Abstract：
OBJECTIVE: The aims of the current study were to describe clinical and biochemical features of patients with Paget disease of bone (PDB) followed at our medical center, and to examine the long-term effectiveness of zoledronate.
METHODS: Retrospective cohort study included consecutive patients≥18 years with a diagnosis of PDB, followed in the Rabin Medical Center (RMC) Institute of Endocrinology from 1973 to 2023. The cohort comprised two groups: patients treated/not treated with zoledronic acid (ZOL/NZOL). The primary outcome was the percentage of patients who achieved a biochemical therapeutic response.
RESULTS: Overall, 101 patients with PDB were included, 68 in the ZOL group and 33 in the NZOL group. The mean age was 65.2 ± 10.0 years, and 47% were female. Notably, 77% exhibited monostotic involvement, and only 3% had experienced fractures attributed to PDB. Mean ALP level at diagnosis was 160 ± 70.6 U/L. The median follow-up duration was 17 years since PDB diagnosis, comparable between the groups. Primary outcome was more prevalent in the ZOL compared to the NZOL group [42 patients (88%) VS 11 patients (52%) respectively, P = 0.004]. At the end of follow-up, mean ALP levels in the NZOL group were significantly higher than the levels in the ZOL group irrespective of the number of infusions received.
CONCLUSIONS: The majority of patients with PDB experience a mild disease course, marked by monostotic involvement and a low prevalence of fractures. Zoledronic acid effectively manages PDB, providing sustained biochemical response. The necessity for multiple zoledronic acid injections remains questionable, often implemented due to osteoporosis.
METHODS: Retrospective cohort study included consecutive patients≥18 years with a diagnosis of PDB, followed in the Rabin Medical Center (RMC) Institute of Endocrinology from 1973 to 2023. The cohort comprised two groups: patients treated/not treated with zoledronic acid (ZOL/NZOL). The primary outcome was the percentage of patients who achieved a biochemical therapeutic response.
RESULTS: Overall, 101 patients with PDB were included, 68 in the ZOL group and 33 in the NZOL group. The mean age was 65.2 ± 10.0 years, and 47% were female. Notably, 77% exhibited monostotic involvement, and only 3% had experienced fractures attributed to PDB. Mean ALP level at diagnosis was 160 ± 70.6 U/L. The median follow-up duration was 17 years since PDB diagnosis, comparable between the groups. Primary outcome was more prevalent in the ZOL compared to the NZOL group [42 patients (88%) VS 11 patients (52%) respectively, P = 0.004]. At the end of follow-up, mean ALP levels in the NZOL group were significantly higher than the levels in the ZOL group irrespective of the number of infusions received.
CONCLUSIONS: The majority of patients with PDB experience a mild disease course, marked by monostotic involvement and a low prevalence of fractures. Zoledronic acid effectively manages PDB, providing sustained biochemical response. The necessity for multiple zoledronic acid injections remains questionable, often implemented due to osteoporosis.
摘要：
目的：本研究的目的是描述在我们的医疗中心随访的Paget骨病（PDB）患者的临床和生化特征。并检查唑来膦酸的长期有效性。
方法：回顾性队列研究包括诊断为PDB的≥18岁的连续患者，随后于1973年至2023年在拉宾医学中心（RMC）内分泌学研究所。该队列包括两组:用唑来膦酸(ZOL/NZOL)治疗/未用唑来膦酸治疗的患者。主要结果是获得生化治疗反应的患者百分比。
结果：总体而言，包括101例PDB患者，ZOL组68和NZOL组33。平均年龄为65.2±10.0岁,47%是女性。值得注意的是,77%表现出单骨受累，只有3%的人经历了归因于PDB的骨折。诊断时的平均ALP水平为160±70.6U/L。自PDB诊断以来，中位随访时间为17年，组间比较。与NZOL组相比，ZOL组的主要结局更为普遍[分别为42例（88%）和11例（52%），P=0.004]。在后续行动结束时，无论接受的输注次数如何,NZOL组的平均ALP水平均显著高于ZOL组.
结论：大多数PDB患者的病程较轻，以单骨受累和骨折患病率低为特征。唑来膦酸有效地管理PDB，提供持续的生化反应。多次注射唑来膦酸的必要性仍然值得怀疑，经常由于骨质疏松症而实施。
方法：回顾性队列研究包括诊断为PDB的≥18岁的连续患者，随后于1973年至2023年在拉宾医学中心（RMC）内分泌学研究所。该队列包括两组:用唑来膦酸(ZOL/NZOL)治疗/未用唑来膦酸治疗的患者。主要结果是获得生化治疗反应的患者百分比。
结果：总体而言，包括101例PDB患者，ZOL组68和NZOL组33。平均年龄为65.2±10.0岁,47%是女性。值得注意的是,77%表现出单骨受累，只有3%的人经历了归因于PDB的骨折。诊断时的平均ALP水平为160±70.6U/L。自PDB诊断以来，中位随访时间为17年，组间比较。与NZOL组相比，ZOL组的主要结局更为普遍[分别为42例（88%）和11例（52%），P=0.004]。在后续行动结束时，无论接受的输注次数如何,NZOL组的平均ALP水平均显著高于ZOL组.
结论：大多数PDB患者的病程较轻，以单骨受累和骨折患病率低为特征。唑来膦酸有效地管理PDB，提供持续的生化反应。多次注射唑来膦酸的必要性仍然值得怀疑，经常由于骨质疏松症而实施。
