INSERM UMR 1033, Université de Lyon, Hôpital E Herriot, Lyon cedex 03, France.
INSERM UMR 1033, Université de Lyon, Hôpital E Herriot, Lyon cedex 03, France.
Bone. 2021 Feb;143:115784. doi: 10.1016/j.bone.2020.115784. Epub 2020 Dec 2.
Fibrous dysplasia of bone (FD) is a rare congenital bone disease, due to a somatic mutation of GNAS. This mutation results in a defect of osteoblast differentiation and mineralization and also an increase in bone resorption by large active osteoclasts. Bone pain is present in half of patients and is the main determinant of quality of life of patients with FD. Bisphosphonates are known to reduce bone pain and reduce the risk of fracture in patients with bone metastases or Paget's disease. Bisphosphonates may have similar effects in FD. In this article, we have reviewed the therapeutic potential of bisphosphonates to reduce bone pain due to FD, improve bone strength and reduce the occurrence of fracture.
We have reviewed 234 articles examining the effect of bisphosphonates on FD/McCune Albright Syndrome with no date limit, in PubMed and selected the articles with highest quality of methodology.
Pamidronate therapy significantly decreased bone pain and bone resorption (urinary NTX, urinary and serum CTX). Pamidronate may improve radiological lesions of FD patients (filling of osteolytic lesion and/or cortical thickening). This data with intravenous pamidronate, however, has been obtained from observational studies and no randomized controlled trial is available. Randomized placebo-controlled trials of oral bisphosphonates (alendronate or risedronate) have failed to demonstrate a significant decrease in bone pain over placebo. Several studies including one randomized controlled trial have shown an increase in bone mineral density (BMD) at FD sites with oral and intravenous bisphosphonate treatment. No effect on occurrence of fracture has been reported.
In conclusion, intravenous bisphosphonates may be proposed to treat persistent, moderate to severe bone pain of FD, e.g., according to the guidelines from the FD/MAS International Consortium. Oral bisphosphonates should not be used in this indication.
骨纤维发育不良（FD）是一种罕见的先天性骨疾病，由于 GNAS 的体细胞突变引起。该突变导致成骨细胞分化和矿化缺陷，并导致大而活跃的破骨细胞增加骨吸收。一半的患者存在骨痛，是 FD 患者生活质量的主要决定因素。双膦酸盐已知可减轻骨转移或 Pagets 病患者的骨痛并降低骨折风险。双膦酸盐在 FD 中可能具有类似的作用。在本文中，我们回顾了双膦酸盐减少 FD 引起的骨痛，增强骨强度和降低骨折发生率的治疗潜力。
我们在 PubMed 中检索了 234 篇关于双膦酸盐对 FD/McCune Albright 综合征影响的文章，未设定时间限制，并选择了方法学质量最高的文章。
帕米膦酸盐治疗可显著减轻骨痛和骨吸收（尿 NTX、尿和血清 CTX）。帕米膦酸盐可能改善 FD 患者的放射学病变（溶骨性病变填充和/或皮质增厚）。然而，这些静脉注射帕米膦酸盐的数据是从观察性研究中获得的，没有随机对照试验可用。口服双膦酸盐（阿仑膦酸盐或利塞膦酸盐）的随机安慰剂对照试验未能证明骨痛减轻与安慰剂相比有显著差异。几项研究，包括一项随机对照试验，表明口服和静脉内双膦酸盐治疗可增加 FD 部位的骨密度（BMD）。尚未报告对骨折发生的影响。
总之，静脉内双膦酸盐可用于治疗 FD 的持续性、中度至重度骨痛，例如，根据 FD/MAS 国际联合会的指南。不建议在这种情况下使用口服双膦酸盐。
