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McCune-Albright综合征

McCune-Albright syndrome

定义 英文原文(暂无中文)

A rare mosaic syndrome characterized by the combination of two or more of the following: fibrous dysplasia of bone (FD), hyperpigmented macules, and hyperfunctioning endocrinopathies (precocious puberty, hyperthyroidism, growth hormone excess, endogenous Cushing syndrome).

别名

促性腺激素非依赖性女性性早熟

基本事实

遗传方式
不适用
发病年龄
儿童期
患病率
1-9 / 1 000 000(Europe)

相关基因 1

基因名称关联类型
GNASGNAS complex locusDisease-causing somatic mutation(s) in

临床表型 53

极常见 99–80%4

  • 内分泌生理学异常 HP:0031072
  • 边缘不规则的较大咖啡牛奶斑 HP:0005605
  • 卵巢囊肿 HP:0000138
  • 性早熟 HP:0000826

常见 79–30%14

  • 睾丸形态异常 HP:0000035
  • 面部骨骼形态异常 HP:0011821
  • 股骨形态异常 HP:0002823
  • 颅底形态异常 HP:0002693
  • 甲状腺异常 HP:0000820
  • 骨骼成熟加速 HP:0005616
  • 骨纤维发育不良 HP:0010734
  • 生长异常 HP:0001507
  • 甲状腺功能亢进症 HP:0000836
  • 血清睾酮水平增高 HP:0030088
  • 巨睾 HP:0000053
  • 单骨性骨纤维发育不良 HP:0010736
  • 肾小管功能障碍 HP:0000124
  • 脊柱侧弯 HP:0002650

偶见 29–5%23

  • 脸部异常 HP:0000271
  • 良性胃肠道肿瘤 HP:0006719
  • 骨折 HP:0020110
  • 骨痛 HP:0002653
  • 生育能力下降 HP:0000144
  • 牙齿错位咬合 HP:0000689
  • 生长激素水平升高 HP:0000845
  • 面部不对称 HP:0000324
  • 胃食管反流 HP:0002020
  • 甲状腺肿 HP:0000853
  • 听力受损 HP:0000365
  • 肝细胞腺瘤 HP:0012028
  • 睾丸间质细胞增生 HP:0010791
  • 异常性行为 HP:0008768
  • 泌乳素水平升高 HP:0000870
  • 月经不调 HP:0000858
  • 鼻塞 HP:0001742
  • 软骨病。 HP:0002749
  • 胰腺炎 HP:0001733
  • 感觉异常 HP:0003401
  • 多骨性骨纤维发育不良 HP:0010735
  • 复发性骨折 HP:0002757
  • 肾源性磷酸盐流失 HP:0000117

罕见 <4–1%12

  • 动脉瘤性骨囊肿 HP:0012063
  • 骨髓细胞减少 HP:0005528
  • 乳腺癌 HP:0003002
  • 胆汁淤积 HP:0001396
  • 皮肤粘液瘤 HP:0030428
  • 肝炎 HP:0012115
  • 高磷酸盐尿症 HP:0003109
  • 低磷血症 HP:0002148
  • 血皮质醇水平增加 HP:0003118
  • 全血细胞减少症 HP:0001876
  • 原发性皮质醇增多症 HP:0001579
  • 视力丧失 HP:0000572

近两年的全球研究 390L2

2024/10 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。

  • 2026-10
    Outcomes of Surgical Interventions in Children With Fibrous Dysplasia/McCune-Albright Syndrome
    Journal of pediatric orthopedics · DOI · Europe PMC
  • 2026-09
    Physical disability and quality-of-life burden in McCune-Albright syndrome: a nationwide survey in Japan
    Endocrine journal · DOI · Europe PMC
  • 2026-09综述开放获取
    Risk factors and preventive strategies for precocious puberty: a narrative review
    Frontiers in pediatrics · DOI · Europe PMC
  • 2026-09病例报告开放获取
    Case Report: Total hip arthroplasty via the direct anterior approach using a cementless revision stem without corrective osteotomy for fibrous dysplasia
    Frontiers in surgery · DOI · Europe PMC
  • 2026-09
    Letrozole in Paediatric Females for Peripheral Precocious Puberty: A Systematic Review of Efficacy and Safety
    The Journal of clinical endocrinology and metabolism · DOI · Europe PMC
  • 2026-09开放获取
    Automated segmentation and length measurement of metacarpal and phalangeal bones for hand radiograph evaluation
    Scientific reports · DOI · Europe PMC
  • 2026-09综述开放获取
    Oral Pigmentation in Children: A Narrative Review and Practical Diagnostic Framework
    Cureus · DOI · Europe PMC
  • 2026-09开放获取
    Inherited and somatic components in the pathogenetics of common diseases
    Vavilovskii zhurnal genetiki i selektsii · DOI · Europe PMC
  • 2026-08病例报告开放获取
    Orthopaedic diagnostic pitfalls in fibroblast growth factor 23-mediated hypophosphatemic rickets/osteomalacia in fibrous dysplasia/McCune-Albright syndrome: Two burosumab-treated cases
    Bone reports · DOI · Europe PMC
  • 2026-08临床试验开放获取
    Palovarotene impact on fibrodysplasia ossificans progressiva (FOP): data from month 48 of the phase III MOVE trial
    JBMR plus · DOI · Europe PMC
  • 2026-08
    Lesion Expansion in Craniofacial Fibrous Dysplasia: Natural History and Indicators of Progression in the Frontal, Zygomatic, and Sphenoid Bones
    Journal of bone and mineral research : the official journal of the Ame · DOI · Europe PMC
  • 2026-08开放获取
    Rising incidence of central precocious puberty in Thailand: a nationwide population-based study before, during, and after the COVID-19 pandemic (2018-2024)
    Clinical and experimental pediatrics · 被引 1 · DOI · Europe PMC
  • 2026-08开放获取
    Impact of endoscopy and related technologies on indications for transsphenoidal pituitary surgery for growth hormone-producing pituitary neuroendocrine tumors in McCune-Albright syndrome: illustrative case
    Journal of neurosurgery. Case lessons · DOI · Europe PMC
  • 2026-08综述开放获取
    Modern Determinants of Earlier Menarche and Mental Health: A Translational Review for Clinicians
    Children (Basel, Switzerland) · DOI · Europe PMC
  • 2026-08开放获取
    Quantitative validation of the "coastline" heuristic for café-au-lait macule borders in neurofibromatosis type 1 and McCune-Albright syndrome
    Journal of the American Academy of Dermatology · DOI · Europe PMC
  • 2026-08综述开放获取
    Phosphate: An Anion Controlling Metabolic Functions
    Nutrients · DOI · Europe PMC
  • 2026-08开放获取
    Fibrosarcoma of bone masquerading as fibrous dysplasia: diagnostic pitfall and molecular correlates of aggressive behavior
    Journal of pathology and translational medicine · DOI · Europe PMC
  • 2026-07开放获取
    Abstracts of the ERICA ERN research conference
    Orphanet journal of rare diseases · DOI · Europe PMC
  • 2026-07开放获取
    Abstracts of the 12th International Conference on Children's Bone Health
    JBMR plus
  • 2026-07病例报告开放获取
    Fibrous Dysplasia Presenting as an Exophytic Gingival Mass: A Rare Clinical Presentation
    Journal of clinical medicine · DOI · Europe PMC

中国境外的在招试验 4L2

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2荷兰1意大利1

共 4 项。

  • 招募中NCT05966064
    DEnosumab for the Treatment of FIbrous Dysplasia/McCune-Albright Syndrome in Adults (DeFiD)
    IV 期 · 干预性 · 2023/06/13Natasha Appelman-Dijkstra
    荷兰
  • 招募中NCT07569731
    Fibrous Dysplasia: An Epidemiological and Correlational Evaluation of Multimodal Data
    观察性 · 2022/05/12Istituto Ortopedico Rizzoli
    意大利
  • 招募中NCT03231644
    Fibrous Dysplasia, McCune-Albright Syndrome Patient Registry
    观察性 · 2016/10/31Tovah Burstein
    美国
  • 招募中NCT00001727
    Screening and Natural History of Patients With Polyostotic Fibrous Dysplasia and the McCune-Albright Syndrome
    观察性 · 1998/12/13National Institute of Dental and Craniofacial Research (NIDCR)
    美国

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)