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 0.51 mg/day (naive patients) and 0.39 and 0.46 mg/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).

中文翻译：

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在真实世界的法国生长激素缺乏症成年患者队列中遵守生长激素治疗指南

客观的

使用来自生长激素缺乏症 (GHD) 患者的真实数据，我们评估了法国的临床实践是否遵守关于生长激素剂量调整的国际指南，并评估了生长激素的长期有效性和安全性。

方法

数据来自一项国家前瞻性系统纵向常规随访计划，该计划针对儿童期发病 (CO) 或成人发病 (AO) GHD 的初治/非初治成人接受 Norditropin® (Novo Nordisk A/S) 治疗。

结果

2003 年至 2006 年间，招募了 331 名初治和非初治的患有严重 GHD 的成年人，并对其进行了中位持续时间约 5 年的随访；328 名患者可供分析。基线时，患者的平均年龄为 39.2 岁；初始患者中胰岛素样生长因子-1 (IGF-1) 水平的中位标准差评分 (SDS) 为 -2.2，随后在研究期间在 -0.1 和 +0.3 SDS 之间波动。平均 GH 剂量范围在 0.25 和 0.51 毫克/天（初治患者）和 0.39 和 0.46 毫克/天（非初治患者）之间。尽管通常接受较高的生长激素剂量，但女性（初治/非初治）的 IGF-1 水平往往低于男性。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) 没有系统地伴随生长激素剂量调整。大约三分之一的患者腰围有所改善，平均为 3.5 岁。生长激素耐受性良好；在 5 年的分析期内没有发生心血管或脑血管事件。

结论

目前法国医生的临床实践遵循关于成人 GHD 生长激素剂量调整的国际指南。然而，剂量调整并不总是足够的，尤其是在女性中，由于生长激素剂量低，治疗效果可能会延迟（临床试验注册 NCT01580605）。