Hormone Research in Paediatrics最新文献

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A dual etiology of neonatal hypoglycemia secondary to FOXA2 heterozygous deletion. 新生儿低血糖继发于FOXA2杂合缺失的双重病因。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-26 DOI: 10.1159/000546342
Margaux Stamm, Carine Villanueva, Lucie Bazus, Sara Cabet, Marc Nicolino, Kevin Perge
{"title":"A dual etiology of neonatal hypoglycemia secondary to FOXA2 heterozygous deletion.","authors":"Margaux Stamm, Carine Villanueva, Lucie Bazus, Sara Cabet, Marc Nicolino, Kevin Perge","doi":"10.1159/000546342","DOIUrl":"https://doi.org/10.1159/000546342","url":null,"abstract":"<p><p>Neonatal hypoglycemia is a critical pediatric condition that requires prompt diagnosis and treatment to prevent long-term neurological damage. This case study reports a female newborn with recurrent hypoglycemia within hours of birth. There was no family history of hypoglycemia or diabetes. The pregnancy was unremarkable, with no gestational diabetes and no abnormalities were detected on prenatal ultrasounds. The girl was born at 36 weeks of gestation by emergency caesarean section due to an abnormal fetal heart rate and with normal parameters. A biological assessment carried out at the time of hypoglycemia revealed somatotropic, thyrotropic and corticotropic insufficiencies. A brain MRI showed hypoplasia of the anterior pituitary gland and interruption of the pituitary stalk, as well as an ectopic posterior pituitary gland on the floor of the third ventricle. Altogether, these findings led to the diagnosis of congenital panhypopituitarism secondary to pituitary stalk interruption syndrome. Hormone replacement therapy was initiated. Despite that treatment, hypoglycemia persisted, new assessments revealed congenital hyperinsulinism, and treatment with diazoxide was initiated, leading to stabilization of blood glucose levels. Genetic testing identified a FOXA2 heterozygous deletion resulting in both congenital hyperinsulinism and congenital panhypopituitarism. The role of the FOXA2 gene in both conditions highlights the need for awareness and precise diagnosis to ensure timely and appropriate treatment. This case underscores the complexity of neonatal hypoglycemia, where dual endocrine pathologies can co-exist, necessitating comprehensive and careful evaluation.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-13"},"PeriodicalIF":2.6,"publicationDate":"2025-05-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144150281","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Hepatic Steatosis and Stiffness in Adolescent Obesity are Linked to Androgenemia, Insulin Sensitivity, and Inflammation. 青少年肥胖中的肝脂肪变性和硬化与雄激素血症、胰岛素敏感性和炎症有关。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-23 DOI: 10.1159/000546577
Mary Ellen Vajravelu, Nabiha Shahid, Marianne Chebli, Theresa Stauffer, James E Squires, Sameh Tadros, Selma F Witchel, Silva Arslanian
{"title":"Hepatic Steatosis and Stiffness in Adolescent Obesity are Linked to Androgenemia, Insulin Sensitivity, and Inflammation.","authors":"Mary Ellen Vajravelu, Nabiha Shahid, Marianne Chebli, Theresa Stauffer, James E Squires, Sameh Tadros, Selma F Witchel, Silva Arslanian","doi":"10.1159/000546577","DOIUrl":"https://doi.org/10.1159/000546577","url":null,"abstract":"<p><strong>Introduction: </strong>Metabolic dysfunction-associated steatotic liver disease (MASLD) is driven by obesity and is common among girls with polycystic ovary syndrome (PCOS). Higher testosterone concentrations are associated with more severe histological MASLD features in girls, but less severe in boys. This proof-of-concept study tested the hypotheses that MRI-based hepatic fat and stiffness are higher among girls with PCOS versus girls without PCOS but not boys and are associated with testosterone concentration (differing by sex), insulin sensitivity, and inflammation.</p><p><strong>Methods: </strong>This proof-of-concept cross-sectional study at an academic pediatric center included pubertal girls (n=25; 10 without PCOS, 15 with PCOS) and boys (n=10) with obesity, ages 12-18 years. Outcomes were (primary) MRI hepatic fat fraction (HFF %) and stiffness and (secondary) insulin sensitivity index (ISI), 2-hour OGTT and inflammatory markers.</p><p><strong>Results: </strong>HFF was higher in girls with PCOS versus those without but not different from boys. Stiffness did not differ by group. HFF and stiffness were both directly associated with OGTT 2-hour glucose and inversely with ISI. Stiffness was directly associated with testosterone (total, free) in girls without PCOS and with interleukin (IL)-6 and IL-18 in the full cohort.</p><p><strong>Conclusions: </strong>HFF was higher in girls with PCOS versus those without, but not different from boys. While stiffness did not differ by group, its association with testosterone concentrations in girls without PCOS may be an additional risk biomarker for MASLD in addition to inflammation and insulin resistance. Future larger, longitudinal studies should determine if addressing these alterations, separately or collectively, in youth with obesity could improve hepatic outcomes.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-20"},"PeriodicalIF":2.6,"publicationDate":"2025-05-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144142435","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Exploring the Heterogeneity of Pediatric Neuroendocrine Tumors: Single-Center Experience. 探索儿童神经内分泌肿瘤的异质性:单中心经验。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-20 DOI: 10.1159/000546507
Cansu Koc, Yaprak Ece Yola Atalah, Sedat Bayrakoglu, Melek Yildiz, Semen Onder, Gulcin Yegen, Basak Erginel, Nihat Aksakal, Yasemin Giles, Asli Derya Kardelen, Sukran Poyrazoglu, Firdevs Bas, Feyza Darendeliler
{"title":"Exploring the Heterogeneity of Pediatric Neuroendocrine Tumors: Single-Center Experience.","authors":"Cansu Koc, Yaprak Ece Yola Atalah, Sedat Bayrakoglu, Melek Yildiz, Semen Onder, Gulcin Yegen, Basak Erginel, Nihat Aksakal, Yasemin Giles, Asli Derya Kardelen, Sukran Poyrazoglu, Firdevs Bas, Feyza Darendeliler","doi":"10.1159/000546507","DOIUrl":"https://doi.org/10.1159/000546507","url":null,"abstract":"<p><strong>Introduction: </strong>Neuroendocrine tumors (NETs) are rare, slow-growing tumors with distinct clinical manifestations that can be challenging to diagnose and treat, particularly in children. This retrospective study aimed to improve diagnosis and treatment of NETs by evaluating their clinical course, hormonal activity, and long-term effects in children.</p><p><strong>Methods: </strong>A retrospective study was conducted on 26 pediatric patients diagnosed with NETs between 2000 and 2024. The cohort included patients with pheochromocytoma and paraganglioma (PPGL), gastroenteropancreatic neuroendocrine tumors (GEP-NETs) medullary thyroid carcinoma (MTC), C cell hyperplasia. The data included demographic, clinical, laboratory, and imaging results.</p><p><strong>Results: </strong>The study cohort included 26 patients (57.6% male). The median age for diagnosis of NETs was 8.8 (range: 1.0-16.6) years. Among the NETs, 15.4% were GEP-NETs, 15.4% were PPGL, 15.4% were C cell hyperplasia and 53.8% were MTC. The median follow-up was 5 (range: 1-14) years. Among the patients in the study cohort, only one patient died due to MTC. Genetic analysis revealed mutations in the RET gene in 69.2% of patients, mutations in the VHL gene in 3.8%, and no mutations in 3.8%. 23% of the patients did not undergo genetic testing.</p><p><strong>Conclusion: </strong>Early diagnosis, genetic screening, and long-term follow-up are crucial in managing pediatric NETs to reduce morbidity and mortality. Genetic testing is essential for identifying comorbidities and screening family members at risk.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-13"},"PeriodicalIF":2.6,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110377","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Type 1 Diabetes Polygenic Scores Improve Diagnostic Accuracy in Pediatric Diabetes Care. 1型糖尿病多基因评分提高儿童糖尿病诊断准确性
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-20 DOI: 10.1159/000546445
Raymond J Kreienkamp, Aaron J Deutsch, Alicia Huerta-Chagoya, Erin M Borglund, Jose C Florez, Jason Flannick, Miriam S Udler
{"title":"Type 1 Diabetes Polygenic Scores Improve Diagnostic Accuracy in Pediatric Diabetes Care.","authors":"Raymond J Kreienkamp, Aaron J Deutsch, Alicia Huerta-Chagoya, Erin M Borglund, Jose C Florez, Jason Flannick, Miriam S Udler","doi":"10.1159/000546445","DOIUrl":"10.1159/000546445","url":null,"abstract":"<p><strong>Background: </strong>Accurately classifying pediatric diabetes can be challenging for providers, and misclassification can result in suboptimal care. In recent years, type 1 diabetes (T1D) polygenic scores, which quantify one's genetic risk for T1D based on T1D risk allele burden, have been developed with good discriminating capacity between T1D and not-T1D. These tools have the potential to improve significantly diagnostic provider accuracy if used in clinic.</p><p><strong>Methods: </strong>We applied T1D polygenic scores to a group of pediatric patients (n=1846) with genetic data available in the Boston Children's Hospital PrecisionLink Biobank, including 96 individuals diagnosed with T1D.</p><p><strong>Results: </strong>Patients with a clinical diagnosis of T1D had higher T1D polygenic scores compared to controls (Wilcoxon rank-sum P<0.0001). Sixty-nine of the 74 individuals with diabetes and a T1D polygenic score exceeding an externally validated cutoff for distinguishing T1D from not-T1D were confirmed to have T1D. There were multiple cases where T1D polygenic scores would have clinical utility. An elevated T1D polygenic score suggested T1D in a pancreatic autoantibody (PAA)- negative individual with negative MODY genetic testing and a phenotype matching T1D. A low T1D polygenic score accurately indicated atypical diabetes in an individual found to have HNF1B-MODY. One individual had positive PAA, but the provider noted that the patient may not have classic T1D, as later suggested by a low T1D polygenic score.</p><p><strong>Conclusion: </strong>T1D polygenic scores already have clinical utility to aid in the accurate diagnosis of pediatric diabetes. Efforts are now needed to advance their use in clinical practice.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-17"},"PeriodicalIF":2.6,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110463","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Obesity in Childhood and Adolescence: Epidemiology and Financial Implications. 儿童和青少年肥胖:流行病学和经济意义。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-20 DOI: 10.1159/000546506
Konstantinos Zisis, Kostas Athanasakis
{"title":"Obesity in Childhood and Adolescence: Epidemiology and Financial Implications.","authors":"Konstantinos Zisis, Kostas Athanasakis","doi":"10.1159/000546506","DOIUrl":"https://doi.org/10.1159/000546506","url":null,"abstract":"<p><strong>Objective: </strong>Childhood obesity is a growing global health concern with significant epidemiological and economic consequences. This study aims to provide an overview of the prevalence, socioeconomic determinants, and financial burden of childhood obesity, emphasizing its impact on healthcare systems and society.</p><p><strong>Methods: </strong>A non-systematic targeted literature review was conducted using PubMed and institutional sources such as the World Health Organization (WHO), Centers for Disease Control and Prevention (CDC), and Organization for Economic Co-operation and Development (OECD). Two independent reviewers screened and extracted relevant data, which were categorized into three domains: epidemiology (prevalence and trends), socioeconomic determinants (income, education, ethnicity, and family factors), and economic impact (direct, indirect, and intangible costs).</p><p><strong>Findings: </strong>Childhood obesity prevalence has been rising globally, with higher rates observed in high-income countries. Socioeconomic disparities play a crucial role, with lower-income families and certain ethnic groups experiencing higher obesity rates. Direct costs include increased medical expenses due to obesity-related comorbidities, while indirect costs result from productivity losses and long-term healthcare needs. Intangible costs, such as psychological distress and reduced quality of life, further highlight the burden of childhood obesity. Economic evaluations indicate that childhood obesity leads to substantial healthcare expenditures and productivity losses over a lifetime, emphasizing the need for early intervention.</p><p><strong>Conclusion: </strong>Childhood obesity imposes a significant public health and economic burden, necessitating urgent policy interventions. A multi-sectoral approach, including public health strategies, socioeconomic support, and cost-effective interventions, is essential to mitigate its impact.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-10"},"PeriodicalIF":2.6,"publicationDate":"2025-05-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144110461","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Co-occurrence of DNET and Hodgkin's Lymphoma in a patient with Noonan syndrome and mutation in the PTPN11 gene. Noonan综合征患者并发DNET和霍奇金淋巴瘤,PTPN11基因突变。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-17 DOI: 10.1159/000546262
Caroline Rosa Pellicciari, Adriana Aparecida Siviero Miachon, Angela Maria Spinola E Castro, Andrea Maria Cappellano, Nasjla Saba da Silva, Frederico Adolfo Benevides Silva, Flavio Augusto Vercillo Luisi, Alexander A L Jorge, Alexsandra C Malaquias
{"title":"Co-occurrence of DNET and Hodgkin's Lymphoma in a patient with Noonan syndrome and mutation in the PTPN11 gene.","authors":"Caroline Rosa Pellicciari, Adriana Aparecida Siviero Miachon, Angela Maria Spinola E Castro, Andrea Maria Cappellano, Nasjla Saba da Silva, Frederico Adolfo Benevides Silva, Flavio Augusto Vercillo Luisi, Alexander A L Jorge, Alexsandra C Malaquias","doi":"10.1159/000546262","DOIUrl":"https://doi.org/10.1159/000546262","url":null,"abstract":"<p><p>Noonan syndrome (NS) is a genetic disorder which belongs to the RASopathy family, characterized by craniofacial dysmorphisms, short stature, congenital heart defects, and an increased predisposition to malignancies. Although hematologic malignancies, neuroblastomas and certain solid tumors have been documented in NS, the co-occurrence of dysembryoplastic neuroepithelial tumor (DNET) and Hodgkin's lymphoma, has not been previously reported in the literature. We present the case of an 11-year-old boy with NS caused by a pathogenic variant in the PTPN11 gene who developed both a DNET and Hodgkin's Lymphoma. Notably, the patient had been receiving recombinant human growth hormone (rhGH) therapy prior to tumor diagnosis, raising concerns about potential contributing factors. Through a literature review, we identified reports of DNETs and lymphomas in patients with NS, highlighting the variability in genetic mutations and clinical presentations. However, no predominant PTPN11 variant was associated with a specific tumor predisposition. This case underscores the complex relationship between NS and tumor development, reinforcing the importance of individualized surveillance strategies, particularly in patients undergoing rhGH therapy. Further studies are needed to clarify the oncogenic potential of specific NS-associated mutations and to establish evidence-based guidelines for cancer surveillance in these patients.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-15"},"PeriodicalIF":2.6,"publicationDate":"2025-05-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093357","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Persistent hyperparathyroidism in vitamin D-dependent rickets type 2A does not prevent normalization of hypophosphatemia or healing of the rickets. 维生素d依赖性2A型佝偻病患者持续甲状旁腺功能亢进并不妨碍低磷血症的正常化或佝偻病的愈合。
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-16 DOI: 10.1159/000546444
Maria Lodefalk, Ola Nilsson
{"title":"Persistent hyperparathyroidism in vitamin D-dependent rickets type 2A does not prevent normalization of hypophosphatemia or healing of the rickets.","authors":"Maria Lodefalk, Ola Nilsson","doi":"10.1159/000546444","DOIUrl":"https://doi.org/10.1159/000546444","url":null,"abstract":"<p><p>Introduction Vitamin D-dependent rickets type 2A (VDDR2A) is a rare, autosomal recessive disorder caused by pathogenic variants of the VDR gene encoding the vitamin D receptor. It has been proposed to be a form of parathyroid hormone (PTH)-dependent rickets. Here we describe in detail a girl with VDDR2A who developed a long-standing, tertiary hyperparathyroidism that did not prevent healing of the rickets nor normalization of hypophosphatemia. Case Presentation A girl who presented with seizures at 9 months of age was diagnosed with VDDR2A. She had poor growth, alopecia, severe hypocalcemia, hypophosphatemia, elevated levels of alkaline phosphatase (ALP), PTH and 1,25-dihydroxyvitamin D, and severe rickets. Genetic studies revealed a novel homozygous microdeletion that included exon 9 of the VDR gene. She responded only partially to high oral doses of calcium, cholecalciferol and alfacalcidol. Upon the initiation of IV calcium infusions, bone pain resolved, and the rickets healed within weeks. In parallel with decreasing ALP values, her phosphate levels normalized even though her PTH levels remained markedly elevated. PTH levels remained elevated for approximately 1 year after the normalization of S-Ca2+. Calcium infusions, despite rendering her mildly hypercalcemic, mostly failed to suppress her PTH into the normal range, consistent with tertiary hyperparathyroidism. The hyperparathyroidism eventually resolved spontaneously with continued high oral doses of calcium, cholecalciferol and alfacalcidol, which promoted sustained normocalcemia without the need for either cinacalcet or surgery. Conclusion Persistent tertiary hyperparathyroidism can develop in children with VDDR2A, but does not seem to prevent the healing of rickets nor normalization of hypophosphatemia. High doses of calcium, preferably administered intravenously, seem to be sufficient for the healing of rickets. We speculate that IV calcium compared to oral calcium increases intestinal phosphorus uptake, and once rickets has healed, improved appetite and dietary phosphorus intake together with reduced phosphorus demands due to saturated bones contribute to the normalization of phosphate levels despite persistent hyperparathyroidism.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-16"},"PeriodicalIF":2.6,"publicationDate":"2025-05-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144093461","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Somapacitan is Effective and Well Tolerated in Chinese Children with Growth Hormone Deficiency: a Randomised Controlled Phase 3 Study. Somapacitan对中国生长激素缺乏症儿童有效且耐受性良好:一项随机对照3期研究
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-05-05 DOI: 10.1159/000545786
Junfen Fu, Xinran Cheng, Michael Højby, Chunxiu Gong, Tina Lund Leunbach, Yanhong Li, Haiyan Wei, Yu Zhu, Yining Zhang, Yan Zhong
{"title":"Somapacitan is Effective and Well Tolerated in Chinese Children with Growth Hormone Deficiency: a Randomised Controlled Phase 3 Study.","authors":"Junfen Fu, Xinran Cheng, Michael Højby, Chunxiu Gong, Tina Lund Leunbach, Yanhong Li, Haiyan Wei, Yu Zhu, Yining Zhang, Yan Zhong","doi":"10.1159/000545786","DOIUrl":"https://doi.org/10.1159/000545786","url":null,"abstract":"<p><strong>Introduction: </strong>REAL6 is a randomized, multi-centre, open-labelled, active-controlled parallel group phase 3 trial (NCT04970654) investigating once-weekly somapacitan, a reversible albumin-binding growth hormone (GH) derivative, in Chinese children with GH deficiency (GHD).</p><p><strong>Methods: </strong>110 treatment-naïve patients were randomized 2:1 to somapacitan (0.16 mg/kg/week) or daily GH (Norditropin®; 0.034 mg/kg/day) for 52 weeks. Annualized height velocity (HV; cm/year) at week 52 was the primary measurement. Additional assessments included HV standard deviation score (SDS), height SDS, bone age, insulin-like growth factor-I (IGF¬ I) SDS, and observer-reported outcomes.</p><p><strong>Results: </strong>Estimated mean HV at week 52 was 11.0 and 10.4 cm/year for somapacitan and daily GH, respectively. Non-inferiority of somapacitan compared to daily GH was confirmed. Changes in HVSDS, height SDS, bone age, and IGF-I SDS from baseline to week 52 were similar between groups. At week 52, mean (SD) IGF-I SDS was within intended reference range (-2.0 to +2.0) and comparable between groups: +0.5 (1.4) for somapacitan versus +0.1 (1.2) for daily GH. Somapacitan was well-tolerated with a safety profile consistent with the well-known safety profile of daily GH. A low proportion of injection-site reactions were reported for somapacitan (2.7%), with no reports of injection-site pain during the 52-week treatment period. Disease burden was reduced from baseline to week 52 for both treatments. Somapacitan reduced treatment burden compared to daily GH.</p><p><strong>Conclusions: </strong>Efficacy and safety profiles were comparable for Chinese children with GHD treated with somapacitan or daily GH. Both treatments similarly reduced disease burden, while treatment burden was reduced with somapacitan.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-21"},"PeriodicalIF":2.6,"publicationDate":"2025-05-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017350","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Endocrine-Related Adverse Conditions in Pediatric Patients Treated with Immune Checkpoint Inhibitors: a Position Statement from the Clinical Practice Committee of the European Society for Pediatric Endocrinology (ESPE). 接受免疫检查点抑制剂治疗的儿科患者的内分泌相关不良状况:欧洲儿科内分泌学会(ESPE)临床实践委员会的立场声明
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-04-29 DOI: 10.1159/000546146
Christa E Flück, Dulanjalee Kariyawasam, Francesco Ceppi, Shlomit Shalitin, Kanetee Busiah
{"title":"Endocrine-Related Adverse Conditions in Pediatric Patients Treated with Immune Checkpoint Inhibitors: a Position Statement from the Clinical Practice Committee of the European Society for Pediatric Endocrinology (ESPE).","authors":"Christa E Flück, Dulanjalee Kariyawasam, Francesco Ceppi, Shlomit Shalitin, Kanetee Busiah","doi":"10.1159/000546146","DOIUrl":"https://doi.org/10.1159/000546146","url":null,"abstract":"","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-5"},"PeriodicalIF":2.6,"publicationDate":"2025-04-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143990184","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Numeracy skills and glycemic control in an observational, multi-centre, cross-sectional, and international study of children with Type 1 Diabetes. 一项观察性、多中心、横断面和国际1型糖尿病儿童研究:计算技能和血糖控制
IF 2.6 3区 医学
Hormone Research in Paediatrics Pub Date : 2025-04-24 DOI: 10.1159/000545419
Ioanna Kosteria, Przemyslawa Jarosz-Chobot, Carine de Beaufort, Timothy G Barrett, Marianne Becker, Fergus Cameron, Luis A Castaño, Cintia Castro-Correia, Mark Palmert, Joanna Polanska, Stefan Särnblad, Timothy C Skinner, Jannet Svensson
{"title":"Numeracy skills and glycemic control in an observational, multi-centre, cross-sectional, and international study of children with Type 1 Diabetes.","authors":"Ioanna Kosteria, Przemyslawa Jarosz-Chobot, Carine de Beaufort, Timothy G Barrett, Marianne Becker, Fergus Cameron, Luis A Castaño, Cintia Castro-Correia, Mark Palmert, Joanna Polanska, Stefan Särnblad, Timothy C Skinner, Jannet Svensson","doi":"10.1159/000545419","DOIUrl":"https://doi.org/10.1159/000545419","url":null,"abstract":"<p><strong>Introduction: </strong>This study examined the possible association between numeracy skills and glycemic outcomes in children with type 1 diabetes.</p><p><strong>Methods: </strong>The study used a cross-sectional design and collected data from 7 centres of the Hvidoere Study Group. HbA1c was measured centrally. Numeracy was assessed using the specific 5-item Diabetes Numeracy Test (DNT-5) and the international, general Wordless Mathematical Test (WMT). The HbA1c predictive multivariate generalised linear model was constructed using the adjusted R-squared index for model selection. Pearson's correlation coefficient was calculated between observed and predicted HbA1c levels in the training and testing datasets.</p><p><strong>Results: </strong>306 adolescents aged 12-18 (mean age 14.96 ± 1.68) years and diabetes duration of 6.57 (± 3.75) participated in this study. Numeracy skills, as assessed by the WMT but not DNT 5, predicted the HbA1c levels after adjustment for sociodemographic and clinical factors. The correlation between observed and predicted HbA1c levels was consistent in both datasets and was 0.34 (N=155) and 0.37 (N=61) for the training and test datasets, respectively (p=0.412). The effect size for the WMT-based predictive model of HbA1c adjusted for clinical and socioeconomic factors was significantly higher (p<0.05) than the single-parameter-based model.</p><p><strong>Conclusion: </strong>Numeracy, as assessed by an international general math test, is a good predictor of HbA1c in children and adolescents with type 1 diabetes. The basic and short WMT is a potentially effective tool in personalised clinical pediatric diabetes practice. Therapy planning should consider adjusting therapy to compensate for lower numeracy skills and/or training to improve the patient's numerical proficiency.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-15"},"PeriodicalIF":2.6,"publicationDate":"2025-04-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144003478","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
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