Endocrine Practice最新文献_第3页

Reliable Monitoring of Patients With Metabolic Dysfunction-Associated Steatotic Liver Disease Using Imaging: A Systematic Literature Review and Meta-Analysis on Measurement Repeatability 影像学对MASLD患者的可靠监测：系统的文献回顾和测量可重复性的荟萃分析。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.205

Michael Ndaa MSc , Prashant K. Pandya DO, FAASLD , Jordan Swensson MD , Niharika Samala MD , Saima Ajaz BSc, MBBS, MPhil, PhD, Dip, IBLM/BSLM , Deepak Joshi PhD, FRCP , Walid S. Ayoub MD, FAASLD, AGAF , Fatih Akisik MD

Introduction

As treatments for patients with metabolic dysfunction-associated steatotic liver disease (MASLD) emerge, patient eligibility assessment and monitoring is increasingly important. To assess the reliability of noninvasive imaging technologies in MASLD, we systematically reviewed the literature for studies on technical repeatability.

Methods

PubMed Central and MEDLINE were searched (2015-2025) for studies in MASLD that examined the repeatability of: magnetic resonance-derived iron-corrected T1 (cT1), liver fat content (LFC), and magnetic resonance elastography (MRE); ultrasound-based vibration controlled transient elastography liver stiffness measurement (VCTE LSM), controlled attenuation parameter and shear wave elastography. The relative repeatability coefficient (%RC) for same-day or different-day (%RC_DD) repeat tests were summarized, and available data pooled using random-effects-meta-analysis.

Results

Our search identified 19 studies including 1040 individuals (mean age 45 years; 43% female). The pooled random-effects average %RC_DD was 7% for cT1, 12% for LFC, 22% for MRE, 73% for VCTE LSM, and 26% for controlled attenuation parameter, with a median interval of 14 days between repeat scans. Relative repeated measures on the same day values were consistently lower across all tests. In the context of MASLD monitoring, the %RC_DD for MRE and VCTE LSM overlapped with previously reported thresholds for clinically-meaningful change for treatment responders (15% and 30%, respectively). In contrast, the %RCs for cT1 and LFC were below established thresholds for clinically-meaningful change (9% and 30%, respectively).

Conclusion

This systematic review and meta-analysis showed that cT1 and LFC are more reliable in detecting change in liver health in people living with MASLD-metabolic dysfunction-associated steatohepatitis, compared to liver stiffness. Liver stiffness measurements are less suited to monitoring individual patients.

随着代谢功能障碍相关脂肪变性肝病（MASLD）患者治疗的出现，患者资格评估和监测变得越来越重要。为了评估非侵入性成像技术在MASLD中的可靠性，我们系统地回顾了技术可重复性研究的文献。PubMed Central和MEDLINE检索了2015-2025年MASLD的研究，这些研究检查了磁共振（MR）衍生的铁校正T1 （cT1）、肝脏脂肪含量（LFC）和弹性成像（MRE）的可重复性；基于超声振动控制的瞬态弹性成像肝脏刚度测量（VCTE LSM）、可控衰减参数（CAP）和剪切波弹性成像（SWE）。总结当日（%RCSD）或隔日（%RCDD）重复试验的相对可重复性系数（%RC），并采用随机效应荟萃分析合并可用数据。我们的搜索确定了19项研究，包括1,040名个体（平均年龄45岁，43%为女性）。cT1组随机效应平均%RCDD为7%，LFC组为12%，MRE组为22%，VCTE组为73%，CAP组为26%，重复扫描的中位间隔为14天。%RCSD值在所有测试中始终较低。在MASLD监测的背景下，MRE和VCTE LSM的%RCDD与先前报道的治疗应答的临床意义变化阈值（分别为15%和30%）重叠。相比之下，cT1和LFC的RCs百分比低于临床意义变化的既定阈值（分别为9%和30%）。该系统综述和荟萃分析显示，与肝脏僵硬度相比，cT1和LFC在检测MASLD-MASH患者肝脏健康变化方面更可靠。肝硬度测量不太适合监测个别患者。

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引用次数: 0

Approach and Challenges for Patients With Advanced Radioiodine-Refractory Differentiated Thyroid Cancer 晚期放射性碘难治性分化型甲状腺癌的治疗方法与挑战。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.203

Dipen C. Patel MD , Irina Azaryan MD , Bhavana Konda MD, MPH

Objective

Differentiated thyroid cancers (DTCs), including papillary, follicular, and oncocytic subtypes, are generally associated with an excellent prognosis due to their responsiveness to conventional therapies, including surgery, thyroid-stimulating hormone suppression, and radioactive iodine (RAI) therapy. However, a subset of patients develops RAI-refractory disease, characterized by tumor dedifferentiation and loss of iodine avidity, resulting in disease progression despite standard interventions. This group poses a considerable therapeutic challenge and is associated with markedly poorer outcomes. This review summarizes contemporary approaches to advanced RAI-refractory DTC.

Methods

This review consolidates the current evidence for diagnosing, evaluating, and managing advanced RAI-refractory DTC.

Results

Recent advancements have transformed the treatment landscape with the emergence of systemic therapies such as antiangiogenic multikinase inhibitors and genotype-directed therapies that offer improved disease control in advanced settings. It explores the pathophysiological basis of refractoriness, prognostic implications, treatment selection strategies—including active surveillance, locoregional therapies, redifferentiation approaches, and systemic therapies—and outlines practical considerations for clinicians.

Conclusion

Future directions including immunotherapy, novel agents, and personalized therapy strategies are also discussed. Emphasis is placed on precision oncology, toxicity management, and the role of multidisciplinary care in optimizing patient outcomes.

分化型甲状腺癌（DTC），包括乳头状、滤泡和嗜瘤细胞亚型，由于其对常规治疗的反应性，包括手术、促甲状腺激素（TSH）抑制和放射性碘（RAI）治疗，通常具有良好的预后。然而，一小部分患者发展为放射性碘难治性（RAI-R）疾病，其特征是肿瘤去分化和碘亲和力丧失，尽管有标准干预，但仍导致疾病进展。这一组具有相当大的治疗挑战，并且与明显较差的结果相关。随着抗血管生成多激酶抑制剂（aaMKIs）和基因型导向疗法等系统性疗法的出现，最近的进展已经改变了治疗领域，这些疗法可以改善晚期疾病的控制。本文综述了目前诊断、评估和治疗晚期rar - r DTC的证据。它探讨了难治性的病理生理学基础、预后影响、治疗选择策略——包括积极监测、局部区域治疗、再分化方法和全身治疗——并概述了临床医生的实际考虑。未来的发展方向包括免疫治疗、新型药物和个性化治疗策略。重点放在精确肿瘤学、毒性管理和多学科护理在优化患者结果中的作用。

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引用次数: 0

Growth Hormone Assay-Adjusted Standardization Reveals Distinct Clinical Phenotypes in Acromegaly 生长激素测定-调整标准化揭示肢端肥大症不同的临床表型。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.10.006

Betina Biagetti MD , Pedro Marques MD , Roser Ferrer MD , Luís Miguel Cardoso MD , Eva Venegas Moreno MD , Carmen Fajardo-Montañana MD , Laura Gonzalez-Fernandez MD , Marta María Pérez Pena MD , Rogelio García-Centeno MD , Claudia Lozano-Aida MD , Iría Novoa-Testa MD , Eider Pascual-Corrales MD , Raúl Sánchón MD , Fernando Guerrero-Pérez MD , Rosario Oliva Rodríguez MD , Beatriz Rodríguez Jiménez MD , María Dolores Ollero García MD , Ana Irigaray Echarri MD , Andreu Simó-Servat MD , María Dolores Moure Rodríguez MD , Marta Araujo-Castro MD

Objective

To identify distinct clinical phenotypes in acromegaly based on growth hormone (GH) assay standardization and unsupervised machine learning.

Methods

This was a multicenter cross-sectional analysis of 416 patients diagnosed with acromegaly from 2010 onward. Patients were stratified according to baseline serum GH levels standardized to the assay-specific upper limit of normal (GHxULN) using a binary classification (GH-B: <1.0×ULN vs ≥1.0×ULN) and a four-tier classification (GH-4: <0.25, 0.25-0.99, 1.0-9.9, ≥10×ULN). Unsupervised cluster analysis included age, GHxULN, insulin-like growth factor 1 (IGF-1)xULN, tumor diameter, and T2-weighted signal intensity.

Results

Overall, 36% of patients had GH levels within the normal reference range for their assay (GH-B <1.0×ULN). Microadenomas (23.1%) were more frequent in older patients and associated with lower GH/IGF-1 levels. Across GH-4 categories, significant gradients were observed for age (z = −5.34, P < .001), tumor size (z = 8.01, P < .001), IGF-1 (z = 9.00, P < .001), and symptom duration (z = 4.34, P < .001). Higher GH categories were associated with greater odds of arthropathy (odds ratio 3.5, P = .015 for 1.0-9.9×ULN and odds ratio 6.58, P = .002 for ≥10×ULN). Cluster analysis revealed 3 phenotypes: cluster 1 (49.0%) [older age, lower GH/IGF-1, intermediate tumor size]; cluster 2 (44.4%) [intermediate age, moderate biochemical activity, smaller tumors]; cluster 3 (6.6%) [younger age, markedly elevated GH/IGF-1, large aggressive tumors].

Conclusion

GH standardization to assay-specific ULN reveals clinically meaningful phenotypes in acromegaly that correlate with age, tumor characteristics, and disease severity (particularly arthropathy). GHxULN complements IGF-1 by capturing tumor secretory activity, and this stratification approach may support more individualized clinical decision-making.

目的：基于生长激素测定标准化和无监督机器学习识别肢端肥大症的不同临床表型。方法：对2010年以来诊断为肢端肥大症的416例患者进行多中心横断面分析。采用二分类(GH- b：结果：总体而言，36%的患者的GH水平在其测定的正常参考范围内（GH- b结论：GH标准化以测定特异性ULN揭示了肢端肥大症与年龄、肿瘤特征和疾病严重程度（特别是关节病）相关的临床有意义的表型）。GHxULN通过捕获肿瘤分泌活性来补充IGF-1，这种分层方法可能支持更个性化的临床决策。

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引用次数: 0

Impairment of Bone Mineral Density in Pituitary Thyrotropin-Secreting Adenomas: A Retrospective, Controlled Study 垂体tsh分泌腺瘤骨密度损害：一项回顾性对照研究。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.202

Jie Liu MD , Yanying Li MD , Jifang Liu MD , Zhang Ye MD , He Liu MD , Xiaofeng Chai MD , Huijuan Zhu MD , Bing Xing MD , Wei Lian MD , Xiaolan Lian MD , Naishi Li MD , Lin Lu MD , Mei Zhang MD , Lian Duan MD, PhD , Yong Yao MD , Kan Deng MD

Objective

Hyperthyroidism can harm bone health, though it is seldom reported in thyroid-stimulating hormone/thyrotropin (TSH)-secreting adenomas (TSHomas). This study assessed bone mineral density (BMD) and bone turnover markers (BTMs) in TSHoma patients versus euthyroid controls and evaluated the impact of surgical treatment on bone metabolism.

Methods

We retrospectively reviewed 85 TSHoma patients who underwent BMD tests. Of these, 71 had baseline BMD data and were matched with 71 euthyroid healthy controls by age, sex, and body mass index. BMD and BTMs were measured.

Results

TSHoma patients demonstrated significantly reduced BMD compared to matched euthyroid controls across all skeletal sites, with reductions of 12.4% at the lumbar spine (1.06 ± 0.19 vs 1.21 ± 0.14 g/cm², P < .001), 8.5% at the femoral neck (0.86 ± 0.14 vs 0.94 ± 0.11 g/cm², P < .001), and 14.8% at the total hip (0.86 ± 0.13 vs 1.01 ± 0.12 g/cm², P < .001). Baseline BTMs revealed elevated osteoblastic (procollagen type 1 N-terminal propeptide 108 [60, 202] ng/mL) and osteoclastic markers (β-CTX 0.84 [0.62, 1.30] ng/mL). Besides, free triiodothyronine showed strong positive correlations with BTMs: alkaline phosphatase (r = 0.45, P = .005), β-CTX (r = 0.61, P < .001), and procollagen type 1 N-terminal propeptide (r = 0.73, P = .004). Following tumor resection, BTMs decreased significantly: alkaline phosphatase (91 [74.25, 119.5] to 71 [68, 94] U/L, P = .003) and β-CTX (0.75 [0.57, 0.94] to 0.22 [0.21, 0.45] ng/mL, P = .008). Postoperative BMD revealed stabilization with nonsignificant improvements at all measured skeletal sites.

Conclusion

TSHoma patients exhibit significant BMD deficits compared to euthyroid controls. Surgery effectively reduces BTMs while stabilizing BMD, preventing further deterioration rather than restoring bone density.

目的：甲状腺机能亢进对骨健康有危害，但在tshoma中很少报道。本研究评估了TSHoma患者与正常甲状腺对照者的骨密度（BMD）和骨转换标志物（BTM），并评估了手术治疗对骨代谢的影响。方法：我们回顾性分析了85例接受BMD检查的TSHoma患者。其中，71人有基线BMD数据，并与71名甲状腺功能正常的健康对照者按年龄、性别和BMI进行匹配。测量BMD和btm。结果：与匹配的甲状腺正常对照组相比，TSHoma患者在所有骨骼部位的骨密度均显著降低，腰椎降低12.4%(1.06±0.19比1.21±0.14 g/cm2, p < 0.001)，股骨颈降低8.5%(0.86±0.14比0.94±0.11 g/cm2, p < 0.001)，全髋关节降低14.8%（0.86±0.13比1.01±0.12 g/cm2, p < 0.001）。基线BTMs显示成骨细胞[P1NP 108 (60,202) ng/mL]和破骨细胞标志物[β-CTX 0.84 (0.62, 1.30) ng/mL]升高。FT3与BTMs呈显著正相关：ALP （r = 0.45, p = 0.005）、β-CTX （r = 0.61, p < 0.001）、P1NP （r = 0.73, p = 0.004）。肿瘤切除后，BTMs显著降低：ALP[91（74.25, 119.5）至71 (68,94)U/L, p = 0.003], β-CTX[0.75（0.57, 0.94）至0.22 (0.21,0.45)ng/mL, p = 0.008]。术后骨密度显示稳定，所有测量的骨骼部位均无明显改善。结论：与甲状腺功能正常的对照组相比，TSHoma患者表现出明显的BMD缺陷。手术有效地减少脑转移，同时稳定骨密度，防止进一步恶化，而不是恢复骨密度。

{"title":"Impairment of Bone Mineral Density in Pituitary Thyrotropin-Secreting Adenomas: A Retrospective, Controlled Study","authors":"Jie Liu MD , Yanying Li MD , Jifang Liu MD , Zhang Ye MD , He Liu MD , Xiaofeng Chai MD , Huijuan Zhu MD , Bing Xing MD , Wei Lian MD , Xiaolan Lian MD , Naishi Li MD , Lin Lu MD , Mei Zhang MD , Lian Duan MD, PhD , Yong Yao MD , Kan Deng MD","doi":"10.1016/j.eprac.2025.09.202","DOIUrl":"10.1016/j.eprac.2025.09.202","url":null,"abstract":"<div><h3>Objective</h3><div>Hyperthyroidism can harm bone health, though it is seldom reported in thyroid-stimulating hormone/thyrotropin (TSH)-secreting adenomas (TSHomas). This study assessed bone mineral density (BMD) and bone turnover markers (BTMs) in TSHoma patients versus euthyroid controls and evaluated the impact of surgical treatment on bone metabolism.</div></div><div><h3>Methods</h3><div>We retrospectively reviewed 85 TSHoma patients who underwent BMD tests. Of these, 71 had baseline BMD data and were matched with 71 euthyroid healthy controls by age, sex, and body mass index. BMD and BTMs were measured.</div></div><div><h3>Results</h3><div>TSHoma patients demonstrated significantly reduced BMD compared to matched euthyroid controls across all skeletal sites, with reductions of 12.4% at the lumbar spine (1.06 ± 0.19 vs 1.21 ± 0.14 g/cm<sup>2</sup>, <em>P</em> < .001), 8.5% at the femoral neck (0.86 ± 0.14 vs 0.94 ± 0.11 g/cm<sup>2</sup>, <em>P</em> < .001), and 14.8% at the total hip (0.86 ± 0.13 vs 1.01 ± 0.12 g/cm<sup>2</sup>, <em>P</em> < .001). Baseline BTMs revealed elevated osteoblastic (procollagen type 1 N-terminal propeptide 108 [60, 202] ng/mL) and osteoclastic markers (β-CTX 0.84 [0.62, 1.30] ng/mL). Besides, free triiodothyronine showed strong positive correlations with BTMs: alkaline phosphatase (<em>r</em> = 0.45, <em>P</em> = .005), β-CTX (<em>r</em> = 0.61, <em>P</em> < .001), and procollagen type 1 N-terminal propeptide (<em>r</em> = 0.73, <em>P</em> = .004). Following tumor resection, BTMs decreased significantly: alkaline phosphatase (91 [74.25, 119.5] to 71 [68, 94] U/L, <em>P</em> = .003) and β-CTX (0.75 [0.57, 0.94] to 0.22 [0.21, 0.45] ng/mL, <em>P</em> = .008). Postoperative BMD revealed stabilization with nonsignificant improvements at all measured skeletal sites.</div></div><div><h3>Conclusion</h3><div>TSHoma patients exhibit significant BMD deficits compared to euthyroid controls. Surgery effectively reduces BTMs while stabilizing BMD, preventing further deterioration rather than restoring bone density.</div></div>","PeriodicalId":11682,"journal":{"name":"Endocrine Practice","volume":"32 2","pages":"Pages 172-178"},"PeriodicalIF":4.6,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145212113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Effectiveness of Bisphosphonates and Denosumab on Risk of Vertebral Fractures in Prostate Cancer Patients Under Androgen Deprivation Therapies: A Real-World Prospective Study 双膦酸盐和地诺单抗对雄激素剥夺治疗前列腺癌患者椎体骨折风险的有效性：一项真实世界的前瞻性研究。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.201

Alberto Piasentier MD , Maria Francesca Birtolo MD , Bianca Turci MD , Walter Vena MD , Alessandro Fanti MD , Emanuela Morenghi MD , Lucrezia MS. Gentile MD , Luca Balzarini MD , Antonio C. Bossi MD , Alfredo Berruti MD , Giovanni Lughezzani MD , Ciro Franzese MD , Fabio De Vincenzo MD , Marta Scorsetti MD , Paolo A. Zucali MD , Andrea G. Lania MD, PhD , Gherardo Mazziotti MD, PhD

Objective

This prospective study investigated the real-world effectiveness of bisphosphonates and denosumab in reducing vertebral fracture (VF) risk induced by androgen deprivation therapies (ADTs).

Methods

Two hundred twenty-six consecutive men (mean age 74.8 ± 6.9 years) undergoing ADT were evaluated for morphometric VFs at baseline and after 26.1 ± 10.7 months of follow-up (range 18-64). Following enrollment, 153 patients commenced bisphosphonates (98 cases) or denosumab (55 cases), while 73 patients (32.3%) received only vitamin D and calcium supplementation. As an additional control group, we enrolled 62 consecutive men (mean age 74.9 ± 7.2 years) under chronic ADTs who had been neither evaluated nor treated for skeletal fragility before study entry.

Results

Incident VFs were found in 16 of 226 (7.1%) patients enrolled in the prospective study and in 18 of 62 (29.0%) control subjects (P < .001). The risk of VFs was significantly lower in patients treated with bone-active drugs as compared to those treated with vitamin D (hazard ratio 0.26, 95% CI 0.09-0.73; P = .01), without significant difference between denosumab and bisphosphonate treatment (P = .423), although denosumab-treated patients had lower bone mineral density, higher serum C-terminal telopeptide of type I collagen values, and more prevalent VFs. Noteworthy, incident VFs were significantly lower in patients receiving vitamin D with calcium as compared to those subjects under chronic ADT who had never been evaluated and treated for skeletal fragility prior to the study (12.3% vs 29.0%; P = .016).

Conclusion

In real-world clinical practice, bisphosphonates or denosumab might be effective in reducing the risk of VFs related to ADTs.

目的：这项前瞻性研究探讨了双膦酸盐和地诺单抗在降低雄激素剥夺疗法（ADTs）诱导的椎体骨折（VFs）风险方面的实际有效性。方法：226名连续接受ADT的男性（平均年龄74.8±6.9岁）在基线和26.1±10.7个月的随访（范围18-64）后评估形态计量学VFs。入组后，153例患者开始服用双膦酸盐（98例）或denosumab（55例），而73例患者（32.3%）仅服用维生素D和钙补充剂。作为额外的对照组，我们连续招募了62名患有慢性ADTs的男性（平均年龄74.9±7.2岁），他们在研究开始前从未接受过骨骼脆性评估或治疗。结果：在前瞻性研究中，16/226（7.1%）的患者和18/62（29.0%）的对照组中发现了偶发性室性心律失常。结论：在现实世界的临床实践中，双膦酸盐或地诺单抗可能有效降低与ADTs相关的室性心律失常的风险。

{"title":"Effectiveness of Bisphosphonates and Denosumab on Risk of Vertebral Fractures in Prostate Cancer Patients Under Androgen Deprivation Therapies: A Real-World Prospective Study","authors":"Alberto Piasentier MD , Maria Francesca Birtolo MD , Bianca Turci MD , Walter Vena MD , Alessandro Fanti MD , Emanuela Morenghi MD , Lucrezia MS. Gentile MD , Luca Balzarini MD , Antonio C. Bossi MD , Alfredo Berruti MD , Giovanni Lughezzani MD , Ciro Franzese MD , Fabio De Vincenzo MD , Marta Scorsetti MD , Paolo A. Zucali MD , Andrea G. Lania MD, PhD , Gherardo Mazziotti MD, PhD","doi":"10.1016/j.eprac.2025.09.201","DOIUrl":"10.1016/j.eprac.2025.09.201","url":null,"abstract":"<div><h3>Objective</h3><div>This prospective study investigated the real-world effectiveness of bisphosphonates and denosumab in reducing vertebral fracture (VF) risk induced by androgen deprivation therapies (ADTs).</div></div><div><h3>Methods</h3><div>Two hundred twenty-six consecutive men (mean age 74.8 ± 6.9 years) undergoing ADT were evaluated for morphometric VFs at baseline and after 26.1 ± 10.7 months of follow-up (range 18-64). Following enrollment, 153 patients commenced bisphosphonates (98 cases) or denosumab (55 cases), while 73 patients (32.3%) received only vitamin D and calcium supplementation. As an additional control group, we enrolled 62 consecutive men (mean age 74.9 ± 7.2 years) under chronic ADTs who had been neither evaluated nor treated for skeletal fragility before study entry.</div></div><div><h3>Results</h3><div>Incident VFs were found in 16 of 226 (7.1%) patients enrolled in the prospective study and in 18 of 62 (29.0%) control subjects (<em>P</em> < .001). The risk of VFs was significantly lower in patients treated with bone-active drugs as compared to those treated with vitamin D (hazard ratio 0.26, 95% CI 0.09-0.73; <em>P</em> = .01), without significant difference between denosumab and bisphosphonate treatment (<em>P</em> = .423), although denosumab-treated patients had lower bone mineral density, higher serum C-terminal telopeptide of type I collagen values, and more prevalent VFs. Noteworthy, incident VFs were significantly lower in patients receiving vitamin D with calcium as compared to those subjects under chronic ADT who had never been evaluated and treated for skeletal fragility prior to the study (12.3% vs 29.0%; <em>P</em> = .016).</div></div><div><h3>Conclusion</h3><div>In real-world clinical practice, bisphosphonates or denosumab might be effective in reducing the risk of VFs related to ADTs.</div></div>","PeriodicalId":11682,"journal":{"name":"Endocrine Practice","volume":"32 2","pages":"Pages 164-171"},"PeriodicalIF":4.6,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145212123","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Belzutifan for HIF2A-Related Pheochromocytoma and Paraganglioma: A Retrospective Study of Real-World Data Belzutifan治疗hif2a相关嗜铬细胞瘤和副神经节瘤：真实世界数据的回顾性研究。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.204

Hussam Alkaissi MD, MS , Sara Talvacchio RN , Alberta Derkyi CRNP , Sriram Gubbi MD , Alberto Pappo MD , Catherine M. Gordon MD, MS , John Glod MD, PhD , Zhengping Zhuang MD, PhD , Karel Pacak MD, PhD, DSc

Objectives

Pheochromocytoma and paraganglioma (PPGL) are rare neuroendocrine tumors of the adrenal medulla and autonomic ganglia, respectively. Germline and somatic genetic drivers are identified in up to 70% of cases. Hypoxia-inducible factor 2α (HIF-2α), encoded by the EPAS1 (HIF2A) gene, plays a central role in PPGL pathogenesis and can be stabilized directly or indirectly by pathogenic variants of several genes, collectively called pseudohypoxia cluster (or Cluster 1). Belzutifan, a small-molecule HIF-2α inhibitor, has demonstrated efficacy in von Hippel–Lindau-related cancers, renal cell carcinoma, and few case reports of PPGL. We report real-world outcomes of belzutifan therapy in 5 patients with recurrent, multifocal, or metastatic EPAS1 (HIF2A)-related PPGL.

Methods

Clinical parameters, biochemical markers, tumor burden (RECIST 1.1), and treatment-related adverse events were carefully monitored, and recorded.

Results

Four of 5 patients (80%) achieved a partial response, and one patient had stable disease, with no disease progression to date. The average reduction in the sum of tumor diameters was 36.8%. Chromogranin A, erythropoietin, and hemoglobin declined by 69%, 97%, and 13%, respectively, eliminating the need for therapeutic phlebotomy in patients with prior erythrocytosis. Catecholamine normalization allowed discontinuation or reduction of antihypertensive medications in 2 patients. Adverse events included hypoxia and anemia (1/5), mild transaminase elevation (2/5), and fatigue with weight gain (1/5).

Conclusions

Belzutifan is a highly effective and well-tolerated therapeutic option for EPAS1(HIF2A)-related PPGL, producing substantial biochemical and radiographic responses, improved blood pressure control, and resolution of erythrocytosis. These findings support belzutifan as a promising genotype-driven therapy for pseudohypoxia-driven PPGL.

目的：嗜铬细胞瘤和副神经节瘤（PPGL）分别是发生在肾上腺髓质和自主神经节的罕见神经内分泌肿瘤。在高达70%的病例中发现了生殖系和体细胞遗传驱动因素。由EPAS1 （HIF2A）基因编码的缺氧诱导因子2α (HIF-2α)在PPGL发病中起核心作用，可通过几种基因的致病变异直接或间接稳定，统称为假缺氧簇（或簇1）。Belzutifan是一种小分子HIF-2α抑制剂，已被证明对von hipel - lindau （VHL）相关癌症、肾细胞癌和PPGL有疗效。我们报告了贝祖替芬治疗5例复发性、多灶性或转移性EPAS1（HIF2A）相关PPGL患者的实际结果。方法：仔细监测临床参数、生化指标、肿瘤负荷（RECIST 1.1）、治疗相关不良事件。结果：5例患者中有4例（80%）达到部分缓解，1例患者病情稳定，迄今无疾病进展。肿瘤直径总和平均缩小36.8%。嗜铬粒蛋白A、促红细胞生成素和血红蛋白分别下降了69%、97%和13%，使既往有红细胞增多症的患者无需进行治疗性放血。儿茶酚胺正常化允许两名患者停用或减少抗高血压药物。不良事件包括缺氧和贫血（1/5），轻度转氨酶升高（2/5），体重增加时疲劳（1/5）。结论：对于EPAS1（HIF2A）相关PPGL， Belzutifan是一种高效且耐受性良好的治疗选择，可产生显著的生化和放射学反应，改善血压控制，并解决红细胞增多症。这些发现支持贝祖替芬作为一种有希望的基因型驱动治疗假性缺氧驱动的PPGL。

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引用次数: 0

A Review of Automated Insulin Delivery Use in Type 1 Diabetes During Pregnancy 妊娠期1型糖尿病患者自动胰岛素输送的研究综述

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.10.015

Elaine Young AB , Andrew R. Goyette AB , Jeremy F. Alkire AB , Nour L. Khachemoune AB , Natalie Bellini DNP, BC-ADM, CDCES , Diana Isaacs PharmD, BCPS, BCACP, CDCES, BC-ADM, FADCES, FCCP

Objective

To review the clinical evidence for automated insulin delivery (AID) use during pregnancy in people with pregestational diabetes, provide an overview of AID systems available in the United States, and offer practical tips and considerations for clinicians working with pregnant patients on these systems.

Methods

We synthesized findings from all randomized controlled trials investigating AID use in people with pregestational diabetes. We also compared the features of 6 AID systems and shared clinical insights on how their settings can be adjusted to better meet pregnancy-specific glycemic targets.

Results

Six randomized controlled trials were included, all in type 1 diabetes. Some demonstrated better glycemic outcomes in patients using AID compared to sensor-augmented pump therapy or standard insulin therapy. Others found no significant differences. Maternal and neonatal outcomes were similar to standard care, though some studies found that AID users had reduced gestational weight gain among other improvements. CamAPS FX is the only food and Drug Administration-cleared AID system for use in type 1 diabetes pregnancy, though it is not yet available in the United States. While no AID system has demonstrated the ability to meet all pregnancy-specific glycemic targets, some systems are more customizable making it easier to achieve the tighter glycemic targets in pregnancy.

Conclusion

For pregnant women using AID systems, there are strategies and workarounds to aid in achieving pregnancy-specific glycemic targets. However, no system consistently meets all targets. More research is needed to understand how AID use during pregnancy impacts maternal and fetal outcomes and patient-reported outcomes, especially for pre-existing type 2 diabetes.

本综述旨在：(i)总结妊娠期糖尿病患者使用自动胰岛素输送（AID）的临床证据，（ii）概述美国可用的AID系统，（iii）为临床医生使用这些系统治疗妊娠患者提供实用提示和注意事项。我们综合了调查妊娠期1型糖尿病患者使用艾滋病药物的所有六项随机对照试验的结果。一些研究表明，与传感器增强泵（SAP）治疗或标准胰岛素治疗相比，使用AID的患者血糖结局更好。其他人则没有发现显著差异。产妇和新生儿的结局与标准护理相似，尽管一些研究发现，使用艾滋病药物可以减少妊娠期体重增加。我们还比较了六种AID系统的特点，并分享了如何调整其设置以更好地满足妊娠特异性血糖目标的临床见解。CamAPS FX是fda唯一批准用于T1D妊娠的AID系统，尽管它尚未在美国上市。虽然没有AID系统能够满足所有妊娠期特定的血糖目标，但一些系统更具可定制性，使其更容易达到妊娠期更严格的血糖目标。总之，对于使用AID系统的孕妇，有一些策略和方法可以帮助实现妊娠特异性血糖目标。然而，没有一个系统能够始终满足所有的目标。需要更多的研究来了解妊娠期间使用抗艾滋病药物如何影响母体和胎儿的结局以及患者报告的结局，特别是对于已经存在的2型糖尿病患者。

{"title":"A Review of Automated Insulin Delivery Use in Type 1 Diabetes During Pregnancy","authors":"Elaine Young AB , Andrew R. Goyette AB , Jeremy F. Alkire AB , Nour L. Khachemoune AB , Natalie Bellini DNP, BC-ADM, CDCES , Diana Isaacs PharmD, BCPS, BCACP, CDCES, BC-ADM, FADCES, FCCP","doi":"10.1016/j.eprac.2025.10.015","DOIUrl":"10.1016/j.eprac.2025.10.015","url":null,"abstract":"<div><h3>Objective</h3><div>To review the clinical evidence for automated insulin delivery (AID) use during pregnancy in people with pregestational diabetes, provide an overview of AID systems available in the United States, and offer practical tips and considerations for clinicians working with pregnant patients on these systems.</div></div><div><h3>Methods</h3><div>We synthesized findings from all randomized controlled trials investigating AID use in people with pregestational diabetes. We also compared the features of 6 AID systems and shared clinical insights on how their settings can be adjusted to better meet pregnancy-specific glycemic targets.</div></div><div><h3>Results</h3><div>Six randomized controlled trials were included, all in type 1 diabetes. Some demonstrated better glycemic outcomes in patients using AID compared to sensor-augmented pump therapy or standard insulin therapy. Others found no significant differences. Maternal and neonatal outcomes were similar to standard care, though some studies found that AID users had reduced gestational weight gain among other improvements. CamAPS FX is the only food and Drug Administration-cleared AID system for use in type 1 diabetes pregnancy, though it is not yet available in the United States. While no AID system has demonstrated the ability to meet all pregnancy-specific glycemic targets, some systems are more customizable making it easier to achieve the tighter glycemic targets in pregnancy.</div></div><div><h3>Conclusion</h3><div>For pregnant women using AID systems, there are strategies and workarounds to aid in achieving pregnancy-specific glycemic targets. However, no system consistently meets all targets. More research is needed to understand how AID use during pregnancy impacts maternal and fetal outcomes and patient-reported outcomes, especially for pre-existing type 2 diabetes.</div></div>","PeriodicalId":11682,"journal":{"name":"Endocrine Practice","volume":"32 2","pages":"Pages 286-292"},"PeriodicalIF":4.6,"publicationDate":"2026-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145408480","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

引用次数: 0

Redefining Thyroid Nodule Evaluation: Demographics, Misleading Symptoms, and Diagnostic Challenges. Insights From a Multicenter Study 重新定义甲状腺结节评估：人口统计学、误导性症状和诊断挑战。来自多中心研究的见解。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.08.007

Laura Croce MD, PhD , Rosaria Maddalena Ruggeri MD, PhD , Marsida Teliti MD , Luca Rossi MD , Elena Petrosino MD , Paolo Caccavale MD , Martina Laganà MD , Spyridon Chytiris MD , Carlo Cappelli MD , Salvatore Cannavò MD , Mario Rotondi MD, PhD

Objective

Thyroid ultrasound (US) is the cornerstone for diagnosing nodular thyroid disease, yet many US examinations are prompted by nonspecific local symptoms (LS) like dysphagia or a palpable neck mass (NM). The clinical utility of such referrals remains debated.

Methods

This multicenter retrospective study analyzed 614 patients diagnosed with thyroid nodules (TNs) via US from 2 endocrinology centers in Italy between December 2021 and October 2022. Patients were grouped based on referral reason: symptomatic TNs, further subdivided into NM and LS, versus nonsymptomatic TNs. Clinical, ultrasonographic, and management data were compared.

Results

Symptomatic TNs accounted for 28.7% of cases (19.2% NM, 9.5% LS). NM patients were younger, more often female, and had larger, often cystic or isthmic-located nodules than nonsymptomatic TN patients. Conversely, LS patients had no significant differences in thyroid volume or nodule size but showed a higher prevalence of gastroesophageal reflux disease. Fine-needle aspiration was more common in the NM group because of larger nodules, but malignancy rates did not differ across groups. Surgical rates were similar, whereas thermal ablation was more frequent in the NM group.

Conclusions

A third of TNs are diagnosed during US prompted by LS, yet only NMs are associated with distinct nodule characteristics. Dysphagia and dysphonia were nonspecific and more related to gastroesophageal reflux disease than TNs. These findings support caution against overuse of US. Demographics, nodule features, and location should guide clinical suspicion and imaging decisions to avoid unnecessary imaging and interventions.

目的：甲状腺超声（US）是诊断甲状腺结节性疾病的基础，然而许多US检查是由非特异性的局部症状引起的，如吞咽困难或可触及的颈部肿块。这种转诊的临床效用仍然存在争议。方法：这项多中心回顾性研究分析了2021年12月至2022年10月期间意大利两个内分泌中心通过US诊断为甲状腺结节（TNs）的614例患者。患者根据转诊原因分组：有症状的TNs (STN)，进一步细分为颈部肿块（NM）和局部症状（LS），与无症状的TNs （nSTN）。比较临床、超声和治疗资料。结果：STNs占28.7%，其中NM 19.2%, LS 9.5%。NM患者较年轻，多为女性，与nSTN患者相比，结节较大，常为囊性或峡部结节。相反，LS患者在甲状腺体积和结节大小上没有显著差异，但胃食管反流的患病率更高。由于较大的结节，FNA在NM组中更常见，但各组间的恶性率没有差异。手术率相似，而热消融在NM组更频繁。结论：三分之一的TNs是由局部症状引起的，但只有颈部肿块与明显的结节特征相关。吞咽困难和发音困难是非特异性的，与胃食管反流的相关性大于tnn。这些发现支持了对过度使用US。人口统计学、结节特征和位置应指导临床怀疑和影像学决定，以避免不必要的影像学和干预。

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引用次数: 0

Insulin Discontinuation in Youth With New-Onset Type 2 Diabetes Presenting With and Without Diabetic Ketoacidosis 伴有或不伴有糖尿病酮症酸中毒的新发2型糖尿病青年患者胰岛素停药

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.10.001

Shuai Hao MD , Adrianna L. Westbrook MPH , Cynthia Sinha PhD , Daniel S. Hsia MD , Priyathama Vellanki MD

Objective

Heterogeneity in the clinical course of type 2 diabetes in youth presenting with and without diabetic ketoacidosis (DKA) at diagnosis is not well described. We aimed to characterize if presentation of type 2 diabetes with DKA affects rates of insulin discontinuation compared with type 2 diabetes without ketosis (non-DKA).

Methods

This single-center retrospective cohort study included patients (body mass index z-score 2.4, IQR 2.07, 2.65) with a mean age of 15 years (IQR 13, 16), hospitalized for new-onset diabetes with DKA (n = 79) or non-DKA (n = 356) at an academic pediatric institution from January 2019 to December 2021 with follow-up until May 2023. All patients were initiated on insulin and titrated per their care team. Type 1 diabetes was excluded by presence of autoimmune antibodies. Primary outcomes were time to insulin discontinuation of insulin therapy and maintenance of discontinuation.

Results

Time to insulin discontinuation and proportion who discontinued did not differ between DKA and non-DKA groups (48.1% vs 44.7% respectively, P = .58). Trajectory analyses combining DKA and non-DKA groups identified 3 insulin discontinuation groups: early (20.1% within 2 months), late (12.5% within 6 months), and never (67.4%). Multinomial regression shows that DKA at presentation is not associated with early (P = .48) or late insulin discontinuation (P = .70) compared with never discontinuation. Insulin was restarted in 37 participants with median of 20 months (IQR 16, 24) after insulin discontinuation.

Conclusions

Despite not showing differences in insulin discontinuation in youth with new-onset type 2 diabetes with and without DKA at presentation, we identified heterogeneity in duration of insulin treatment in the combined group.

目的：青年2型糖尿病在诊断时有和没有糖尿病酮症酸中毒（DKA）的临床过程的异质性尚未得到很好的描述。我们的目的是确定与没有酮症的2型糖尿病（非DKA）相比，2型糖尿病合并DKA是否会影响胰岛素停药率。方法：这项单中心回顾性队列研究纳入了2019年1月至2021年12月在一家儿科学术机构因新发糖尿病合并DKA （n= 79）或非DKA （n=356）住院的患者（BMI Z-score 2.4, IQR 2.07,2.65），平均年龄15岁（IQR 13,16），随访至2023年5月。所有患者都开始使用胰岛素，并按护理小组滴定。自身免疫抗体排除了1型糖尿病。主要结局是胰岛素停药的时间和停药的维持时间。结果：胰岛素停药时间和停药比例在DKA组和非DKA组之间无差异（分别为48.1 vs 44.7%, p=0.58）。结合DKA组和非DKA组进行轨迹分析，确定了3个胰岛素停药组：早期（2个月内20.1%）、晚期（6个月内12.5%）和从未停药（67.4%）。多项回归显示，与从未停药相比，出现时的DKA与早期（p=0.48）或晚期胰岛素停药（p=0.70）无关。37名受试者在胰岛素停药后中位时间为20个月（IQR 16,24）重新开始使用胰岛素。结论：尽管新发2型糖尿病青年伴和不伴DKA患者在胰岛素停药方面没有差异，但我们确定了联合组胰岛素治疗持续时间的异质性。

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引用次数: 0

Glucocorticoid Resistance Syndrome: A Systematic Review of the Genotypes, Phenotypes, and Their Relationships 糖皮质激素抵抗综合征：基因型、表型及其关系的系统综述。

IF 4.6 3区医学 Q2 ENDOCRINOLOGY & METABOLISM

Endocrine Practice

Pub Date : 2026-02-01 DOI: 10.1016/j.eprac.2025.09.199

Shirui Wang MD, Wan Su MBBCH, Lian Duan MD, PhD, Fengying Gong PhD, Shi Chen MD, Linjie Wang MD, PhD, Hui Pan MD, Lin Lu MD, Huijuan Zhu MD

Objective

Generalized glucocorticoid residestance syndrome is a rare disorder caused by mutations in NR3C1. We aimed to systematically characterize its clinical, biochemical, and genetic features and quantitatively evaluate their inter-relationships.

Methods

We conducted a systematic literature review, identifying 67 cases from 33 published reports, and included 4 unreported cases from Peking Union Medical College Hospital.

Results

In total, 71 cases from 43 unrelated families were analyzed, encompassing 39 distinct NR3C1 mutations. The most frequent clinical manifestations were symptoms of androgen excess (43.3%), followed by mineralocorticoid excess (38.8%) and glucocorticoid deficiency (14.9%). Elevated post-low-dose dexamethasone suppression test cortisol was the predominant hormonal abnormality (97.8%). Adrenal computed tomography revealed hyperplasia or adenomas in 68.8% of evaluated patients. Compared with heterozygotes, those with homozygous/compound heterozygous mutations presented earlier (19.9 vs 35.5 year old, P = .007), with markedly higher prevalence of hypertension/hypokalemia (90.9% vs 28.6%, odds ratio 25.00 [95% confidence interval 2.95 -211.61], P < .001) and higher cortisol (3.20 [1.19, 2.63] vs 1.40 [0.59, 1.20] × upper limit of normal [ULN], P = .002), adrenocorticotropic hormone (4.81 [1.71, 13.00] vs 1.04 [1.00, 1.76] × ULN, P = .001), and testosterone levels (1.84 [1.34, 2.79] vs 0.89 [0.55, 1.77] × ULN, P = .047). Sensitivity analyses restricted to probands confirmed these associations. Clinical manifestations correlated with elevated cortisol, and lower renin was observed in patients with hypertension/hypokalemia.

Conclusion

This study provided the most comprehensive quantitative synthesis to date of glucocorticoid resistance syndrome, highlighting genotype-phenotype correlations and advancing understanding of its clinical and hormonal spectrum.

目的：广泛性糖皮质激素抵抗综合征是一种由NR3C1基因突变引起的罕见疾病。我们旨在系统地描述其临床、生化和遗传特征，并定量评估它们之间的相互关系。方法：我们进行了系统的文献综述，从33篇已发表的报告中筛选出67例病例，并纳入4例来自北京协和医院的未报告病例。结果：共分析了43个不相关家族的71例病例，包括39种不同的NR3C1突变。最常见的临床表现是雄激素过量（43.3%），其次是矿皮质激素过量（38.8%）和糖皮质激素缺乏（14.9%）。ldst后皮质醇升高是主要的激素异常（97.8%）。肾上腺CT显示68.8%的患者有增生或腺瘤。与杂合的相比,那些纯合子/复合的杂合突变了早些时候(19.9 vs 35.5岁,P = 0.007),高血压患病率明显更高/低血钾(90.9%比28.6%,或25.00 (95% CI 2.95 - -211.61), P < 0.001)和更高的皮质醇(3.20[1.19,2.63]和[0.59,1.20]×1.40 ULN, P = 0.002), ACTH(4.81[1.71, 13.00]和[1.00,1.76]×1.04 ULN, P = 0.001),和睾酮水平(1.84[1.34,2.79]和[0.55,1.77]×0.89 ULN, P = 0.047)。局限于先证者的敏感性分析证实了这些关联。高血压/低血钾患者的临床表现与皮质醇升高、肾素降低相关。结论：本研究提供了迄今为止最全面的糖皮质激素抵抗综合征的定量合成，突出了基因型-表型相关性，并推进了对其临床和激素谱的理解。

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引用次数: 0