Standard Deviation Score Research Articles

Emotional and Behavioural Adjustment in Children and Adolescents with Short Stature vs. Their Normal-Statured Peers

Background/Objectives: the aim of the current cross-sectional study is to explore and compare the emotional and behavioural conditions of children and adolescents with short stature (i.e., familial short stature and constitutional delay of growth), idiopathic growth hormone deficiency (GHD), and normal height. Methods: Twenty-nine participants (15 males, 14 females; mean age ± standard deviation (SD) = 11.2 ± 1.96 years) with short stature (height standard deviation score (SDS): −2.10 ± 0.57), 10 age-matched participants (4 males, 6 females; mean age ± SD = 10.9 ± 2.35 years) with growth hormone deficiency (GHD; height SDS: −2.44 ± 0.29), and 36 age-matched participants (19 males, 17 females; mean age ± SD = 11.3 ± 1.93 years) with normal stature (height SDS: 0.56 ± 0.78) were admitted to this study. Psychological distress was evaluated using the Depression Anxiety and Stress Scale (DASS-21), and emotional and behavioural problems using the Child Behaviour Checklist for Children (CBCL) and the Strengths and Difficulties Questionnaire (SDQ). Results: Participants with GHD exhibited higher levels of the “withdrawn/depressed subscale” score of CBCL (H = 7.794, df = 2, p = 0.020), compared to their peers with normal height, while no significant differences were observed between participants with short stature and those with normal stature. Normal-statured participants reported higher levels of the “conduct problems” subscale score (H = 10.421, df = 2, p = 0.005) and the “rule-breaking behaviour” subscale score of CBCL (H = 10.358, df = 2, p = 0.006) compared to both short-statured groups. No significant differences among the three subgroups were found in the DASS-21 and SDQ scores questionnaires, suggesting the lack of short stature-dependent effects on psychological distress and emotional and behavioural adjustment. Conclusions: Although participants with GHD exhibited higher levels of the “withdrawn/depressed subscale” score of CBCL (H = 7.794, df = 2, p = 0.020) compared to their peers with normal height, participants with short stature did not experience the same problems. Although short-statured participants had lower levels of “conduct problems” and “rule-breaking behaviours” scores than their normal-statured peers, the psychological distress and emotional and behavioural adjustment were not affected by short stature, being comparable to those recorded in normal-statured participants.

IGF-1 assessment during weekly somatrogon treatment in pediatric patients with growth hormone deficiency

Abstract Context In patients with growth hormone deficiency (GHD) receiving GH treatment, IGF-1 concentrations are used by physicians to monitor treatment safety and efficacy and guide dosing decisions. Somatrogon is a long-acting GH approved as a once-weekly treatment for pediatric GHD. Somatrogon administration results in characteristic changes in the IGF-1 profile, with values measured at 96 hours post-dose representing mean IGF-1 concentrations that best reflect overall somatrogon exposure. Objective To develop a simple method to enable physicians to predict mean IGF-1 concentrations following somatrogon dosing, based on a single IGF-1 measurement taken at any point during the 7-day dosing interval. Methods Data from phase 2 and phase 3 somatrogon studies were used to develop a 2-compartment pharmacokinetic model with delayed first-order absorption. An indirect-response pharmacokinetic/pharmacodynamic model was applied to the predicted somatrogon concentrations, and model simulations were used to predict IGF-1 and IGF-1 SD score (SDS) levels for participants in both studies. Results A total of 16,213 dosing records (from 42 and 109 participants in the phase 2 and 3 studies, respectively) were used for the simulations, generating predicted values for IGF-1 and IGF-1 SDS. Predicted values were scaled against the respective values at 96 hours (Day 4). These values were used to create a table showing the adjustments required to predict mean IGF-1 and IGF-1 SDS values depending on time after dose. Conclusion We developed a simple method enabling physicians to predict mean weekly IGF-1 values using IGF-1 values measured at any point in the dosing interval. Clinicaltrials.gov: NCT01592500, NCT02968004

Changes in body composition and muscle strength in girls with idiopathic central precocious puberty during gonadotropin-releasing hormone agonist therapy

Aims: Our objective is to explore changes in body fat distribution and muscle strength among a cohort of girls with idiopathic central precocious puberty (ICPP) undergoing the gonadotropin-releasing hormone analogs (GnRHa) therapy. Methods: A total of 50 patients who were newly diagnosed with ICPP and treated with GnRHa were included in the study. Patients were investigated at baseline, 6th months and 12th months. Results: Body-mass index (BMI) standard deviation score (SDS) was similar throughout the treatment duration. The percentage of body fat (PBF) increased from 24.2±5.1% at the beginning to 26.3±5.3% at the 6th month and to 27.7±5.43% at the 12th month (p

Novel Gel Formulation and Deep Injection Techniques for Lifting Effects in Cosmetic Dermatology.

In recent years, the field of aesthetic dermatology has witnessed a surge in demand for minimally invasive procedures aimed at rejuvenating aging skin. This study aims to address this demand by evaluating the effectiveness of the injectable gel in rejuvenating aging skin, particularly by targeting collagen regeneration and lifting effect. The study involved 43 participants who underwent three monthly injection sessions targeting retaining ligaments. The injections were administered deeply to ensure proper targeting. Follow-up assessments were conducted after each treatment session and three months after the final injection. Evaluation methods included subjective assessments by both patients and investigators using the Global Aesthetic Improvement Scale (GAIS), as well as objective assessments using a 3D photosystem to measure wrinkle conditions and vectors of traction. All participants completed the study, with no significant adverse effects observed apart from mild swelling at the injection sites. Despite the high viscosity of HA necessitating the use of a 27 G needle, the injection process was generally comfortable and minimally painful. Subjective evaluations revealed consistent improvements in skin appearance from the first application, which continued to increase throughout the study and remained high even 3 months post-treatment. Objective evaluations demonstrated significant improvements in wrinkle conditions and lifting effects, with a substantial increase in the standard deviation score for wrinkle conditions and the average traction vector length measuring 1.6 mm. The study findings confirm the safety and efficacy of the injectable formula, with high patient satisfaction, noticeable lifting effects, and significant improvements in wrinkle conditions. These results support the use of the injectable as a promising option for non-invasive skin rejuvenation treatments.

Longitudinal analysis of anthropometric measures over 5 years in patients with Friedreich ataxia in the EFACTS natural history study.

Friedreich ataxia is a rare neurodegenerative disorder caused by frataxin deficiency. Both underweight and overweight occur in mitochondrial disorders, each with adverse health outcomes. We investigated the longitudinal evolution of anthropometric abnormalities in Friedreich ataxia and the hypothesis that both weight loss and weight gain are associated with faster disease progression. Participants were drawn from the European Friedreich's Ataxia Consortium for Translational Studies (EFACTS). Age- and sex-specific BMI and height scores were calculated using the KIGGS-BMI percentiles for children. Height correction was applied for scoliosis. Longitudinal data were analysed using linear mixed effects models and incremental standard deviation scores and growth mixture models identified subclasses with varying BMI trajectories. Five hundred and forty-three adults and fifty-nine children were assessed for up to 5 years. In children, severe underweight (26%), underweight (7%), severe short stature (16%) and short stature (23%) were common. The corrected BMI percentile was stable in children, although 48% had negative incremental BMI scores over 1 year and 63% over 3 years versus 10%/year in a normal reference cohort. Overweight was common in adults (19%), with a slight increase in BMI over time. Longer GAA repeat size was linked to lower BMI in adults. Weight trajectory was not associated with ataxia progression in adults. Significant anthropometric abnormalities were identified, with underweight and short stature prevalent in children and overweight in adults. These findings highlight the need for regular nutritional monitoring and interventions to manage underweight in children and promote healthy weight in adults.

Age of menarche and final height in patients with permanent congenital hypothyroidism.

We compared the age at menarche and standard deviation score (SDS) of final height (FH) in permanent congenital hypothyroidism (CH) patients with those of healthy female adolescents and assessed their associations with CH screening-related variables or demographic factors. In this cross-sectional study, we included 207 female CH patients and 598 healthy age-matched female adolescents. Ages at puberty onset and menarche, height at puberty and menarche, and the FH and its SDS were evaluated in the 2 groups and compared. Associations between screening variables and anthropometric data with age at menarche and SDS of FH were also assessed in CH patients. In the included population, 113 patients with CH and 453 healthy girls attained their FH. The mean ages at puberty onset and menarche in CH patients were higher than those in the healthy population (P<0.05). The mean height at menarche and the FH and its SDS were not different between the 2 groups (P>0.05). There was no significant association between FH SDS in CH patients and age of treatment (P=0.30). Age at menarche was significantly higher in CH patients with delayed age at treatment initiation (P=0.04). The difference between FH and target height was not significantly different among CH patients (P=0.83). While CH patients had a significantly higher age at menarche compared to the healthy population, appropriate treatment changed this age to be similar to that in the healthy group. However, CH patients who experienced delayed treatment had a higher age at menarche. Age at treatment initiation was the only screening-related variable related to age at onset of menarche and puberty.

Pituitary abnormalities in patients with pediatric growth hormone deficiency in a single tertiary center.

There is controversy as to whether brain magnetic resonance imaging (MRI) should be performed on all children with growth hormone deficiency (GHD) including those judged to have mild GHD. This study was aimed to determine the frequency of pituitary or intracranial abnormalities in pediatric GHD and to identify risk factors that may predict pituitary or intracranial abnormalities. A total of 95 pediatric GHD patients were included. Their medical records and brain magnetic resonance (MR) images were reviewed retrospectively. Abnormal pathogenic MR images were found in 14 patients (14.7%), including 10 (10.5%) with pituitary hypoplasia and 4 (4.2%) with pituitary stalk interruption syndrome. Serum levels of insulin-like growth factor-I (IGF-I), IGF-I standard deviation score (SDS), insulin-like growth factor binding protein 3 (IGFBP3), and growth hormone (GH) peak level of GH stimulation test were statistically significantly lower in the group with abnormal brain MRI. The frequency of abnormal MRI was statistically significantly higher in the complete GHD group. IGF-1 SDS showed the highest area under the curve which can predict the presence of brain abnormality with a sensitivity of 85% and a specificity of 71.4%, if IGF-1 SDS was less than -1.365. IGF-1, IGFBP3, and GH peak levels also showed good sensitivity of over 80% for predicting brain abnormalities with cutoff values of 70.285 ng/mL, 1,604 ng/mL, and 4.205 ng/mL, respectively. The sensitivity and specificity of each cutoff value of IGF-1, IGF-1 SDS, IGFBP3, and GH peak levels were good and statistically significant in predicting brain MRI abnormalities. However, it was insufficient to predict all brain abnormalities with these variables. Therefore, we would like to recommend performing a brain MRI if a child is diagnosed with GHD.

EFFECTS OF PILATES EXERCISES ON BALANCE AND GAIT IN SCHOOL GOING CHILDREN

Background: Physical activity is a cornerstone of childhood development, contributing significantly to physical, mental, and social well-being. In the context of modern sedentary lifestyles, integrating structured exercise programs is essential for fostering motor skills, balance, and overall health in children. Pilates exercises, with their focus on core stability and neuromuscular control, are increasingly being recognized as an effective intervention to enhance motor function, particularly in improving balance and gait in school-aged children. Objective: To evaluate the effects of Pilates exercises combined with conventional exercises on balance and gait in school-going children. Methods: This randomized controlled trial included 96 school-going children aged 6–9 years, recruited using non-probability convenience sampling. Participants were randomly allocated into two groups of 48 children each. The control group performed conventional exercises, including flexibility, strength, and endurance training for 45 minutes per session, three times a week. The experimental group followed the same routine for 15 minutes, supplemented by 45 minutes of Pilates exercises emphasizing core stability, postural control, and coordination. Interventions lasted eight weeks. Outcomes were assessed using the Pediatric Balance Scale, 6-Minute Walking Test, and Functional Reach Test. Data were analyzed with SPSS version 25, using non-parametric statistical tests, with significance set at p≤0.05. Results: The Pediatric Balance Scale improved significantly in both groups, with mean±SD scores increasing from 36.53±4.04 to 53.60±1.55 in the experimental group and from 35.73±3.2 to 47.3±1.4 in the control group (p&lt;0.05). The 6-Minute Walking Test results showed greater improvements in the experimental group (172.22±19.9 to 407.3±82.1) compared to the control group (166.4±23.9 to 316.6±24.2) (p&lt;0.05). Similarly, the Functional Reach Test improved from 18.46±2.30 to 38.6±2.00 in the experimental group and from 19.08±2.6 to 30.7±3.13 in the control group (p&lt;0.05). Conclusion: Both Pilates-based and conventional exercises were effective in improving balance and gait in school-going children. However, children in the Pilates group demonstrated superior improvements across all measured parameters, emphasizing the value of incorporating Pilates into pediatric exercise programs.

Using case-based role-play to learn professionalism in Pharmacology

Background: Professionalism has been identified as a key competency for physicians to conduct, effective and ethical practice. The current competency-based medical education curriculum lays great emphasis on the development of attitude, ethics, and communication (AETCOM) among medical students which is embedded in the core concepts of professionalism. Comprehending these concepts early in the course of medical training is especially important as such a change may lead to reducing future incidents of professional misconduct. However, teaching this complex topic to undergraduate students through routine didactic lectures alone is challenging. Methods: To address this, we divided the batch of second-year MBBS students into 5 random groups and assigned 1 case scenario for role play to each group with sub-questions for discussion and reflection. After a short introductory lecture on professionalism, each group presented their role-play and discussed the sub-questions. Assessment for the improvement in knowledge was done using pre and post-test multiple choice questions. Results: Our findings show a statistically significant improvement in the mean (± Standard Deviation) scores for post-test (16.76±3.00) vs the pre-test (11.56±2.81) (p&lt; 0.0001). Participant feedback was overwhelmingly positive based on the 5-point Likert scale. Conclusion: This shows that an interactive and engaging model, such as relevant case scenarios and role-play with reflection, along with assessment and feedback, could be effective for medical students, in learning the complex topic of professionalism.

Over-the-counter procured emergency contraception among undergraduate students in a public University in Ghana

BackgroundEmergency Contraception (EC) is more popular among young people today, yet, there is little research on Over-the-counter procured EC among undergraduate students in African universities. This study's primary goal is to fill that gap.MethodsThis was an exploratory-cross-sequential study conducted in a public university in Ghana, using 145 purposively sampled sexually active undergraduate students. A structured questionnaire and in-depth interview guide designed by the author were used for data collection during the University’s 2021/2022 Academic year. The questionnaire was administered online via students’ social media platforms. In contrast, four group discussions were held with 20 female participants (5 each from levels 100, 200, 300, and 400). Quantitative data were analyzed using Stata Version 14.0 to generate the Median, Standard deviation, Chi-square, and Fisher Exact Test scores, Whilst Content analysis was done with the qualitative data to generate verbal quotes.ResultsThere is demand and abuse of ECs among the students studied with about 99% usage to prevent pregnancy. Friends (81%) played a significant role in providing information about ECs to most participants (96%). About 80% of participants have used an EC at least once during the academic year and upon recommendation by friends (44%) and partners (39%). The most preferred ECs were Postinor-2 (61%) and Lydia post-pills (38%). Pharmacies on campus are the major (86%) suppliers at a cost between ghs5 and ghs15 cedis respectively. There were relationships between gender and choice of ECs (p = 0.001); age group and choice of ECs (p = 0.02); marital status and choice of ECs (p < 0.0001) as well as employment status of students and choice of ECs (p = 0.003). The qualitative findings provided relevant justifications for students’ EC choices, sources, dosage, frequency, and motivations for use. The EC pills were taken routinely and soon after sexual intercourse sometimes overdosed for a perceived efficacy to prevent failure, regulate menses, or as an abortifacient. Male partners played a critical role in procuring and supervising the intake soon after sexual intercourse.ConclusionThere is a high demand, usage, and general abuse of over-the-counter procured ECs among undergraduate students in the study area. These findings suggest that sexually active students are concerned about pregnancy prevention and need health education programs on modern contraceptive methods to prevent unwanted pregnancy and possible STIs. From unprotected sex.

Growth Prediction Model for Prepubertal Children With Idiopathic Growth Hormone Deficiency: An Analysis of LG Growth Study Data.

Growth hormone (GH) treatment is effective in restoring normal growth in children with GH deficiency (GHD). However, individual responses to GH treatment vary, necessitating predictive models to estimate growth outcomes. This study aimed to develop and validate a predictive model for GH treatment response during the first 2 years in patients with idiopathic GHD using the LG growth study (LGS) database. This observational study included 669 prepubertal patients with idiopathic GHD from the LGS registry who received GH treatment for at least 2 years. Clinical and laboratory data were collected at baseline and every 6 months thereafter. Stepwise multivariate regression analysis was performed to develop prediction models for the treatment period. The mean age of patients with GDH was 6.0 ± 1.8 years. Height standard deviation score (SDS) significantly increased from -2.50 ± 0.71 to -1.66 ± 0.71 in the first year and -1.35 ± 0.71 in the second year. The first-year growth velocity was 9.06 ± 1.51 cm, decreasing to 7.42 ± 1.37 cm in the second year. The prediction models incorporated variables such as age, birth weight, bone age, initial height SDS, body mass index SDS, mid-parental height, GH dose and first year of height after GH treatment, explaining 76.9% and 84.1% of the variability in height SDS changes in the first and second years, respectively. GH treatment significantly improves height outcomes in prepubertal children with GHD. The developed predictive models demonstrated accuracy, facilitating personalized GH therapy. Future research should focus on refining these models and exploring the long-term effects of GH treatment in pubertal patients. ClinicalTrials.gov identifier: NCT01604395.

Powdered human milk-derived versus bovine milk-derived breastmilk fortification: A multi-centre preterm randomised controlled trial.

To compare faecal calprotectin, plasma amino acids and clinical outcomes in preterm infants receiving powdered human milk-based fortifier (PHMF) compared to powdered bovine milk-based fortifier (PBMF) in preterm infants on an otherwise exclusive human milk diet (EHMD). A randomised controlled trial in infants <32 weeks of gestation or <1500 g who only received human milk and had reached full enteral feeds (150 mL/kg/day), without pre-existing gastrointestinal morbidity. Primary outcome was faecal calprotectin within 21 days of starting fortification; secondary outcomes were calprotectin at discharge, plasma amino acids and clinical outcomes, including growth and neonatal morbidities. The trial stopped early after the manufacturer's withdrawal of PHMF. Thirty-one infants were enroled, three without informative sampling, leaving 14 per group. No statistical differences were seen in faecal calprotectin on Day 7 (236 mcg/g PHMF vs. 303 mcg/g PBMF, p = 0.90) or 21 (135 mcg/g PHMF vs. 315 mcg/g PBMF, p = 0.21). Adjusting for gestation and day of life, and including all time points after enrolment to discharge, fortifier type did not impact faecal calprotectin (coefficient estimate -7.13, 95% confidence interval = -172 to 158, p = 0.93). Rates of key neonatal morbidities did not differ. PHMF infants grew more slowly reaching statistical significance in change in weight standard deviation score at discharge compared to PBMF infants (mean (standard deviation) -0.94 (0.7) PHMF vs. -0.24 (0.8) PBMF, p = 0.02). We did not detect reduced gut inflammation as measured by faecal calprotectin in PHMF compared to PBMF but weight gain was slower, of potential clinical importance.

Взаимосвязь миокинов и саркопенического ожирения с неалкогольной жировой болезнью печени при конституционально-экзогенном ожирении у детей

Obesity in children is associated with high risk for development and progression of nonalcoholic fatty liver disease (NAFLD). NAFLD has common pathogenetic mechanisms with sarcopenic obesity (SO), in the development of which such myokines as fibroblast growth factor 21 (FGF-21) and irisin play an important role. The study of their secretion peculiarities is relevant to the prospects of using myokines as diagnostic markers or indicators for drug therapy and physical activity control as well as in the development of target therapy for NAFLD. The connection between NAFLD and SO and changes in myokine profile in children is an understudied issue. The purpose of this research was to determine differences between groups of pediatric patients with the presence v. those with the absence of NAFLD in constitutional-exogenous obesity in myokine and SO levels. Methods used: a single-stage comparative single-center uncontrolled study of 95 children aged 12 to 18 y/o with constitutional-exogenous obesity: body mass index (BMI) standard deviation score (SDS)≥+2.0. Results: NAFLD was detected in 48% (n=50/95) [95% CI (42; 63)] of whom steatohepatitis was detected in 40% (n=20/50) [95% CI (26; 55)]. SO was detected in 96% (n=48/50) [95% CI (86; 99)] with NAFLD including children with steatohepatitis (n=20). The combination of SO with elevated FRF-21 levels and decreased irisin was associated with NAFLD (AUC=0.761, [95% CI (0.65; 0.87)]). The SO frequency was statistically significantly higher in NAFLD than in the SO absence (p&lt;0.001). The predictive ability of the SO presence in relation to the detection of NAFLD was found to be high (PCPR=96%, [95% CI (88; 99)]). FRF-21 values above 60 pg/mL (AUC=0.689, [95% CI (0.57; 0.81)]) and irisin values below 7.86 μg/mL (AUC=0.703, 95% CI (0.58; 0.82)]) were associated with the presence of NAFLD. When comparing operational characteristics, no differences were found for AUC value while the best performance was demonstrated by regression model and cut-off point of irisin equal to 7.86 μg/mL. Conclusion: SO combined with changes in myokine profile is associated with NAFLD. Identification of the main factors influencing the NAFLD development in childhood is crucial in preventing the progression of liver damage.

Long-Term Treatment for Laron Syndrome with IGF-1 Injection over 22 Years in Saudi Arabia: A Cohort Study

Introduction: Laron syndrome (LS) is a rare autosomal recessive disorder caused by mutations in the growth hormone (GH) receptor gene, resulting in GH resistance and reduced levels of insulin-like growth factor 1 (IGF-1). Patients with LS exhibit severe growth retardation, low IGF-1 levels, elevated basal GH, and poor response to GH stimulation. Recombinant IGF-1 is the only approved treatment and has been shown to improve linear growth. This study evaluates the long-term efficacy and safety of IGF-1 therapy in a large cohort of LS patients treated at King Faisal Specialist Hospital and Research Center (KFSH & RC), Riyadh, Saudi Arabia over 22 years. Methods: We conducted a retrospective review of medical records for 28 patients with growth hormone insensitivity syndrome, including 12 males and 16 females, treated with IGF-1 from 1998 to 2020. Patients were selected based on criteria including age over 2 years, height standard deviation score (SDS) ≤−2.8, normal or elevated GH secretion (>2.5 ng/mL), IGF-1 levels <50 ng/mL, and insensitivity to exogenous GH. IGF-1 was administered initially at 40 μg/kg/dose subcutaneously twice daily, escalating to a maximum of 120 μg/kg/dose as tolerated. Dosage was adjusted to minimize hypoglycemia risk, with blood glucose monitored frequently during hospitalization. In addition, molecular genetic results were reviewed for each patient in the cohort. Results: IGF-1 treatment significantly increased height velocity (HV) from a baseline of 3.4 cm/year to 6.5 cm/year in the first year (mean difference of 3.1 cm/year, p < 0.0001). In the second year, HV remained elevated at 5 cm/year (mean difference of 1.6 cm/year, p = 0.0015). Long-term follow-up over 10 years demonstrated sustained improvements in HV compared to baseline, with the most substantial gains occurring within the initial 5 years. Weight SDSs also showed significant improvement. Age at the start of therapy did not notably affect growth outcomes, though longer treatment durations were associated with greater growth. Ten disease-causing variants in the GHR gene were identified in 24 of the 28 LS patients. Conclusion: IGF-1 therapy significantly enhanced linear growth in children with Laron syndrome and was generally well tolerated. Although many patients did not reach normal adult height, the growth achieved with IGF-1 treatment was markedly better than expected without therapy. This study underscores the effectiveness of IGF-1 in improving growth outcomes and highlights the need for continued longitudinal studies to optimize treatment strategies and manage potential complications.

Bone Health Determinants in Ambulant Prepubertal Boys With Duchenne Muscular Dystrophy Treated With Deflazacort: Findings From a 3-Year Study.

Duchenne muscular dystrophy (DMD) is complicated by bone fragility. This study aimed to elucidate changes in bone mineral density (BMD) and body composition over time and to explore associations with adiposity measures in DMD. A three-year follow-up analysis was performed of total body (TB) and lumbar spine (LS) dual-energy x-ray absorptiometry (DXA) measurements, anthropometric measures, Tanner stage and bone turnover biomarkers assessments, and the incidence of fragility fractures in 26 ambulant prepubertal DMD patients treated with deflazacort (DFZ). Age at baseline was 7.7 years (interquartile range: 6-9.2). The TB BMD Z-score declined over time and was negatively related to the TB fat mass percentage and fat mass index (p < 0.05), but not to body mass index (BMI) standard deviation score (SDS). In contrast LS bone mineral apparent density (BMAD) Z-score remained stable and normal. The cumulative incidence of fragility fractures was 19.2%; DMD boys with fractures displayed a 1.5-fold higher decline of TB BMD Z-score/year (p < 0.05) and a worse adiposity profile compared to fracture-free patients. No difference was found in DFZ dose or duration between the two groups. We observed a high incidence of fragility fractures, and identified fat tissue as a potential detrimental factor for bone health, suggesting a need for monitoring in DMD patients with excessive adiposity. Fat mass measures assessed by DXA could help to identify those at risk, enabling targeted interventions for better bone health. The co-occurrence of multiple glucocorticoid side effects might characterize patients at higher risk of fractures.

Association between Slow and Rapid Weight Gain before 36 Weeks of Corrected Age and 6-Year-Developmental Quotient in Infants Born at

The postnatal growth of preterm infants is influenced by various factors; however, the association between growth and long-term outcomes in extremely preterm infants remains unclear. Therefore, we aimed to determine the association between 6-year development and body weight (BW) at 36 weeks of corrected age (CA) in extremely preterm infants. We conducted a retrospective observational study including extremely preterm infants born at < 26 weeks of gestation. We defined the key standard deviation score (SDS) using BW at birth and evaluated the association between BW-related values at 36 weeks of CA and the 6-year developmental quotient (DQ). This study included 90 infants. The absolute difference between changes in the BW SDS from birth and the median changes in DQ ≥ 85 infants was significantly greater in infants with a DQ < 85 than ≥85 (median [interquartile range]: 0.80 [0.29, 1.31] vs. 0.34 [0.13, 0.55]; p = 0.001). After adjustment by the logistic regression analysis for DQ < 85, the absolute difference was statistically significant (odds ratio: 4.99, 95% confidence interval: 1.71-14.5). The correlation coefficient between the absolute difference and DQ was -0.35 (p < 0.001). There were no significant associations between a DQ < 85 and SDS or changes in SDS from birth. For infants born at <26 weeks of gestation, slow and rapid weight gain were associated with a low 6-year DQ when using values representing individual body sizes at birth. Individual backgrounds should be considered to avoid both slow and rapid weight gain for the growth management of these infants for at least up to 36 weeks of CA. Further research is needed to determine which body size has a positive impact on the long-term outcomes of infants. · The median change in BW SDS was a key factor for DQ.. · Slow/rapid weight gain were related to a low 6-year DQ.. · Individual backgrounds may be important in neonatal intensive care unit care..

Early-onset neonatal infection and school performance: a Danish nationwide population-based cohort study.

We aimed to study the association between early-onset neonatal infection in near-term and term children and school performance based on mandatory tests in reading and mathematics. We conducted a nationwide register-based cohort study including all Danish near-term and term singletons born from 1997 to 2009. Early-onset infection was defined as an invasive bacterial infection during the first week of life. Infections were categorized into diagnosed sepsis or meningitis, and culture-positive sepsis or meningitis verified by bacteria cultured from blood or cerebrospinal fluid. Multivariable mixed model linear regression was used to estimate mean differences in test scores, expressed as standard deviation scores (SDS) with 95% confidence intervals (CI). Among 638,402 children, 2,362,046 test scores were available from 9 to 15years of age. A total of 5,347 children were diagnosed with sepsis and 73 with meningitis, while 135 also had culture-positive sepsis and 20 had culture-positive meningitis. Diagnosed sepsis was associated with lower test scores with mean differences in reading of 0.08 SDS (95% CI: -0.10, -0.05) and mathematics of -0.08 SDS (95% CI: -0.10, -0.05). Diagnosed meningitis was associated with even lower test scores with mean differences in reading of -0.22 SDS (95% CI: -0.43, 0.00) and mathematics of -0.31 SDS (95% CI: -0.55, -0.07). These findings remained consistent even in sibling-matched analyses. Similar results were also found when only culture-positive infections were compared with the reference population. Sepsis caused by Escherichia coli showed the largest reduction in test scores, while GBS appeared not to affect point estimates. Early-onset sepsis was associated with modest reductions in test scores. This may be insignificant for the individual but could be important on a public health level. Early-onset meningitis was associated with more substantial reductions, emphasizing the severity of this condition even in children able to attend public school.

ChatGPT-4o's performance on pediatric Vesicoureteral reflux.

Vesicoureteral reflux (VUR) is a common congenital or acquired urinary disorder in children. Chat Generative Pre-trained Transformer (ChatGPT) is an artificial intelligence-driven platform offering medical information. This research aims to assess the reliability and readability of ChatGPT-4o's answers regarding pediatric VUR for general, non-medical audience. Twenty of the most frequently asked English-language questions about VUR in children were used to evaluate ChatGPT-4o's responses. Two independent reviewers rated the reliability and quality using the Global Quality Scale (GQS) and a modified version of the DISCERN tool. The readability of ChatGPT responses was assessed through the Flesch Reading Ease (FRE) Score, Flesch-Kincaid Grade Level (FKGL), Gunning Fog Index (GFI), Coleman-Liau Index (CLI), and Simple Measure of Gobbledygook (SMOG). Median mDISCERN and GQS scores were 4 (4-5) and 5 (3-5), respectively. Most of the responses of ChatGPT have moderate (55%) and good (45%) reliability according to the mDISCERN score and high quality (95%) according to GQS. The mean±standard deviation scores for FRE, FKGL, SMOG, GFI, and CLI of the text were 26±12, 15±2.5, 16.3±2, 18.8±2.9, and 15.3±2.2, respectively, indicating a high level of reading difficulty. While ChatGPT-4o offers accurate and high-quality information about pediatric VUR, its readability poses challenges, as the content is difficult to understand for a general audience. ChatGPT provides high-quality, accessible information about VUR. However, improving readability should be a priority to make this information more user-friendly for a broader audience.

EVALUATION OF THE RELATIONSHIP BETWEEN IRON LOAD, AGE, AND CARDIAC FUNCTION IN CHILDREN AND YOUNG ADULTS WITH THALASSEMIA MAJOR: A PULSED-WAVE DOPPLER AND TISSUE DOPPLER IMAGING STUDY

Objective: To determine the structural and functional cardiac differences in children and young adults with thalassemia major (TM) compared to healthy subjects using pulsed-wave Doppler and tissue Doppler imaging methods and determine the relationship between iron overload and these differences. Materials and Methods: We analyzed the data of pediatric and young adult TM patients (n = 44) aged 4–22 years and an age- and gender-matched control group (n = 40) in our hospital data system between Oct.01.2023 and Oct.01.2024. Height, weight, body mass index (BMI), systolic–diastolic blood pressure measurements, complete blood count, ferritin, cardiac T2* magnetic resonance imaging (MRI) values, and echocardiography results were recorded. In addition to comparisons between the two groups, correlation analysis was performed between ferritin–cardiac T2* MRI results and echocardiographic parameters and age in TM patients. Results: Our study showed growth retardation (low height standard deviation score (SDS), low weight SDS and low BMI SDS), dilatation of the left cavities (high left ventricular internal diameter end diastole (LVIDd)), increased left ventricular muscle mass (high left ventricular mass index (LVMI)), cardiac distinctive diastolic (restrictive pattern: left ventricular (LV) peak early diastolic flow (E)/peak late diastolic flow (A) and E/early diastolic myocardial peak flow (E') high), and subclinical systolic (LV peak systolic flow low and LV Tei index high) dysfunction. In addition, iron load (ferritin and cardiac T2* MRI) was correlated with LVMI, and cardiac diastolic and systolic function indicators. As age increased, ferritin value did not change, but cardiac T2* MRI value decreased and diastolic–systolic parameters worsened. Conclusion: Periodic cardiac T2* MRI and Doppler echocardiography examinations of patients with TM may detect subclinical myocardial dysfunction at an early stage, thus providing a window of opportunity for intervention.

Contribution of Sex Steroids in Management of Tall Stature: Is it Effective or Not?

Reduction in adult height by high-dose sex steroids was introduced decades ago. Here, we present the impact of lower doses of sex steroids on the predicted adult height in tall stature. This single-center retrospective observational study included 22 tall children treated with low-dose sex steroids. Patients with familial tall stature, constitutional advance of growth or Marfan syndrome were included. Anthropometric measurements at the commencement of treatment, six-monthly intervals on treatment, cessation of treatment, and at final assessment, were evaluated. Bone age (BA) determination, and adult height predictions (PAH) were made using both the tables of Bayley-Pinneau (BP) and Tanner Whitehouse (TW) mark II methods. The standard deviation scores (SDS) of height over time were not statistically significant. The final height was significantly lower than the predicted height in girls whereas it was not significantly lower than predicted height in boys. In Marfan cases, the final height was only lower than the prediction of TW rather than BP. Non Marfan cases had significantly lower final height than both the predicted heights. Starting treatment in early BA (<10 years) did not affect the last height SDS or the difference between predicted height and final height. Sex difference, sex steroid dosage, differences in treatment duration and differences in BA measurement method, PAH method, BA and chronological age at the start of treatment may all influence the therapy response. Shortcomings about these influences can be overcome in future prospective studies with a larger sample size.