Effectiveness and safety of zoledronate in the treatment of 24 Chinese patients with Paget disease of bone
10.3760/cma.j.cn311282-20240607-00250
- VernacularTitle:唑来膦酸治疗24例中国人Paget骨病的疗效及安全性分析
- Author:
An SONG
1
;
Jing LIU
;
Jiajia WANG
;
Yingyu CHEN
;
Yan JIANG
;
Mei LI
;
Weibo XIA
;
Ou WANG
;
Xiaoping XING
;
Xunwu MENG
Author Information
1. 中国医学科学院北京协和医学院,北京协和医院内分泌科,协和转化医学中心,国家罕见病疑难重症重点实验室,国家卫生健康委员会内分泌重点实验室,北京 100730
- Publication Type:Journal Article
- Keywords:
Paget′s disease of bone;
Zoledronic acid;
Chinese;
Efficacy;
Adverse effects
- From:
Chinese Journal of Endocrinology and Metabolism
2025;41(3):226-232
- CountryChina
- Language:Chinese
-
Abstract:
Objective:To evaluate the effectiveness and safety of zoledronic acid in patients with Paget′s disease of bone based on clinical data from a single medical center.Methods:This retrospective study included 24 patients diagnosed with Paget′s disease of bone and treated with zoledronic acid at Peking Union Medical College Hospital between January 2009 and June 2020. Demographic data, clinical symptoms, treatment efficacy, and safety outcomes were collected. The primary efficacy measure was serum alkaline phosphatase(ALP) levels. Treatment was considered effective if ALP levels returned to normal or decreased by more than 75% from baseline in the difference between the ALP level and its normal median value.Results:Among the 24 patients with Paget′s bone disease, the most commonly affected site was the skull(in 17 cases). All patients received a single 5 mg intravenous infusion of zoledronic acid. Serum ALP levels significantly decreased after treatment. Among the 15 patients who completed at least 3 months of follow-up, all achieved treatment success. The median time for serum ALP levels to reach the target was 13.1(9.4, 26.1) weeks. In 12 patients, ALP levels normalized within a medium of 16.9(11.5, 37.3) weeks, and remained stable over a medium follow-up of 4.56(2.42, 5.71) years. The most common side effects were hypocalcemia(21 cases, 87.5%) and flu-like symptoms(17 cases, 70.8%). Seven patients had severe hypocalcemia(serum calcium<1.75 mmol/L), and they had higher baseline levels of ALP, calcium, and phosphorus compared to those with mild hypocalcemia.Conclusions:Zoledronic acid 5 mg intravenous infusion effectively controlled disease activity in patients with Paget′s disease of bone. Generally, Most patients achieved treatment goals within 3-4 months, with sustained remission for a median of 4 years. Hypocalcemia was the most frequent side effect, underscoring the importance of timely calcium and vitamin D supplementation.