布罗索尤单抗治疗儿童X连锁低磷性佝偻病疗效评估  

Evaluation of Burosumab in the treatment of X-linked hypophosphatemic rickets in children

作　　者：方韶晗 邓芳[1] 袁悦 李旭[3] 赵振 朱颖[1] 彭寅 张罗丹 Fang Shaohan;Deng Fang;Yuan Yue;Li Xu;Zhao Zhen;Zhu Ying;Peng Yin;Zhang Luodan(The Fifth Clinical College of Anhui Medical University,Department of Pediatric Nephrology,Anhui Provincial Children′s Hospital,Hefei 230000,China;The Fifth Clinical College of Anhui Medical University,Department of Pediatric Orthopedics,Anhui Provincial Children′s Hospital,Hefei 230000,China;The Fifth Clinical College of Anhui Medical University,Department of Radiology,Anhui Provincial Children′s Hospital,Hefei 230000,China)

机构地区：[1]安徽医科大学第五临床医学院,安徽省儿童医院儿童肾脏科,合肥230000 [2]安徽医科大学第五临床医学院,安徽省儿童医院儿童骨科,合肥230000 [3]安徽医科大学第五临床医学院,安徽省儿童医院放射科,合肥230000

出　　处：《中华内分泌代谢杂志》2024年第1期11-16,共6页Chinese Journal of Endocrinology and Metabolism

基　　金：安徽省自然科学基金(2108085MH262)。

摘　　要：目的评估布罗索尤单抗在X连锁低磷性佝偻病患儿中应用的有效性及安全性。方法回顾性分析2021年11月至2023年9月在安徽省儿童医院儿童肾脏科住院确诊的9例X连锁低磷性佝偻病并接受布罗索尤单抗治疗患儿的临床资料,包括患儿一般情况、临床表现、辅助检查、布罗索尤单抗治疗及随访情况等。结果 9例患儿中男性5例,女性4例,确诊中位年龄为2岁,传统治疗后血磷波动在0.7~0.9 mmol/L。开始使用布罗索尤单抗治疗中位年龄为2.8岁,药物初始使用剂量为0.8 mg/kg,每2周皮下注射1次。布罗索尤单抗治疗6个月后实验室及影像学指标得到改善,1个月后血磷从开始平均(0.81±0.14)mmol/L上升到(1.02±0.10)mmol/L(t=3.85,P=0.001),6个月后上升到(1.14±0.25)mmol/L(t =3.58,P=0.002)。碱性磷酸酶(ALP)由治疗前平均(509.89±110.10)U/L,1个月后下降到(447.89±106.76)U/L(t=1.21,P=0.243),6个月后ALP显著下降到(385.89±60.33)U/L(t=2.96,P=0.009)。身高百分位由治疗前平均18.42±10.09到6个月后26.56±16.59(t=1.26,P=0.227)。双下肢佝偻病严重程度评分由治疗前平均(4.61±1.36)分到6个月后的(3.06±1.51)分(t=2.29,P=0.036)。治疗期间未出现严重不良事件。结论布罗索尤单抗治疗X连锁低磷性佝偻病安全、有效,不良反应少,具有良好的临床应用价值。Objective To evaluate the efficacy and safety of Burosumab in patients with X-linked hypophosphatemic rickets.Methods Clinical data of 9 children diagnosed with X-linked hypophosphatemic rickets and treated with Burosumab in the Department of Pediatric Nephrology,Anhui Children′s Hospital from November 2021 to September 2023 were retrospectively analyzed,including the general information,clinical manifestations,auxiliary examination,Burosumab treatment and follow-up.Results Among the 9 cases,there were 5 males and 4 females,with a median age at diagonosis of 2 years.After traditional treatment,the fluctuation of serum phosphorus ranged from 0.7 to 0.9 mmol/L.The median age at the initiation of Burosumab treatment was 2.8 years,and the initial dosage was 0.8 mg/kg,administrated subcutaneously every 2 weeks.The laboratory and imaging indexes were improved after 6 months of Burosumab treatment,and the mean serum phosphorus level increased from(0.81±0.14)mmol/L to(1.02±0.10)mmol/L at 1 month(t=3.85,P=0.001)and(1.14±0.25)mmol/L at 6 months(t=3.58,P=0.002).The average alkaline phosphatase(ALP)level decreased from(509.89±110.10)U/L before treatment to(447.89±106.76)U/L after 1 month(t=1.21,P=0.243).After 6 months,the ALP level significantly decreased to(385.89±60.33)U/L(t=2.96,P=0.009).The average height percentile increased from 18.42±10.09 before treatment to 26.56±16.59 after 6 months(t=1.26,P=0.227).Rachitis severity scores of both lower limbs ranged from 4.61±1.36 before treatment to 3.06±1.51 after 6 months(t=2.29,P=0.036).No serious adverse events occurred during treatment.Conclusion Burosumab is safe and effective in treating X-linked hypophosphatemic rickets,exhibiting minimal side effects and significant clinical applicability value.

关 键 词：X连锁低磷性佝偻病 儿童 布罗索尤单抗 疗效 

分 类 号：R725.9[医药卫生—儿科]