Journal of pediatric endocrinology & metabolism : JPEM最新文献

{"title":"Refractory diabetic ketoacidosis in Rabson-Mendenhall syndrome due to a novel mutation: contrasting outcomes in a sibling - case series.","authors":"J Mohammed Azarudeen, R Narayanan, Anjana Kar, Gowri Rao, Ashwin Dalal, Laxi Vairavan","doi":"10.1515/jpem-2026-0389","DOIUrl":"https://doi.org/10.1515/jpem-2026-0389","url":null,"abstract":"<p><strong>Objectives: </strong>Rabson-Mendenhall syndrome (RMS) is a rare monogenic insulin resistance disorder characterized by progressive dysglycemia and early mortality. Management of diabetic ketoacidosis (DKA) in RMS is particularly challenging because standard insulin doses may be inadequate, and pediatric-specific experience is limited.</p><p><strong>Case presentation: </strong>We report two siblings with genetically confirmed RMS. The index child, a 7-year-old girl, presented with severe refractory DKA requiring progressive escalation of intravenous insulin to 12 IU/kg/h before meaningful metabolic response was achieved. Insulin was titrated according to serial glycemic and acid-base responses, without bicarbonate therapy, with close potassium monitoring. Her 3-year-old brother was evaluated proactively after the diagnosis in his sister and was found to have hyperglycemia and the same homozygous <i>INSR</i> variant before an episode of metabolic decompensation. He was treated with subcutaneous insulin and insulin sensitizers. At 1-year follow-up, HbA_1c was 10.4 % in the index child and 8.9 % in the sibling.</p><p><strong>Conclusions: </strong>Refractory DKA in RMS may require rapid, response-guided escalation of insulin beyond conventional pediatric dosing. The detailed temporal relationship between insulin dose and metabolic response in this child provides practical clinical insight, while early recognition of RMS permits evaluation and treatment of affected siblings before presentation with acute metabolic decompensation.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149122703","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Papillary thyroid carcinoma in children: prevalence, morphology, molecular and cytogenetic characteristics, and prognostic determinants.","authors":"Alireza Abdollahi, Masoumeh Avarjeh","doi":"10.1515/jpem-2026-0277","DOIUrl":"https://doi.org/10.1515/jpem-2026-0277","url":null,"abstract":"<p><p>Papillary thyroid carcinoma (PTC) stands as the most frequently encountered endocrine malignancy in children and adolescents. A notable clinical feature of pediatric PTC is that while young patients often present with advanced disease - including lymph node involvement and distant metastases - long-term survival rates remain high. This presentation underscores pediatric PTC as a biologically distinct entity. The molecular landscape differs fundamentally from adult disease, with gene fusions involving <i>RET/PTC</i>, <i>NTRK</i>, and <i>ALK</i> serving as primary drivers, while <i>BRAF</i> V600E point mutations play a lesser role. This narrative review examines the prevalence, morphological features, molecular and cytogenetic characteristics, and prognostic determinants of pediatric papillary thyroid carcinoma. We explore how these children frequently present with multifocal tumors, extensive nodal metastases, and pulmonary spread yet maintain favorable clinical outcomes. The review critically evaluates current approaches to surgery, radioactive iodine therapy, and risk-adapted surveillance strategies. We also consider the growing role of targeted therapies - particularly RET and NTRK inhibitors - in managing refractory or metastatic disease, while acknowledging that long-term safety data in children remain limited. Available evidence indicates that pediatric PTC represents a biologically and clinically distinct entity requiring a tailored management paradigm. While outcomes are generally favorable, significant questions remain about epidemiology, molecular drivers, treatment resistance, and long-term survivorship effects. Moving forward, progress will depend on international collaboration, standardized data collection, and extended follow-up to develop pediatric-specific biomarkers and therapeutic approaches.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-10-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149122792","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"From guidelines to real-world practice: clinical, biochemical, and genetic insights in a series of Moroccan patients with acid sphingomyelinase deficiency.","authors":"Imane Assiri, Miloud Hammoud, Abdelmalek Hakmaoui, Samira Najeh, Maroua Jakani, Es-Said Sabir, Karima Lafhal, Abdelaati Berrachid, Sana Elfoutat, Francisco J Del Castillo, Najwa Imad, Imane Ait Sab, Aicha Bourrahouat, Alice M S Rodrigues, Emeline Houel, Dedier Stien, Naima Fdil","doi":"10.1515/jpem-2026-0194","DOIUrl":"https://doi.org/10.1515/jpem-2026-0194","url":null,"abstract":"<p><strong>Objectives: </strong>Niemann-Pick disease types A and B (NPD-A, NPD-B) are lysosomal storage disorders caused by acid sphingomyelinase (ASM) deficiency, leading to sphingomyelin (SM) accumulation. NPD-A presents with rapidly progressive neurodegeneration in infancy, whereas NPD-B spares the nervous system. This study aimed to define the biochemical profile of Moroccan patients, delineate the <i>SMPD1</i> mutation spectrum, and illustrate a practical diagnostic pathway suited to a resource-limited setting.</p><p><strong>Methods: </strong>Genetic and biochemical investigations were performed to identify <i>SMPD1</i> variants and evaluate sphingomyelin excretion. Molecular analysis was conducted using Sanger sequencing. Urinary lipid extracts were analyzed by thin-layer chromatography (TLC) as an orientation tool, and exploratory high-performance liquid chromatography-mass spectrometry (HPLC-MS/MS) was performed in two representative cases. Five genetically confirmed Moroccan patients are described in this cohort.</p><p><strong>Results: </strong>Five patients were diagnosed with ASM deficiency (ASMD) using this integrated approach. TLC analysis revealed abnormal urinary sphingomyelin patterns, providing an early biochemical orientation toward the diagnosis. Enzymatic assays confirmed reduced ASM activity, and molecular analysis identified two pathogenic <i>SMPD1</i> variants: the in-frame deletion NM_000543.4:c.1826_1828delGCC (p.Arg610del) in a patient with NPD-B and the nonsense mutation NM_000543.4:c.1624C>T (p.Arg542*) in patients with NPD-A. The exploratory HPLC-MS/MS analysis highlighted a marked signal for the SM(d18:1/16:0) isoform in urine.</p><p><strong>Conclusions: </strong>This study proposes a practical diagnostic workflow adapted to a resource-limited setting and provides new insights into the <i>SMPD1</i> mutation spectrum and urinary SM species in the Moroccan population. Future studies on larger cohorts are needed to better define biochemical variability and confirm genotype-phenotype associations.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149067024","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Successful management of paediatric chronic SIAD with oral urea: report of two cases.","authors":"Suki Suk-Yan Chan, Gloria Shir-Wey Pang, Sarah Wing-Yiu Poon, Wai-Lan Yeung, Tak-Loi Ku, Fiona Fung-Yee Lai, Joanna Yuet-Ling Tung","doi":"10.1515/jpem-2026-0398","DOIUrl":"https://doi.org/10.1515/jpem-2026-0398","url":null,"abstract":"<p><strong>Objectives: </strong>Syndrome of inappropriate antidiuretic hormone secretion (SIAD) causes euvolemic hyponatraemia and is managed with fluid restriction, which can be difficult to maintain in children with chronic disease. Urea is an osmotic diuretic recommended as a second-line therapy, but paediatric experience remains limited.</p><p><strong>Case presentation: </strong>Patient 1 was a 1-year-old male infant diagnosed with SIAD secondary to an optic pathway pilocytic astrocytoma, following therapeutic exposure to carboplatin and vincristine/vinblastine. Initial management with fluid restriction and sodium supplementation proved insufficient, resulting in persistent hyponatraemia and compromised physical growth. Crucially, because the patient's nutritional intake was derived primarily from milk, standard fluid restriction inherently imposed severe nutritional restriction. Following the introduction and gradual titration of urea therapy, plasma sodium normalised, urine output increased and sodium supplementation was discontinued. This clinical improvement allowed for the liberalisation of fluid intake, thereby restoring adequate nutritional delivery and subsequently improved growth. Patient 2 was a 16-year-old girl with severe neurological impairment, panhypopituitarism and chronic SIAD following neonatal meningitis and hypoxic-ischaemic injury. Her hyponatraemia persisted despite fluid restriction and sodium supplementation, and prolonged restriction probably contributed to renal calcium cysts. After starting urea, plasma sodium normalised, fluid restriction was eased and sodium supplementation was discontinued.</p><p><strong>Conclusions: </strong>In conclusion, oral urea represents a safe, cost-effective maintenance therapy for paediatric chronic SIAD. By mitigating strict fluid restriction, it facilitates adequate nutritional delivery essential for growth. Additionally, its minimal potential for drug-drug interactions offers a distinct practical advantage in medically complex children.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-22","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149013510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anti-Müllerian hormone levels in girls with precocious puberty.","authors":"Merve Nur Hepokur, Aşan Önder Çamaş, Gözde Taniş, Burçin Çiçek, Sare Betül Kaygusuz, Ferruh Kemal İşman, Hamdi Cihan Emeksiz","doi":"10.1515/jpem-2026-0340","DOIUrl":"https://doi.org/10.1515/jpem-2026-0340","url":null,"abstract":"<p><strong>Objectives: </strong>We aimed to evaluate the relationship between anti-Müllerian hormone (AMH) levels in cases of central precocious puberty (CPP), premature adrenarche (PA), and isolated premature thelarche (IPT) and those of healthy controls (HC).</p><p><strong>Methods: </strong>A total of 153 girls (40 PA, 39 CPP, 39 IPT, and 35 HC) were included in the study. AMH levels were assessed using the ELISA method.</p><p><strong>Results: </strong>The mean age of all participants was 7.19 ± 0.63 (5.0-8.0) years. Height SDS was higher in the puberty and puberty variant groups compared with the healthy group. This difference was found to be statistically significant between the CPP and HC groups. Obesity was present in 20 of all participants (13 %). AMH levels were 0.125 ± 0.154, 0.086 ± 0.072, 0.084 ± 0.074, and 0.069 ± ;0.060 ng/mL in PA, PT, CPP, and HC groups, respectively. Although AMH levels were higher in the PA group, there was no statistically significant difference among the four groups. A weak negative correlation was found between AMH levels and BMI SDS (body mass index standard deviation score).</p><p><strong>Conclusions: </strong>AMH levels do not differ between prepubertal girls and those diagnosed with central precocious puberty or a variant of puberty of the same age.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149009741","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Allgrove syndrome in Colombia: clinical characterization and evidence of a possible founder effect.","authors":"Johana Andrea Botero Hernandez, Eder Donadoni Varela Macias, María Victoria Lopera Cañaveral, Adriana Carolina Forero Torres, Juan José Ramírez Jiménez, Gina Marcela González Valencia, Juan Manuel Alfaro Velázquez, Nicolas Pineda Trujillo","doi":"10.1515/jpem-2026-0358","DOIUrl":"https://doi.org/10.1515/jpem-2026-0358","url":null,"abstract":"<p><strong>Objectives: </strong>Allgrove syndrome is a rare autosomal recessive disorder caused by pathogenic variants in the <i>AAAS</i> gene, characterized by achalasia, alacrima, and adrenal insufficiency, with variable neurological involvement. Genotype-phenotype correlations remain poorly characterized. This study aimed to describe the clinical and genetic features of Colombian patients with Allgrove syndrome and to explore potential genotype-phenotype differences.</p><p><strong>Methods: </strong>A multicenter retrospective cohort study was conducted between January 2010 and July 2025. A total of 1,587 clinical records were screened across three tertiary referral centers in Antioquia, complemented with structured telephone interviews, identifying 30 patients meeting diagnostic criteria. Genetic testing results, including whole-exome sequencing or targeted AAAS gene analysis retrieved from clinical records, were analyzed by genotype.</p><p><strong>Results: </strong>Twenty-nine of 30 patients (96.7 %) presented the complete classical triad. Median age at diagnosis was 5.5 years, glucocorticoid and mineralocorticoid deficiencies were diagnosed at a median of 4 and 3 years, respectively; achalasia at a mean of 7.75 years. Among 19 genotyped patients, two homozygous pathogenic variants were identified: c.1331+1G>A and c.1300C>T (p.Arg434*) in one patient, who exhibited earlier onset and more severe neurological involvement. Clustering in the Paisa region suggests a potential founder effect.</p><p><strong>Conclusions: </strong>This is the largest reported Colombian cohort of Allgrove syndrome. A highly prevalent AAAS variant suggestive of a possible founder effect was identified in the Paisa population. Clinically meaningful differences in disease severity were observed between variants. These findings expand the phenotypic spectrum and underscore the importance of early genetic testing and systematic multidisciplinary surveillance.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-21","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149013533","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Ketone metabolism defects in childhood: a spectrum of overlapping presentations and clinical features.","authors":"Burcu Koseci, Ezgi Burgac, Ahmet Yöntem, Ilknur Arslan, Kanay Yararbas, Merve Yoldas Celik","doi":"10.1515/jpem-2026-0252","DOIUrl":"https://doi.org/10.1515/jpem-2026-0252","url":null,"abstract":"<p><strong>Objectives: </strong>Ketone body metabolism defects are rare inherited disorders that may present with life-threatening metabolic crises in childhood. This study aimed to describe and compare clinical, biochemical, and genetic findings in patients with three ketone metabolism defects.</p><p><strong>Methods: </strong>In this retrospective study, data from eight genetically confirmed patients were analyzed: 3 with 3-hydroxy-3-methylglutaryl-CoA synthase deficiency (HMGCS2), 4 with beta-ketothiolase deficiency (BKTD), and 1 with succinyl-CoA:3-oxoacid CoA transferase deficiency (SCOT). Patients were followed at the Pediatric Nutrition and Metabolism Department of Adana City Training and Research Hospital since October 2023.</p><p><strong>Results: </strong>Eight patients (5 female, 3 male) were included; consanguinity was present in 7 families. Triggers included vaccination, infections, gastroenteritis, and dietary changes. All presented with severe metabolic acidosis requiring hemodialysis. Ketone levels were absent/very low in HMGCS2 deficiency and markedly elevated in BKTD and SCOT deficiency. Hypoglycemia occurred only in HMGCS2, while hyperglycemia was seen in BKTD and SCOT. Hepatomegaly was present in all HMGCS2, SCOT patients and in half of BKTD cases. Elevated 3-hydroxybutyrylcarnitine/3-hydroxyisobutyrylcarnitine (C4-OH) was found in all BKTD patients. One HMGCS2 patient had thrombocytopenia and coagulopathy. Genetic analysis identified homozygous pathogenic/likely pathogenic variants in <i>HMGCS2, ACAT1</i>, and <i>OXCT1</i>, including three novel variants. All patients remained clinically stable on dietary treatment.</p><p><strong>Conclusions: </strong>HMGCS2 deficiency, BKTD, and SCOT deficiency show overlapping features but can be distinguished by ketone levels, glucose patterns, and acylcarnitine profiles. Novel variants expand the mutational spectrum. Early diagnosis and management are crucial to prevent irreversible neurological damage.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148973045","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sociodemographic predictors of follow-up adherence and metabolic control in children with type 1 diabetes: a 2-year retrospective cohort study.","authors":"Selen Duygu Arık, Semra Çağlar Çetinkaya","doi":"10.1515/jpem-2026-0197","DOIUrl":"https://doi.org/10.1515/jpem-2026-0197","url":null,"abstract":"<p><strong>Objectives: </strong>Early metabolic outcomes in children with type 1 diabetes mellitus (T1DM) are shaped not only by clinic follow-up frequency but also by family-level capacity for daily disease management. We evaluated adherence to scheduled outpatient visits during the first 2 years after diagnosis and identified sociodemographic and clinical predictors of adherence and HbA₁ _c-based metabolic control.</p><p><strong>Methods: </strong>In this retrospective, single-center cohort study, 150 children with newly diagnosed T1DM who were followed for at least 2 years were studied. Sociodemographic characteristics, family structure, visit frequency, hospitalizations, and serial HbA_1c values were extracted from medical records. Adequate adherence was defined as ≥3 visits per year.</p><p><strong>Results: </strong>Adherence was high in both the first (97.3 %) and second (91.3 %) years, yet mean HbA_1c rose significantly from year 1 to year 2 (6.54 vs. 7.05 %, p<0.001). Sibling count was the only independent predictor of adherence: vs. ≥3 siblings, children with 0-1 siblings (odds ratio [OR] 2.885, 95 % confidence interval [CI] 1.136-7.323, p=0.026), and 2 siblings (OR 3.527, 95 % CI 1.354-9.186, p=0.010) were more likely to attend. Low maternal education was associated with higher hospitalization risk and poorer second-year control (both p<0.001); a family history of diabetes was protective against hospitalization (OR 0.372, 95 % CI 0.164-0.845, p=0.018).</p><p><strong>Conclusions: </strong>Despite high clinic attendance, glycemic control deteriorated, indicating that regular attendance alone is insufficient. Family structure and maternal educational capacity appear central to early diabetes trajectories, underscoring the need for family-centered, context-sensitive interventions.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148957801","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Clinical heterogeneity and disease progression during adolescence in AGPAT2-associated congenital generalized lipodystrophy: a case series of four patients.","authors":"Jantje Weiskorn, Olga Kordonouri","doi":"10.1515/jpem-2026-0516","DOIUrl":"https://doi.org/10.1515/jpem-2026-0516","url":null,"abstract":"<p><strong>Objectives: </strong>This case series describes the clinical presentation and longitudinal course of four adolescents with genetically confirmed <i>AGPAT2</i>-associated congenital generalized lipodystrophy (CGL) followed at a pediatric lipodystrophy center.</p><p><strong>Case presentation: </strong>Despite shared phenotypic features - including generalized lipoatrophy, muscular hypertrophy, acromegaloid features, and hyperphagia - metabolic severity varied markedly, even between siblings carrying the same homozygous <i>AGPAT2</i> variant. All three female patients experienced rapid metabolic deterioration during puberty, developing insulin resistance, diabetes, and severe hypertriglyceridemia, while the male patient remained relatively metabolically stable. Three patients developed progressive albuminuria during adolescence, and two required ACE inhibitor therapy for hypertension, highlighting early renal involvement independent of diabetes. Metreleptin therapy led to substantial metabolic improvement in all treated patients, with normalization of glycemic control, triglyceride levels, and liver enzymes, as well as reduced hyperphagia and, in one case, spontaneous menarche. However, metabolic decompensation occurred during acute illness in one patient, necessitating treatment escalation, indicating that metreleptin does not fully protect against metabolic stress. Notably, pronounced daytime fatigue persisted in three patients despite metabolic stabilization, suggesting an underrecognized, potentially central component of the disease.</p><p><strong>Conclusions: </strong>These findings underscore the phenotypic variability of <i>AGPAT2</i>-associated CGL, the critical impact of puberty - especially in females - the frequent early onset of nephropathy, and the partial but incomplete response to leptin replacement. Close monitoring during adolescence and individualized management are essential.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148957749","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"GLP-1 receptor agonist use and mental health outcomes in adolescents with obesity: a retrospective review.","authors":"Jeries Said, Melodee Liegl, Amy Y Pan, Elizabeth Dabrowski","doi":"10.1515/jpem-2026-0227","DOIUrl":"https://doi.org/10.1515/jpem-2026-0227","url":null,"abstract":"<p><strong>Objectives: </strong>Adolescent obesity is associated with increased rates of depression and suicidality. Glucagon-like peptide-1 receptor agonists (GLP-1RAs) are increasingly used to treat pediatric obesity, yet concerns remain regarding potential psychiatric effects. This study evaluated changes in depressive symptoms and suicidality screening scores among adolescents with obesity treated with GLP-1RAs in a pediatric endocrinology setting. We evaluated changes in depressive symptoms and suicidality following initiation of GLP-1RA therapy in adolescents with obesity.</p><p><strong>Methods: </strong>We performed a retrospective chart review of adolescents aged 12-21 years with obesity treated with liraglutide or semaglutide at a single academic center between June 2022 and December 2024. Demographic, clinical, and psychosocial data were extracted from the electronic health record at baseline and follow-up. Depressive symptoms or suicidality were screened at clinic visits per protocol using either the Patient Health Questionnaire-9 (PHQ-9) or the Ask Suicide-Screening Questions (ASQ), respectively. Analyses were restricted to patients with paired baseline and follow-up assessments using the same instrument. Paired nonparametric tests were used for longitudinal comparisons.</p><p><strong>Results: </strong>Forty-four adolescents met inclusion criteria. Among patients with paired PHQ-9 data (n=15), the median PHQ-9 score decreased significantly from 6 (IQR 2-14) at baseline to 1 (IQR 0-5) at follow-up (p=0.003), independent of changes in BMI. Among patients with paired ASQ data (n=23), no increase in suicidality or new suicidal ideation was observed.</p><p><strong>Conclusions: </strong>In this small, exploratory retrospective review, no evidence of short-term psychological harm or increased suicidality was observed among adolescents receiving GLP-1RA therapy. These findings support the short-term psychosocial safety of GLP-1RA therapy in adolescents with obesity. Larger prospective case-control studies are needed to better characterize psychiatric safety and psychosocial outcomes.</p>","PeriodicalId":520684,"journal":{"name":"Journal of pediatric endocrinology & metabolism : JPEM","volume":" ","pages":""},"PeriodicalIF":1.0,"publicationDate":"2026-09-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148957809","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}