真实世界中法国成年生长激素缺乏症患者队列中对生长激素治疗指南的依从性。

Adherence to growth hormone therapy guidelines in a real-world French cohort of adult patients with growth hormone deficiency.

机构信息

Fédération d'endocrinologie, de diabétologie et des maladies métaboliques, Hospices Civils de Lyon, Hôpital Louis Pradel, Université Lyon1 et EA 7425 HESPER, Lyon, France.

Service d'endocrinologie, diabétologie et nutrition, Centre Hospitalier Universitaire de Grenoble, Boulevard de la Chantourne, 38700 La Tronche, France.

出版信息

Ann Endocrinol (Paris). 2021 Feb;82(1):59-68. doi: 10.1016/j.ando.2020.11.007. Epub 2020 Dec 5.

DOI:10.1016/j.ando.2020.11.007

PMID:33290752

Abstract

OBJECTIVE

Using real-world data from patients with growth hormone deficiency (GHD), we evaluated whether clinical practice in France adheres to international guidelines regarding somatropin dose adjustment, and assessed the long-term effectiveness and safety of somatropin.

METHODS

Data were obtained from a national prospective systematic longitudinal routine follow-up programme of naive/non-naive adults with childhood-onset (CO) or adult-onset (AO) GHD treated with Norditropin® (Novo Nordisk A/S).

RESULTS

Between 2003 and 2006, 331 treatment-naive and non-naive adults with severe GHD were enrolled and followed for a median duration of approximately 5 years; 328 patients were available for analysis. At baseline, mean patient age was 39.2 years; median standard deviation score (SDS) for insulin-like growth factor-1 (IGF-1) level was -2.2 in naive patients, subsequently fluctuating between -0.1 and +0.3 SDS during the study period. Mean GH doses ranged between 0.25 and 0.51mg/day (naive patients) and 0.39 and 0.46mg/day (non-naive patients). Despite generally receiving a higher somatropin dose, women (naive/non-naive) tended to have lower IGF-1 levels than men. Median somatropin dose was consistently higher in patients with CO-GHD than patients with AO-GHD. Extreme IGF-1 values (<-2 or >+2 SDS) were not systematically accompanied by somatropin dose adjustments. Waist circumference improved in approximately one third of patients, at a mean 3.5 years. Somatropin was well tolerated; there were no cardiovascular or cerebrovascular events during the 5-year analysis period.

CONCLUSION

Current clinical practice of physicians in France follows international guidelines regarding somatropin dose adjustment in adults with GHD. However, dose adjustments are not always sufficient, notably in women, and treatment effects may have been delayed due to low somatropin dose (Clinical trial registration NCT01580605).

摘要

目的

利用生长激素缺乏症（GHD）患者的真实世界数据，评估法国的临床实践是否符合关于生长激素剂量调整的国际指南，并评估生长激素的长期疗效和安全性。

方法

数据来自一项针对接受诺泽生长激素（Novo Nordisk A/S）治疗的儿童期起病（CO）或成年期起病（AO）GHD 的初治/非初治成年患者的全国前瞻性系统纵向常规随访计划。

结果

2003 年至 2006 年，纳入了 331 名初治和非初治的严重 GHD 成年患者，并进行了大约 5 年的中位随访；328 名患者可进行分析。基线时，患者平均年龄为 39.2 岁；初治患者的胰岛素样生长因子-1（IGF-1）水平的中位数标准差（SDS）为-2.2，在研究期间波动在-0.1 至+0.3 SDS 之间。平均 GH 剂量范围为 0.25 至 0.51mg/天（初治患者）和 0.39 至 0.46mg/天（非初治患者）。尽管女性（初治/非初治）接受的生长激素剂量普遍较高，但 IGF-1 水平却低于男性。CO-GHD 患者的中位生长激素剂量始终高于 AO-GHD 患者。极端 IGF-1 值（<-2 或>+2 SDS）并不总是伴随着生长激素剂量调整。大约三分之一的患者腰围得到改善，平均在 3.5 年内。生长激素耐受性良好；在 5 年的分析期间，未发生心血管或脑血管事件。