Centre for Genomic and Experimental Medicine, MRC Institute of Genetics and Molecular Medicine, University of Edinburgh, Edinburgh, UK.
Internal Medicine Department, Hospital Universitario Río Hortega, University of Valladolid, Valladolid, Spain.
J Bone Miner Res. 2019 Apr;34(4):579-604. doi: 10.1002/jbmr.3657. Epub 2019 Feb 25.
An evidence-based clinical guideline for the diagnosis and management of Paget's disease of bone (PDB) was developed using GRADE methodology, by a Guideline Development Group (GDG) led by the Paget's Association (UK). A systematic review of diagnostic tests and pharmacological and nonpharmacological treatment options was conducted that sought to address several key questions of clinical relevance. Twelve recommendations and five conditional recommendations were made, but there was insufficient evidence to address eight of the questions posed. The following recommendations were identified as the most important: 1) Radionuclide bone scans, in addition to targeted radiographs, are recommended as a means of fully and accurately defining the extent of metabolically active disease in patients with PDB. 2) Serum total alkaline phosphatase (ALP) is recommended as a first-line biochemical screening test in combination with liver function tests in screening for the presence of metabolically active PDB. 3) Bisphosphonates are recommended for the treatment of bone pain associated with PDB. Zoledronic acid is recommended as the bisphosphonate most likely to give a favorable pain response. 4) Treatment aimed at improving symptoms is recommended over a treat-to-target strategy aimed at normalizing total ALP in PDB. 5) Total hip or knee replacements are recommended for patients with PDB who develop osteoarthritis in whom medical treatment is inadequate. There is insufficient information to recommend one type of surgical approach over another. The guideline was endorsed by the European Calcified Tissues Society, the International Osteoporosis Foundation, the American Society of Bone and Mineral Research, the Bone Research Society (UK), and the British Geriatric Society. The GDG noted that there had been a lack of research on patient-focused clinical outcomes in PDB and identified several areas where further research was needed. © 2019 The Authors. Journal of Bone and Mineral Research Published by Wiley Periodicals Inc.
一个基于证据的临床指南，用于诊断和管理佩吉特病的骨骼（PDB）是使用 GRADE 方法开发的，由一个由 Paget 协会（英国）领导的指南开发小组（GDG）。进行了一项系统的诊断测试和药物及非药物治疗选择的审查，旨在解决几个关键的临床相关问题。提出了 12 项建议和 5 项有条件的建议，但没有足够的证据来解决提出的 8 个问题。确定以下建议是最重要的：1）放射性核素骨扫描，除了有针对性的射线照相外，建议作为一种全面准确地定义代谢活跃性疾病患者疾病范围的方法。2）血清总碱性磷酸酶（ALP）建议作为第一线生化筛选试验，与肝功能试验结合用于筛查代谢活跃性 PDB 的存在。3）双膦酸盐推荐用于治疗与 PDB 相关的骨痛。唑来膦酸被推荐为最有可能产生有利疼痛反应的双膦酸盐。4）建议对 PDB 患者采用旨在改善症状的治疗方法，而不是采用旨在使总 ALP 正常化的针对目标的治疗策略。5）建议对患有代谢活跃性 PDB 的关节炎患者进行全髋关节或全膝关节置换，如果药物治疗不足。没有足够的信息来推荐一种手术方法优于另一种。该指南得到了欧洲钙化组织协会、国际骨质疏松基金会、美国骨与矿物研究协会、骨骼研究协会（英国）和英国老年学会的认可。GDG 指出，在 PDB 患者的以患者为中心的临床结果方面缺乏研究，并确定了需要进一步研究的几个领域。
