普拉德-威利综合征的长期生长激素影响:系统性审查和元分析
Mahmoud N Almutadares1, Nour M Gazzaz1, Naseem Y Alyahyawi1
1From the Department of Genetic Medicine (Almutadares), Faculty of Medicine, King Abdulaziz University; from the Department of Pediatrics (Gazzaz, Alyahyawi, Hothan, Bukhari, A. Mazi), Faculty of Medicine, King Abdulaziz University; from the Medical Laboratory Sciences (Alrayes), Faculty of Applied Medical Sciences, King Abdulaziz University; from Princess Al-Jawhara Center of Excellence in Research of Hereditary Disorders (Alrayes); from the Primary Health Care (E. Mazi), King Abdullah Complex Center, Jeddah Second Health Cluster, Ministry of Health; from the Department of Emergency Medicine (Mansouri), Faculty of Medicine, King Abdulaziz University; and from the Faculty of Medicine in Rabigh (Hamadallah), King Abdulaziz University Jeddah, Kingdom of Saudi Arabia.
在普拉德-威利综合征 (PWS) 中,生长激素 (GH) 治疗显著提高身高和胰岛素类生长因子1 (IGF-1) 水平,同时降低体重指数 (BMI). 在PWS患者的GH治疗期间,仔细监测代谢变化是必不可少的.
科学领域:
- 内分泌学和代谢障碍 代谢障碍
- 遗传学和罕见疾病
背景情况:
- 普拉德-威利综合征 (PWS) 是一种罕见的遗传疾病,通常与生长激素 (GH) 缺乏和其他内分泌异常有关.
- 了解GH治疗的长期影响对于管理PWS患者至关重要.
研究的目的:
- 评估GH治疗对PWS患者生长,身体组成和代谢参数的长期影响.
- 为了确定与GH治疗相关的死亡率在这个人群中.
主要方法:
- 在PubMed,Scopus,Web of Science和Cochrane数据库中进行了系统的文献搜索.
- 使用的关键词是"生长激素"和"普雷德-威利综合征".
- 结果措施包括身高-SDS,体重-SDS,BMI-SDS,IGF1,死亡率,LDL胆固醇和血糖.
主要成果:
- 与基线和未接受治疗的患者相比,GH治疗导致身高-SDS (MD 1.05-1.53) 和IGF1-SDS显著增加.
- GH治疗与BMI-SDS (MD-1.02) 的降低有关,这表明肌肉质量有所改善.
- 代谢变化包括增加的LDL胆固醇和血糖水平. 估计的死亡率为1.5%.
结论:
- 在PWS患者中,GH疗法有效地改善了身高和IGF-1水平,对BMI产生有益影响.
- 由于可能增加LDL胆固醇和血糖,建议对代谢进行密切监测.
- GH疗法支持PWS患者更健康的发育,但需要仔细管理相关的代谢风险.
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