Despite the current debate on the best therapeutic approach, i.e. symptomatic vs intensive strategy, one zoledronate (Zol) infusion is effective in most patients with Paget’s disease of bone (PDB), whereas few need retreatment, whose predictors are not well established. We aimed to evaluate long-term efficacy of intensive Zol therapy and predictors of retreatment in PDB. Pagetic complications, clinical and biochemical response to Zol together with frequency of retreatment were retrospectively assessed in forty-seven PDB patients (age, mean ± SD: 72.5 ± 8.9 years, M/F: 24/23; symptomatic/asymptomatic: 16/31). Statistical analysis for retreatment prediction were based on Mann–Whitney U test, Pearson’s Χ2 and ROC curve analysis. During seven-year follow-up, all patients achieved pain relief and only one underwent arthroplasty. Bone alkaline phosphatase (BAP) detected three non-responder (6%) and six relapsing (13%) patients needing retreatment. Retreated patients had less old age (66.1 ± 11.2 vs 74.0 ± 7.7 years), higher frequency of polyostotic disease (78% vs 40%) and higher baseline (96.5 ± 24.8 vs 44.9 ± 27.7 mcg/l) and post-Zol nadir BAP levels (24.7 ± 24.1 vs 8.1 ± 4.1 mcg/l) than patients treated once (p < 0.05 for all comparisons). In multivariate analysis both serum baseline and post-Zol nadir BAP significantly predicted retreatment (OR 1.09, 95%CI 1.01–1.17 and 1.29, 1.03–1.62, respectively), with ROC curve analysis showing the greatest accuracies for threshold values of 75.6 and 9.9 mcg/l (sensitivity 88 and 90%, specificity 94 and 86%, AUC 0.92 and 0.93, respectively). Our data in mostly asymptomatic, metabolically active PDB patients treated with intensive Zol therapy show a negligible incidence of pagetic complications and long-term optimal disease control, with BAP being the best predictor of retreatment.

尽管目前就最佳治疗方法（即对症治疗还是强化治疗）存在争议，但一次唑来膦酸盐 (Zol) 输注对大多数佩吉特骨病 (PDB) 患者有效，而很少需要再治疗，其预测指标尚不明确。我们旨在评估强化 Zol 治疗的长期疗效和 PDB 再治疗的预测因素。对 47 名 PDB 患者（年龄，平均值 ± SD：72.5 ± 8.9 岁，M/F：24/23；有症状/无症状：16/31 ）。再治疗预测的统计分析基于 Mann-Whitney U测试、Pearson's Χ2 和 ROC 曲线分析。在七年的随访中，所有患者的疼痛均得到缓解，只有一名患者接受了关节置换术。骨碱性磷酸酶 (BAP) 检测到 3 名无反应 (6%) 和 6 名复发 (13%) 需要再治疗的患者。接受复治的患者年龄较小（66.1 ± 11.2 vs 74.0 ± 7.7 岁），多发性骨病发生率较高（78% vs 40%）和基线较高（96.5 ± 24.8 vs 44.9 ± 27.7 mcg/l）和 Zol 最低点 BAP水平 (24.7 ± 24.1 vs 8.1 ± 4.1 mcg/l) 比接受一次治疗的患者 ( p < 0.05 对于所有比较）。在多变量分析中，血清基线和 Zol 最低点 BAP 均显着预测再治疗（OR 1.09，95%CI 分别为 1.01-1.17 和 1.29，1.03-1.62），ROC 曲线分析显示阈值的最大准确性为 75.6 和 9.9 mcg/l（灵敏度分别为 88 和 90%，特异性 94 和 86%，AUC 分别为 0.92 和 0.93）。我们在接受 Zol 强化治疗的大多数无症状、代谢活跃的 PDB 患者中的数据显示，癫痫并发症的发生率和长期的最佳疾病控制可忽略不计，而 BAP 是再治疗的最佳预测指标。