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葡萄牙关于沙芬酰胺用于帕金森病治疗的专家观点:葡萄牙德尔菲共识的见解

Expert perspectives on the use of safinamide for Parkinson's disease in Portugal: insights from a Portuguese Delphi Consensus.

作者信息

Rodrigues Ana Margarida, Costa Cristina, Gago Miguel, Grunho Miguel, Guedes Leonor Correia, Morgadinho Ana, Rosas Maria José, Simões Rita, Velon Ana Graça

机构信息

CNS - Campus Neurológico (Braga), 4710-452 Braga, Portugal.

Department of Neurology, Hospital Professor Doutor Fernando Fonseca, Unidade Local de Saúde Amadora/Sintra, 2720-276 Amadora, Portugal.

出版信息

J Comp Eff Res. 2025 Jul;14(7):e240228. doi: 10.57264/cer-2024-0228. Epub 2025 Jun 6.

Abstract

Safinamide is an approved medication for managing motor fluctuations in Parkinson's disease (PD). However, limited data exist regarding its application in clinical practice in Portugal and the perspectives of Portuguese neurologists on its use. To address this, a group of Portuguese specialists with recognized expertise in PD management convened to compile the insights on various aspects of safinamide use in PD patients among the field and to develop recommendations aimed at informing and guiding physicians in Portugal on its optimal clinical application. A focus group composed of nine Portuguese PD experts developed a questionnaire building on the 2022 European Delphi study, and employed a Delphi methodology approach to gather the views of Portuguese neurologists with a minimum of 5 years of clinical experience with safinamide (n = 35). A final online questionnaire comprising 35 statements was administered in a single-round Delphi format, utilizing a 5-point Likert scale. Consensus was defined as achieving ≥66% agreement or disagreement among the panelists. A strong consensus emerged among Portuguese neurologists regarding the therapeutic efficacy of safinamide in addressing motor symptoms, motor fluctuations and quality of life. Additionally, agreement was reached on its positive effects on nonmotor symptoms such as sleep, fatigue, mood, quality of life and pain management. However, no consensus was achieved regarding safinamide's efficacy in managing orthostatic hypotension, cognitive issues, urinary and sexual dysfunction, as well as its safety profile in PD patients with hallucinations. Overall, the opinions of Portuguese neurologists aligned closely with those of their European counterparts. This Delphi study highlights the consensus among Portuguese neurologists on the efficacy of safinamide for managing motor symptoms in PD. The considerations presented herein offer essential guidance for effectively managing PD with safinamide, ultimately enhancing patient care in Portugal.

摘要

沙芬酰胺是一种已获批用于治疗帕金森病(PD)运动波动的药物。然而,关于其在葡萄牙临床实践中的应用以及葡萄牙神经科医生对其使用的看法的数据有限。为了解决这一问题,一群在PD管理方面具有公认专业知识的葡萄牙专家齐聚一堂,汇编了该领域关于PD患者使用沙芬酰胺各个方面的见解,并制定了相关建议,旨在为葡萄牙的医生提供关于其最佳临床应用的信息和指导。一个由九名葡萄牙PD专家组成的焦点小组在2022年欧洲德尔菲研究的基础上设计了一份问卷,并采用德尔菲方法收集了至少有5年沙芬酰胺临床经验的葡萄牙神经科医生的意见(n = 35)。最终的在线问卷包含35条陈述,采用单轮德尔菲形式,使用5点李克特量表。共识被定义为专家小组成员中达成≥66%的同意或不同意。葡萄牙神经科医生在沙芬酰胺治疗运动症状、运动波动和生活质量的疗效方面达成了强烈共识。此外,对于其对睡眠、疲劳、情绪、生活质量和疼痛管理等非运动症状的积极影响也达成了一致意见。然而,在沙芬酰胺治疗体位性低血压、认知问题、泌尿和性功能障碍的疗效以及其在有幻觉的PD患者中的安全性方面未达成共识。总体而言,葡萄牙神经科医生的意见与欧洲同行的意见密切一致。这项德尔菲研究突出了葡萄牙神经科医生对沙芬酰胺治疗PD运动症状疗效的共识。本文提出的考虑因素为有效使用沙芬酰胺治疗PD提供了重要指导,最终改善了葡萄牙的患者护理。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/26b8/12224116/d9bed6d60962/cer-14-240228-g1.jpg

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