Endocrine Unit and Neuroendocrinology Research Center, Medical School and Hospital Universitário Clementino Fraga Filho - Universidade Federal do Rio de Janeiro, Rio de Janeiro, 21941-913, Brazil.
Neuroendocrine Unit - Instituto Estadual do Cérebro Paulo Niemeyer, Secretaria Estadual de Saúde, Rio de Janeiro, 20231-092, Brazil.
J Clin Endocrinol Metab. 2022 Jan 18;107(2):297-308. doi: 10.1210/clinem/dgab726.
Currently, the first-generation somatostatin receptor ligands (fg-SRLs), octreotide LAR and lanreotide autogel, are the mainstays of acromegaly treatment and achieve biochemical control in approximately 40% of patients and tumor shrinkage in over 60% of patients. Pasireotide, a second-generation SRL, shows higher efficacy with respect to both biochemical control and tumor shrinkage but has a worse safety profile. In this review, we discuss the future perspectives of currently available SRLs, focusing on the use of biomarkers of response and precision medicine, new formulations of these SRLs and new drugs, which are under development. Precision medicine, which is based on biomarkers of response to treatment, will help guide the decision-making process by allowing physicians to choose the appropriate drug for each patient and improving response rates. New formulations of available SRLs, such as oral, subcutaneous depot, and nasal octreotide, may improve patients' adherence to treatment and quality of life since there will be more options available that better suit each patient. Finally, new drugs, such as paltusotine, somatropin, ONO-5788, and ONO-ST-468, may improve treatment adherence and present higher efficacy than currently available drugs.
目前,第一代生长抑素受体配体(fg-SRLs),奥曲肽 LAR 和兰瑞肽微球,是治疗肢端肥大症的主要药物,可使约 40%的患者实现生化控制,超过 60%的患者肿瘤缩小。作为第二代 SRL,培高利特在生化控制和肿瘤缩小方面显示出更高的疗效,但安全性较差。在这篇综述中,我们讨论了目前可用的 SRL 的未来前景,重点讨论了反应生物标志物和精准医学的应用、这些 SRL 的新制剂和正在开发的新药。基于治疗反应生物标志物的精准医学将通过允许医生为每个患者选择合适的药物并提高反应率来帮助指导决策过程。现有的 SRL 的新制剂,如口服、皮下储库和鼻喷奥曲肽,可能会提高患者对治疗的依从性和生活质量,因为将有更多适合每个患者的选择。最后,新的药物,如培高利特、生长激素、ONO-5788 和 ONO-ST-468,可能会提高治疗的依从性,并表现出比现有药物更高的疗效。