Understanding the Patient Experience of Advanced Parkinson's Disease: Qualitative Research with Patients and Expert Clinicians to Identify Symptoms and Associated Health-Related Quality of Life Impacts.

IF 3.9 3区医学 Q1 CLINICAL NEUROLOGY

Neurology and Therapy Pub Date : 2025-05-13 DOI:10.1007/s40120-025-00747-5

Nicola Williamson, Thomas Morel, Helena Bradley, Aoife Lydon, Molly Clifford, Patrick Cronin, Milton Biagioni

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引用次数: 0

Abstract

Introduction: Parkinson's disease (PD) is a progressive neurodegenerative condition, characterized by motor symptoms (MS) and non-motor symptoms (NMS). Currently, there is no global definition of advanced PD, but those in advanced stages experience diverse and severe symptomatology, and significant impacts to their health-related quality of life (HRQoL). This qualitative interview study aimed to explore the experience of symptoms and HRQoL impacts of advanced PD from the patient and clinician perspective and identify key concepts for assessment in PD clinical trials.

Methods: A targeted review of literature and qualitative concept elicitation interviews conducted with people with advanced PD (N = 20) and expert clinicians (N = 3) in the US aimed to identify symptoms of advanced PD and their impact on HRQoL. People with advanced PD (PwP) were defined as those diagnosed ≥ 5 years, experiencing ON/OFF motor fluctuations despite levodopa therapy, and classified as modified Hoehn and Yahr stages 2-4.

Results: In total, 65 symptoms (48 NMS, 17 MS) were identified across the reviewed literature and qualitative interviews. The most frequently reported MS included tremor, rigidity, balance issues, and slowness of movement. Several NMS were also frequently reported by participants, including fatigue, cognitive dysfunction (e.g., difficulty thinking), neuropsychiatric symptoms (e.g., apathy), pain, sleep problems, urinary dysfunction, autonomic dysfunction, excessive daytime sleepiness, gastrointestinal dysfunction, and sensory dysfunction. Participants reported these to impact HRQoL, including daily activities, emotional functioning, physical functioning, social functioning, work/study, and financial well-being.

Conclusion: The study provides in-depth insights into the symptoms and HRQoL impacts experienced by those with advanced PD. A comprehensive conceptual model of the patient experience of advanced PD was developed to support the identification of relevant concepts for assessment in PD clinical trials.

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了解晚期帕金森病患者的经历：与患者和专家临床医生定性研究，以确定症状和相关的健康相关的生活质量影响。

简介：帕金森病（PD）是一种进行性神经退行性疾病，以运动症状（MS）和非运动症状（NMS）为特征。目前，对晚期PD没有统一的定义，但晚期PD的症状多样且严重，其健康相关生活质量（HRQoL）受到显著影响。本定性访谈研究旨在从患者和临床医生的角度探讨晚期PD的症状体验和HRQoL影响，并确定PD临床试验中评估的关键概念。方法：对美国晚期PD患者（N = 20）和专家临床医生（N = 3）进行有针对性的文献回顾和定性概念启发访谈，旨在确定晚期PD的症状及其对HRQoL的影响。晚期PD （PwP）患者被定义为诊断≥5年，尽管左旋多巴治疗仍出现ON/OFF运动波动，并被归类为改良Hoehn和Yahr期2-4。结果：通过回顾文献和定性访谈，共确定了65种症状（48种NMS， 17种MS）。最常见的多发性硬化症包括震颤、僵硬、平衡问题和行动迟缓。参与者也经常报告几种NMS，包括疲劳、认知功能障碍（例如，思维困难）、神经精神症状（例如，冷漠）、疼痛、睡眠问题、泌尿功能障碍、自主神经功能障碍、白天过度嗜睡、胃肠功能障碍和感觉功能障碍。参与者报告了影响HRQoL的因素，包括日常活动、情绪功能、身体功能、社会功能、工作/学习和财务状况。结论：该研究为晚期PD患者的症状和HRQoL影响提供了深入的见解。开发了晚期PD患者体验的综合概念模型，以支持PD临床试验中评估的相关概念的识别。

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来源期刊

Neurology and Therapy CLINICAL NEUROLOGY-

CiteScore

5.40

自引率

8.10%

发文量

103

审稿时长

6 weeks

期刊介绍： Aims and Scope Neurology and Therapy aims to provide reliable and inclusive, rapid publication for all therapy related research for neurological indications, supporting the timely dissemination of research with a global reach, to help advance scientific discovery and support clinical practice. Neurology and Therapy is an international, open access, peer reviewed, rapid publication journal dedicated to the publication of high-quality clinical (all phases), observational, real-world and health outcomes research around the discovery, development, and use of neurological and psychiatric therapies, (also covering surgery and devices). Studies relating to diagnosis, pharmacoeconomics, public health, quality of life, and patient care, management, and education are also welcomed. The journal is of interest to a broad audience of healthcare professionals and publishes original research, reviews, case reports, trial designs, communications and letters. The journal is read by a global audience and receives submissions from all over the world. Neurology and Therapy will consider all scientifically sound research be it positive, confirmatory or negative data. Submissions are welcomed whether they relate to an international and/or a country-specific audience, something that is crucially important when researchers are trying to target more specific patient populations. This inclusive approach allows the journal to assist in the dissemination of all scientifically and ethically sound research. Rapid Publication The journal’s rapid publication timelines aim for a peer review decision within 2 weeks of submission. If an article is accepted, it will be published online 3-4 weeks from acceptance. These rapid timelines are achieved through the combination of a dedicated in-house editorial team, who closely manage article workflow, and an extensive Editorial and Advisory Board who assist with rapid peer review. This allows the journal to support the rapid dissemination of research, whilst still providing robust peer review. Combined with the journal’s open access model, this allows for the rapid and efficient communication of the latest research and reviews to support scientific discovery and clinical practice. Open Access All articles published by Neurology and Therapy are open access. Personal Service The journal’s dedicated in-house editorial team offer a personal “concierge service” meaning that authors will always have a personal point of contact able to update them on the status of their manuscript. The editorial team check all manuscripts to ensure that articles conform to the most recent COPE and ICMJE publishing guidelines. This supports the publication of ethically sound and transparent research. We also encourage pre-submission enquiries and are always happy to provide a confidential assessment of manuscripts. Digital Features and Plain Language Summaries Neurology and Therapy offers a range of additional features designed to increase the visibility, readership and educational value of the journal’s content. Each article is accompanied by key summary points, giving a time-efficient overview of the content to a wide readership. Articles may be accompanied by plain language summaries to assist readers who have some knowledge of, but not in-depth expertise in, the area to understand the scientific content and overall implications of the article. The journal also provides the option to include various types of digital features including animated abstracts, video abstracts, slide decks, audio slides, instructional videos, infographics, podcasts and animations. All additional features are peer reviewed to the same high standard as the article itself. If you consider that your paper would benefit from the inclusion of a digital feature, please let us know. 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Peer Review Process Upon submission, manuscripts are assessed by the editorial team to ensure they fit within the aims and scope of the journal and are also checked for plagiarism. All suitable submissions are then subject to a comprehensive single-blind peer review. Reviewers are selected based on their relevant expertise and publication history in the subject area. The journal has an extensive pool of editorial and advisory board members who have been selected to assist with peer review based on the afore-mentioned criteria. At least two extensive reviews are required to make the editorial decision, with the exception of some article types such as Commentaries, Editorials and Letters which are generally reviewed by one member of the Editorial Board. Where reviews conflict, an Editorial Board Member will be contacted for further advice and a presiding decision. Manuscripts are then either accepted, rejected or authors are required to make major or minor revisions (both reviewer comments and editorial comments may need to be addressed. Once a revised manuscript is re-submitted, it is assessed along with the responses to reviewer comments and if it has been adequately revised, it will be accepted for publication. Accepted manuscripts are then copyedited and typeset by the production team before online publication. Appeals against decisions following peer review are considered on a case-by-case basis and should be sent to the journal editor, and authors are welcome to make rebuttals against individual reviewer comments, if appropriate. Preprints We encourage posting of preprints of primary research manuscripts on preprint servers, authors'' or institutional websites, and open communications between researchers whether on community preprint servers or preprint commenting platforms. 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