PDF(Pubmed)
Abstract：
BACKGROUND: Paget\'s disease of bone is a focal skeletal disorder causing bone deformities and impairing bone quality. Despite the prevalence of asymptomatic cases is increasing, the progression of the disease can lead to invalidating complications that compromise the quality of life. Doubts on clinical and therapeutic management aspects exist, although beneficial effects of antiresorptive drugs, particularly bisphosphonates are known. However, limited information is available from randomized controlled trials on the prevention of disease complications so that somewhat contrasting positions about treatment indications between expert panels from the main scientific societies of metabolic bone diseases exist. This task force, composed by expert representatives appointed by the Italian Society of Osteoporosis, Mineral Metabolism and Skeletal Diseases and members of the Italian Association of Paget\'s disease of bone, felt the necessity for more specific and up to date indications for an early diagnosis and clinical management.
METHODS: Through selected key questions, we propose evidence-based recommendations for the diagnosis and treatment of the disease. In the lack of good evidence to support clear recommendations, available information from the literature together with expert opinion of the panel was used to provide suggestions for the clinical practice.
CONCLUSIONS: Description of the evidence quality and support of the strength of the statements was provided on each of the selected key questions. The diagnosis of PDB should be mainly based on symptoms and the typical biochemical and radiological features. While treatment is mandatory to all the symptomatic cases at diagnosis, less evidence is available on treatment indications in asymptomatic as well as in previously treated patients in the presence of biochemical recurrence. However, given the safety and long-term efficacy of potent intravenous bisphosphonates such as zoledronate, a suggestion to treat most if not all cases at the time of diagnosis was released.
METHODS: Through selected key questions, we propose evidence-based recommendations for the diagnosis and treatment of the disease. In the lack of good evidence to support clear recommendations, available information from the literature together with expert opinion of the panel was used to provide suggestions for the clinical practice.
CONCLUSIONS: Description of the evidence quality and support of the strength of the statements was provided on each of the selected key questions. The diagnosis of PDB should be mainly based on symptoms and the typical biochemical and radiological features. While treatment is mandatory to all the symptomatic cases at diagnosis, less evidence is available on treatment indications in asymptomatic as well as in previously treated patients in the presence of biochemical recurrence. However, given the safety and long-term efficacy of potent intravenous bisphosphonates such as zoledronate, a suggestion to treat most if not all cases at the time of diagnosis was released.
摘要：
背景：Paget骨病是一种引起骨畸形和骨质量损害的局灶性骨骼疾病。尽管无症状病例的患病率正在增加，疾病的进展可能导致无效的并发症，从而影响生活质量。在临床和治疗管理方面存在疑问，虽然抗吸收药物的有益作用，特别是双膦酸盐是已知的。然而,关于预防疾病并发症的随机对照试验提供的信息有限，因此来自代谢性骨病主要科学学会的专家小组之间关于治疗适应症的立场存在一定的差异。这个特遣部队,由意大利骨质疏松学会任命的专家代表组成，矿物质代谢和骨骼疾病以及意大利佩吉特骨病协会的成员，认为有必要为早期诊断和临床管理提供更具体和最新的适应症。
方法：通过选定的关键问题，我们为该疾病的诊断和治疗提出了循证建议.在缺乏良好证据支持明确建议的情况下，从文献中获得的信息以及专家小组的专家意见被用来为临床实践提供建议。
结论：对每个选定的关键问题提供了证据质量的描述和对陈述强度的支持。PDB的诊断应主要基于症状和典型的生化和放射学特征。虽然诊断时所有有症状的病例都必须接受治疗，在无症状的患者以及在存在生化复发的以前接受过治疗的患者中，关于治疗适应症的证据较少。然而,鉴于有效的静脉注射双膦酸盐如唑来膦酸盐的安全性和长期疗效，建议在诊断时治疗大多数病例，即使不是所有病例。
方法：通过选定的关键问题，我们为该疾病的诊断和治疗提出了循证建议.在缺乏良好证据支持明确建议的情况下，从文献中获得的信息以及专家小组的专家意见被用来为临床实践提供建议。
结论：对每个选定的关键问题提供了证据质量的描述和对陈述强度的支持。PDB的诊断应主要基于症状和典型的生化和放射学特征。虽然诊断时所有有症状的病例都必须接受治疗，在无症状的患者以及在存在生化复发的以前接受过治疗的患者中，关于治疗适应症的证据较少。然而,鉴于有效的静脉注射双膦酸盐如唑来膦酸盐的安全性和长期疗效，建议在诊断时治疗大多数病例，即使不是所有病例。
