Woelfle Joachim, Kreitschmann-Andermahr I, Strasburger C J, Pittrow D B, Pausch C, Schnabel D
Department of Pediatrics and Adolescent Medicine, University Hospital Erlangen, Loschgestr. 15, Erlangen, Germany.
Department of Neurosurgery and Spine Surgery, University Hospital Essen, University of Duisburg-Essen, Essen, Germany.
Orphanet J Rare Dis. 2025 Jul 23;20(1):372. doi: 10.1186/s13023-025-03898-8.
The development of long-acting growth hormone (LAGH) formulations offers a promising approach to reduce injection frequency and to improve adherence in growth hormone deficiency (GHD) treatment. INSIGHTS-GHT is the first product-independent registry to document the real-world use of recombinant human (rh) growth hormone (GH) replacement therapy within the labelling. Following the market launch of three LAGH products in Germany (lonapegsomatropin, somapacitan, and somatrogon) we aimed to provide early real-world evidence on their use in order to obtain an initial picture on patient selection and physician preferences outside of clinical trials.We report in this interim analysis on 70 pediatric patients from 15 centers across Germany as well as 31 adult patients from 6 German centers under LAGH treatment. The majority of the pediatric patients (76%) were male, with a mean age at LAGH initiation of 9.2 years. About half of the pediatric patients (54%) were switch patients transitioning from daily GH therapy. Notably, 82% of patients received a LAGH starting dose below the manufacturer's recommendation, with a median dose of 92% of the recommended level. In the group of adult patients, 65% were male, with a mean age of 38.2 years at LAGH initiation. In pediatric patients, before start of GH therapy, mean IGF-I (SDS) was - 2.1 ± 1.1 SDS, mean IGFBP-3 (SDS) was - 2.0 ± 1.5 SDS.All adult patients switched from daily GH therapy. More than half (55%) received the LAGH starting dose according to the manufacturer's recommendation, while 41% began with a lower-than-recommended dose. Our findings provide early insights into LAGH therapy adoption and highlight the need for continued follow-up to evaluate long-term efficacy, adherence, and safety in real-world settings.
长效生长激素（LAGH）制剂的研发为减少注射频率以及提高生长激素缺乏症（GHD）治疗的依从性提供了一种很有前景的方法。INSIGHTS-GHT是首个记录重组人生长激素（rhGH）替代疗法在标签范围内实际应用情况的产品独立登记处。在德国三种LAGH产品（洛那派索马促生长素、索马帕西坦和索马罗贡）上市后，我们旨在提供关于其使用情况的早期真实世界证据，以便初步了解临床试验之外的患者选择和医生偏好。我们在这项中期分析中报告了来自德国15个中心的70名儿科患者以及来自6个德国中心的31名接受LAGH治疗的成年患者的情况。大多数儿科患者（76%）为男性，开始使用LAGH时的平均年龄为9.2岁。约一半的儿科患者（54%）是从每日生长激素治疗转换而来的转换患者。值得注意的是，82%的患者开始使用的LAGH起始剂量低于制造商的推荐剂量，中位剂量为推荐水平的92%。在成年患者组中，65%为男性，开始使用LAGH时的平均年龄为38.2岁。在儿科患者中，开始生长激素治疗前，平均胰岛素样生长因子-I（SDS）为-2.1±1.1 SDS，平均胰岛素样生长因子结合蛋白-3（SDS）为-2.0±1.5 SDS。所有成年患者均从每日生长激素治疗转换而来。超过一半（55%）的患者根据制造商的推荐接受LAGH起始剂量，而41%的患者开始时使用低于推荐剂量。我们的研究结果为LAGH疗法的应用提供了早期见解，并强调需要持续随访以评估真实世界环境中的长期疗效、依从性和安全性。
