Division of Endocrinology and the Rotterdam Pituitary Centre, Department of Medicine, Erasmus University MC, Rotterdam, The Netherlands.
Assistance Publique-Hôpitaux de Paris, Hôpitaux Universitaires Paris-Saclay, Hôpital de Bicêtre, Service d'Endocrinologie et des Maladies de la Reproduction, Centre de Référence des Maladies Rares de l'Hypophyse, Le Kremlin Bicêtre, France.
Eur J Endocrinol. 2020 Jun;182(6):D17-D29. doi: 10.1530/EJE-19-0998.
Current guidelines recommend the use of long-acting somatostatin receptor ligands (SRLs) first when surgery fails to correct GH/IGF-I hypersecretion in patients with acromegaly. In this issue of the journal, a pro- and contra debate will outline which arguments are in favour and which are against positioning pegvisomant (PEGV), a GH receptor antagonist, as the first-line treatment modality of acromegaly. The task of the pros was to promote a paradigm shift towards repositioning PEGV as first-line treatment as PEGV is safe and more effective than the first- and second-generation of SRLs. SRLs, when prescribed together with PEGV can still reduce tumour size when necessary, while they decrease the necessary dose of PEGV by around 50% in the average patient. They conclude that PEGV must move up towards the first-line treatment. For the cons, SRLs remain the first-line medical treatment. Indeed, even if, in recent studies, the remission rate is lower than initially claimed, SRLs are still effective not only for normalizing GH/IGF-I levels in half of the patients but also for inducing tumour shrinkage, improving comorbidities and headaches and reversing excess mortality. They are more convenient for use with their monthly administration and have a remarkable safety profile as demonstrated by the very prolonged experience acquired by more than 30 years of use. Finally, the cost-effectiveness of first-generation SRLs is better than that of PEGV. For all these reasons, cons consider that SRLs remain the best first medical treatment in patients requiring medical therapy.
目前的指南建议,当手术未能纠正肢端肥大症患者的 GH/IGF-I 过度分泌时,首先使用长效生长抑素受体配体(SRL)。在本期杂志中,正反双方将围绕哪些论点支持将 pegvisomant(PEGV),一种生长激素受体拮抗剂,定位为肢端肥大症的一线治疗方法展开辩论。正方的任务是推动将 PEGV 重新定位为一线治疗的范式转变,因为 PEGV 既安全又比第一代和第二代 SRL 更有效。当与 PEGV 一起使用时,SRL 仍然可以在必要时缩小肿瘤大小,同时使平均患者的 PEGV 剂量减少约 50%。他们得出的结论是,PEGV 必须向一线治疗方向发展。对于反方来说,SRL 仍然是一线的药物治疗。事实上,即使在最近的研究中,缓解率低于最初声称的水平,SRL 仍然有效,不仅可以使一半患者的 GH/IGF-I 水平正常化,还可以诱导肿瘤缩小,改善合并症和头痛,并逆转过度死亡。它们每月给药,使用更方便,并且具有卓越的安全性,这得益于超过 30 年的使用所获得的非常长期的经验。最后,第一代 SRL 的成本效益优于 PEGV。基于所有这些原因,反方认为 SRL 仍然是需要药物治疗的患者的最佳一线治疗药物。