Skeletal Clinical Studies Unit, Craniofacial and Skeletal Disease Branch, National Institute of Dental and Craniofacial Research (NIDCR), National Institutes of Health (NIH), Bethesda, MD, USA.
Clinical Center, National Institutes of Health (NIH), Bethesda, MD, USA.
J Bone Miner Res. 2017 Aug;32(8):1667-1671. doi: 10.1002/jbmr.3162. Epub 2017 Jun 12.
Tumor-induced osteomalacia (TIO) is a rare paraneoplastic syndrome in which unregulated hypersecretion of fibroblast growth factor 23 (FGF23) by phosphaturic mesenchymal tumors (PMT) causes renal phosphate wasting, hypophosphatemia, and osteomalacia. The resulting mineral homeostasis abnormalities and skeletal manifestations can be reversed with surgical resection of the tumor. Unfortunately, PMTs are often difficult to locate, and medical treatment with oral phosphate and vitamin D analogues is either insufficient to manage the disease or not tolerated. Octreotide has been proposed as a potential treatment for TIO due to the presence of somatostatin receptors (SSTR) on PMTs; however, the role of somatostatin signaling in PMTs and the efficacy of treatment of TIOs with somatostatin analogues is not clear. In an effort to evaluate the efficacy of octreotide therapy in TIO, five subjects with TIO were treated with octreotide for 3 days. Blood intact FGF23, phosphate, and 1,25(OH) D , and tubular reabsorption of phosphate (TRP) were measured at frequent time points during treatment. Octreotide's effects were assessed by comparing group means of the biochemical parameters at each time-point to mean baseline values. There were no significant changes in blood phosphate, FGF23, 1,25(OH) D , or TRP during octreotide treatment, consistent with a lack of efficacy of octreotide in treating TIO. © 2017 American Society for Bone and Mineral Research.
肿瘤相关性骨软化症（TIO）是一种罕见的副瘤综合征，磷酸酯酶成纤维细胞生长因子 23（FGF23）的无调节性分泌过盛，由磷酸酯酶成纤维细胞瘤（PMT）引起，导致肾脏磷酸盐丢失、低磷酸盐血症和骨软化症。手术切除肿瘤可逆转由此产生的矿物质稳态异常和骨骼表现。不幸的是，PMT 通常难以定位，口服磷酸盐和维生素 D 类似物的药物治疗要么不足以治疗疾病，要么不能耐受。奥曲肽被提议作为 TIO 的潜在治疗方法，因为 PMT 上存在生长抑素受体（SSTR）；然而，SSTR 信号在 PMT 中的作用以及生长抑素类似物治疗 TIO 的疗效尚不清楚。为了评估奥曲肽治疗 TIO 的疗效，对 5 例 TIO 患者进行了 3 天的奥曲肽治疗。在治疗过程中频繁的时间点测量血液完整的 FGF23、磷酸盐和 1,25(OH)D 和磷酸盐的肾小管重吸收（TRP）。通过比较每个时间点的生化参数的组平均值与平均基线值来评估奥曲肽的作用。奥曲肽治疗期间，血液磷酸盐、FGF23、1,25(OH)D 或 TRP 均无明显变化，表明奥曲肽治疗 TIO 无效。©2017 美国骨与矿物质研究协会。
