Evaluation of Burosumab in the treatment of X-linked hypophosphatemic rickets in children
10.3760/cma.j.cn311282-20231011-00113
- VernacularTitle:布罗索尤单抗治疗儿童X连锁低磷性佝偻病疗效评估
- Author:
Shaohan FANG
1
;
Fang DENG
;
Yue YUAN
;
Xu LI
;
Zhen ZHAO
;
Ying ZHU
;
Yin PENG
;
Luodan ZHANG
Author Information
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
- Keywords:
X-linked hypophosphatemic rickets;
Children;
Burosumab;
Efficacy
- From:
Chinese Journal of Endocrinology and Metabolism
2024;40(1):11-16
- CountryChina
- Language:Chinese
-
Abstract:
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.
