生长激素缺乏症儿童使用长效生长激素制剂的长期有效性和安全性。

IF 1.3 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM Journal of Pediatric Endocrinology & Metabolism Pub Date : 2024-10-18 DOI:10.1515/jpem-2024-0351
Eungu Kang, Lindsey Yoojin Chung, Young-Jun Rhie, Kee-Hyoung Lee, Hyo-Kyoung Nam
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引用次数: 0

摘要

目的评估生长激素缺乏症儿童每周与每天服用生长激素的长期疗效:本研究是 "LG 生长研究 "的一部分,共纳入了 996 名生长激素缺乏症儿童(其中 773 名每天服用生长激素,193 名每周服用生长激素)。在基线期和每 12 个月收集一次人体测量数据;在基线期和整个研究期间收集临床和实验室数据:结果:在基线时,每周 GH 组与每日 GH 组相比,年龄更大、父母身高中位数(MPH)更矮、青春期男孩更多(年龄:8.46±3.44 岁 vs. 7.46±2.89 岁,p):对于GH缺乏症患儿来说,每周一次的GH治疗似乎是一种有效且安全的替代每日GH治疗的方法,疗程长达4年。为了证实这些研究结果,并评估 LAGH 的长期安全性和有效性,还需要进行样本量更大、随访时间更长的进一步研究。
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Long-term effectiveness and safety of long-acting growth hormone preparation in children with growth hormone deficiency.

Objectives: To evaluate the long-term effectiveness of weekly vs. daily growth hormone (GH) administration in children with GH deficiency.

Methods: This study, part of the "LG Growth Study", included a total of 996 children with GH deficiency (773 receiving daily GH and 193 receiving weekly GH). Anthropometric data were collected at baseline and every 12 months; clinical and laboratory data were collected at baseline and throughout the study.

Results: At baseline, the weekly GH group was older, shorter in mid-parental height (MPH), and had more pubertal boys compared to the daily GH group (age: 8.46 ± 3.44 vs. 7.46 ± 2.89 years, p<0.001; MPH: -0.88 ± 0.73 SD vs. -1.02 ± 0.84 SD, p=0.044; pubertal boys: 34.0 vs. 16.9 %, p=0.006). Height velocity and change in height SDS during the first 12 months were higher in the daily GH group (height velocity: 9.06 ± 1.72 vs. 8.67 ± 1.98 cm/year, p=0.028; height SDS change: 0.78 ± 0.39 vs. 0.61 ± 0.41, p<0.001). However, height SDS at 24 and 48 months were similar between groups. No significant differences in overall height velocity, annualized treatment continuation rate, and safety profile were observed over 48 months.

Conclusions: Weekly GH therapy appears to be an effective and safe alternative to daily GH treatment in children with GH deficiency over a 4-year period. Further research with larger sample sizes and longer follow-up is needed to confirm these findings and assess the extended safety and effectiveness of LAGH.

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来源期刊
CiteScore
2.70
自引率
7.10%
发文量
176
审稿时长
3-6 weeks
期刊介绍: The aim of the Journal of Pediatric Endocrinology and Metabolism (JPEM) is to diffuse speedily new medical information by publishing clinical investigations in pediatric endocrinology and basic research from all over the world. JPEM is the only international journal dedicated exclusively to endocrinology in the neonatal, pediatric and adolescent age groups. JPEM is a high-quality journal dedicated to pediatric endocrinology in its broadest sense, which is needed at this time of rapid expansion of the field of endocrinology. JPEM publishes Reviews, Original Research, Case Reports, Short Communications and Letters to the Editor (including comments on published papers),. JPEM publishes supplements of proceedings and abstracts of pediatric endocrinology and diabetes society meetings.
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