AP-HP, Paris Saclay University, INSERM U1185, Endocrinology and Diabetes for children, Reference Center for rare diseases of calcium and phosphate metabolism, OSCAR network, Platform of expertise for rare diseases of Paris Saclay Hospital, Bicêtre Paris Saclay Hospital, Le Kremlin-Bicêtre, France.
Service d'Endocrinologie et Diabétologie Pédiatrique, Centre de Référence des Maladies Rares de la Thyroïde et des Récepteurs Hormonaux, Centre de Référence (constitutif) des Maladies Rares de l'Hypophyse, FIRENDO network, CHU Angers, 49000 Angers, France.
Arch Pediatr. 2024 Aug;31(6):357-364. doi: 10.1016/j.arcped.2024.03.009. Epub 2024 Jul 18.
Approximately 10,000 children in France with growth hormone deficiency (GHD) are being administered daily recombinant human growth hormone (rhGH). Although this treatment has long proved efficient for restoring children's growth and metabolism, daily injections of rhGH have a few limitations, such as difficulties in terms of adherence to treatment, which may compromise growth during childhood but also metabolism in adulthood. In addition to the disease burden and besides the adherence hurdles, the obligations related to daily injection have a negative impact on the quality of life of patients and their families. The hypothesis that injections administered at intervals of 1 week, or even 1 month, could improve compliance, reduce treatment discontinuations, and optimize quality of life and therapeutic effectiveness has led to the emergence of new long-acting growth hormone (LAGH). Recent access to LAGHs (somatrogon MA) on the European and French market will likely be followed by a high demand from the families concerned and may raise questions on their effectiveness, safety, and practical use. Numerous practical and practice-related points are needed to guide prescribing physicians while many concerns are still left unresolved (treatment effectiveness or ineffectiveness endpoints, long-term effectiveness, etc.). These issues can only be addressed in the future by compiling registries and conducting long-term real-world studies.
在法国，大约有 10000 名患有生长激素缺乏症（GHD）的儿童每天接受重组人生长激素（rhGH）治疗。虽然这种治疗方法长期以来被证明可以有效地恢复儿童的生长和代谢，但 rhGH 的每日注射存在一些局限性，例如治疗的依从性困难，这可能会影响儿童时期的生长，也会影响成年后的代谢。除了疾病负担和治疗障碍之外，与每日注射相关的义务对患者及其家庭的生活质量产生负面影响。每周甚至每月注射一次可以提高依从性、减少治疗中断、优化生活质量和治疗效果的假设，催生了新的长效生长激素（LAGH）。最近，LAGHs（somatrogon MA）在欧洲和法国市场的上市，可能会引起相关家庭的高度需求，并可能引发对其有效性、安全性和实际应用的质疑。需要解决许多实际问题和实践相关问题，为处方医生提供指导，同时仍有许多问题悬而未决（治疗效果或无效终点、长期效果等）。只有通过编制登记册和开展长期真实世界研究，才能解决这些问题。
