Hormone Research in Paediatrics最新文献

{"title":"Growth hormone (GH) deficiency and subsequent replacement therapy trigger differential expression of specific miRNAs in males and females: not just a matter of height.","authors":"Valentina Bianco, Gloria Ravegnini, Cecilia Catellani, Anna-Mariia Shulhai, Sabrina Angelini, Maria Elisabeth Street","doi":"10.1159/000551459","DOIUrl":"https://doi.org/10.1159/000551459","url":null,"abstract":"<p><p>Sex differences influence various physiological processes, including growth, body composition, and hormonal regulation, underscoring the importance of sex-specific therapeutic strategies. Growth hormone (GH) plays a pivotal role in growth and metabolism. While sex differences in response to GH therapy (GHT) are already documented in adults, data in pediatric patients remain sparse and often conflicting. Additionally, the role of epigenetic regulators, such as microRNAs (miRNAs), in mediating sex-specific responses to GHT has been largely unexplored. In our study, we investigated gender-specific changes in the miRNA profile that occur before and after 3 months on GHT in a cohort of prepubertal children with GH deficiency (GHD). We found that 3 miRNAs (hsa-miR-185-5p, hsa-miR-380-3p, hsa-miR-486-5p) in females and 9 miRNAs (hsa-miR-30b-5p, hsa-miR-30c-5p, hsa-miR-132-3p, hsa-miR-139-5p, hsa-miR-142-5p, hsa-miR-195-5p, hsa-miR-197-3p, hsa-miR-200c-3p, hsa-miR-340-5p) in males were significantly upregulated before the start of GHT. After 3 months, 3 miRNAs (hsa-miR-124-3p, hsa-miR-215-5p, hsa-miR-525-3p) in females and 4 (hsa-miR-30c-5p, hsa-miR-30e-5p, hsa-miR-551b-3p, hsa-miR-671-3p) in males were significantly upregulated. Performing pathway enrichment analyses, we found that the most affected pathways among others were Wnt, TGFβ, Jak-STAT, and p53 signaling pathways. Overall, in addition to the shared changes previously documented changes of miRNAs on GH treatment by our group, this study shows that prepubertal males and females with isolated idiopathic GHD have distinct miRNA profiles before and after three months on GHT suggesting gender effects that are not currently taken into consideration while treating pediatric patients.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-18"},"PeriodicalIF":2.7,"publicationDate":"2026-03-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147490814","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":"Erratum.","authors":"","doi":"10.1159/000551299","DOIUrl":"https://doi.org/10.1159/000551299","url":null,"abstract":"<p><p>The article \"Bone Health in Young Individuals with Primary Ciliary Dyskinesia: Insights from a Comparison with Cystic Fibrosis and Healthy Controls\" [Horm Res Paediatr. 2025; https://doi.org/10.1159/000549604] by Gaupmann et al. was published with the wrong open access license. The correct license of the article is CC-BY.The original article has been updated.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1"},"PeriodicalIF":2.7,"publicationDate":"2026-03-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147485548","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":"High-intensity interval training: safe and beneficial physical activity strategy in adolescents with type 1 diabetes.","authors":"Julio Soto, Katherine Rozas, Alba León, Natalia Ponce, Rosario Venegas, Julie Pelicand, Oriana Paiva, Alfonso Galderisi, Larry A Fox, Andrea Gleisner","doi":"10.1159/000551568","DOIUrl":"https://doi.org/10.1159/000551568","url":null,"abstract":"<p><strong>Introduction: </strong>Cardiovascular risk factors and depressive symptoms are common in adolescents and young adults with type 1 diabetes (T1D). While exercise improves cardiovascular health and metabolic control, its mental health benefits remain unclear. High intensity interval training (HIIT) has shown an improvement in aerobic capacity without a detrimental decline in blood glucose and home-based HIIT has reduced barriers towards exercise in adults with T1D. There is scant evidence in children with T1D.</p><p><strong>Aims: </strong>Evaluate metabolic effects of HIIT (time in range, glycemic variability, HbA1c, lipid profile, blood pressure, and body composition). Secondary outcomes included assessing aerobic capacity, strength endurance, depressive symptoms, and barriers towards physical activity.</p><p><strong>Methods: </strong>Prospective, longitudinal, multicenter study involving adolescents (aged 12 and 19) with T1D diagnosed for ≥1 year, at least Tanner stage III, HbA1c between 7-11.5%, and a total daily insulin dose ≥0.7 units/kg/day. Outcomes were assessed at baseline, 1, 3, and 3 months after intervention.</p><p><strong>Results: </strong>22 participants completed intervention (mean age: 14.32±1.62 years) showing no changes in HbA1c or time in range. Mean blood glucose significantly declined during sessions (191.61±13.69 to 159.95±13.04 mg/dL, p<0.001), with no increase in time in hypoglycemia. Body lean mass increase and fat mass decline after 3 months of training (40.84 ± 8.53 to 42.35 ± 8.34, p<0.001 and 29.94 ± 8.99 to 28.85 ± 9.20, p=0.010, respectively). Maximal oxygen consumption improved once the intervention was completed (33.86±4.00 to 36.43±3.64, <0.001). Lack of motivation and concerns regarding weather decreased after training (73% vs. 59% and 27% vs 14%, respectively).</p><p><strong>Conclusions: </strong>HIIT improved physical fitness and body composition maintaining glycemic stability, being a safe regimen to encourage physical activity in adolescents with TD1.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-20"},"PeriodicalIF":2.7,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473557","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":"Screening and Management of Adrenal Suppression in Youth - A Pediatric Endocrinology Society Survey.","authors":"Laurence Bastien, Mimi S Kim, Cathrine Constantacos, Preneet Cheema Brar, Neha Patel, Anshu Gupta, Ewa Sucha, Nicholas Mitsakakis, Alexandra Ahmet","doi":"10.1159/000551569","DOIUrl":"https://doi.org/10.1159/000551569","url":null,"abstract":"<p><strong>Background: </strong>Adrenal suppression is a potential side effect of glucocorticoid (GC) therapy. Timely diagnosis and management can significantly reduce morbidity and mortality from adrenal crisis. There is a need for pediatric-specific guidelines to provide non-endocrinologist GC prescribers with diagnostic and management tools to keep children and youth safe.</p><p><strong>Objectives: </strong>To assess the adrenal suppression screening and management practices amongst surveyed pediatric endocrinology members of the Pediatric Endocrinology Society (PES), to support the need for national pediatric adrenal suppression guidelines.</p><p><strong>Methods: </strong>A survey (19 questions, implied consent model), developed by members of the PES Adrenal Special Interest Group and the PES Drugs and Therapeutics Committee, was distributed to PES members.</p><p><strong>Results: </strong>Among 154 survey respondents, 90.9% were pediatric endocrinology attendings, predominantly working in the US (91.6%) in an academic setting (86.4%). A majority (70.1%) have seen adrenal crisis due to adrenal suppression. Only 48.1% participants follow any pediatric adrenal suppression guidelines (within which 32.5% used a local guideline); 27.9% use adult guidelines. While 52.6% reported that adrenal suppression cases are managed by both endocrinologists and non-endocrinologists, only 38.3% of respondents provide resources to non-endocrinologists. Most screen for adrenal suppression with early morning cortisol (88.3%) or a low-dose ACTH stimulation test (55.8%). Notably, 8.4% of respondents believe that use of inhaled corticosteroids is not associated with a risk of adrenal suppression. Nearly all respondents (99.4%) agree that an official PES guideline on adrenal suppression would reduce morbidity and mortality by raising awareness and standardizing management.</p><p><strong>Conclusion: </strong>Adrenal crisis due to adrenal suppression remains a preventable yet persistent threat. Screening and management practices vary widely among North American pediatric endocrinologists, underscoring the need for pediatric-specific guidelines. Establishing such guidelines would fill a critical care gap and promote a standardized approach to diagnosis and management across clinicians.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-9"},"PeriodicalIF":2.7,"publicationDate":"2026-03-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147473482","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":"Prediabetes and Type 2 Diabetes in Youth following Premature Adrenarche.","authors":"Varsha Thomas, Natalie Segev, Amy S Shah, Nancy A Crimmins","doi":"10.1159/000551458","DOIUrl":"10.1159/000551458","url":null,"abstract":"<p><strong>Introduction: </strong>Type 2 diabetes mellitus (T2DM) is more aggressive when diagnosed in childhood, and its incidence continues to rise. Premature adrenarche (PA) is often considered benign; however, emerging data suggest a complex interplay among subsequent hyperandrogenism, obesity, and insulin resistance. Our objective was to determine whether PA in childhood is a risk factor for future youth-onset prediabetes (PreDM) or T2DM.</p><p><strong>Methods: </strong>We conducted a retrospective chart review from 2011 to 2022 of youth diagnosed with benign PA, with subsequent assessment for PreDM or T2DM.</p><p><strong>Results: </strong>There were 229 patients with PA identified (60 males, 169 females), of whom 82% were overweight or obese at PA diagnosis. Follow-up hemoglobin A1c (HbA1c) were available for 90 patients (24 males, 66 females). Among these, 38% of both males and females developed PreDM, while 8% of males and 6% of females developed T2DM, yielding an overall dysglycemia frequency of 44%. In males, those with PreDM were more likely to be of non-Hispanic Black race/ethnicity compared to those with normal HbA1c (p = 0.31). In females, height, weight, and BMI at initial PA evaluation were higher in those who later developed PreDM compared to those with normal HbA1c (all p < 0.01).</p><p><strong>Conclusions: </strong>Compared with national estimates, we observed youth with PA demonstrated higher frequency of dysglycemia and obesity. While prior studies have shown increased insulin resistance in PA compared to all youth with obesity, this study is the first to demonstrate higher rates of subsequent youth-onset T2DM in this population. These findings support close longitudinal monitoring for dysglycemia in children with PA.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-7"},"PeriodicalIF":2.7,"publicationDate":"2026-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147377233","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":"Double Trouble: When Focal and Diffuse Hyperinsulinism Occur Simultaneously.","authors":"Victoria R Sanders, Katherine Lord, Winnie Sigal, Heather McKnight, Emily Wilkinson, Lauren M Mitteer, Kara E Boodhansingh, N Scott Adzick, Lisa J States, Tricia R Bhatti, Diva D De Leon","doi":"10.1159/000550909","DOIUrl":"10.1159/000550909","url":null,"abstract":"<p><strong>Introduction: </strong>Congenital hyperinsulinism (HI) may be diazoxide-responsive or diazoxide-unresponsive. Patients with diazoxide-unresponsive HI are further classified based on having the diffuse or focal form of the disease, with different management strategies associated with each form. While patients with HI typically have only one form of the disease, here we report 3 patients who had both focal and diffuse HI.</p><p><strong>Case presentation: </strong>Three patients with diazoxide-unresponsive HI were transferred to our hospital for further management. All 3 patients had genetic testing which was equivocal or initially negative. Given their diazoxide status and not wanting to miss a focal lesion that was amenable to a surgical cure, all 3 patients underwent imaging with 18 F-L 3,4-dihydroxyphenylalanine positron emission tomography which identified focal pancreatic lesions in all 3 patients. They all had surgical resection of the lesions. Biopsies of the pancreas outside of the lesions noted rare or scattered islet cell nucleomegaly, indicative of diffuse disease, and subsequent fasting studies to determine if patients were cured revealed ongoing HI. Further review of genetic results suggested a mechanism for both focal and diffuse HI occurring in each of the patients. For all 3 patients, their ongoing HI could be managed with diazoxide following removal of the focal lesion.</p><p><strong>Conclusion: </strong>Focal and diffuse HI can occur in the same patient. Following resection of focal pancreatic lesions, the patients require careful evaluations for evidence of ongoing HI, and depending on the genetic results, diazoxide may be a management option for ongoing HI in a subset of these patients.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-10"},"PeriodicalIF":2.7,"publicationDate":"2026-03-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13006072/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147377194","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":"Autoantibody-Positive versus Autoantibody-Negative Type 1 Diabetes in Children: Clinical and Biochemical Characteristics.","authors":"Roos I van Rhijn, A S Paul van Trotsenburg, Rosaline Mentink","doi":"10.1159/000550312","DOIUrl":"10.1159/000550312","url":null,"abstract":"<p><strong>Introduction: </strong>This study aimed to investigate differences in clinical and biochemical characteristics between autoantibody-positive and autoantibody-negative type 1 diabetes mellitus (T1DM) in children, to get a better understanding of autoantibody-negative T1DM.</p><p><strong>Methods: </strong>This study is a monocenter retrospective cross-sectional cohort study conducted at Diaboss, a Dutch pediatric diabetes clinic at OLVG hospital. Patients diagnosed with T1DM before the age of 18 years were included. Patients tested positive for one or more diabetes-associated autoantibodies (GADA, IA2A, ZnT8, IAA, and/or ICA) were considered autoantibody positive. Patients solely positive for anti-ICA were excluded.</p><p><strong>Results: </strong>A total of 562 patients were recruited. Eight of 562 (1.4%) patients tested positive for a monogenic cause of diabetes at a later stage and were excluded. A total of 11 of 488 (2.2%) autoantibody-positive patients tested solely positive for anti-ICA and were excluded. In 15 of 66 patients (23%) with autoantibody-negative T1DM, monogenic causes were ruled out by genetic testing. The other 51 patients (77%) were not tested. Autoantibody-negative patients with a positive family history for diabetes were more likely to be tested for monogenic causes (p = 0.002). No significant differences in demographic and clinical characteristics, diabetes presentation, and long-term diabetes regulation were found between autoantibody-positive and autoantibody-negative patients.</p><p><strong>Conclusion: </strong>In our study, autoantibody-positive and -negative T1DM patients were similar. This implies that both patient groups can be approached in the same way regarding treatment, education, and prognosis. Misdiagnosis of monogenic causes within T1DM could occur; therefore, clinicians should be aware of and perform genetic tests according to international guidelines.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-8"},"PeriodicalIF":2.7,"publicationDate":"2026-03-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13061391/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147348249","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":"Once-Weekly Lonapegsomatropin Was Efficacious and Well Tolerated in Chinese Children with Growth Hormone Deficiency: Results from a Phase 3 Randomized Trial.","authors":"Yanqin Ying, Haiyan Wei, Yan Zhong, Yanhong Li, Yu Yang, Guimei Li, Wei Gu, Xinran Cheng, Chunxiu Gong, Xu Xu, Hua Zhang, Junfen Fu, Hui Yao, Li Zhou, Xiuying Ge, Tang Li, Shunye Zhu, Yan Wang, Jing Li, Ying Li, Xiaoping Luo","doi":"10.1159/000550980","DOIUrl":"10.1159/000550980","url":null,"abstract":"<p><strong>Introduction: </strong>Lonapegsomatropin, a prodrug of somatropin, is approved for once-weekly treatment of paediatric growth hormone deficiency (GHD) in the USA and Europe. Here, we report the first trial to assess the efficacy and safety of weekly lonapegsomatropin compared to daily somatropin in treatment-naive Chinese children with GHD.</p><p><strong>Methods: </strong>The briGHt trial was a randomized, open-label, active-controlled, 52-week, phase 3 trial (NCT04326374; CTR20200399) conducted at 17 sites across China. Treatment-naive, prepubertal children with GHD were enrolled and randomized 2:1 to either lonapegsomatropin 0.24 mg hGH/kg/week or equivalent weekly dose of daily somatropin 0.034 mg/kg/day. The primary endpoint was annualized height velocity (AHV) at week 52. Secondary endpoints included change in height standard deviation score (ΔHT SDS) from baseline, insulin-like growth factor 1, and safety.</p><p><strong>Results: </strong>A total of 153 participants received treatment. Least squares (LS) mean ± standard error (±SE) of AHV at week 52 was 10.66 ± 0.22 cm/year for weekly lonapegsomatropin and 9.75 ± 0.26 cm/year for daily somatropin; weekly lonapegsomatropin demonstrated non-inferiority and superiority over daily somatropin, with a difference of 0.91 ± 0.28 cm/year (95% confidence interval: 0.37-1.45; p = 0.0010). LS mean (±SE) of ΔHT SDS was 1.01 ± 0.04 for weekly lonapegsomatropin and 0.83 ± 0.05 for daily somatropin at week 52, favouring lonapegsomatropin from week 13 (p < 0.05) onward. The safety profile was similar between treatment groups.</p><p><strong>Conclusions: </strong>Weekly lonapegsomatropin demonstrated non-inferiority and superiority in efficacy compared to daily somatropin among treatment-naive Chinese children with GHD. The treatment groups showed comparable safety and tolerability profiles.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-12"},"PeriodicalIF":2.7,"publicationDate":"2026-02-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13099130/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147316662","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":"Sixteen Years of Clinical Data Including Genetic Analysis to Explain Delayed Puberty in a Chinese Boy with 21-Hydroxylase Deficiency: A Case Report.","authors":"Xinmeng Wang, Zheng Yuan, Miao Qin, Ming Cheng, Bingyan Cao, Chunxiu Gong","doi":"10.1159/000551139","DOIUrl":"https://doi.org/10.1159/000551139","url":null,"abstract":"<p><strong>Introduction: </strong>In 21-hydroxylase deficiency (21-OHD), impaired 21-hydroxylase activity causes 17-hydroxyprogesterone (17-OHP) accumulation and androgen excess, typically manifesting as hyperandrogenism. The POR gene encodes cytochrome P450 oxidoreductase (POR), the essential electron donor for all microsomal cytochrome P450 enzymes. While POR deficiency impairs multiple steroidogenic enzymes, its phenotypic impact on 21-OHD patients remains poorly characterized.</p><p><strong>Case presentation: </strong>We report a male 21-OHD patient with homozygous CYP21A2 variant (c.293-13C>G) who presented atypically with absent hyperandrogenism and persistently low testosterone levels before puberty. Medication withdrawal revealed significantly elevated adrenocorticotropic hormone and 17-OHP, but only mildly elevated androstenedione (AD) and relatively low testosterone, suggesting impaired 17-OHP-to-AD conversion due to 17,20-lyase deficiency. Whole-exome sequencing identified a concurrent heterozygous pathogenic POR variant (c.1660C>T, p.Arg554Ter). The patient presented with delayed puberty during disease progression but achieved spontaneous puberty after 1.3 years of cumulative GnRH pump therapy, consistent with disease-related functional delayed puberty.</p><p><strong>Conclusion: </strong>This 16-year follow-up case demonstrates that heterozygous POR variants may modulate the phenotype and hormonal profile in 21-OHD. These findings highlight the importance of whole-exome sequencing in atypical 21-OHD cases to identify potential modifiers of steroidogenic pathways.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-17"},"PeriodicalIF":2.7,"publicationDate":"2026-02-26","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"147305187","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":"European Society for Paediatric Endocrinology and European Society of Endocrinology Joint Clinical Practice Guidance for Healthcare Transition from Paediatric to Adult Endocrine Care.","authors":"Enora Le Roux, Kirstine Stochholm, Martin Fassnacht, Imelda Coyne, Philippe Touraine, Krystallenia I Alexandraki, Malgorzata Wasniewska, Judith P van Eck, Victoria Di Guisto, Amanda Helm, Elena Valassi, Joke Marlier, Martine Cools, Rasha T Hamza, Tommaso Aversa, Andrea M Isidori, Anders Juul, Charlotte Verroken, Marie Juul Ornstrup, Lina Zabuliene, Kirsten Davidse, Stefano Cianfarani, Sebastian J C M M Neggers","doi":"10.1159/000550744","DOIUrl":"10.1159/000550744","url":null,"abstract":"<p><strong>Background: </strong>The transition from paediatric to adult healthcare is a critical period for young individuals with endocrine conditions. Despite numerous published recommendations, Europe still lacks recent, comprehensive, evidence-based, and practically applicable guidelines for endocrine healthcare transition.</p><p><strong>Objective: </strong>The aim of the study was to develop European consensus guidance for transition from paediatric to adult care in endocrine conditions through a structured, evidence-based approach.</p><p><strong>Methods: </strong>A systematic literature review identified 351 recommendations from 55 articles (2011-2023). Articles were included if they provided recommendations on the transition from paediatric to adult care for patients with endocrine diseases or general (non-disease-specific) transition guidance. The guidance was developed by a core multidisciplinary group (n = 7) and refined through focus groups with 18 experts from 10 European countries, representing both paediatric and adult care settings. Patient representatives have reviewed and approved it.</p><p><strong>Results: </strong>The guidance includes recommendations across 11 domains: structure of transition service, patient empowerment, patient-professional relationship, multidisciplinary team organisation, healthcare provider education, timing and planning, care coordination, management of non-attendance, psychological support, parent/caregiver role, and readiness tools. Each recommendation was rated as either \"recommend\" (strong) or \"suggest\" (conditional) based on expert consensus and available evidence.</p><p><strong>Conclusion: </strong>This ESE-ESPE guidance provides a comprehensive, practical framework for endocrine healthcare transition, applicable across different European healthcare settings. The recommendations emphasise structured programs, care coordination, and patient-centred approaches to optimise transition outcomes.</p>","PeriodicalId":13025,"journal":{"name":"Hormone Research in Paediatrics","volume":" ","pages":"1-11"},"PeriodicalIF":2.7,"publicationDate":"2026-02-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12919983/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"146226678","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}