Eileen H Koh, Susan K Ewing, Sigurdur Sigurdsson, Vilmundur Gudnason, Trisha F Hue, Eric Vittinghoff, Claes Ohlsson, Åsa Tivesten, Louise Grahnemo, Tony Yuen, Mone Zaidi, Clifford J Rosen, Ann V Schwartz, Anne L Schafer
{"title":"Higher FSH Level Is Associated With Increased Risk of Incident Hip Fracture in Older Adults, Independent of Sex Hormones.","authors":"Eileen H Koh, Susan K Ewing, Sigurdur Sigurdsson, Vilmundur Gudnason, Trisha F Hue, Eric Vittinghoff, Claes Ohlsson, Åsa Tivesten, Louise Grahnemo, Tony Yuen, Mone Zaidi, Clifford J Rosen, Ann V Schwartz, Anne L Schafer","doi":"10.1210/clinem/dgae690","DOIUrl":"10.1210/clinem/dgae690","url":null,"abstract":"<p><strong>Context: </strong>Higher levels of FSH are associated with bone loss among women during the perimenopausal transition and among older men, independent of estradiol and testosterone levels, but whether higher FSH is an independent fracture risk factor is unknown.</p><p><strong>Objective: </strong>To determine whether baseline FSH level predicts subsequent hip fracture in older adults.</p><p><strong>Setting, design, participants: </strong>Using a case-cohort design, we randomly sampled 295 participants stratified by sex from the Age, Gene/Environment Susceptibility-Reykjavik cohort, including 25 participants with incident hip fracture within 10 years after baseline. We sampled an additional 230 sex-stratified participants with incident hip fracture. Serum FSH and sex hormone levels were measured at baseline. Robust weighted Cox proportional hazards models were used to determine the relationship between FSH and hip fracture risk.</p><p><strong>Main outcome: </strong>Incident hip fracture.</p><p><strong>Results: </strong>As no interaction was identified between FSH and sex for the relationship with fracture, men and women were pooled for analysis. Higher levels of FSH were associated with a significantly increased risk of incident hip fracture in models adjusted for age and sex [hazard ratio (HR) 1.24 (95% CI 1.04-1.48, P = .02)] and after further adjustment for estradiol, testosterone, and SHBG levels [HR 1.20 (95% CI 1.01-1.44, P = .04) per sex-specific SD increase in FSH level].</p><p><strong>Conclusion: </strong>Higher FSH is associated with an increased risk of subsequent hip fracture. Our findings support a growing body of evidence for the direct pleiotropic effects of FSH on bone and for a role for FSH in aging and disability independent of sex hormone levels.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"1888-1895"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12187201/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142479565","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"NAFLD vs MASLD (Metabolic Dysfunction-Associated Steatotic Liver Disease)-Why the Need for a Change of Nomenclature?","authors":"Amalia Gastaldelli, Philip N Newsome","doi":"10.1210/clinem/dgaf094","DOIUrl":"10.1210/clinem/dgaf094","url":null,"abstract":"<p><p>Several reasons led to the change in the nomenclature from nonalcoholic fatty liver disease (NAFLD) to metabolic dysfunction-associated steatotic liver disease (MASLD); the most important being limitations due to the reliance on exclusionary confounder terms and the use of potentially stigmatizing language (the terms \"nonalcoholic\" and \"fatty\"). The new name was decided through a Delphi process and now includes in the name, and definition, the metabolic origin (the presence of at least 1 of 5 cardiometabolic risk factors) without the stigmatizing terms. The recognition of a new category termed \"metabolic and alcohol related/associated liver disease\" (MetALD) opens up a new area for exploration although the relative contribution of alcohol and metabolic risk factors requires further evaluation as does the evidencing at a patient rather than population level.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2407-e2410"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143460507","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Georg Semmler, Clemens Baumgartner, Matthäus Metz, Sophie Gensluckner, Hansjörg Habisch, Hannah Hofer, Winfried März, Felix Offner, Andreas Völkerer, Oleksandr Petrenko, Bernhard Wernly, Sophie Draxler-Dworzak, Manuela Neyer, Charlotte Nigmann, Susanne Greber-Platzer, Harald Esterbauer, Tobias Madl, Elmar Aigner, Thomas Scherer, Christian Datz
{"title":"Lipid Dysregulation in Tangier Disease: A Case Series and Metabolic Characterization.","authors":"Georg Semmler, Clemens Baumgartner, Matthäus Metz, Sophie Gensluckner, Hansjörg Habisch, Hannah Hofer, Winfried März, Felix Offner, Andreas Völkerer, Oleksandr Petrenko, Bernhard Wernly, Sophie Draxler-Dworzak, Manuela Neyer, Charlotte Nigmann, Susanne Greber-Platzer, Harald Esterbauer, Tobias Madl, Elmar Aigner, Thomas Scherer, Christian Datz","doi":"10.1210/clinem/dgaf131","DOIUrl":"10.1210/clinem/dgaf131","url":null,"abstract":"<p><strong>Context: </strong>Tangier disease (TD) is a rare, autosomal recessive genetic disorder associated with a deficiency in cellular cholesterol export leading to cholesterol accumulation in peripheral tissues. With approximately 150 described cases, the disease is significantly understudied, and the clinical presentation appears to be heterogenous.</p><p><strong>Objective: </strong>To investigate the phenotype and lipid metabolism in TD.</p><p><strong>Design: </strong>Multicenter cohort study.</p><p><strong>Patients: </strong>Four patients with TD.</p><p><strong>Main outcome measures: </strong>Nuclear magnetic resonance (NMR)-based lipidomic and metabolomic analyses were performed in patients with TD and healthy controls.</p><p><strong>Results: </strong>While showing similar laboratory patterns with respect to high-density lipoprotein (HDL) depletion, the clinical presentation of 4 TD patients was heterogenous with 2 patients diagnosed at 47 and 72 years having predominantly gastrointestinal and neurological phenotypes. Two previously undescribed variants (c.2418G > A, c.5055.del) were reported.Apart from pathognomonic changes in HDL composition, NMR spectroscopy revealed an increased abundance of very low-density lipoprotein (VLDL) with higher total lipid and cholesterol concentrations, pointing toward an impaired clearance of triglyceride-rich lipoproteins. Increased triglyceride-rich intermediate-density lipoprotein supports impaired hepatic lipase activity, together with a cholesteryl ester transfer protein-mediated increase in low-density lipoprotein (LDL)-triglycerides at higher abundance of large LDL subtypes and decreased small dense LDL.The lipid composition of HDL particles and LDL-1/LDL-4 remained the strongest differentiating factors as compared to healthy controls.</p><p><strong>Conclusion: </strong>Clinical phenotypes of TD can be heterogeneous including gastrointestinal and neurological manifestations. Impaired triglyceride-rich lipoprotein clearance and hepatic lipase activity could be a pathophysiological hallmark of TD.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2146-e2156"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143558694","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Arturo Vega-Beyhart, Betina Biagetti, Mónica Marazuela, Manel Puig-Domingo, Marta Araujo-Castro
{"title":"Efficacy and Safety of Pasireotide in Insulinoma-Associated Hypoglycemia.","authors":"Arturo Vega-Beyhart, Betina Biagetti, Mónica Marazuela, Manel Puig-Domingo, Marta Araujo-Castro","doi":"10.1210/clinem/dgaf195","DOIUrl":"10.1210/clinem/dgaf195","url":null,"abstract":"<p><strong>Context: </strong>Persistent hypoglycemia is a life-threatening complication in insulinoma patients. When tumor excision is not possible, medical treatments are the main option. Pasireotide has shown promise in managing refractory hypoglycemia, but its use has been reported only in case series and reports.</p><p><strong>Objective: </strong>This work aimed to assess the efficacy and safety of pasireotide in treating insulinoma-associated hypoglycemia.</p><p><strong>Methods: </strong>We conducted a systematic review on using pasireotide to treat insulinoma-associated hypoglycemia, following a predeveloped protocol. We searched MEDLINE, Scopus, Google Scholar, and references forward and backward from database inception to March 30, 2024.</p><p><strong>Results: </strong>Of 490 identified studies, 137 were reviewed, and 17 cases from 13 studies met the inclusion criteria. Patients' ages ranged from 52 to 71 years (9 women). Five patients (30%) underwent surgical tumor resection. Pasireotide was never the initial treatment. The most common doses were 40 to 60 mg/month for pasireotide long-acting release and 0.6 mg/12 h for short-acting pasireotide. Six patients (35%) showed no improvement, 4 (23%) had partial improvement, and 7 (41%) had complete resolution. Patients with aggressive insulinomas had a lower response rate, with 55% showing no improvement compared to 16% in indolent cases. Larger tumors were significantly associated with poorer response (P = .043). Hyperglycemia was the most common side effect (n = 3).</p><p><strong>Conclusion: </strong>Pasireotide effectively restored glucose levels in insulinoma patients who failed prior treatments. However, its efficacy was lower in aggressive insulinomas, emphasizing the need for alternative or combinatory strategies in metastatic cases. Given that pasireotide was never used as a first-line therapy in the reviewed cases, earlier administration in selected patients may improve outcomes.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2109-e2120"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143755614","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Are CELSR2 Intronic Variants Genetic Determinants of Energy Balance?","authors":"Kimberley D Bruce, Robert H Eckel","doi":"10.1210/clinem/dgae658","DOIUrl":"10.1210/clinem/dgae658","url":null,"abstract":"","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2397-e2398"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142331505","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Jinghua Zhang, Wilson Wai San Tam, Jinhua Lu, Junjie Chen, Joji Kusuyama, Yanhong Dong, Xin Yi Yap, Wentao Zhou, Na Wang, Hui Nan Yeo, Frena Jia Sy Lee, Vivien Xi Wu
{"title":"Response to Letter to the Editor From Bai and Zhang: \"Cognitive Risk Stratification Score in Middle-aged and Older Adults With Type 2 Diabetes: A Cross-sectional Study\".","authors":"Jinghua Zhang, Wilson Wai San Tam, Jinhua Lu, Junjie Chen, Joji Kusuyama, Yanhong Dong, Xin Yi Yap, Wentao Zhou, Na Wang, Hui Nan Yeo, Frena Jia Sy Lee, Vivien Xi Wu","doi":"10.1210/clinem/dgaf241","DOIUrl":"10.1210/clinem/dgaf241","url":null,"abstract":"","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2423-e2424"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144065120","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Rebecca J Johnson, Simon Bergford, Robin L Gal, Peter Calhoun, Karissa Neubig, Corby K Martin, Michael C Riddell, Ananta Addala
{"title":"The Role of Whole Food Plant-Based Food Intake on Postprandial Glycemia in Type 1 Diabetes.","authors":"Rebecca J Johnson, Simon Bergford, Robin L Gal, Peter Calhoun, Karissa Neubig, Corby K Martin, Michael C Riddell, Ananta Addala","doi":"10.1210/clinem/dgae725","DOIUrl":"10.1210/clinem/dgae725","url":null,"abstract":"<p><strong>Context: </strong>A whole food plant-based diet (WFPBD), minimally processed foods with limited consumption of animal products, is associated with improved health outcomes. The benefits of WFPBD are underexplored in individuals with type 1 diabetes (T1D).</p><p><strong>Objective: </strong>The primary objective of this analysis is to evaluate the association between WFPBD on glycemia in individuals with T1D.</p><p><strong>Methods: </strong>Utilizing prospectively collected meal events from the Type 1 Diabetes Exercise Initiative, we examined the effect of WFPBD intake on glycemia, determined by the plant-based diet index (PDI). The PDI calculates overall, healthful (hPDI), and unhealthy PDI (uPDI) to evaluate for degree of processed foods and animal products (ie, WFPBD). Mixed effects linear regression model assessed time in range (TIR), time above range, and time below range.</p><p><strong>Results: </strong>We analyzed 7938 meals from 367 participants. TIR improved with increasing hPDI scores, conferring a 4% improvement in TIR between highest and lowest hPDI scores (high hPDI: 75%, low hPDI: 71%; P < .001). Compared with meals with low hPDI, meals with high hPDI had lower glucose excursion (high hPDI: 53 mg/dL, low hPDI: 62 mg/dL; P < .001) and less time >250 mg/dL (high hPDI: 8%, low hPDI: 14%; P < .001). These effects were present but less pronounced by PDI (high PDI: 74%, low PDI: 71%; P = .01). No differences in time below 70 mg/dL and 54 mg/dL were observed by PDI or hPDI.</p><p><strong>Conclusion: </strong>Meal events with higher hPDI were associated with 4% postprandial TIR improvement. These benefits were seen primarily in WFPBD meals (captured by hPDI) and less pronounced plant-based meals (captured by PDI), emphasizing the benefit of increasing unprocessed food intake over limiting animal products alone.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"1828-1835"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12187185/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142479586","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
{"title":"Heterogeneity of Gestational Diabetes and Risk for Adverse Pregnancy Outcome: A Cohort Study.","authors":"Yixin Gong, Qunhua Wang, Suyu Chen, Yujie Liu, Chenghua Li, Rong Kang, Jing Wang, Tian Wei, Qin Wang, Xianming Li, Sihui Luo, Jianping Weng, Xueying Zheng, Yu Ding","doi":"10.1210/clinem/dgae754","DOIUrl":"10.1210/clinem/dgae754","url":null,"abstract":"<p><strong>Context: </strong>Diabetes is increasingly recognized as a heterogeneous disease, with clinical characteristics and outcome risks varying across different phenotypes. Evidence on heterogeneity of gestational diabetes (GDM) is yet to be provided.</p><p><strong>Objective: </strong>To investigate the insulin physiology and pregnancy outcomes of GDM phenotypes characterized by fasting hyperglycemia or postload hyperglycemia.</p><p><strong>Methods: </strong>A total of 2050 women who underwent a 75-g oral glucose tolerance test were prospectively recruited and followed until delivery. Women were categorized into normoglycemia (NGT, n = 936), isolated impaired fasting glucose (gestational-IFG, n = 378), and isolated impaired postload glucose tolerance (gestational-IGT, n = 736) groups. Fasting blood samples at mid-pregnancy were collected to measure C-peptide and insulin concentrations. Homeostasis model assessment (HOMA) and quantitative insulin sensitivity check index (QUICKI) were used to evaluate insulin physiology. Maternal and neonatal outcomes were recorded.</p><p><strong>Results: </strong>Gestational-IFG had greater insulin resistance (HOMA-IR 3.11 vs 2.25, QUICKI-C-peptide 0.94 vs 1.03, both P < .01), and gestational-IGT had worse β-cell function (C-peptide 2.00 vs 2.26 ng/mL, P < .05), when compared to one another. Gestational-IFG was more strongly associated with excessive gestational weight gain (risk ratio [RR] 1.62; 95% CI, 1.18-2.23) and large-for-gestational-age infants (RR 1.45; 95% CI, 1.03-2.03) than gestational-IGT. The risk for neonatal brain injury was increased in gestational-IGT (RR 2.03; 95% CI, 1.04-4.09), but not in gestational-IFG (P = .439). Gestational-IGT showed a stronger association with the risk of preterm birth compared to gestational-IFG (RR 1.80; 95% CI, 1.02-3.36).</p><p><strong>Conclusion: </strong>GDM exhibits distinct insulin physiology profiles. Pregnancy outcome varies between each phenotype. These findings provide evidence on risk stratification and diverse strategies for the treatment of GDM.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2264-e2272"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12187195/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142512185","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Loren S van der Hoeven, Tessa N A Slagboom, Arjan Malekzadeh, Jantien Hoogmoed, Madeleine L Drent, Eleonora Aronica, Dirk Jan Stenvers, Alberto M Pereira
{"title":"Cell Lineage-Specific Differences in Clinical Behavior of Non-Functioning Pituitary Adenomas.","authors":"Loren S van der Hoeven, Tessa N A Slagboom, Arjan Malekzadeh, Jantien Hoogmoed, Madeleine L Drent, Eleonora Aronica, Dirk Jan Stenvers, Alberto M Pereira","doi":"10.1210/clinem/dgaf112","DOIUrl":"10.1210/clinem/dgaf112","url":null,"abstract":"<p><strong>Context: </strong>Immunohistochemistry (IHC) of cell lineage-specific transcription factors (TFs) has been added to the histopathological classification of pituitary adenomas since 2017, resulting in new histopathological subtypes of TF+/hormone-non-functioning pituitary adenomas (NFPAs) and a reduction in the prevalence of null cell adenomas (NCAs).</p><p><strong>Objective: </strong>This work aimed to evaluate associations between expression of cell lineage-specific TFs by IHC and radiological invasion and prognosis of NFPAs.</p><p><strong>Data sources: </strong>A literature search in Medline, Embase, and CENTRAL was performed from inception up to July 11, 2023.</p><p><strong>Study selection: </strong>Eligible studies were cohort studies reporting on radiological invasion, recurrence, and/or radiotherapy in patients with NFPAs who tested positive for one cell lineage-specific TF or negative for all 3. Finally, 27 out of 1985 studies were included.</p><p><strong>Data extraction: </strong>Two authors independently extracted data and critically appraised risk of bias using the Quality In Prognostic Studies (QUIPS) tool.</p><p><strong>Data synthesis: </strong>Random-effects inverse variance models were used to pool effect sizes. Prevalence rate ratios (PRRs) were calculated using the Mantel-Haenszel method. Cavernous sinus invasion was more prevalent in NCAs and TPIT+ NFPAs compared with SF1+ NFPAs (PRR 1.60; 95% CI, 1.22-2.08, I2 10%, 95% prediction interval [PrI] 1.23-2.06; P = .0036, and PRR 1.43; 95% CI, 1.21-1.70, I2 0%, 95% PrI 1.17-1.76; P = .0017, respectively), and in NCAs compared with PIT1+ (PRR 1.44; 95% CI, 1.01-2.06, I2 0%, 95% PrI 0.83-2.50; P = .0454). A limited number of studies precluded data syntheses of recurrence and radiotherapy.</p><p><strong>Conclusion: </strong>The use of cell lineage-specific TFs by IHC enables to detect histopathological subtypes of NFPAs with distinct clinical behavior.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"e2362-e2382"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143473168","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
Noha Behairy, Anthony J Leonardi, Sriram Gubbi, Sonam Kumari, Mateus Pascoal, Ashwin Bharadwaj, Amr Dorgham, Elizabeth C Wright, Tomilowo Abijo, Chandra Nayan Uttarkar Vikram, Padmasree Veeraraghavan, Craig Cochran, Srivandana Akshintala, John Glod, Joanna Klubo-Gwiezdzinska
{"title":"Tumor Volume Doubling Time of Less Than 1 Year Is Associated With a Higher Risk of Death From Medullary Thyroid Cancer.","authors":"Noha Behairy, Anthony J Leonardi, Sriram Gubbi, Sonam Kumari, Mateus Pascoal, Ashwin Bharadwaj, Amr Dorgham, Elizabeth C Wright, Tomilowo Abijo, Chandra Nayan Uttarkar Vikram, Padmasree Veeraraghavan, Craig Cochran, Srivandana Akshintala, John Glod, Joanna Klubo-Gwiezdzinska","doi":"10.1210/clinem/dgae733","DOIUrl":"10.1210/clinem/dgae733","url":null,"abstract":"<p><strong>Context: </strong>Tumor volume doubling time (TVDT) is emerging as a useful tool in predicting oncologic outcomes. There are limited data on the prognostic role of TVDT in metastatic medullary thyroid cancer (MTC).</p><p><strong>Objective: </strong>The goal of this study was to assess the value of TVDT in predicting disease-specific survival (DSS) in patients with hereditary and sporadic MTC.</p><p><strong>Methods: </strong>This was an Institutional Review Board-approved cohort study including patients with metastatic MTC having at least 3 consecutive imaging studies. TVDT of up to the 5 largest lesions per organ was calculated using a standardized formula. The association between TVDT and DSS was analyzed using Kaplan-Meier survival curves. Cox proportional regression model was used to account for confounding factors.</p><p><strong>Results: </strong>The study sample consisted of 51 patients presenting with 286 metastatic lesions measured with 457 scans during the follow-up of 51 (IQR, 25-102) months. Median age was 19 years (IQR, 15-41), 53% female patients. Cumulative volumes of all metastatic lesions and proportion of patients with TVDT of < 1 year were higher in patients with sporadic as compared with hereditary MTC (P < .01). Factors independently associated with shorter DSS were TVDT of < 1 year based on 3 initial and 3 last scans as well as lung, brain, and prostate as the organs with the fastest growing tumor. TVDT based on 2-dimensional and 3-dimensional measurements showed strong correlation (r = 0.94, P < .05).</p><p><strong>Conclusion: </strong>Measurements from 3 baseline and 3 most recent scans preceding follow-up visit enable calculation of TVDT and can be used as predictors of mortality from MTC.</p>","PeriodicalId":50238,"journal":{"name":"Journal of Clinical Endocrinology & Metabolism","volume":" ","pages":"1854-1864"},"PeriodicalIF":5.0,"publicationDate":"2025-06-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12187190/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142479587","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}