Karolinska Institutet, Karolinska University Hospital, Solna, Sweden.
Hôpital Necker Enfants Malades, Paris, France.
J Clin Endocrinol Metab. 2019 Oct 1;104(10):4730-4742. doi: 10.1210/jc.2019-00775.
Understanding real-world prescribing of GH may help improve treatment of eligible patients.
Overall: to assess real-world effectiveness and safety of GH (Norditropin). This analysis: to compare clinical characteristics of GH-treated children in the United States and Europe.
The American Norditropin Studies: Web-Enabled Research Program (ANSWER; 2002 to 2016, United States) and the NordiNet International Outcome Study (NordiNet IOS; 2006 to 2016, Europe) were multicenter longitudinal observational cohort studies.
Data were recorded in 207 (United States) and 469 (Europe) clinics.
Patients with GH deficiency, Turner syndrome, Noonan syndrome, idiopathic short stature, Prader-Willi syndrome, or born small for gestational age, who commenced GH treatment aged <18 years.
GH was prescribed by treating physicians according to local practice.
Baseline data and drug doses were recorded. Data on effectiveness and safety were collected.
ANSWER had 19,847 patients in the full analysis set (FAS; patients with birthdate information and one or more GH prescription) and 12,660 in the effectiveness analysis set (EAS; GH-naive patients with valid baseline information). NordiNet IOS had 17,711 (FAS) and 11,967 (EAS). Boys accounted for 69% (ANSWER) and 57% (NordiNet IOS). Treatment start occurred later than optimal to improve growth. The proportion of boys treated was generally larger, children were older at treatment start, and GH doses were higher in the United States vs Europe. No new safety signals of concern were noted.
In most indications, more boys than girls were treated, and treatment started late. Earlier diagnosis of GH-related disorders is needed. The data support a favorable benefit-risk profile of GH therapy in children.
了解 GH 的实际应用情况可能有助于改善符合条件的患者的治疗效果。
总体而言，评估 GH（诺德）的实际疗效和安全性。本分析：比较美国和欧洲接受 GH 治疗的儿童的临床特征。
美国诺德研究：网络支持研究计划（ANSWER；2002 年至 2016 年，美国）和诺地网国际结果研究（NordiNet IOS；2006 年至 2016 年，欧洲）是多中心纵向观察队列研究。
数据记录于 207 个（美国）和 469 个（欧洲）诊所。
接受 GH 缺乏症、特纳综合征、努南综合征、特发性身材矮小症、普拉德-威利综合征或宫内生长受限的患者，年龄<18 岁开始接受 GH 治疗。
GH 由主治医生根据当地实践开具处方。
记录基线数据和药物剂量。收集有效性和安全性数据。
ANSWER 的全分析集（FAS；有出生日期信息和一次或多次 GH 处方的患者）有 19847 例患者，有效分析集（EAS；有有效基线信息且 GH 初治的患者）有 12660 例。NordiNet IOS 有 17711 例（FAS）和 11967 例（EAS）。男孩占 69%（ANSWER）和 57%（NordiNet IOS）。为了改善生长，治疗开始时间晚于最佳时间。美国治疗男孩的比例普遍较大，治疗开始时儿童年龄较大，GH 剂量较高。未发现新的严重安全信号。
在大多数适应证中，接受治疗的男孩多于女孩，且治疗开始较晚。需要更早诊断 GH 相关疾病。这些数据支持 GH 治疗儿童的良好获益风险特征。
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