Clinical outcomes of switching to lonapegsomatropin from somatropin for treatment of pediatric growth hormone deficiency.

IF 1 4区 医学 Q4 ENDOCRINOLOGY & METABOLISM
Emma Coyne, Gajanthan Muthuvel, Iris Gutmark-Little
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引用次数: 0

Abstract

Objectives: This study aimed to assess differences in insulin-like growth factor 1 (IGF-1) levels following the transition from somatropin to lonapegsomatropin in patients with pediatric growth hormone deficiency (GHD). Secondary objectives included the evaluation of dose titrations based on IGF-1 levels, changes in annualized height velocity (AHV) and body mass index (BMI), and assessing reported adverse effects associated with lonapegsomatropin therapy.

Methods: A single-center, retrospective review was conducted including patients diagnosed with pediatric GHD initially treated with somatropin who transitioned to lonapegsomatropin between January 1, 2022, and December 31, 2023.

Results: Fourteen patients (median age: 9 years) were included. The median somatropin dose was 0.18 mg/kg/week (range, 0.09 to 0.29) at the time of transition and patients were initiated on a median lonapegsomatropin dose of 0.23 mg/kg/week (range, 0.15 to 0.26). This resulted in an IGF-1 increase of 2.3 SDS post-switch. Dose adjustments were made based on IGF-1 levels. Five patients required immediate dose reductions; four of these required further adjustments due to persistent elevation. There were no serious adverse effects reported.

Conclusions: Lonapegsomatropin may be a favorable option to reduce injection burden for those with pediatric GHD, though the manufacturer's recommended starting dose of 0.24 mg/kg/week may require individualization. Careful monitoring and dose adjustment based on IGF-1 levels are necessary to maintain safety and efficacy.

生长激素缺乏症儿童生长激素改用lonapegsomatropin治疗的临床效果。
目的:本研究旨在评估儿童生长激素缺乏症(GHD)患者从生长激素过渡到lonapegsomatropin后胰岛素样生长因子1 (IGF-1)水平的差异。次要目标包括基于IGF-1水平、年化身高速度(AHV)和身体质量指数(BMI)变化的剂量滴定评估,以及评估lonapegsomatropin治疗相关的不良反应。方法:在2022年1月1日至2023年12月31日期间,对最初接受生长激素治疗的诊断为儿童GHD的患者进行单中心回顾性研究,这些患者转为使用lonapegsomatropin。结果:纳入14例患者(中位年龄:9岁)。在过渡时期,生长激素的中位剂量为0.18 mg/kg/周(范围,0.09至0.29),患者开始使用lonapegsomatropin的中位剂量为0.23 mg/kg/周(范围,0.15至0.26)。这导致开关后IGF-1增加2.3 SDS。根据IGF-1水平调整剂量。5名患者需要立即减量;其中四个由于持续升高需要进一步调整。没有严重的不良反应报告。结论:Lonapegsomatropin可能是减少儿童GHD患者注射负担的有利选择,尽管制造商推荐的起始剂量为0.24 mg/kg/周可能需要个体化。基于IGF-1水平的仔细监测和剂量调整对于维持安全性和有效性是必要的。
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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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