Cusano Natalie E, Rubin Mishaela R, Bilezikian John P
Department of Medicine, Division of Endocrinology, College of Physicians and Surgeons, Columbia University, New York, NY, USA.
Expert Rev Endocrinol Metab. 2015 Jan 1;10(1):5-13. doi: 10.1586/17446651.2015.971755.
Hypoparathyroidism is a rare disease characterized by hypocalcemia and insufficient circulating levels of parathyroid hormone (PTH). Conventional therapy includes calcium and active vitamin D supplementation, often in large doses. Therapy with calcium and vitamin D, however, does not address certain problematic aspects of the disease, including abnormal bone metabolism and reduced quality of life. Hypoparathyroidism is the only classic endocrine deficiency disease for which the missing hormone, PTH, is not yet an approved treatment. PTH(1-84) may soon become a therapeutic option for patients with hypoparathyroidism. PTH (1-84) has been demonstrated to maintain serum calcium while reducing or eliminating requirements for calcium and active vitamin D supplementation. Data from bone densitometry, bone turnover markers and histomorphometry of bone biopsy specimens show positive structural and dynamic effects on the skeleton. PTH replacement therapy may also be associated with improved quality of life. PTH(1-84) replacement therapy for hypoparathyroidism is promising, although further acquisition of long-term data is needed.
甲状旁腺功能减退症是一种罕见疾病,其特征为低钙血症和循环中的甲状旁腺激素(PTH)水平不足。传统治疗方法包括补充钙和活性维生素D,通常剂量较大。然而,钙和维生素D治疗并不能解决该疾病的某些问题,包括异常的骨代谢和生活质量下降。甲状旁腺功能减退症是唯一一种经典的内分泌缺乏疾病,其缺失的激素PTH尚未成为一种获批的治疗方法。PTH(1-84)可能很快成为甲状旁腺功能减退症患者的一种治疗选择。已证明PTH(1-84)可维持血清钙水平,同时减少或消除对钙和活性维生素D补充剂的需求。骨密度测定、骨转换标志物以及骨活检标本组织形态计量学的数据显示,其对骨骼具有积极的结构和动态影响。甲状旁腺激素替代疗法也可能与生活质量改善相关。尽管需要进一步获取长期数据,但PTH(1-84)替代疗法治疗甲状旁腺功能减退症前景广阔。