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中国帕金森病患者中依维莫司的性别效应。

The effects of safinamide according to gender in Chinese parkinsonian patients.

机构信息

Neuroscience Section, Department of Medicine, Surgery and Dentistry "Scuola Medica Salernitana", University of Salerno, 84131, Salerno, Italy.

Medical Department, Zambon SpA, Bresso, Italy.

出版信息

Sci Rep. 2023 Nov 23;13(1):20632. doi: 10.1038/s41598-023-48067-8.

Abstract

The incidence and prevalence of Parkinson's disease (PD) is expected to raise dramatically over the next decades. Gender-related differences are not yet widely recognized, particularly regarding the response to dopaminergic medications. To analyse gender differences in the clinical effects of safinamide, compared to placebo, in Chinese PD patients of the pivotal XINDI trial. The XINDI study was a phase III, randomized, double-blind, placebo-controlled, multicenter trial. Patients were followed for 16 weeks receiving safinamide or placebo as add-on to levodopa. The primary efficacy endpoint was the change in the mean total daily OFF time. Secondary efficacy endpoints included total daily ON time, ON time with no/non-troublesome dyskinesia, Unified Parkinson's Disease Rating Scale and Parkinson's Disease Questionnaire-39 items. A post-hoc analysis was performed to describe the efficacy of safinamide in both genders on motor symptoms, motor fluctuations and quality of life. 128 (42%) out of 305 patients enrolled were women and 177 (58%) men. Our additional analyses of the XINDI study have shown that safinamide, compared to placebo, was associated with improvements in motor symptoms, motor fluctuations and quality of life in both genders, with some differences in the response that did not reach statistical significance, possibly due to sample size limitation and post-hoc design of the study. The changes from baseline at week 16 were > 50% higher in the females compared to males for the total daily OFF time (- 1.149 h vs - 0.764 h in males), the total daily ON time (1.283 h vs 0.441 h in males), the UPDRS total score (- 8.300 points vs - 5.253 points in males) and the UPDRS part II score (- 2.574 points vs - 1.016 points in males). The changes from baseline at week 16 were higher in the females compared to males in the "ADL" domain (- 6.965 points vs - 5.772 points in males), the "Emotional well-being" domain (- 6.243 points vs - 4.203 in males), the "Stigma" domain (- 6.185 points vs - 4.913 points in males) and the "Bodily discomfort" domain (- 5.196 points vs 1.099 points in males), while were higher in males in the "Mobility" score (- 6.523 points vs - 4.961 points in females) and the "Communication" score (- 3.863 points vs - 1.564 points in females). Safinamide was shown to improve PD symptoms and quality of life in both male and female Chinese patients. Possible differences in the response between genders need to be further studied in larger and different ethnic populations.

摘要

帕金森病(PD)的发病率和患病率预计在未来几十年内会大幅上升。性别相关差异尚未得到广泛认识,特别是在对多巴胺能药物的反应方面。本研究旨在分析中国 XINDI 试验中,与安慰剂相比,司来吉兰对帕金森病患者的临床疗效是否存在性别差异。

XINDI 研究是一项 III 期、随机、双盲、安慰剂对照、多中心试验。患者接受司来吉兰或安慰剂添加治疗,为期 16 周。主要疗效终点为平均每日总关闭时间的变化。次要疗效终点包括总每日开启时间、无/非麻烦性异动症的开启时间、统一帕金森病评定量表和帕金森病问卷-39 项。进行了一项事后分析,以描述两性患者在运动症状、运动波动和生活质量方面司来吉兰的疗效。305 名入组患者中,128 名(42%)为女性,177 名(58%)为男性。我们对 XINDI 研究的额外分析表明,与安慰剂相比,司来吉兰可改善两性患者的运动症状、运动波动和生活质量,反应存在一些差异,但未达到统计学意义,可能是由于样本量限制和研究的事后设计所致。与男性相比,女性在第 16 周时的总每日关闭时间(-1.149 小时 vs. 男性-0.764 小时)、总每日开启时间(1.283 小时 vs. 男性 0.441 小时)、UPDRS 总分(-8.300 分 vs. 男性-5.253 分)和 UPDRS 第二部分评分(-2.574 分 vs. 男性-1.016 分)的变化幅度均高出 50%。与男性相比,女性在第 16 周时的“日常生活活动”评分(-6.965 分 vs. 男性-5.772 分)、“情绪健康”评分(-6.243 分 vs. 男性-4.203 分)、“耻辱感”评分(-6.185 分 vs. 男性-4.913 分)和“身体不适”评分(-5.196 分 vs. 男性 1.099 分)的变化幅度均更高,而男性在“移动性”评分(-6.523 分 vs. 女性-4.961 分)和“沟通”评分(-3.863 分 vs. 女性-1.564 分)的变化幅度更高。司来吉兰可改善中国男性和女性帕金森病患者的 PD 症状和生活质量。两性之间的反应差异需要在更大的不同种族人群中进一步研究。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/f848/10667246/9b2fa1c09e78/41598_2023_48067_Fig1_HTML.jpg

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