背景:促生长素治疗适用于多种疾病,包括生长激素(GH)缺乏症,普拉德-威利和特纳综合征,慢性肾功能不全和其他。迄今为止,几乎所有的研究都仅限于单一的GH产品,并且没有建立跨适应症和生长激素产品的独立注册。
目的:本研究人员发起的名为INSIGHTS-GHT的注册表旨在提供有关德国在常规临床实践中批准的适应症中生长激素治疗的各个方面的全面信息:药物利用,有效性(包括实际最终高度,身体成分),耐受性,生活质量,其他患者相关结果(PRO),和健康经济变量。
方法:注册(前瞻性观察研究)在德国专门的儿科和成人内分泌中心。任何年龄的患者都有资格获得文件,如果他们正在使用标签中的任何批准的生长激素或生长激素相关产品进行正在进行或新开始的治疗,可用于长期随访文件,如果他们提供知情同意。受试者可能会转换,随时停止/中断或启动生长激素产品。随访至少3年(最短研究时间)。计划每年一次或两次记录生长激素的使用情况(产品,给药),其他药物,实验室状态(葡萄糖,脂质,GH功能,包括刺激测试,IGF-I,IGFBP3),如果适用,青春期发育,辅助参数,身体成分和骨龄。患者报告结果(PRO)措施包括,但不限于,14岁及以上的成人和青少年的简短形式12。安全性报告包括不良事件。
结论:注册表在一个联合注册表中记录了儿童和成人,包括,目前,德国的患者,并允许所有批准的生长激素和其他生长激素制剂的患者记录。它将允许描述受试者从青春期到成年期的过渡(治疗和身高),描述生长激素制剂之间的转换,进行响应者分析,并分析生长激素利用的异同(按年龄组,性别,设置,和PRO仪器)。INSIGHTS-GHT提供了广泛的,综合研究平台,以评估生长激素治疗和结果的多个相关方面(包括受试者从青春期到成年的过渡),允许所有GH产品的文档,包括长效GH制剂后,并将独立于资助者评估数据。试用注册BfArMNr.NIS7492,DRKS注册表DRKS00027394。
Somatropin treatment is indicated in a variety of disorders including growth hormone (GH) deficiency, Prader-Willi and Turner syndrome, chronic renal insufficiency and others. To date, almost all studies have been limited to single GH products, and no independent registry across indications and
somatropin products was ever established.
The present investigator-initiated registry named INSIGHTS-GHT aims to provide comprehensive information on various aspects of somatropin treatment in Germany in approved indications within routine clinical practice: drug utilization, effectiveness (including real final height, body composition), tolerability, quality of life, other patient related outcomes (PRO), and health economic variables.
Registry (prospective observational study) in specialised pediatric and adult endocrinology centres in Germany. Patients of any age are eligible for documentation, if they are on ongoing or newly initiated treatment with any approved somatropin or
somatropin-related product within the labelling, available for long term follow-up documentation, and if they provided informed consent. Subjects may switch, discontinue/interrupt or initiate
somatropin products at any time. They are followed up for at least 3 years (minimal study duration). Documentation is planned once or twice per year to record
somatropin utilisation (product, dosing), other medications, laboratory status (glucose, lipids, GH function including stimulation tests, IGF-I, IGFBP3), if applicable, pubertal development, auxological parameters, body composition and bone age. Patient reported outcome (PRO) measures include, but are not limited to, Short Form 12 in adults and adolescents aged 14 years and over. Safety reporting includes adverse events.
The registry documents children and adults in one joint registry, includes, at present, patients in Germany and allows documentation of patients on all approved somatropin and other growth hormone preparations. It will allow to describe the transition of subjects from adolescence to adulthood (treatment and height), to describe switches between somatotropin preparations, to perform responder analyses, and to analyse differences and similarities of somatropin utilization (by age group, sex, setting, and PRO instrument). INSIGHTS-GHT offers a broad, comprehensive research platform to assess multiple relevant aspects of somatropin treatment and outcomes (including the transition of subjects from adolescence to adulthood), allows the documentation of all GH products including long-acting GH preparations after their introduction, and will evaluate the data independently of funders. Trial registration BfArM Nr. NIS7492, DRKS registry DRKS00027394.