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嗜铬细胞瘤-副神经节瘤

Pheochromocytoma-paraganglioma

ORPHA:573163疾病组中国目录 第2批 · 60

定义

一种罕见的神经内分泌肿瘤,产生于肾上腺髓质的嗜铬细胞瘤(嗜铬细胞瘤)或交感神经节和副交感神经节(副神经节瘤)。这些肿瘤最常见的是良性的,可能会产生过多的儿茶酚胺,引起高血压,有时还会引起严重的急性心血管并发症。

近两年的全球研究 402L2

2024/08 起在 Europe PMC 检索所得,按发表时间倒序显示最近 20 篇。标题未译成中文——自动翻译需要接入 LLM 服务,尚未引入。

  • 2026-07
    Histological spectrum of resected adrenal tumors in the 11-20 HU grey zone on unenhanced CT: a retrospective cohort study
    Updates in surgery · DOI · Europe PMC
  • 2026-07病例报告
    Expansion of Germline Variants in Primary Hyperparathyroidism: Fumarate Hydratase Deficiency as a Cause of Parathyroid Adenomas
    Endocrine pathology · DOI · Europe PMC
  • 2026-07
    A multistakeholder call for belzutifan biomarker data in advanced pheochromocytoma and paraganglioma
    Trends in endocrinology and metabolism: TEM · DOI · Europe PMC
  • 2026-07
    Clinical Impact of Germline Multigene Sequencing in Pediatric Cohorts with a Wide Spectrum of Neoplasms
    International journal of molecular sciences · DOI · Europe PMC
  • 2026-07
    OncoMimic peptide-based immunotherapy EO2401 induces shared T cell clusters in patients with adrenal cancer
    Communications medicine · DOI · Europe PMC
  • 2026-07
    Systemic management of adrenal malignancies: adrenocortical carcinoma and pheochromocytoma/paraganglioma
    Endocrine connections · DOI · Europe PMC
  • 2026-07
    Clinical presentation and outcomes of sporadic neuroendocrine neoplasms in young adults
    European journal of endocrinology · DOI · Europe PMC
  • 2026-06
    Intense Incidental F-18 DOPA Uptake in Low-grade Prostate Adenocarcinoma: A Potential Diagnostic Pitfall
    Clinical nuclear medicine · DOI · Europe PMC
  • 2026-06综述
    Adrenal tumor microenvironment: hormone-immune crosstalk, molecular heterogeneity, and immunotherapeutic opportunities
    Frontiers in immunology · DOI · Europe PMC
  • 2026-06开放获取
    Recurrent PRES in a Patient With Cyclic Vomiting Syndrome
    Case reports in radiology · DOI · Europe PMC
  • 2026-06开放获取
    Retroperitoneal Extra-Adrenal Paraganglioma Presenting With Secondary Amenorrhea and Hypertension in an Adolescent: A Rare Case From Sub-Saharan Africa
    Clinical case reports · DOI · Europe PMC
  • 2026-06
    Outcomes With Peptide Receptor Radionuclide Therapy in Patients With Pheochromocytoma/Paraganglioma: A Center of Excellence Experience
    American journal of therapeutics · DOI · Europe PMC
  • 2026-06病例报告
    Synchronous Bilateral Carotid Body and Bilateral Vagal Paragangliomas: A Case Report and Literature Review
    Journal of clinical medicine · DOI · Europe PMC
  • 2026-06综述开放获取
    The cGAS-STING pathway in cancer immunotherapy: prognostic value and therapeutic potential
    Frontiers in immunology · DOI · Europe PMC
  • 2026-06病例报告开放获取
    Synchronous Bilateral Carotid Body and Bilateral Vagal Paragangliomas: A Case Report and Literature Review
    Journal of clinical medicine
  • 2026-06开放获取
    Can clinicians predict individual patient outcomes in neuroendocrine tumors treated with [177Lu]Lu-DOTATATE?
    The oncologist · DOI · Europe PMC
  • 2026-06
    Pan-cancer atlas of ITGA6 unveils its multifaceted oncogenic roles and therapeutic potential in uveal melanoma
    BMC cancer · DOI · Europe PMC
  • 2026-06综述开放获取
    Genetics of familial acromegaly and pituitary gigantism
    The Journal of clinical endocrinology and metabolism · DOI · Europe PMC
  • 2026-06综述
    Effects of catecholamines on bone and mineral metabolism in patients with pheochromocytoma and paraganglioma
    The Journal of clinical endocrinology and metabolism · DOI · Europe PMC
  • 2026-05综述开放获取
    Updates on Minimally Invasive Treatment of Adrenal Tumors
    Cancers · DOI · Europe PMC

境外已获批用于本病的药物 1L2

欧盟 0 项、美国 1 项。同一药物在两地各批一次的,会分别列出。

「境外已获批」不等于「在中国能用」。中间隔着进口注册、临床急需境外新药通道、海南博鳌乐城国际医疗旅游先行区等几条路径,各有各的条件与费用。这一节能确定地告诉你的只有一件事:这个病在世界范围内已经有获得批准的药物,它叫什么名字。拿这个名字去问主治医生,是下一步最省力的做法。

药名一律保留英文原文,不作翻译——中国的药品通用名与英文名的音译经常不一致,译错会让人去找一个不存在的药。

数据来自欧洲药品管理局(EMA)的药品与孤儿药资格公开导出表,以及美国 FDA 孤儿药资格数据库。两边口径不同:欧盟一侧取的是当前状态仍为「已授权」的药品;美国一侧记录的是「曾获批准」这一事实,FDA 的公开表不追踪药物此后是否退市(例如 Relyvrio 于 2024 年撤市,表中仍记为已获批)。请以官方记录页为准。

在中国开展的临床试验 20L2

按病名在 ClinicalTrials.gov 检索、并校验研究中心含中国大陆而来。登记状态不等于现在真的能入组——务必按 NCT 号到原站核实,并与主治医生商量。

登记为可入组 8

  • 招募中NCT04924075
    Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
    II 期 · 干预性 · 2021/08/12Merck Sharp & Dohme LLC
    中国研究中心 4 个:Beijing、Chengdu、Guangzhou、Shanghai
  • 招募中NCT05069220
    18F-MFBG PET/CT in the Evaluation of Neural Crest Tumor
    早期 I 期 · 干预性 · 2021/09/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 招募中NCT06813742
    18F-mFBG PET Imaging in the Evaluation of Pheochromocytoma
    观察性 · 2023/05/01First Affiliated Hospital of Zhejiang University
    中国研究中心 2 个:Hangzhou
  • 招募中NCT07288931
    Al18F-NOTA-LM3 PET/CT in Patients With Pheochromocytoma and Paraganglioma
    不适用 · 干预性 · 2024/08/06Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 仅邀请入组NCT07182175
    Comparative Study of Perioperative Cardiac Injury in Patients With Functional Adrenal Tumors
    观察性 · 2024/09/19Peking University First Hospital
    中国研究中心 1 个:Beijing
  • 招募中NCT06683846
    Ivonescimab in the Treatment of Multiple Advanced Tumors
    II 期 · 干预性 · 2024/11/20Fudan University
    中国研究中心 1 个:Shanghai
  • 招募中NCT07195500
    Clinical Application of Somatostatin Receptor and Norepinephrine Transporter Targeted Imaging for Diagnosis and Staging of Neuroblastoma and Pheochromocytoma/Paraganglioma
    观察性 · 2024/12/25Nanjing First Hospital, Nanjing Medical University
    中国研究中心 1 个:Nanjing
  • 尚未开始招募NCT06429397
    Anlotinib Combined With Benmelstobart for Advanced Pheochromocytoma
    II 期 · 干预性 · 2025/05/01Sun Yat-sen University
    中国研究中心 1 个:Guangzhou
其他状态的试验(12 项)
  • 已完成NCT07412158
    Development and Clinical Application of a Novel Imaging Grading System for Pheochromocytoma and Paraganglioma (PPGL)
    观察性 · 2017/01/01Peking University First Hospital
    中国研究中心 1 个:Beijing
  • 已完成NCT05858177
    The Efficacy and Safety of Temozolomide in Patients With MPPGL
    II 期 · 干预性 · 2019/10/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 已完成NCT06669481
    Correlation Between Preoperative Images of PPGL and Intraoperative Hemodynamics
    观察性 · 2020/01/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 已完成NCT04860700
    The Efficacy and Safety of Anlotinib in Patients With Metastatic Pheochromocytoma or Paraganglioma
    II 期 · 干预性 · 2021/04/30Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 状态未知NCT04890444
    China Adrenal Disease Registry
    观察性 · 2021/06/01Shanghai Jiao Tong University School of Medicine
    中国研究中心 1 个:Shanghai
  • 状态未知NCT05133349
    A Prospective Phase II Efficacy and Safety Study of Anlotinib in Metastatic or Locally Advanced Pheochromocytoma/ Paraganglioma : Open-label Single-arm, Exploratory Trial
    II 期 · 干预性 · 2021/09/14Nanjing First Hospital, Nanjing Medical University
    中国研究中心 1 个:Nanjing
  • 状态未知NCT05883085
    A Study on the Safety and Effectiveness of Anlotinib for Neoadjuvant Treatment of PPGL
    II 期 · 干预性 · 2022/05/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 状态未知NCT05451134
    Predictors and Outcomes of Dysglycemia in Pheochromocytoma and Paraganglioma
    观察性 · 2022/06/30China National Center for Cardiovascular Diseases
    中国研究中心 1 个:Beijing
  • 状态未知NCT05752773
    Prediction of Myocardial Injury After Laparoscopic Pheochromocytoma/ParaGangLioma Resection
    观察性 · 2023/02/06Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 状态未知NCT06015061
    Prediction and Evaluation of Anlotinib Treatment Response Using Contrast Enhanced Ultrasound PPGL Patients
    观察性 · 2023/03/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 状态未知NCT05885386
    A Study on the Safety and Effectiveness of Temozolomide for Neoadjuvant Treatment of PPGL
    II 期 · 干预性 · 2023/04/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing
  • 状态未知NCT05885399
    The Efficacy and Safety of Penpulimab in the Treatment of Metastatic PPGL Patients Who Fail to Other Systemic Treatment
    II 期 · 干预性 · 2023/04/01Peking Union Medical College Hospital
    中国研究中心 1 个:Beijing

中国境外的在招试验 47L2

这些试验在中国没有研究中心,通常无法直接报名——入组一般要求在当地居住并接受随访。列在这里是因为它另有用处:看清楚全世界正在试哪些药、做到了哪一期、由谁在做。把药名和 NCT 编号记下来去问主治医生,或据此进一步查该药是否已在境外获批、是否有拓展性用药(expanded access)通道。

美国22意大利3加拿大2英国2西班牙1阿根廷1荷兰1巴西1丹麦1韩国1墨西哥1瑞士1澳大利亚1新西兰1另有 3 个国家/地区

CT.gov 报告命中 47 项,此处取回并展示最近的 15 项。

  • 尚未开始招募NCT07743138
    STOMP OUT: A Phase 2 Study To Evaluate The Effects Of Ivonescimab In Patients With Unresectable/Metastatic Adrenocortical Carcinoma (ACC) Or Unresectable Pheochromocytoma/Paraganglioma (PPGL)
    II 期 · 干预性 · 2027/02/07M.D. Anderson Cancer Center
    美国
  • 尚未开始招募NCT07714551
    Zanzalintinib in Unresectable and Progressive MPGGs
    II 期 · 干预性 · 2027/01/09Dana-Farber Cancer Institute
    美国
  • 尚未开始招募NCT07621146
    A Comparison of [68Ga]DOTATATE and [18F]AmBF3TATE for the Staging and Assessment of Neuroendocrine Malignancies
    观察性 · 2026/09/15British Columbia Cancer Agency
    加拿大
  • 尚未开始招募NCT07538791
    Italian Multicenter Experience With Radioreceptor-assisted Therapy (PRRT)
    观察性 · 2026/08/30Azienda USL Reggio Emilia - IRCCS
    意大利
  • 尚未开始招募NCT07661420
    211At-MABG in Adults With Advanced Neuroendocrine Cancers
    I 期 · 干预性 · 2026/08/01University of Pennsylvania
    美国
  • 招募中NCT07680205
    An Experimental Study of Belzutifan Impact on Catecholamine Metabolism
    II 期 · 干预性 · 2026/04/27Cambridge University Hospitals NHS Foundation Trust
    英国
  • 尚未开始招募NCT07516275
    Prospective Cohort Study of Pheochromocytoma/Paraganglioma
    观察性 · 2026/04Peking Union Medical College Hospital
  • 招募中NCT07282587
    Study of ONC206 (JZP3507) in Advanced Pheochromocytoma and Paraganglioma
    II 期 · 干预性 · 2026/01/02Jazz Pharmaceuticals
    美国
  • 招募中NCT04119024
    Gene Modified Immune Cells After Conditioning Regimen for the Treatment of Stage IIIC or IV Melanoma or Metastatic Solid Tumors
    I 期 · 干预性 · 2025/10/07Anusha Kalbasi
    美国
  • 尚未开始招募NCT07200245
    Cardiovascular Complications After Adrenalectomy for Pheochromocytoma and Non-secreting Tumors
    观察性 · 2025/10/01Central Hospital, Nancy, France
  • 招募中NCT06503146
    18F-Fibroblast Activation Protein Inhibitor ([18F]FAPI-74) PET Imaging for Cancer Detection
    II 期 · 干预性 · 2025/09/09National Cancer Institute (NCI)
    美国
  • 招募中NCT06479811
    [212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers
    I 期 · 干预性 · 2025/08/19National Cancer Institute (NCI)
    美国
  • 招募中NCT06427798
    Somatostatin-Receptors (SSTR)-Agonist [212Pb]VMT-alpha-NET in Metastatic or Inoperable SSTR+ Gastrointestinal Neuroendocrine Tumor and Pheochromocytoma/Paraganglioma Previously Treated With Systemic Targeted Radioligand Therapy
    I 期、II 期 · 干预性 · 2025/02/07National Cancer Institute (NCI)
    美国
  • 招募中NCT07072143
    An International Study on Pediatric Patients With Rare Tumors.
    观察性 · 2025/01/25Azienda Ospedaliera di Padova
    意大利
  • 招募中NCT06607692
    Study in Children and Adolescents of 177Lu-DOTATATE (Lutathera®) Combined With the PARP Inhibitor Olaparib for the Treatment of Recurrent or Relapsed Solid Tumours Expressing Somatostatin Receptor (SSTR) (LuPARPed).
    I 期、II 期 · 干预性 · 2024/09/20Fundación de investigación HM
    西班牙

外部标识与链接

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本页数据来源

  • 疾病定义、同义词、基因、表型、流行病学:Orphanet(CC BY 4.0)
  • 中文病名:Orphanet 中文包,冻结于 2020-06-01
  • 表型中文标签:HPO 简体中文翻译(CHPO 上游成果)