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从生长激素转换为洛那索肽治疗儿童生长激素缺乏症的临床结局

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

作者信息

Coyne Emma, Muthuvel Gajanthan, Gutmark-Little Iris

机构信息

Department of Pharmacy, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

Department of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.

出版信息

J Pediatr Endocrinol Metab. 2025 Jan 23;38(3):248-253. doi: 10.1515/jpem-2024-0496. Print 2025 Mar 26.

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.

摘要

目的

本研究旨在评估小儿生长激素缺乏症(GHD)患者从生长激素转换为长效生长激素后胰岛素样生长因子1(IGF-1)水平的差异。次要目标包括根据IGF-1水平评估剂量滴定、年化身高增长速度(AHV)和体重指数(BMI)的变化,以及评估与长效生长激素治疗相关的报告不良反应。

方法

进行了一项单中心回顾性研究,纳入了最初接受生长激素治疗、在2022年1月1日至2023年12月31日期间转换为长效生长激素的小儿GHD患者。

结果

纳入了14名患者(中位年龄:9岁)。转换时生长激素的中位剂量为0.18mg/kg/周(范围为0.09至0.29),患者开始使用长效生长激素的中位剂量为0.23mg/kg/周(范围为0.15至0.26)。这导致转换后IGF-1增加了2.3 SDS。根据IGF-1水平进行了剂量调整。5名患者需要立即降低剂量;其中4名由于持续升高需要进一步调整。没有报告严重不良反应。

结论

对于小儿GHD患者,长效生长激素可能是减轻注射负担的有利选择,尽管制造商推荐的起始剂量0.24mg/kg/周可能需要个体化。基于IGF-1水平进行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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