Coyne Emma, Muthuvel Gajanthan, Gutmark-Little Iris
Department of Pharmacy, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
Department of Endocrinology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA.
J Pediatr Endocrinol Metab. 2025 Jan 23;38(3):248-253. doi: 10.1515/jpem-2024-0496. Print 2025 Mar 26.
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.
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.
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.
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.
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。
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。
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。
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。