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.

由 Paget 协会（英国）领导的指南制定小组 (GDG) 使用 GRADE 方法制定了 Paget 骨病诊断和管理的循证临床指南 (PDB)。对诊断试验以及药物和非药物治疗选择进行了系统综述，试图解决几个具有临床相关性的关键问题。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其中8个问题。以下建议被确定为最重要的：1）放射性核素骨扫描，除靶向 X 线片外，被推荐为全面准确定义 PDB 患者代谢活动性疾病范围的一种手段。2）血清总碱性磷酸酶 (ALP) 在筛选是否存在代谢活性 PDB 时，建议作为一线生化筛查试验，与肝功能检查相结合。3）推荐双膦酸盐用于 PDB 相关骨痛的治疗。推荐唑来膦酸作为最有可能产生有利疼痛反应的双膦酸盐。4）在 PDB 中，推荐旨在改善症状的治疗，而不是旨在使总 ALP 正常化的治疗达标策略。5）对于药物治疗不充分的 PDB 患者，建议进行全髋关节或膝关节置换。没有足够的信息来推荐一种类型的手术方法而不是另一种。该指南得到了欧洲钙化组织学会、国际骨质疏松基金会、美国骨与矿物质研究学会、骨研究学会（英国）和英国老年学会的认可。GDG指出，PDB中缺乏以患者为中心的临床结局研究，并确定了需要进一步研究的几个领域。