Grover Ashna, Jha Smita
Metabolic Diseases Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, MD 20892, USA.
Metabolic Diseases Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Building 10, Room 9C432A, 10 Center Drive, Bethesda, MD 20892, USA.
Best Pract Res Clin Endocrinol Metab. 2025 Mar;39(2):101984. doi: 10.1016/j.beem.2025.101984. Epub 2025 Mar 1.
Familial or heritable hyperparathyroidism (FHPT) is seen in approximately 10-15 % of patients with primary hyperparathyroidism (PHPT). Once the diagnosis of PHPT is established, consideration of heritable forms should be made in patients with positive family history, young onset, multi-glandular disease, and recurrent or persistent disease. FHPT encompasses both syndromic and non-syndromic forms. Syndromic forms include multiple endocrine neoplasia (MEN) types 1, 2, 3 and 4, hyperparathyroidism-jaw tumor syndrome, hereditary pheochromocytoma and paraganglioma, and the more recently reported, Birt-Hogg-Dubé (BHD) syndrome, and X-linked intellectual disability syndrome. Non-syndromic forms include familial hypocalciuric hypercalcemia (FHH)- types 1,2, and 3, neonatal severe hyperparathyroidism, GCM2-mediated hyperparathyroidism, transient neonatal hyperparathyroidism, and familial isolated hyperparathyroidism. In this review we aim to review the heritable forms of PHPT and highlight the important genetics insights and clinical implications.
家族性或遗传性甲状旁腺功能亢进症(FHPT)约占原发性甲状旁腺功能亢进症(PHPT)患者的10% - 15%。一旦确诊为PHPT,对于有家族史阳性、发病年龄较轻、多腺体疾病以及复发或持续性疾病的患者,应考虑遗传性形式。FHPT包括综合征型和非综合征型。综合征型包括1、2、3和4型多发性内分泌腺瘤病(MEN)、甲状旁腺功能亢进 - 颌骨肿瘤综合征、遗传性嗜铬细胞瘤和副神经节瘤,以及最近报道的Birt - Hogg - Dubé(BHD)综合征和X连锁智力障碍综合征。非综合征型包括1、2和3型家族性低钙血症性高钙血症(FHH)、新生儿重症甲状旁腺功能亢进症、GCM2介导的甲状旁腺功能亢进症、短暂性新生儿甲状旁腺功能亢进症以及家族性孤立性甲状旁腺功能亢进症。在本综述中,我们旨在回顾PHPT的遗传性形式,并强调重要的遗传学见解和临床意义。