摘要
本文通过检索北京协和医院于2021年1月至2022年12月期间诊断X-连锁低磷性佝偻病(XLH)且接受布罗索尤单抗治疗的患者,对患者的临床特征、布罗索尤单抗治疗前后的临床指标变化、治疗期间的药品不良反应等进行描述,初步评价布罗索尤单抗用于XLH患者的有效性和安全性。结果显示,3例儿童XLH患者和1例成年XLH患者接受布罗索尤单抗治疗,全部患者血磷水平较治疗前提高,3例儿童患者血磷均达到参考值范围下限以上,全部患者较治疗前血碱性磷酸酶(ALP)降低,1例成年患者治疗2.5年后血ALP接近正常范围。1例儿童患者治疗48周后肾脏超声可见小结晶,1例儿童患者和1例成年患者治疗前出现血甲状旁腺激素(PTH)水平升高,用药后血PTH进一步升高。总之,布罗索尤单抗用于XLH患者,提高了血磷水平并保持相对稳定,同时降低了血ALP水平,治疗期间未观察到严重不良反应,为布罗索尤单抗这一新药用于XLH患者提供参考。
We studied the patients diagnosed with X-linked hypophosphatemicrickets(XLH)and treated with burosumab in Peking Union Medical College Hospital from January 2021 to December 2022.In ad-dition,we described the clinical characteristics of the patients,the changes of clinical indexes before and after burosumab treatment,and the adverse drug reactions during treatment.We also evaluated the efficacy and safety of burosumab for XLH.The results showed that three children XLH patients and one adult XLH patients re-ceived burosumab treatment.After treatment,the serum phosphorus level of all patients increased;the serum phosphorus of 3 children patients increased above the lower limit of the reference value range;the serum alkaline phosphatase(ALP)of all patients was lower than that of before treatment;the serum ALP of one adult patient was close to the normal range after 25 years of treatment.One child patient showed small crystals in kidney through ultrasound 48 weeks after treatment;one child and one adult showed increased serum parathy-roid hormone(PTH)level before treatment and serum PTH continued increasing after treatment.Finally,it may be concluded that burosumab increased serum phosphorus levels in XLH patients,kept the level relatively stable,and reduced serum ALP levels.No serious adverse reactions occurred during treatment,in order to pro-vide reference for the use of burosumab in patients with XLH.
作者
刘清扬
后子靖
杨紫霞
刘鑫
张波
唐彦
LIU Qingyang;HOU Zijing;YANG Zixia;LIU Xin;ZHANG Bo;TANG Yan(Department of Pharmacy,Peking Union Medical College Hospital,Chinese Academy of Medical Sciences&Peking Union Medical College,Beijing 100730,China;Department of Pharmacy,Dongzhimen Hospital,Beijing University of Chinese Medicine,Beijing 100700,China)
出处
《罕见病研究》
2024年第1期108-113,共6页
Journal of Rare Diseases
基金
中央高水平医院临床科研业务费(2022-PUMCH-B-059)
中国医学科学院医学与健康科技创新工程(2021-I2M-1-003)。
