相关实验视频

Updated: May 11, 2026

Assessment of Antibody-based Drugs Effects on Murine Bone Marrow and Peritoneal Macrophage Activation

Assessment of Antibody-based Drugs Effects on Murine Bone Marrow and Peritoneal Macrophage Activation

Published on: December 26, 2017

从活性维生素d和酸盐补充剂转换为布罗苏马布显著纠正儿科X链接低度症下肢不整齐

Leanne M Ward¹, Erik A Imel², David Frumberg³

¹Department of Pediatrics, University of Ottawa, Division of Endocrinology and Metabolism, Children's Hospital of Eastern Ontario, Ottawa, Ontario, Canada.

Journal of bone and mineral research : the official journal of the American Society for Bone and Mineral Research

|June 13, 2025

概括

与活性维生素D和酸盐补充剂相比,切换到布罗苏马布显著改善了X链接低度血症 (XLH) 的儿童下肢不整齐. 早期的布罗苏马布治疗为骨形变提供了最大的益处.

科学领域:

儿科内分泌学儿科内分泌学
骨发育不良症骨发育不良症
罕见的遗传疾病罕见的遗传疾病

背景情况:

与X相关的低血症 (XLH) 是一种罕见的遗传疾病,其特点是脏酸盐浪费和维生素D代谢异常.
患者通常会患上恶心病,骨质疏松症和明显的下肢形,如真 (genu valgum/varum).
目前使用活性维生素D和补充剂 (活性D/Pi) 的治疗可能无法完全纠正不对齐,往往需要手术.

研究的目的:

评估从活性D/Pi切换到布罗苏马布对儿科XLH患者下肢不整齐的影响.
为了比较布罗苏马布与持续活跃的D/Pi在纠正机械股骨角 (mFTA) 的疗效.
为了确定与改善mFTA相关的因素,在XLH患者治疗布罗苏马布.

主要方法:

来自XLH疾病监测计划 (DMP) 的数据分析,包括转换为布罗苏马布或继续活跃D/Pi的患者.
在基线和第3年对机械股骨角 (mFTA) 的放射性评估.
多变量因子分析以确定mFTA变化的预测因素.

主要成果:

在那些转换为布罗苏马布的患者中,在mFTA中改善的四肢比例明显高于那些仍在使用活性D/Pi的患者 (p < .023).
转换为布罗苏马布显示,肢体改善的可能性是持续活跃D/Pi的4.38倍 (p = .0469).
开始服用布罗苏马布的年龄较小和基线较低的身高Z-score显著与更大的mFTA改善有关.

结论:

切换到布罗苏马布可显著改善小儿XLH患者下肢不对齐的情况,超出了活跃D/Pi的好处.
早期启动布罗苏马布治疗与骨形变的最大纠正有关.
布罗苏马布代表了一种有价值的治疗选择,用于管理XLH的下肢不对齐.

关键词:

与X相关的低酸血症.有活性维生素D的维生素D.这就是Burosumab.真正的选举权.真正的瓦鲁姆.下肢不整齐下肢不整齐的情况酸盐酸盐的使用方法

更多相关视频

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Measurements of Motor Function and Other Clinical Outcome Parameters in Ambulant Children with Duchenne Muscular Dystrophy

Published on: January 12, 2019

Preparation Of Gushukang GSK Granules for In Vivo and In Vitro Experiments

Preparation Of Gushukang GSK Granules for In Vivo and In Vitro Experiments

Published on: May 9, 2019

相关概念视频

Introduction to Electrolytes

Introduction to Electrolytes

In humans, electrolytes play a vital role in various physiological processes. Balancing electrolyte levels is essential for normal body functions; their imbalance can be life-threatening. The major electrolytes include sodium, potassium, chloride, calcium, phosphate, and bicarbonate. They are primarily involved in physiological processes, such as nerve signal transmission, membrane trafficking, muscle contraction, buffering body fluids, and balancing water levels in the body.
Role of Sodium
One...

Bone Disorders

Bone Disorders

Aging and its effect on bone remodeling is the most common cause of bone disorders. In young and healthy people, bone deposition and resorption happen at an equal rate to maintain optimal bone health.
Bone deposition is also affected by the levels of sex hormones like estrogen and testosterone that promote osteoblast activity and bone matrix synthesis. When the level of these hormones decreases due to aging, it causes a reduction in bone deposition. As a result, bone resorption by osteoclasts...

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Pharmacokinetics in Pediatric Patients: Overview and Drug Absorption

Understanding the physiological differences in the pediatric population is crucial for effective pharmacotherapy. Neonates, infants, and children exhibit significant variations in gastric pH, gastric emptying time, intestinal transit time, and biliary function. These variations profoundly affect oral drug absorption, necessitating a nuanced approach to pediatric dosing.Neonates present with a unique physiological profile, having a gastric pH greater than 4 and faster and more irregular gastric...