T1D) can lead to serious health outcomes if not detected early. Despite the availability of clinical data, there remains a gap in interpretable tools that support risk stratification in this age group, particularly in alignment with local clinical guidelines. OBJECTIVE: The purpose of this study is to develop a clinically interpretable model that classifies the risk levels of T1D complications-acute, chronic, and low-using real-world data and expert clinical rules derived from the Saudi Diabetes Clinical Practice Guidelines. METHODS: A pediatric T1D dataset comprising of 306 patients was preprocessed through structured cleaning and feature engineering. Risk labels were constructed using Saudi Diabetes Clinical Practice Guidelines-derived rules. Feature selection was performed using a hybrid approach that combined the SHAP (Shapley Additive Explanations) analysis with exhaustive feature selection. A decision tree model was trained and optimized via cross-validation, using the F1-score as the primary performance metric. RESULTS: The final model achieved a high mean F1-score of 0.9876 with a low variance of 0.0189, using only 5 clinical features: BMI, hypoglycemia, disease duration, hemoglobin A1c, and impaired glucose metabolism. These features were consistently ranked as the most influential. The resulting decision tree offered a transparent logic path, enhancing its clinical interpretability and usability. CONCLUSIONS: This study demonstrates that a simple and interpretable model, guided by national clinical guidelines, can effectively predict the risk levels of T1D complications in children and adolescents. Its strong performance, clarity, and reliance on a small number of clinically meaningful features make it a promising candidate for integration into clinical decision support systems. This supports a shift toward predictive and personalized diabetes care.
17 May 2026
Read appraisal →BMJ open
Regular human insulins versus rapid-acting insulin analogues in children and adolescents with type 1 diabetes: a systematic review with meta-analysis and trial sequential analysis
OBJECTIVES: To assess the beneficial and harmful effects of regular human insulins versus rapid-acting insulin analogues in children and adolescents with type 1 diabetes. DESIGN: Systematic review of randomised clinical trials with meta-analysis and trial sequential analysis. DATA SOURCES: CENTRAL, MEDLINE, Embase, LILACS and other sources from inception to 30 January 2026. STUDY SELECTION: Randomised clinical trials comparing regular human insulins versus rapid-acting insulin analogues (insulin aspart, lispro, glulisine) in children and adolescents with type 1 diabetes. ANALYSES: Data were analysed using meta-analysis and trial sequential analysis. Risk of bias was assessed using the Cochrane Risk of Bias tool, V.2, and the certainty of the evidence was assessed using the Grading of Recommendations Assessment, Development and Evaluation (GRADE) approach. PRIMARY OUTCOMES: Severe hypoglycaemia, ketoacidosis and serious adverse events. RESULTS: 10 trials randomising 1107 participants were included. The certainty of evidence was very low mainly due to high risk of bias and small sample sizes. Meta-analysis showed no evidence of a difference between regular human insulins and rapid-acting insulin analogues on severe hypoglycaemia (risk ratio (RR) 1.28, 95% CI 0.81 to 2.03; I2=0.0%; p=0.2851; nine trials), ketoacidosis (RR 0.88, 95% CI 0.26 to 2.93; I2=0.0%; p=0.8593; two trials) and serious adverse events (RR 1.00, 95% CI 0.44 to 2.25; I2=0.0%; p=0.9958; two trials). Trial sequential analysis showed that all meta-analyses of primary outcomes were underpowered. CONCLUSIONS: Current research shows no differential effects between regular human insulins and rapid-acting insulin analogues for children and adolescents with type 1 diabetes, but the evidence is very uncertain. PROSPERO REGISTRATION NUMBER: CRD42024508625.
16 May 2026
Read appraisal →Bone research
A phase 2 trial of burosumab for treatment of fibroblast growth factor-23-mediated hypophosphatemia in children and adults with fibrous dysplasia
Fibrous dysplasia (FD) is a rare disorder associated with fractures and deformities. FD lesions produce excess phosphaturic hormone fibroblast growth factor 23 (FGF23), leading to hyperphosphaturia in most patients, and hypophosphatemia in those with high FD burden. Skeletal complications are associated with both low-normophosphatemia and frank hypophosphatemia. Burosumab is approved for other forms of FGF23 excess, but there is little evidence to inform use in FD. A phase 2 study investigated the safety and efficacy of burosumab in patients with FD. The primary endpoint was the proportion of participants achieving phosphate levels within the mid to upper part of the normal range (age and sex-adjusted Z-score -1 to +2). 12 participants (7 children, 5 adults) received burosumab for 48 weeks. Median phosphate Z-score increased from -2.88 (1.65) to 0.22 (1.37), meeting the target in 100% of participants. Alkaline phosphatase levels were elevated at baseline in 8 participants [median 846 U/L (464)] and declined by 49% at week 48, representing a median decline of -364 (244.5) U/L. PROMIS questionnaires showed trends toward improvements in all domains in children; adult scores showed no identifiable trends. Two children experienced transformational mobility gains, including advancement from full-time wheelchair use to independent ambulation. Lesion biopsies showed no changes in cellularity or composition, and 18F-NaF PET/CT scans showed no changes in tracer uptake, suggesting burosumab did not adversely impact lesional activity. Adverse events were mild, and none resulted in treatment withdrawal. Burosumab treatment in patients with FD was well-tolerated, restored phosphate homeostasis, and reduced alkaline phosphatase levels. Burosumab has the potential to lead to functional improvements and ambulation gains in severely affected patients and is a valuable tool to reduce the impact of FD-related disability.
2 May 2026
Read appraisal →European journal of pediatrics
Efficacy and safety of vosoritide in children with achondroplasia: a systematic review and meta-analysis
UNLABELLED: Achondroplasia is caused by a gain-of-function mutation in the FGFR3 gene. Vosoritide activates the NPR-B receptor to inhibit the overactive FGFR3 signaling pathway. We aim to pool the efficacy and safety outcomes of vosoritide in children with genetically confirmed achondroplasia who are receiving the approved dose of 15 μg/kg/day. We searched five databases up to February 10, 2026. A systematic review and single-arm meta-analysis were conducted in accordance with the PRISMA guidelines. The primary outcomes were annualized growth velocity (AGV), height gain, and change in height Z-score. Three quality assessment tools were used to assess different study designs of the included studies. All analyses were conducted using OnlineMeta V1.1. Thirteen studies on vosoritide treatment in children with achondroplasia were included, comprising randomized controlled trials, cohort studies, case reports, and case series. A meta-analysis showed that vosoritide was associated with an AGV of 5.72 cm/year (95% CI: 5.51-5.94) at 12 months. The mean height Z-score improvement at 12 months after sensitivity analysis was 0.28 (95% CI: 0.16-0.4), with no significant difference between sexes. Overall, the most common adverse events were injection site reactions (51%) and gastrointestinal symptoms (50%). CONCLUSION: One-year treatment with vosoritide is associated with increased growth velocity, height gain, and a modest improvement in height Z-score, accompanied by a high incidence of mild to moderate adverse events. Larger, longer-term studies are necessary to confirm the treatment's safety and efficacy. WHAT IS KNOWN: • Achondroplasia is a skeletal dysplasia caused by a mutation in the FGFR3 gene, which leads to inhibit endochondral ossification and bone growth. • Vosoritide is a recombinant C-type natriuretic peptide analogue designed to counteract overactive FGFR3 signaling and was approved by the FDA and EMA. WHAT IS NEW: • One-year treatment with vosoritide in achondroplasia children is associated with increased growth velocity and improvement in height Z-score with reported minor adverse events.
26 Apr 2026
Read appraisal →Archives of disease in childhood. Fetal and neonatal edition
Cerebral fuels within the first week of life in very preterm infants: a cohort study
BACKGROUND: Ketones and lactate may contribute towards overall cerebral fuel availability in term infants, yet the availability of such cerebral fuels in very preterm infants is unclear. We undertook a prespecified substudy to explore ketone and lactate concentrations in the first week of life in infants recruited to the REACT trial (real-time continuous glucose monitoring in the newborn): an international multicentre randomised controlled trial of 182 very low birth weight infants investigating the use of continuous glucose monitoring in glycaemic care. METHODS: Ketone and lactate measurements were prospectively collected over the first week of life using the Nova Biomedical point-of-care meter. A longitudinal analysis was undertaken to explore lactate and ketone concentration trends across time and their relationships with blood glucose, baseline demographics, nutritional support and insulin treatment. RESULTS: Data were available for 168 infants (85 females) including 2902 blood glucose, 2084 ketone and 2017 lactate samples. The mean (SD) gestational age was 27.4 (2.0) weeks. Lactate concentrations were higher initially, with mean (SD) 1.72 (1.26) mmol/L on day 2 and lowered to 1.19 (1.1) mmol/L on day 7. Ketone concentrations remained consistently low at 0.1 mmol/L. Neither simultaneous blood glucose concentrations, macronutrient intake nor receipt of insulin was consistently related to ketone or lactate concentrations. CONCLUSION: In this cohort of very preterm infants, there were persistently low concentrations of ketones and relatively higher concentrations of lactate throughout the first week of life. Future research should evaluate changes in these metabolites during episodes of acute hypoglycaemia or hyperglycaemia over more prolonged periods of neonatal intensive care.
20 Apr 2026
Read appraisal →European journal of pediatrics
The impact of growth hormone therapy on ocular structural parameters in idiopathic short stature: a systematic review and meta-analysis
This study aimed to conduct a systematic review and meta-analysis of the effects of growth hormone (GH) therapy on ocular structures and biometric parameters in children with idiopathic short stature (ISS), synthesizing existing evidence for future research. We systematically searched PubMed, EMBASE, the Cochrane Library, CNKI, Wanfang, and VIP databases from inception to September 2025. Randomized controlled trials and observational studies comparing the effects of GH therapy versus control on ocular biometric and refractive outcomes in children with ISS were included. Study quality was assessed using the Cochrane Risk of Bias Tool (RoB 2) and the NOS. Meta-analysis was performed using R software version 4.3.2, with effect sizes expressed as MD or SMD and 95% CI. Nine studies were included (4 RCTs, 5 cohort studies). Meta-analysis showed no significant association of GH therapy with corneal curvature (SMD = 0.13, 95% CI: -0.34 to 0.61). For axial length, the random-effects model showed no significant difference (MD = 0.72, 95% CI: -0.36 to 1.81), with high heterogeneity (I2 = 91.7%). Similarly, no significant association was found for intraocular pressure (MD = 1.38, 95% CI: -0.66 to 3.43; I2 = 95.9%). However, a significant association was observed for refractive status (MD = 0.53, 95% CI: 0.20 to 0.86; I2 = 0.0%). Sensitivity analysis showed robust results for corneal curvature and refractive outcomes, while axial length and intraocular pressure results were influenced by individual studies. Funnel plots and Egger's test indicated no significant publication bias.Conclusion: The associations between GH therapy and ocular biometric parameters in children with ISS remain uncertain. Current evidence suggests a possible link with refractive changes but shows no significant associations with axial length or intraocular pressure when accounting for high heterogeneity. The findings are inconsistent and should be regarded as hypothesis-generating, insufficient to recommend changes in clinical monitoring practice. Future large-sample, long-term studies are required.
17 Apr 2026
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