PDF(Pubmed)
Abstract：
UNASSIGNED: More than half of patients with Parkinson\'s disease will experience psychosis symptoms in the form of hallucinations or delusions at some point over the course of their disease. These symptoms can significantly impact patients\' health-related quality of life, cognitive abilities, and activities of daily living (ADLs) and function. Clinical assessment of how psychosis impacts these measures is crucial; however, few studies have assessed this sufficiently, in part due to a lack of appropriate scales for comprehensively assessing function.
UNASSIGNED: The objective was to assess how symptoms of Parkinson\'s disease psychosis (PDP) impact ADLs and function, cognitive function, and health-related quality of life.
UNASSIGNED: To address this unmet need, we utilized a modified version of the Functional Status Questionnaire (mFSQ) to measure the impact of psychosis on ADLs and function in patients with PDP treated with pimavanserin, a US Food and Drug Administration-approved medication to treat hallucinations and delusions associated with PDP.
UNASSIGNED: Eligible patients entered a 16-week, single-arm, open-label study of oral pimavanserin (34 mg) taken once daily. The primary endpoint was change from baseline to Week 16 on the mFSQ. Secondary endpoints included the Movement Disorders Society-modified Unified Parkinson\'s Disease Rating Scale (MDS-UPDRS) I and II; Schwab and England ADL; Clinical Global Impression-Severity of Illness (CGI-S), Clinical Global Impression-Improvement (CGI-I), and Patient Global Impression-Improvement (PGI-I), and were also measured as change from baseline to Week 16 using mixed-effects model for repeated measures (MMRM) and least-squares mean (LSM).
UNASSIGNED: Our results in a proof-of-concept, 16-week, open-label clinical study in 29 patients demonstrated that an improvement in psychosis symptoms following treatment with pimavanserin was associated with improvements in multiple measures of ADLs and function. Notably, a significant improvement was found on the primary endpoint, change from baseline to Week 16 in mFSQ score [LSM [SE] 14.0 [2.50], n = 17; 95% CI (8.8, 19.3); p < 0.0001].
UNASSIGNED: These findings highlight the potential for improvement in function with improvement of psychosis symptoms in patients with PDP and suggest that the mFSQ may be a measurement tool to evaluate the level of improvement in function.
UNASSIGNED: ClinicalTrials.gov Identifier: NCT04292223.
UNASSIGNED: The objective was to assess how symptoms of Parkinson\'s disease psychosis (PDP) impact ADLs and function, cognitive function, and health-related quality of life.
UNASSIGNED: To address this unmet need, we utilized a modified version of the Functional Status Questionnaire (mFSQ) to measure the impact of psychosis on ADLs and function in patients with PDP treated with pimavanserin, a US Food and Drug Administration-approved medication to treat hallucinations and delusions associated with PDP.
UNASSIGNED: Eligible patients entered a 16-week, single-arm, open-label study of oral pimavanserin (34 mg) taken once daily. The primary endpoint was change from baseline to Week 16 on the mFSQ. Secondary endpoints included the Movement Disorders Society-modified Unified Parkinson\'s Disease Rating Scale (MDS-UPDRS) I and II; Schwab and England ADL; Clinical Global Impression-Severity of Illness (CGI-S), Clinical Global Impression-Improvement (CGI-I), and Patient Global Impression-Improvement (PGI-I), and were also measured as change from baseline to Week 16 using mixed-effects model for repeated measures (MMRM) and least-squares mean (LSM).
UNASSIGNED: Our results in a proof-of-concept, 16-week, open-label clinical study in 29 patients demonstrated that an improvement in psychosis symptoms following treatment with pimavanserin was associated with improvements in multiple measures of ADLs and function. Notably, a significant improvement was found on the primary endpoint, change from baseline to Week 16 in mFSQ score [LSM [SE] 14.0 [2.50], n = 17; 95% CI (8.8, 19.3); p < 0.0001].
UNASSIGNED: These findings highlight the potential for improvement in function with improvement of psychosis symptoms in patients with PDP and suggest that the mFSQ may be a measurement tool to evaluate the level of improvement in function.
UNASSIGNED: ClinicalTrials.gov Identifier: NCT04292223.
摘要：
■超过一半的帕金森氏病患者在其疾病过程中的某个时刻会出现幻觉或妄想形式的精神病症状。这些症状会显著影响患者的健康相关生活质量，认知能力,以及日常生活活动(ADLs)和功能。对精神病如何影响这些措施的临床评估至关重要；然而，很少有研究对此进行了充分的评估，部分原因是缺乏全面评估功能的适当尺度。
■目的是评估帕金森病精神病（PDP）的症状如何影响ADL和功能，认知功能,和健康相关的生活质量。
■为了解决这个未满足的需求，我们使用功能状态问卷（mFSQ）的修改版本来测量精神病对使用匹马色林治疗的PDP患者的ADL和功能的影响，美国食品和药物管理局批准的治疗与PDP相关的幻觉和妄想的药物。
■符合条件的患者进入16周，单臂,每日一次口服匹马舍林（34毫克）的开放标签研究。主要终点是在mFSQ上从基线到第16周的变化。次要终点包括运动障碍协会修改的帕金森病统一评定量表（MDS-UPDRS）I和II；施瓦布和英格兰ADL；临床总体印象-疾病严重程度（CGI-S），临床总体印象改善（CGI-I），和患者总体印象改善(PGI-I)，并且还使用重复测量的混合效应模型（MMRM）和最小二乘均值（LSM）测量从基线到第16周的变化。
■我们的结果是概念验证，16周,29例患者的开放标签临床研究表明,匹马色林治疗后精神病症状的改善与ADL和功能的多项指标的改善相关.值得注意的是,发现主要终点有显著改善，mFSQ分数从基线到第16周的变化[LSM[SE]14.0[2.50]，n=17；95%CI（8.8，19.3）；p<0.0001]。
■这些发现强调了PDP患者随着精神病症状的改善而改善功能的潜力，并表明mFSQ可能是评估功能改善水平的测量工具。
■ClinicalTrials.gov标识符：NCT04292223。
■目的是评估帕金森病精神病（PDP）的症状如何影响ADL和功能，认知功能,和健康相关的生活质量。
■为了解决这个未满足的需求，我们使用功能状态问卷（mFSQ）的修改版本来测量精神病对使用匹马色林治疗的PDP患者的ADL和功能的影响，美国食品和药物管理局批准的治疗与PDP相关的幻觉和妄想的药物。
■符合条件的患者进入16周，单臂,每日一次口服匹马舍林（34毫克）的开放标签研究。主要终点是在mFSQ上从基线到第16周的变化。次要终点包括运动障碍协会修改的帕金森病统一评定量表（MDS-UPDRS）I和II；施瓦布和英格兰ADL；临床总体印象-疾病严重程度（CGI-S），临床总体印象改善（CGI-I），和患者总体印象改善(PGI-I)，并且还使用重复测量的混合效应模型（MMRM）和最小二乘均值（LSM）测量从基线到第16周的变化。
■我们的结果是概念验证，16周,29例患者的开放标签临床研究表明,匹马色林治疗后精神病症状的改善与ADL和功能的多项指标的改善相关.值得注意的是,发现主要终点有显著改善，mFSQ分数从基线到第16周的变化[LSM[SE]14.0[2.50]，n=17；95%CI（8.8，19.3）；p<0.0001]。
■这些发现强调了PDP患者随着精神病症状的改善而改善功能的潜力，并表明mFSQ可能是评估功能改善水平的测量工具。
■ClinicalTrials.gov标识符：NCT04292223。
