Journal of Pediatric Endocrinology & Metabolism最新文献

{"title":"A systematic review and meta-analysis of the self-reported Pubertal Development Scale's applicability to children.","authors":"Dongxue Pan, Cuilan Lin, Simao Fu","doi":"10.1515/jpem-2024-0407","DOIUrl":"10.1515/jpem-2024-0407","url":null,"abstract":"<p><strong>Introduction: </strong>Assessing pubertal stage can be challenging, particularly in large-scale settings, due to the sensitive nature of Tanner staging by healthcare providers (HCP) or self-reported Tanner stage through photographs or line drawings. The self-reported Pubertal Development Scale (PDS) avoids sensitive issues like genitalia or nudity, is adaptable to various settings, reduces time and cost burdens on researchers. This study aimed to explore the agreement between self-reported PDS and HCP-assessed Tanner staging.</p><p><strong>Content: </strong>Papers for the meta review were retrieved from Pubmed, Embase, Fang Wan, CNKI, and Cochrane Library before January 15, 2025. Quality was assessed using the Quality Assessment of Diagnostic Accuracy Studies-2 tool. Pooled estimates and 95 % confidence intervals (CI) were calculated using random-effects models.</p><p><strong>Summary and outlook: </strong>Five studies with 6024 participants met inclusion criteria. Among stage 1-5, substantial agreement was found among girls (Wk: 0.63 [0.62-0.65]) and overall participants (Wk: 0.68 [0.67-0.69]), while moderate agreement was observed in boys (Wk: 0.58 [0.56-0.61]). Broadening puberty criteria to stages I-III showed substantial agreement for girls (Wk: 0.66 [0.64-0.68]), boys (Wk: 0.64[0.61-0.67]), and overall participants (Wk: 0.69 [0.67-0.70]). For pubertal onset, using Tanner stage as the gold standard, girls showed that the area under the receiver operating characteristic curve (AUC) was 0.86 (0.85-0.87), the sensitivity and positive predictive value (PPV) of self-reported PDS were 0.85 and 84.2 % respectively. Similarly, among boys, the AUC was 0.89 (95 % CI: 0.87-0.92), the sensitivity and PPV were 0.91 and 97.8 % respectively. Our findings indicate moderate to substantial agreement between the two methods, with high sensitivity and PPV for self-reported PDS in assessing puberty onset, PDS may be a reliable and cost-effective method for large-scale epidemiological studies.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"718-724"},"PeriodicalIF":1.0,"publicationDate":"2025-04-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144045192","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Relationships among biological sex, body composition, and bone mineral density in young persons with and without diabetes.","authors":"Carson Platnick, Ye Ji Choi, Phoom Narongkiatikhun, Nathan Bekelman, Callie Rountree-Jablin, Carissa Birznieks, Emily Sell, Kalie L Tommerdahl, Ian de Boer, Viral N Shah, Kristen J Nadeau, Alexandra Sawyer, Laura Pyle, Petter Bjornstad","doi":"10.1515/jpem-2024-0254","DOIUrl":"10.1515/jpem-2024-0254","url":null,"abstract":"<p><strong>Objectives: </strong>Bone mineral density (BMD) is influenced by factors including age, sex, body composition, and diabetes. However, data regarding these relationships in young individuals is limited. The objective of this study was to address this gap in the literature.</p><p><strong>Methods: </strong>We conducted a post-hoc analysis of participants from six cross-sectional cohort studies, encompassing individuals with type 1 diabetes (T1D) and type 2 diabetes (T2D), as well as controls of healthy weight (HWC) and with obesity (OC). Whole-body dual-energy X-ray absorptiometry (DXA) was employed to measure BMD and body composition. Multiple linear regression models assessed sexual dimorphism in BMD, adjusting for age and exploring effect modification by group and sex.</p><p><strong>Results: </strong>A total of 325 participants were included (T1D [n=123, mean age 22.4 years, 50 % male], T2D [n=72, mean age 16.2 years, 33 % male], HWC [n=79, mean age 16.6 years, 41 % male], and OC [n=51, mean age 13.8 years, 53 % male]). Sexual dimorphism in BMD was evident only in T1D and HWC, with males having higher BMD than females (p=0.021; p<0.001, respectively). BMI was positively correlated with BMD in all groups (p<0.001 for HWC; p=0.001 for OC; p<0.001 for T1D; p=0.008 for T2D). Body fat percentage was inversely correlated with BMD in HWC and T1D (p<0.001; p=0.011, respectively), but not in OC or T2D. Additionally, lean mass percentage was significantly associated with higher BMD in HWC and OC (p<0.001; p=0.023, respectively), but not in groups with diabetes.</p><p><strong>Conclusions: </strong>Our study documents sexual dimorphism in BMD in youth, with varied associations between body composition metrics and BMD across groups with diabetes and in controls without diabetes, underscoring the importance of understanding these relationships for optimizing bone health during adolescence.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":"38 5","pages":"476-487"},"PeriodicalIF":1.0,"publicationDate":"2025-04-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12077546/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144043288","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}

{"title":"Hormonal therapy for impaired growth due to pediatric-onset inflammatory bowel disease: a systematic review and meta-analysis with trial sequential analysis.","authors":"Mardhen Catunda Rocha Melo, Rian Vilar Lima, Maryana Modena Strada, João Lucas Maia Rocha, Beatriz Vieira Cavalcante, Maria Lya Pinheiro Bezerra, Lívia Vasconcelos Martins, Maria Clara Parente Torquato, Túlio Veras Veloso, Delanie Bulcao Macedo","doi":"10.1515/jpem-2024-0609","DOIUrl":"10.1515/jpem-2024-0609","url":null,"abstract":"<p><strong>Introduction: </strong>Inflammatory bowel diseases (IBDs) have an increasing incidence in the pediatric population. The dysabsorptive effects of this condition often lead to a decrease in linear growth. However, the effectiveness and safety of growth hormone (GH) therapy in this population is still a topic of debate, with studies showing conflicting results.</p><p><strong>Content: </strong>MEDLINE, Embase, and Cochrane Library databases were systematically searched according to the PRISMA guidelines. All experimental studies featuring children with IBD receiving GH therapy were included. In addition, a trial sequential analysis (TSA) was conducted to determine the sample size required for each outcome. The prospective registry was carried out under protocol CRD42024563079. The total data set comprised eight studies involving 127 patients with IBD, 78 (61.41 %) of whom received GH therapy, with a mean follow-up duration of 1.3 years. A statistically significant effect of GH was found in increasing the height standard deviation score (HtSDS) of children with IBD (standardized mean difference - SMD=1.07; CI=0.58, 1.56; p<0.0001). When comparing children who received GH with controls, no significant improvement in HtSDS was observed (SMD=0.18; CI=-0.73, 1.08; p=0.70). However, meta-regression analysis indicated that a longer follow-up was associated with a greater improvement in the HtSDS (p=0.04). Regarding height velocity (HV), a significant increase was found when comparing measurements before and after the initiation of hormone therapy (mean difference - MD=4.09; CI=2.58, 5.60; p<0.0001). An increase in HV was also noted in children receiving GH compared to the control group (MD=4.47; CI=2.03, 6.90; p=0.0003). No significant changes in the Pediatric Crohn's Disease Activity Index (PCDAI) were detected, comparing values before and after the start of treatment (MD=-10.09; CI=-22.29, 2.10; p=0.10). The overall prevalence of any adverse effect was estimated at 15.51 % (95 % CI: 2.32-58.70 %). Most common reaction was itching at injection sites. TSA indicated a low risk of overestimating or underestimating the intervention's effect on the analyzed outcomes.</p><p><strong>Summary: </strong>Our study points to the effectiveness and safety of GH therapy in children with IBD and growth impairment.</p><p><strong>Outlook: </strong>Further randomized controlled trials (RCT) with standardized methodologies and extended follow-up periods are necessary to confirm these findings.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"562-569"},"PeriodicalIF":1.0,"publicationDate":"2025-04-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143804573","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pubertal disorders in juvenile idiopathic arthritis: a systemic review.","authors":"Soumaya Boussaid, Sonia Rekik, Hanene Lassoued Ferjani, Maissa Abbes, Safa Rahmouni, Khaoula Zouaoui, Rim Dhahri, Wafa Hamdi, Hela Sahli","doi":"10.1515/jpem-2024-0412","DOIUrl":"10.1515/jpem-2024-0412","url":null,"abstract":"<p><strong>Introduction: </strong>Juvenile idiopathic arthritis (JIA) is a chronic inflammatory disease beginning before 16 years. Inflammatory cytokine, medication, and genetic factors may lead to puberty disorders in children with JIA. The main objectives of this systematic review were to elucidate the impact of JIA on puberty and identify the influential factors that disrupt puberty.</p><p><strong>Content: </strong>A systematic literature review was performed on pubertal disorders from Medline, Google Scholar, and Scopus databases. This systematic review followed the preferred reporting items for systematic review guidelines. The initial search yielded 4,011 articles: identified by Google Scholar (3,880), PubMed (99), and Scopus (940). Finally, 11 articles were retained.</p><p><strong>Summary and outlook: </strong>The age of menarche onset, Tanner stage, and pubertal signs were later compared with controls. The mean age of menarche onset ranged from 12.0 ± 0.3 to 13.39 ± 0.93 years for the JIA girls. This delay was more reported in the polyarticular and oligoarticular subtypes. Menstrual irregularities, metrorrhagia with irregular cycles, primary oligomenorrhea, and secondary amenorrhea were also reported. Factors found to influence delayed menarche and puberty were steroid use, JIA subtype, disease duration, and age at onset. Any studies have focused on the effect of puberty on JIA outcomes. Overall, our review revealed that pubertal disorders are frequent in JIA patients with polyarticular and systemic subtypes. However, some influencing factors remain editable if well-assessed and controlled.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"551-561"},"PeriodicalIF":1.0,"publicationDate":"2025-03-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143732656","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Chronotype, sleep, and glycemic control in children and adolescents with type 1 diabetes: a case-control study.","authors":"Gulay Can Yilmaz, Mehmet Karadag","doi":"10.1515/jpem-2024-0492","DOIUrl":"10.1515/jpem-2024-0492","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to explore the relationships between sleep parameters, chronotype preferences, and glycemic control in children and adolescents with type 1 diabetes (T1DM), compared to healthy peers.</p><p><strong>Methods: </strong>A total of 96 children and adolescents with T1DM and 95 healthy controls aged 8-18 years participated in this case-control study. Anthropometric measurements were collected, and participants completed the Munich Chronotype Questionnaire and the Pittsburgh Sleep Quality Index (PSQI). Glycemic control was assessed using HbA_1c levels.</p><p><strong>Results: </strong>Children with T1DM demonstrated significantly shorter sleep durations, poorer sleep quality, and a later chronotype compared to controls (p<0.05). Poor glycemic control (HbA_1c>7.5 %) was observed in 72.9 % of the T1DM group, with 34.3 % exhibiting very poor control (HbA_1c>9 %). Logistic regression identified poor sleep quality (PSQI score, OR: 1.47, p<0.001) and later chronotype (OR: 5.14, p<0.01) as independent predictors of poor glycemic control. Generalized linear modeling (GLM) further revealed significant associations between HbA_1c levels, insulin dosage (p<0.001), and chronotype (p=0.090).</p><p><strong>Conclusions: </strong>Late chronotype and poor sleep quality are closely linked to suboptimal glycemic control in pediatric T1DM populations. These findings underscore the importance of integrating sleep-focused strategies into routine diabetes management.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"442-449"},"PeriodicalIF":1.0,"publicationDate":"2025-03-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143665180","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A recent update on childhood obesity: aetiology, treatment and complications.","authors":"Katherine Hawton, Diksha Shirodkar, Thomas Siese, Julian P Hamilton-Shield, Dinesh Giri","doi":"10.1515/jpem-2024-0316","DOIUrl":"10.1515/jpem-2024-0316","url":null,"abstract":"<p><p>Obesity is a complex, chronic condition characterised by excess adiposity. Rates of obesity in childhood and adolescence are increasing worldwide, with a corresponding increase in adulthood. The aetiology of obesity is multifactorial and results from a combination of endocrine, genetic, environmental and societal factors. Population level approaches to reduce the prevalence of childhood obesity worldwide are urgently needed. There are wide-ranging complications from excess weight affecting every system in the body, which lead to significant morbidity and reduced life expectancy. Treatment of obesity and its complications requires a multi-faceted, biopsychosocial approach incorporating dietary, exercise and psychological treatments. Pharmacological treatments for treating childhood obesity have recently become available, and there is further development of new anti-obesity medications in the pipeline. In addition, bariatric surgery is being increasingly recognised as a treatment option for obesity in adolescence providing the potential to reverse complications related to excess weight. In this review, we present an update on the prevalence, aetiology, complications and treatment of childhood obesity.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"429-441"},"PeriodicalIF":1.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143659609","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Prader-Willi syndrome gene expression profiling of obese and non-obese patients reveals transcriptional changes in CLEC4D and ANXA3.","authors":"Ju Young Yoon, Choong Ho Shin, Murim Choi, Jung Min Ko, Young Ah Lee, Kye Shik Shim, Jun Lee, Suk Dong Yoo, Minji Kim, Yeuni Yu, Joo Young Lee, Yun Hak Kim, Chong Kun Cheon","doi":"10.1515/jpem-2024-0408","DOIUrl":"10.1515/jpem-2024-0408","url":null,"abstract":"<p><strong>Objectives: </strong>We aimed to characterize genetic alterations in Prader-Willi syndrome (PWS) using whole genome microarrays.</p><p><strong>Methods: </strong>We performed mRNA expression microarray analysis using RNA isolated from whole blood of 25 PWS patients and 25 age-matched controls. After preprocessing the data to reduce heterogeneity, differentially expressed genes (DEGs) between groups were identified using a linear regression model package. Reactome pathway analysis was performed for upregulated and downregulated genes using EnrichR. Correlations between gene expression levels and clinical factors were estimated using Spearman's rank correlation coefficient.</p><p><strong>Results: </strong>Of 21,488 probes examined in the microarray analysis, 4,156 were detected. Fifty-two genes had different expression levels in children with PWS compared with healthy controls (36 genes upregulated and 16 downregulated). Twelve genes were upregulated and 13 were downregulated in obese PWS patients compared with normal-weight PWS (NW-PWS) patients. The C-type lectin domain family 4 member D (CLEC4D) was upregulated in both PWS (vs. control) and obese-PWS (vs. NW-PWS) patients, and CLEC4D expression was also correlated with body mass index-standard deviation score in PWS patients. Among the genes upregulated in obese PWS vs. NW-PWS, Annexin A3 (ANXA3), potassium inwardly rectifying channel subfamily J member 15 (KCNJ15), and selenium binding protein 1 (SELENBP1) were upregulated in obese-control vs. NW-control. Gene ontology analysis revealed that upregulated DEGs were significantly enriched in biological processes, including pathways involved in myeloid dendritic cell activation associated with CLEC4D.</p><p><strong>Conclusions: </strong>This study revealed differences in gene expression between obese and NW-PWS patients. The regulation of macrophage infiltration by CLEC4D suggests a possible mechanism associated with obesity-related complications in PWS.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"514-524"},"PeriodicalIF":1.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143659610","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Determinants of childhood and adolescent obesity and it's effect on metabolism in South Indian population.","authors":"Sengottaiyan Palanivel, Egappan Subbiah, K S Raghavan, Subbiah Sridhar","doi":"10.1515/jpem-2024-0340","DOIUrl":"10.1515/jpem-2024-0340","url":null,"abstract":"<p><strong>Objectives: </strong>The primary objective is to determine the risk factors underlying the development of childhood and adolescent obesity. The secondary objective is to determine the predictors of metabolic syndrome (MetS) in childhood and adolescent obesity and its metabolic alterations in the South Indian population.</p><p><strong>Methods: </strong>This is a cross-sectional study conducted over two years. We have screened 3,195 school children and adolescents from lower and lower-middle socioeconomic groups. From this pool, by random cluster sampling technique, we have included 166 overweight and obese individuals and 38 control subjects. We have analyzed their sociodemographic, dietary, lifestyle, anthropometric, clinical, and metabolic parameters.</p><p><strong>Results: </strong>The prevalence of overweight and obesity in rural areas was 14.2 and 7.6 %, respectively, and in urban areas, it was 16.1 and 8.8 %, respectively. The age distribution of the control and study group is 12.3 ± 1.5 and 13.0 ± 1.9 years with a male-to-female ratio of 1.4:1 and 1.6:1, respectively. Our study found a higher average consumption of energy-dense food and screen time in the obese group compared to the control group. The average outdoor play time was 1.5 h per day in the control group and less than 0.5 h per day in the obese group. In our study, the waist-to-height ratio (WHtR) optimum cutoff value of 0.56, has 95 % sensitivity and 84 % specificity, effectively identifying MetS cases. HOMA-IR optimum cutoff value of 2.25, has 96 % sensitivity and 72 % specificity. The triglyceride-glucose index (TGI) optimum cutoff value of 4.51, has 92 % sensitivity and 88 % specificity indicating a strong balance between correctly identifying positive and negative MetS cases.</p><p><strong>Conclusions: </strong>Our study found that even in lower socioeconomic status, there is a higher prevalence of childhood and adolescent obesity due to an urbanized lifestyle in rural areas, a sedentary lifestyle, higher consumption of low-cost energy-dense foods, and higher screening time in this electronic era<b>.</b> We also conclude that WHtR is a simple anthropometric marker that predicts MetS more effectively than BMI and WHR among children and adolescents. HOMA-IR and TGI are effective biochemical markers to identify metabolically unhealthy obesity early.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"450-457"},"PeriodicalIF":1.0,"publicationDate":"2025-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143665182","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The effect of antenatal steroids on metabolic bone disease of prematurity.","authors":"Sara Erol, Mustafa Senol Akin, Nihan Hilal Hosagasi, Sabriye Korkut","doi":"10.1515/jpem-2025-0033","DOIUrl":"10.1515/jpem-2025-0033","url":null,"abstract":"<p><strong>Objectives: </strong>The study aimed to evaluate the impact of antenatal steroid administration, a key intervention for reducing early mortality and morbidity in preterm infants, on the development of metabolic bone disease.</p><p><strong>Methods: </strong>This single-center retrospective study was conducted in a Level III neonatal intensive care unit from October 2020 to December 2023.</p><p><strong>Results: </strong>It included 173 infants born before 32 weeks of gestation, with a mean birth weight of 1,338 ± 293 g. Metabolic bone disease, diagnosed at four weeks of age based on serum phosphorus and alkaline phosphatase levels, was identified in 26 (15 %) of the infants. Regression analysis examined prenatal factors, including birth weight, intrauterine growth restriction, respiratory distress syndrome, gender, and antenatal steroid exposure, revealing that only lower birth weight was an independent risk factor for metabolic bone disease.</p><p><strong>Conclusions: </strong>Antenatal steroid administration did not significantly influence the diagnosis of metabolic bone disease when assessed using biochemical markers at four weeks of age. These findings underscore the importance of birth weight in the risk profile for metabolic bone disease while indicating that antenatal steroids are not a contributing factor.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":"38 5","pages":"509-513"},"PeriodicalIF":1.0,"publicationDate":"2025-03-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144025349","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Early-onset growth hormone treatment in Prader-Willi syndrome attenuates transition to severe obesity.","authors":"Aneta Kodytková, Shenali Anne Amaratunga, Eva El-Lababidi, Ivana Čermáková, Jana Černá, Marcela Dvořáková, Božena Kalvachová, Stanislava Koloušková, Ivana Kotvalová, Olga Magnová, David Neumann, Dana Novotná, Barbora Obermannová, Renata Pomahačová, Štěpánka Průhová, Jiří Strnadel, Jaroslav Škvor, Marta Šnajderová, Zdeněk Šumník, Jirina Zapletalová, Daniela Zemková, Kateřina Kusalová, Jiří Šilar, Jan Lebl","doi":"10.1515/jpem-2024-0463","DOIUrl":"10.1515/jpem-2024-0463","url":null,"abstract":"<p><strong>Objectives: </strong>Subsequent to early life feeding issues, children with Prader-Willi syndrome (PWS) develop hyperphagia and severe obesity. Growth hormone (GH) therapy has been approved in PWS to improve growth, body composition, and BMI. We aimed to clarify the role of age at GH therapy onset on growth and BMI trajectories in children with PWS.</p><p><strong>Methods: </strong>We analyzed height and BMI in 114 patients (58 boys) from REPAR - Czech national GH registry. From them, 69 started GH therapy prior to 2 y/o (age 0.8 ± 0.4 years; mean ± SD; early-onset group [EO]), and 45 later (age 7.1 ± 4.1 years; late-onset group [LO]).</p><p><strong>Results: </strong>Height-SDS before therapy was similar in all (EO: -1.9 ± 1.2 [mean ± SD]; LO: -1.7 ± 1.1). After the first year of GH therapy, height-SDS in the EO group increased to -1.0 ± 1.2, in the LO group to -0.9 ± 1.1. After 5 years, height fully normalized in all (-0.1 ± 1.1 SDS). The LO children were already obese at treatment initiation (BMI-SDS: 2.9 ± 2.2), and their BMI-SDS decreased after 1 year of GH therapy by 0.9 (p=0.003). The weight in EO children was below average before GH treatment (BMI-SDS: -0.9 ± 1.2) and their BMI-SDS increased to the overweight range of 1.3 ± 2.2 (p<0.001) within the oncoming 3 years. Albeit BMI-SDS was around the obesity limit in most children after 5 years on GH therapy, the highest lifetime BMI-SDS was lower in EO (2.2 ± 2.6) than in LO (3.7 ± 2.2; p<0.001).</p><p><strong>Conclusions: </strong>GH treatment in PWS normalizes body height. After 5 years of GH therapy, BMI-SDS in EO and LO groups are similar; however, the EO group is exposed to lower maximal BMI-SDS values.</p>","PeriodicalId":50096,"journal":{"name":"Journal of Pediatric Endocrinology & Metabolism","volume":" ","pages":"525-532"},"PeriodicalIF":1.0,"publicationDate":"2025-03-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143625987","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":4,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}