Hormone Research in Paediatrics最新文献

{"title":"Management of Arginine Vasopressin Deficiency (Central Diabetes Insipidus) in Neonates and Infants.","authors":"Hannah Pearlstein, Allie Dayno, Jessica Zook, Julia Crowley, Craig Alter, Iris Gutmark-Little, Shana E McCormack","doi":"10.1159/000547155","DOIUrl":"10.1159/000547155","url":null,"abstract":"<p><strong>Background: </strong>Arginine vasopressin deficiency (AVP-D), previously called central diabetes insipidus (central DI), is the inability to concentrate urine despite elevated serum osmolality (i.e., volume depletion) related to inadequate production of the posterior pituitary hormone vasopressin. Without treatment, which typically consists of fluids and pharmacologic vasopressin analogs, AVP-D can quickly lead to hypernatremia and dehydration. Management of AVP-D in neonates and infants is particularly challenging for many reasons: their inability to communicate thirst, their limited renal concentrating capacity, the obligate fluids required for nutrition that may cause hyponatremia with anti-diuretic therapy, the lack of FDA-approved formulation of vasopressin analog in this age, the potential need for growth-related adjustments in nutrition, fluids, and vasopressin analogs, and a limited evidence base. Despite these challenges, multiple groups have reported experiences with the available pharmacologic options, including alternative formulations of desmopressin (buccal, standard oral tablet, orally disintegrating tablet [melt], subcutaneous) and thiazide diuretics.</p><p><strong>Summary: </strong>The objective of this mini-review was to provide pragmatic guidance on the options for long-term outpatient management of AVP-D in neonates and infants.</p><p><strong>Key messages: </strong>Management of AVP-D in neonates and infants necessitates special considerations. For each affected patient and family, weighing the relative merits and drawbacks of each approach is critical to identify the most appropriate option, which may also change over time.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-11"},"PeriodicalIF":2.7,"publicationDate":"2025-07-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144553383","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}

{"title":"IDEAL: A Comprehensive Virtual Training Program for Pediatric Diabetes Educators in Low-Resource Settings - Structure, Strengths, and Challenges.","authors":"Anju Virmani, Sirisha K Boddu, Preeti Singh, Sheryl S Salis, Santhosh S Olety, Rakesh Kumar, Aspi J Irani, Ganesh Jevalikar, Shaila Bhattacharyya","doi":"10.1159/000547140","DOIUrl":"10.1159/000547140","url":null,"abstract":"<p><strong>Introduction: </strong>Pediatric diabetes educators (PDEs) are scarce in low-resource settings (LRSs), compromising diabetes care and increasing morbidity and mortality.</p><p><strong>Methods: </strong>The Indian Society for Pediatric &amp; Adolescent Endocrinology (ISPAE) developed ISPAE Diabetes Education And Learning (IDEAL), a 12-week virtual program with 24 interactive sessions of 2 h each, 58 faculty members, practical assignments, a rigorous exit exam, and ongoing post-certification engagement via WhatsApp.</p><p><strong>Results: </strong>Since October 2021, 177 PDEs (128 nonphysicians, 49 physicians) have been trained in 8 batches, 9th batch completing. Teaching is in English, but assignments are accepted in 8 Indian languages. A total of 91% of trainees were women, 24% were persons with type 1 diabetes or their parents, and 50% were from smaller cities. Engagement was high, with a 91% attendance rate. Post-session test scores improved significantly (p < 0.05). IDEAL received the ISPAD Innovation Award (2023) and ISPAD endorsement. Eighty-five of the 177 IDEAL alumni (\"IDEALites\") completed a post-course survey. Of these, 88% are actively contributing to pediatric diabetes care and earning recognition and awards for their efforts.</p><p><strong>Challenges: </strong>This study has the following limitations: limited hands-on experience, a demanding program, and language barriers.</p><p><strong>Conclusion: </strong>IDEAL is a pioneering, structured, intensive, virtual, award-winning PDE training program. Being accessible and sustainable, it can serve as a practical model for other programs in LRS.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-9"},"PeriodicalIF":2.6,"publicationDate":"2025-06-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144505556","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}

{"title":"International Consensus Guideline on the Diagnosis and Management of Endocrine Complications of β and α Thalassemia in Children and Adolescents.","authors":"Abdelhadi Habeb, Asma Deeb, Rasha T Hamza, Lorenzo Iughetti, Muhammad Yazid Jalaludin, Kandi-Catherine Muze, Elizabeth E Oyenusi, Christine Rodda, Preeti Singh, Nicos Skordis, Ashraf T Suliman, Maria G Vogiatzi, Mohammed Zolaly, Evangelia Charmandari","doi":"10.1159/000546904","DOIUrl":"10.1159/000546904","url":null,"abstract":"<p><p>β thalassemia (βT) and α thalassemia (αT) are chronic hemolytic anemias caused by hereditary defects in the β or α chains of hemoglobin, respectively. According to the clinical picture, both forms of thalassemia are subdivided into minor, intermedia, or major. Previous guidelines focused on growth and endocrine dysfunctions in βT major, where the complications reported are consequences of iron toxicity. However emerging evidence shows that patients with other forms of thalassemia are also at risk of some endocrinopathies. This guideline provides consensus on the screening and management of endocrine complications of children and adolescents with different forms of thalassemia. The panel has 14 experts from 13 countries representing 8 societies. They reviewed literature up to 2024 for the highest available evidence on the subject and 42 recommendations were modified until at least 70% vote for agreement was achieved. Hypogonadism, delayed growth, and puberty are common in βT major and transfusion-dependent (TD) αT HbH disease and they are also reported in βT intermedia and non-TD αT HbH disease. Osteopenia, adrenal insufficiency, and reproductive dysfunction are reported only in βT major and TD αT HbH disease. In addition, hypothyroidism, diabetes, and hypoparathyroidism are also reported in TD and non-TD thalassemia. Adherence to modern transfusion and iron chelation can prevent or reverse endocrine complications. Regular screening should be conducted before the age of 10 years in patients with TD thalassemia and from 11 years onward in non-TD thalassemia. Those who received hematopoietic stem cell transplantation for βT major are at risk of endocrinopathies and should be managed similarly to individuals with TD thalassemia.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-24"},"PeriodicalIF":2.7,"publicationDate":"2025-06-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144484091","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}

{"title":"Little Loopers: A Case Series of Automated Insulin Delivery Usage with Standard and Diluted Insulin in Very Young Children with Diabetes Mellitus.","authors":"Benjamin G Fisher, Julia Ware, Paul Geetha Paul Nicholsion, Jennifer Ashford, Helen Hysted, Cliodhna Myles, Eilidh Nicol, M Loredana Marcovecchio, Rachel M Williams","doi":"10.1159/000547035","DOIUrl":"10.1159/000547035","url":null,"abstract":"<p><strong>Introduction: </strong>Management of diabetes mellitus in very young children presents challenges due to variable insulin sensitivity, unpredictable carbohydrate intake, and low insulin requirements. An automated insulin delivery (AID) system addresses some of these challenges and can be used with diluted insulin where indicated.</p><p><strong>Methods: </strong>Retrospective case series of children aged <6 years with diabetes starting CamAPS FX AID with standard (U100) or diluted (U5 or U10) insulin at a single UK clinical centre between October 2020 and April 2022.</p><p><strong>Results: </strong>AID was started for seven children with diluted insulin (median interquartile range [IQR] age 1.5 [0.6, 2.8] years, mean ± standard deviation HbA1c 83 ± 18 mmol/mol) and four with standard insulin (age 4.6 [3.9, 5.4] years, HbA1c 62 ± 13 mmol/mol). AID was started at a median (IQR) of 0.2 (0.1, 0.2) months post-diagnosis in the diluted group and 17.8 (7.7, 23.6) months in the standard group. At the most recent clinic visit (9.3 ± 4.8 months after starting AID in the diluted group and 12.0 ± 2.1 months in the standard group), time in target range (3.9-10.0 mmol/L) was 66.5 ± 6.8% and 54.0 ± 5.0%, respectively. Median time in hypoglycaemia (<3.9 mmol/L) was <4% in both groups. Glucose variability was 37.5 ± 4.2% in the diluted and 43.5 ± 4.7% in the standard group. There were no episodes of diabetic ketoacidosis or severe hypoglycaemia.</p><p><strong>Conclusion: </strong>AID with both standard and diluted insulin can be used to safely manage diabetes in very young children with low total insulin requirements.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-7"},"PeriodicalIF":2.6,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474990","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}

{"title":"Clinical Variability in Congenital Adrenal Hyperplasia: A Distinct Subgroup with a Low Glucocorticoid Dose Requirement.","authors":"Ala Ustyol, Erica A Eugster","doi":"10.1159/000546883","DOIUrl":"10.1159/000546883","url":null,"abstract":"<p><strong>Introduction: </strong>Some children with classic congenital adrenal hyperplasia (CAH) achieve excellent control on very low glucocorticoid doses. We aimed to characterize these patients and assess the timing of their low-dose requirements.</p><p><strong>Methods: </strong>We reviewed charts of patients with salt-wasting CAH due to 21-hydroxylase deficiency, defining low-dose glucocorticoid as <10 mg/m2/day. Demographic and growth data were compared with a matched group on standard doses.</p><p><strong>Results: </strong>Among 154 patients with CAH, 14 (9%) required low-dose glucocorticoid therapy (<10 mg/m2/day), including 8 boys (57%) and 6 girls (43%). The average age at treatment initiation was 2.1 years, comparable to a matched group of 23 patients (48% boys). The low-dose group received 8.8 ± 1.2 mg/m2/day versus 14.9 ± 3.9 mg/m2/day in the matched group (p < 0.001), with similar fludrocortisone doses (0.1 ± 0.05 mg). No differences were observed in weight, height, or height velocity. Of the 14 patients on low-dose treatment, 3 experienced an increase in their glucocorticoid dose requirement above 10 mg/m2/day at ages 10.3, 10.8, and 8.5 years after being on 6.3-9.8 mg/m2/day for 6.4-8.5 years. The remaining 11 patients are currently on 5.89-10 mg/m2/day with a duration on low-dose therapy ranging from 0.48 to 8.65 years.</p><p><strong>Conclusion: </strong>Our findings highlight a subgroup of patients with 21-hydroxylase deficiency who achieve good control on low glucocorticoid doses from early childhood. The factors underlying this and the transient need for low doses in some remain unclear.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-5"},"PeriodicalIF":2.7,"publicationDate":"2025-06-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144474980","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}

{"title":"The Utility of Copeptin Measurement in Hospitalized Pediatric Patients with Syndrome of Inappropriate Antidiuretic Secretion.","authors":"Julianne Gibbons, Daina Dreimane","doi":"10.1159/000547012","DOIUrl":"10.1159/000547012","url":null,"abstract":"<p><strong>Introduction: </strong>Hyponatremia is common in hospitalized pediatric patients, and in many cases, the diagnosis of the syndrome of inappropriate antidiuretic secretion (SIADH) remains challenging, with no gold standard for diagnosis. We assessed factors associated with hyponatremia in pediatric patients clinically diagnosed with SIADH and examined the validity of copeptin level as a useful tool to distinguish SIADH from non-SIADH causes of hyponatremia.</p><p><strong>Methods: </strong>This observational study retrospectively analyzed 19 patients admitted to Children's Hospital of Orange County in 2021-2024 for hyponatremia. ROC analyses assessed the ability of copeptin level to distinguish diagnostic groups, determining the optimal threshold for classification.</p><p><strong>Results: </strong>Pediatric patients with a diagnosis of SIADH had a significantly higher average urine sodium level (135.4 vs. 68.3, p = 0.036) and higher average copeptin level (median = 14.3 vs. 5.7, p = 0.036). ROC analyses determined copeptin had good ability to differentiate a clinical diagnosis of SIADH from non-SIADH causes of hyponatremia with sensitivity 83%, specificity 71%, PVP 83%, NPV 71%. A significantly higher percentage of patients with copeptin level greater than 8.0 pmol/L were diagnosed with SIADH (83.3% vs. 28.6%, p = 0.017).</p><p><strong>Conclusion: </strong>Copeptin levels correlated with a clinical diagnosis of SIADH in hospitalized pediatric patients, particularly if elevated above 8.0 pmol/L at the time of hyponatremia, and the patient met the Schwartz and Bartter clinical criteria for SIADH. However, in some cases of SIADH, copeptin levels may be in normal range and could be considered inappropriately high for the degree of hyponatremia.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-10"},"PeriodicalIF":2.7,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12310180/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368817","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"ADECA: A Novel Course for Training Paediatric Diabetes Nurse Educators in Sub-Saharan Africa.","authors":"Juliana Chizo Agwu, Kandi Catherine Muze, Joyce Mbogo, Emmanuel Ameyaw, Debra Cohen, Anna Lindholm-Olinder, Carole Gelder, Carine de Beaufort, Graham D Ogle","doi":"10.1159/000546936","DOIUrl":"10.1159/000546936","url":null,"abstract":"<p><strong>Introduction: </strong>Care of children with diabetes is best delivered by a specialist multidisciplinary team of paediatric endocrinologists, diabetes nurse educators, dietitians, and psychologists. The Allied Healthcare Paediatric Diabetes Educator Course for Africa (ADECA) is the first specialised paediatric diabetes educator training programme for nurses working in sub-Saharan Africa. The aim of the paper was to describe the course structure and evaluation findings of the first ADECA programme.</p><p><strong>Methods: </strong>The ADECA course is a hybrid 1-year course, organised in six phases, including online modules, in-person modules, and work-based assessments. Fifteen nurses from seven sub-Saharan African countries were selected to undertake the first course. The course was evaluated using the Kirkpatrick model, which rates the results of training courses against four levels of criteria: reaction, learning, behaviour, and results.</p><p><strong>Results: </strong>All nurses successfully completed the course. Overall, 100% strongly agreed that the \"in-person\" modules were beneficial and enjoyable, compared to 87.5% of nurses for the online modules. Eighteen months following completion, the nurses are contributing to care of children and young people with diabetes and taking a lead in training other healthcare professionals. Seventy-three percent have joined national committees, with 27% actively involved in developing national guidelines and influencing policy. Forty percent have presented at either national or international scientific conferences.</p><p><strong>Conclusion: </strong>The ADECA course has successfully created a pool of competent paediatric diabetes nurse educators who can support children and their families as well as train other healthcare personnel in diabetes care and become future faculty members. This bespoke course can be adapted for use in other low-income countries.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-9"},"PeriodicalIF":2.7,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12303553/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368816","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Acute Kidney Injury at the Onset of Type 1 Diabetes Mellitus: A Balance between Kidney Stress and Nephron Mass.","authors":"Paola Tirelli, Stefano Guarino, Mariantonia Braile, Francesca Maisto, Dario Iafusco, Angela Zanfardino, Anna Di Sessa, Emanuele Miraglia Del Giudice, Grazia Cirillo, Pierluigi Marzuillo","doi":"10.1159/000547090","DOIUrl":"10.1159/000547090","url":null,"abstract":"<p><strong>Introduction: </strong>Uromodulin reflects nephron mass, while urinary neutrophil gelatinase-associated lipocalin (NGAL) indicates kidney injury. We hypothesized that low urinary uromodulin at type 1 diabetes mellitus (T1DM) onset may be linked to acute kidney injury (AKI) and that a higher urinary uromodulin-to-NGAL ratio, reflecting the balance between nephron mass and kidney stress, may reduce the risk of AKI. Our aim was to test these hypotheses.</p><p><strong>Method: </strong>In this prospective study, 75 children (mean age: 8.6 ± 4.3 years) hospitalized for new-onset T1DM were enrolled. AKI was defined as a highest-to-basal serum creatinine ratio ≥1.5. Urinary NGAL and uromodulin levels were measured at admission and upon kidney injury resolution.</p><p><strong>Results: </strong>Of 75 patients, 33 (44%) had diabetic ketoacidosis (11 severe, 10 moderate, 12 mild) and 33 (44%) developed AKI. At T1DM onset, patients with AKI had similar urinary uromodulin levels but higher urinary NGAL levels, higher uromodulin-to-creatinine ratio, and lower urinary uromodulin-to-NGAL ratio than those without AKI. The uromodulin-to-NGAL ratio correlated inversely with the highest-to-basal creatinine ratio (r = -0.42; p < 0.001), while NGAL correlated positively (r = 0.36; p = 0.001). Both urinary NGAL and the uromodulin-to-NGAL ratio at T1DM onset predicted the absence of AKI, with an areas under the receiver-operating characteristic curve of 0.66 (95% confidence interval [CI]: 0.54-0.79; p = 0.01) and 0.75 (95% CI: 0.63-0.86; p < 0.001), respectively.</p><p><strong>Conclusion: </strong>Our findings suggest a potential interplay between nephron mass and kidney stress in AKI development at T1DM onset. While uromodulin levels alone were not associated with AKI, a higher urinary uromodulin-to-NGAL ratio - possibly reflecting better-preserved nephron mass under stress - may be linked to a reduced risk of AKI. Further confirmation is needed.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-6"},"PeriodicalIF":2.6,"publicationDate":"2025-06-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144368815","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}

{"title":"Polygenic Childhood Obesity: Integrating Genetics and Environment for Early Intervention.","authors":"Jorrit van Uhm, Elisabeth F C van Rossum, Mieke M van Haelst, Philip R Jansen, Erica L T van den Akker","doi":"10.1159/000546951","DOIUrl":"10.1159/000546951","url":null,"abstract":"<p><strong>Background: </strong>Childhood obesity is a global health challenge driven by a complex interplay of genetic predispositions and environmental exposures. Genome-wide association studies have identified many obesity-associated loci, and polygenic risk scores (PRS) enable quantification of genetic susceptibility. Concurrently, lifestyle factors - including diet, physical activity, sleep, stress, and socioeconomic status - modify these genetic risks.</p><p><strong>Summary: </strong>Healthy lifestyle practices can mitigate genetic risk, while unhealthy diets and sedentary habits amplify it. The review details how PRS, by capturing the cumulative effect of numerous small-effect variants, facilitate risk stratification in children. Furthermore, gene-environment interactions - from diet and exercise to sleep, stress, and socioeconomic conditions - might inform personalized intervention strategies, including tailored nutritional guidance, behavior modification, and targeted physical activity interventions initiated early.</p><p><strong>Key message: </strong>Understanding gene-environment interactions is essential for refining risk assessments and developing personalized, equitable public health strategies. Future research should focus on enhancing multi-ancestry PRS accuracy, elucidating underlying biological pathways, and translating genetic insights into actionable, context-specific interventions to combat childhood obesity.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-9"},"PeriodicalIF":2.7,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12266691/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144316787","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Endocrine Comorbidities in Survivors of Childhood Brain Tumors: Insights from the Slovenian National Cohort.","authors":"Sončka Jazbinšek, Barbara Faganel Kotnik, Lidija Kitanovski, Lorna Zadravec Zaletel, Tadej Battelino, Primož Kotnik","doi":"10.1159/000546392","DOIUrl":"10.1159/000546392","url":null,"abstract":"<p><strong>Introduction: </strong>Endocrine disorders present a major comorbidity in pediatric brain tumors and/or treatment-related damage, which results in impaired function of the hypothalamic-pituitary axes. The aim of the study was to assess the prevalence of endocrine disorders among a complete childhood brain tumor survivor cohort treated between 2008 and 2018 at our national center.</p><p><strong>Methods: </strong>Children with primary brain tumors treated at the University Children's Hospital, University Medical Center Ljubljana, between 2008 and 2018 were included and evaluated by the endocrinologist in the years 2023/2024. Data on demographics, anthropometrics, tumor type, and therapy were gathered. The presence of endocrinopathies was determined by clinical examination and laboratory data.</p><p><strong>Results: </strong>A total of 94 patients (mean age at the most recent follow-up 14.9 +/- 5.5 years, mean follow-up duration 8 +/- 3 years) were included in the study. At the time of final follow-up, 23% were diagnosed with an endocrine disorder. The most prevalent were hyposomatotropism, followed by central hypothyroidism, with panhypopituitarism affecting 10% of the cohort. Endocrine dysfunction was more frequently observed in survivors with tumors located in the suprasellar region, those who underwent radiotherapy, and those who presented with hydrocephalus. A significant decrease in height SDS was noted compared to baseline height at the time of treatment initiation (p < 0.02), with a more pronounced reduction among those who received craniospinal radiotherapy as part of the oncological treatment.</p><p><strong>Conclusion: </strong>The findings highlight a high prevalence of endocrine disorders in childhood brain tumor survivors, particularly those with suprasellar or posterior fossa tumors, or those treated with radiotherapy. These results emphasize the need for regular and ongoing endocrine monitoring in this patient population following the completion of oncological treatment.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-10"},"PeriodicalIF":2.6,"publicationDate":"2025-06-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144301976","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}