Serpil Albayrak, Murat Karaoğlan, Mehmet Keskin, Ahmet Yıldırım
{"title":"Association with Metabolic Syndrome in Children Diagnosed with Type 1 Diabetes Mellitus: A Cross-Sectional Study","authors":"Serpil Albayrak, Murat Karaoğlan, Mehmet Keskin, Ahmet Yıldırım","doi":"10.4274/jcrpe.galenos.2025.2025-2-11","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-2-11","url":null,"abstract":"<p><strong>Objective: </strong>To evaluate the prevalence of metabolic syndrome (MetS) in children with type 1 diabetes mellitus (T1DM) and to determine the predictive value of simple anthropometric measurements, in particular neck circumference (NC) and waist circumference (WC), for identifying MetS.</p><p><strong>Methods: </strong>Children (aged 6-18 years) and diagnosed with T1DM were included in this cross-sectional study. Anthropometric [NC, WC, hip circumference (HC), body mass index (BMI), tri-ponderal mass index (TMI)] and laboratory parameters, including lipid profile and hemoglobin A1c (HbA1c) were recorded. MetS diagnosis was established using the International Diabetes Federation criteria. Receiver operating characteristic (ROC) curve analysis and Least Absolute Shrinkage and Selection Operator (LASSO) regression were employed to identify key predictors.</p><p><strong>Results: </strong>A total of 168 children with T1DM participated, among whom the prevalence of MetS was 8.9%. Children with MetS had significantly higher BMI, WC, NC, HC, and TMI values compared to non-MetS counterparts. ROC analysis identified WC Z-score having the highest discriminative power [area under the curve (AUC)=0.954], followed by NC Z-score (AUC=0.906). LASSO regression identified NC Z-score and BMI percentile as the most robust predictors. A strong positive correlation was observed between NC and WC (r=0.812, p<0.001), and there was a mild inverse correlation between NC and high-density lipoprotein cholesterol.</p><p><strong>Conclusion: </strong>NC and WC are simple, non-invasive, and reliable tools for early detection of MetS risk in pediatric T1DM patients. Their routine measurement may enhance risk stratification and guide preventive interventions targeting obesity and dyslipidemia. These findings support incorporating NC and WC into standard clinical assessments to improve long-term cardiometabolic outcomes in children with T1DM, with NC z>1.04 or WC z>1.41 as actionable thresholds.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"243-253"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325816/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145076286","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}
Chen Chongyang, Zhao Yangting, Li Kai, Lv Xiaoyu, Wang Yawen, Zhen Donghu, Fu Songbo, Ma Lihua, Zhou Liyuan, Liu Jingfang
{"title":"Hereditary Severe Insulin-resistance Syndrome and Acanthosis Nigricans Caused by Novel Mutations in the <i>INSR</i> Gene","authors":"Chen Chongyang, Zhao Yangting, Li Kai, Lv Xiaoyu, Wang Yawen, Zhen Donghu, Fu Songbo, Ma Lihua, Zhou Liyuan, Liu Jingfang","doi":"10.4274/jcrpe.galenos.2024.2024-2-14","DOIUrl":"10.4274/jcrpe.galenos.2024.2024-2-14","url":null,"abstract":"<p><p>Most cases of hereditary severe insulin-resistance syndrome (H-SIRS) are linked to mutations in the insulin receptor (<i>INSR</i>) gene. Patients with H-SIRS typically manifest symptoms of hyperinsulinemia, insulin resistance, and diabetes mellitus. Other symptoms include impaired glucose regulation, hyperandrogenism, and the presence of acanthosis nigricans (AN). In this report, we present two cases of H-SIRS in female children exhibiting various symptoms, including hyperinsulinemia, fasting hypoglycemia, postprandial hyperglycemia, overweight, fatty liver, hyperandrogenism, and varying degrees of AN. One patient also presented with mental retardation. Gene sequencing identified specific mutations in the <i>INSR</i> gene for both patients: c.2663A > G (p.Tyr888Cys) in Patient 1 and c.38_61del (p.Pro13_Ala20del) in Patient 2. These mutations both have the potential to disrupt the interaction between the insulin receptor, <i>INSR</i>, and insulin, leading to abnormal insulin signaling, insulin resistance, and various clinical manifestations.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"358-367"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325959/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141477766","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":"Type 1 Diabetes Mellitus and Transfer from Pediatric to Adult Care: A Single-Center Experience","authors":"Betul Yigit Yalcin, Ummahan Tercan, Melek Yildiz, Hulya Hacisahinogullari, Gulsah Yenidunya Yalin, Nurdan Gul, Ozlem Soyluk Selcukbiricik, Ayse Kubat Uzum, Sukran Poyrazoglu, Firdevs Bas, Kubilay Karsidag, Ilhan Satman, Feyza Darendeliler","doi":"10.4274/jcrpe.galenos.2025.2025-1-21","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-1-21","url":null,"abstract":"<p><strong>Objective: </strong>Type 1 diabetes mellitus (T1DM) requires lifelong follow-up, and the transition from pediatric to adult care may influence clinical outcomes. To evaluate sociodemographic, clinical, and laboratory characteristics associated with the transition process in patients with T1DM and to compare two different transition models.</p><p><strong>Methods: </strong>Patients with T1DM transferred to an adult outpatient clinic between 2001 and 2022 were retrospectively assessed. Demographic data, metabolic parameters, complications, and treatment modalities before and after transition were assessed. Transition was performed either as a single-session model (Model 1) or as a gradual process over 4-6 months (Model 2).</p><p><strong>Results: </strong>A total of 64 patients transitioned over the 21 year study period. The annual number of follow-up visits was significantly lower in adult care (3.0±0.9 vs. 2.1±1.8 visits/year; p=0.009). HbA1c levels were also lower in adulthood (8.9% vs. 8.3%; p=0.007). Total insulin dose was lower (0.95 vs. 0.75 IU/kg/day; p=0.009), whereas the basal insulin ratio was higher (43.1% vs. 52.8%; p<0.0001). Although mean body mass index slightly decreased, obesity prevalence increased. No significant differences were observed between the two transition models in terms of glycemic outcomes, insulin requirements, or complication rates.</p><p><strong>Conclusion: </strong>A structured transition process was associated with improved glycemic control and treatment adaptation in T1DM, regardless of whether it is implemented as a single-session or gradual model. The absence of major differences between models may support the importance of individualized, patient-centered transition strategies.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"224-232"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325666/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145076381","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":"Sleep Hygiene in Pediatric Patients with Steatotic Liver Disease","authors":"Josef Finsterer","doi":"10.4274/jcrpe.galenos.2026.2026-4-16","DOIUrl":"10.4274/jcrpe.galenos.2026.2026-4-16","url":null,"abstract":"","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"370-371"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325955/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148310113","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}
Ayşe Akyüz, Hakan Atalar, Kübra Çilesiz, Aslı İnci, İlyas Okur, Leyla Tümer, Fatih Ezgü
{"title":"Mucopolysaccharidosis or Skeletal Dysplasia? Clinical and Radiologic Clues for Differential Diagnosis Based on Difficult Cases","authors":"Ayşe Akyüz, Hakan Atalar, Kübra Çilesiz, Aslı İnci, İlyas Okur, Leyla Tümer, Fatih Ezgü","doi":"10.4274/jcrpe.galenos.2025.2025-8-11","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-8-11","url":null,"abstract":"<p><strong>Objective: </strong>The skeletal abnormalities of mucopolysaccharidosis (MPS) and skeletal dysplasia (SD) may be similar and even indistinguishable. This study aims to elucidate clinical clues and overlapping features that may assist in the different diagnosis.</p><p><strong>Methods: </strong>The clinical features of patients who were first referred to endocrinology or rheumatology department for short stature or joint abnormalities were addressed and signs were examined upon different diagnosis.</p><p><strong>Results: </strong>Three patients (I, II and III) were diagnosed with SD with overlapping and also distinguishing skeletal features compared with MPS. An atypical presentation defined in patient IV who was diagnosed with Morquio syndrome. Patients V and VI were diagnosed with MPS with early onset and typical skeletal features accompanied with additional systemic manifestations uncommon in SD.</p><p><strong>Conclusion: </strong>In conclusion, this study emphasizes the clinical and radiological evaluation and nuances distinctions in clinical presentations that will highlight the challenges and guide to distinguishing different diagnosis of MPS and SD in atypical presentations for achieving the accurate diagnosis.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"282-287"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325825/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145670340","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}
Özge Bayrak Demirel, Feyza Darendeliler, Ruveyde Bundak
{"title":"Response to the Letter Regarding \"Reference Values for Weight, Height, Head Circumference, and Body Mass Index in Turkish Children\".","authors":"Özge Bayrak Demirel, Feyza Darendeliler, Ruveyde Bundak","doi":"10.4274/jcrpe.galenos.2026.2026-2-25","DOIUrl":"10.4274/jcrpe.galenos.2026.2026-2-25","url":null,"abstract":"","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":"18 2","pages":"369"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325679/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148377666","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}
Emrullah Arslan, Hanife Gül Balkı, Deniz Özalp Kızılay, Günay Demir, Samim Özen, Şükran Darcan, Damla Gökşen
{"title":"The Relationship between Hemoglobin A1c and the Glucose Management Indicator and Glucose Metrics in Children and Adolescents with Type 1 Diabetes Mellitus Using Automated Insulin Delivery Systems","authors":"Emrullah Arslan, Hanife Gül Balkı, Deniz Özalp Kızılay, Günay Demir, Samim Özen, Şükran Darcan, Damla Gökşen","doi":"10.4274/jcrpe.galenos.2025.2025-10-11","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-10-11","url":null,"abstract":"<p><strong>Objective: </strong>Hemoglobin A1c (HbA1c) remains the standard biomarker for long-term glycemic control in type 1 diabetes mellitus (T1D), butis incapable of capturing short-term glucose variability and acute excursions. This limitation is especially relevant in children with T1D who use continuous glucose monitoring systems (CGMS) and automated insulin delivery (AID) systems. The objective of this study was to evaluate the temporal relationship between HbA1c and the glucose management indicator (GMI), and any further associations with CGMS-derived glycemic parameters over a 12 weeks in youth with T1D using AID.</p><p><strong>Methods: </strong>This retrospective, cross-sectional, observational study, included children and adolescents with T1D using the Medtronic MiniMed 780G™ system. CGMS data covering the 12 weeks prior to HbA1c measurement were analyzed in two-week intervals. Correlations between HbA1c, GMI, and CGMS metrics were assessed.</p><p><strong>Results: </strong>The study cohort numbered 81; 46 (57%) were female. Median age at T1D diagnosis was 8.1 (interquartile range: 4.3-10.8) years. HbA1c correlated positively with all GMI values, with the strongest correlation observed for the last six-week GMI (r=0.728, p<0.001). The mean difference between HbA1c and last 12-week GMI was 0.57% (95% confidence interval: -1.13 to 2.27). GMI demonstrated stronger correlations than HbA1c with time in range (TIR), time above range (TAR) and time below range (TBR). Notably, in individuals with similar TIR (~70%), HbA1c values varied widely (6.6-9.6%/48-81 mmol/mol), while GMI remained stable (6.8-7.1%).</p><p><strong>Conclusion: </strong>The strongest correlation between HbA1c and the most recent 6-week GMI suggests that HbA1c reflects relatively recent glycemic trends. GMI also showed closer alignment with CGMS-derived indices such as TIR, TAR and TBR, suggesting better sensitivity in capturing day-today glycemic variability. We suggest GMI offers a more sensitive and clinically actionable estimate of glycemic control, supporting its integration into routine care for children with T1D using AID.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"314-322"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325986/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145953597","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}
Kübra Şen Küçük, Sebla Güneş, Mustafa Dinçer, Sercan Öztürk, Tolga Ünüvar, Ahmet Anık
{"title":"Once-Weekly Somatrogon in Pediatric Growth Hormone Deficiency: Real-World Efficacy, Safety, and Quality-of-Life Findings","authors":"Kübra Şen Küçük, Sebla Güneş, Mustafa Dinçer, Sercan Öztürk, Tolga Ünüvar, Ahmet Anık","doi":"10.4274/jcrpe.galenos.2026.2025-11-4","DOIUrl":"10.4274/jcrpe.galenos.2026.2025-11-4","url":null,"abstract":"<p><strong>Objective: </strong>To report real-world 6- and 12-month outcomes in children with growth hormone deficiency (GHD) treated with somatrogon or somatropin, including those who transitioned from somatropin to somatrogon.</p><p><strong>Methods: </strong>Eligible patients were categorized into three groups [somatrogon-naïve (naïve), somatrogon-switch (switch), and somatropin only] and were followed for 6 or 12 months. Bioimpedance analysis, as well as a standardized, age-appropriate assessment of the Pediatric Quality of Life Inventory (PedsQL), the Child Behavioural Checklist (CBCL) and the Multidimensional Scale of Perceived Social Support (MSPSS), were conducted at baseline and month 6 in the naïve and switch groups. Psychiatric evaluations were also performed according to the Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition, Text Revision (DSM-5-TR) criteria.</p><p><strong>Results: </strong>A total of 58 patients (58.6% male) were included (naïve: n=20; switch: n=18; somatropin: n=20). Mean ages were 11.1±3.0, 9.7±3.4, and 10.5±3.2 years, respectively. After 12 months, mean changes in height standard deviation score (Δheight SDS) were 0.6±0.3, 0.7±0.3, and 0.7±0.4; and height velocities were 10.0±1.9, 9.1±1.7, and 9.8±1.9 cm/year, respectively. Corresponding increases in IGF-1 SDS (ΔIGF-1 SDS) were 2.2±1.2, 0.9±1.2, and 1.3±1.0, respectively. Among the 38 patients receiving somatrogon, 15.8% (n=6; 3 naïve, 3 switch) developed IGF-1 SDS >+2 during follow-up, managed successfully with observation or dose adjustment. No serious adverse events were observed. Bioimpedance analyses demonstrated a favorable but non-significant trend toward improved body composition in somatrogon-naïve children. At six months, PedsQL domains, CBCL scales, and MSPSS scores remained stable (all p>0.05).</p><p><strong>Conclusion: </strong>Once-weekly somatrogon demonstrated efficacy and safety comparable to daily somatropin with stable quality-of-life and psychosocial outcomes in children with GHD.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"323-332"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325961/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147469754","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}
Belma Haliloğlu, Tuba Seven Menevşe, Seda Güleç Yılmaz, Tuba Akdeniz, Büşra Gürpınar Tosun, Serap Demircioğlu Turan, Tülay Güran, Yasemin Gökdemir, Ela Erdem, Bülent Karadağ, Turgay İşbir, Abdullah Bereket
{"title":"Isolated Hypoglycemia in Children with Cystic Fibrosis: Role of Pancreatic Insufficiency and Glucagon Response","authors":"Belma Haliloğlu, Tuba Seven Menevşe, Seda Güleç Yılmaz, Tuba Akdeniz, Büşra Gürpınar Tosun, Serap Demircioğlu Turan, Tülay Güran, Yasemin Gökdemir, Ela Erdem, Bülent Karadağ, Turgay İşbir, Abdullah Bereket","doi":"10.4274/jcrpe.galenos.2025.2025-2-6","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-2-6","url":null,"abstract":"<p><strong>Objective: </strong>Hypoglycemia is one of the comorbidities that adversely affects the quality of life in patients with cystic fibrosis (CF). Isolated hypoglycemia (IsoHypo) is poorly described in patients with CF and its etiopathogenic significance is unclear. To investigate the etiopathogenesis of IsoHypo and the role of pancreatic insufficiency (PI) in IsoHypo in children with CF.</p><p><strong>Methods: </strong>The blood glucose, insulin, and glucagon responses of patients with CF and healthy controls were evaluated during a 3-hour oral glucose tolerance test. Based on the results, the patients were categorized into 5 groups: 1) normal glucose tolerance (NGT); 2) IsoHypo; 3) hypoglycaemia with abnormal glucose tolerance (Hypo+AGT); 4) AGT; and 5) CF-related diabetes. IsoHypo and NGT were sub-classified according to the presence of PI as PI(+) or PI(-). Hypoglycemia was defined as blood glucose <70 mg/dL.</p><p><strong>Results: </strong>A total of 44 patients with CF and 9 controls. Hypoglycaemia was observed in 21 of 44 patients (47.7%), predominantly as IsoHypo (29.5%). Hypo+AGT was found in eight patients (18.2%). The IsoHypo group exhibited undelayed and higher insulin secretion than the Hypo+AGT group, with IsoHypo PI(-) being less impaired compared to IsoHypo PI(+). Both IsoHypo and Hypo+AGT groups exhibited a blunted rise in glucagon at 180 minutes, with the deficiency being more pronounced in the Hypo+AGT group. Insulin and glucagon responses to oral glucose load in IsoHypo PI(+) were similar to Hypo+AGT, whereas they were less impaired in IsoHypo PI(-) patients who had early and higher insulin secretion.</p><p><strong>Conclusion: </strong>IsoHypo is common in children with CF and may precede Hypo+AGT in those with PI(+). The abnormal insulin and glucagon responses to glucose appear to be the most significant contributors to the development of IsoHypo in CF.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"233-242"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13326013/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145294034","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}
Mohammad Hosny Awad, Reham Ghanim, Rania Eladl, Zulf Mughal, Manal Mustafa
{"title":"Real-World Efficacy of Weekly Somatrogon on Growth and Bone Health in Pediatric Growth Hormone Deficiency: A 12-Month Retrospective Cohort Study","authors":"Mohammad Hosny Awad, Reham Ghanim, Rania Eladl, Zulf Mughal, Manal Mustafa","doi":"10.4274/jcrpe.galenos.2025.2025-6-24","DOIUrl":"10.4274/jcrpe.galenos.2025.2025-6-24","url":null,"abstract":"<p><strong>Objective: </strong>Growth hormone deficiency (GHD) in children results in short stature and impaired bone health. While daily growth hormone (GH) injections are effective, they are associated with adherence challenges. Somatrogon, a long-acting, recombinant human GH (rhGH), allows weekly administration, potentially improving treatment compliance.</p><p><strong>Methods: </strong>This retrospective cohort study included prepubertal children with GHD treated with weekly Somatrogon at Al Jalila Children’s Hospital, Dubai. Diagnosis was based on clinical, biochemical, and radiological criteria, including height standard deviation score (SDS) <-2.0, subnormal growth velocity, and subnormal peak GH in one stimulation test (<10 ng/mL), supported by low IGF-1 and/or abnormal cranial magnetic resonance imaging. Growth outcomes and bone health indices were assessed over 12 months using auxology, insulin-like growth factor 1 (IGF-1) levels, and BoneXpert-derived bone health index (BHI) SDS and Metacarpal Index (MCI) SDS.</p><p><strong>Results: </strong>The study cohort consisted of 39 children with GHD. After 12 months of therapy, mean height SDS improved significantly from -2.16±0.80 to -1.65±0.71 (p<0.001). IGF-1 SDS rose from -1.38±1.02 to 0.88±1.57 (p<0.001). Adult predicted height and BHI SDS also improved significantly (p=0.005 and p<0.001, respectively). No significant changes were observed in bone age SDS or MCI SDS.</p><p><strong>Conclusion: </strong>Weekly Somatrogon significantly improved linear growth, IGF-1 levels, and measures of cortical bone health without advancing bone age in children with GHD. These findings support the efficacy of this form of long-acting GH therapy and its potential to optimize growth and skeletal outcomes in clinical practice.</p>","PeriodicalId":48805,"journal":{"name":"Journal of Clinical Research in Pediatric Endocrinology","volume":" ","pages":"254-260"},"PeriodicalIF":1.9,"publicationDate":"2026-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13325584/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145193605","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}