Impact of Daily Growth Hormone Adherence on Height Velocity Among Children With Growth Hormone Deficiency (GHD)

IF 3.7 3区 医学 Q2 ENDOCRINOLOGY & METABOLISM Endocrine Practice Pub Date : 2024-09-01 DOI:10.1016/j.eprac.2024.05.012
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Abstract

Objective

To describe adherence to daily somatropin treatment and impact on height velocity within 1 year of treatment start among patients with pediatric growth hormone deficiency in a real-world US population.

Methods

This retrospective cohort study included pediatric patients aged ≥3 years to <16 years with pediatric growth hormone deficiency prescribed somatropin by a pediatric endocrinologist at a US-based center of excellence between January 1, 2015 and December 31, 2020. Patient data were collected using hospital electronic health records linked to a specialty pharmacy patient prescription records. Adherence, evaluated over 12 months, was measured using the proportion of days covered metric and patients were categorized as adherent if their proportion of days covered ≥80%. Height velocity was annualized to compare across adherent and nonadherent patients.

Results

One hundred eighty-one patients were identified and included in this study, of which 70.2% were male,73.5% were white, and mean age (standard deviation [SD]) at index was 12.1 (2.8). In the height velocity analysis, 174 patients were included and the mean (SD) annualized change in height was 10.2 (5.7) cm/y in the adherent group (n = 108) and 9.8 (7.6) in the nonadherent group (n = 66). The difference in height velocity between the groups was not statistically significant.

Conclusions

Minor improvements in average height velocity were observed in the patient group who were adherent to somatropin therapy, although not statistically significant. Lack of observed significance may be due to small sample sizes, short observation period, a likely heterogenous population in terms of growth hormone prescribing, data bias due to single-center origin, or potential patient misclassification.

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坚持每天使用生长激素对生长激素缺乏症(GHD)儿童身高增长速度的影响。
目的描述美国真实人群中小儿生长激素缺乏症(pGHD)患者对每日索马托品治疗的依从性以及治疗开始一年内对身高速度的影响:这项回顾性队列研究纳入了年龄≥3 岁的儿科患者:其中 70.2% 为男性,73.5% 为白人,指数时的平均年龄(标准差 [SD])为 12.1 (2.8)。在身高速度分析中,共纳入了 174 名患者,坚持治疗组(108 人)和非坚持治疗组(66 人)的平均(标清)年身高变化分别为 10.2 (5.7) 厘米/年和 9.8 (7.6)厘米/年。两组间的身高体重差异无统计学意义:坚持使用索马托品治疗的患者组平均血压略有改善,但无统计学意义。未观察到显著性的原因可能是样本量小、观察期短、在生长激素处方方面可能存在异质性人群、单中心导致的数据偏差或潜在的患者分类错误。
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来源期刊
Endocrine Practice
Endocrine Practice ENDOCRINOLOGY & METABOLISM-
CiteScore
7.60
自引率
2.40%
发文量
546
审稿时长
41 days
期刊介绍: Endocrine Practice (ISSN: 1530-891X), a peer-reviewed journal published twelve times a year, is the official journal of the American Association of Clinical Endocrinologists (AACE). The primary mission of Endocrine Practice is to enhance the health care of patients with endocrine diseases through continuing education of practicing endocrinologists.
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