Morel Thomas, Cleanthous Sophie, Andrejack John, Barker Roger A, Blavat Geraldine, Brooks William, Burns Paul, Cano Stefan, Gallagher Casey, Gosden Lesley, Siu Carroll, Slagle Ashley F, Trenam Kate, Boroojerdi Babak, Ratcliffe Natasha, Schroeder Karlin
UCB Pharma SRL, Brussels, Belgium.
Modus Outcomes, a Division of Thread, London, UK.
Neurol Ther. 2022 Sep;11(3):1319-1340. doi: 10.1007/s40120-022-00375-3. Epub 2022 Jul 1.
Qualitative research on patient experiences in early-stage Parkinson's disease (PD) is limited. It is increasingly acknowledged that clinical outcome assessments used in trials do not fully capture the range of symptoms/impacts that are meaningful to people with early-stage PD. We aimed to conceptualize the patient experience in early-stage PD and identify, from the patient perspective, those cardinal symptoms/impacts which might be more useful to measure in clinical trials.
In a mixed-methods analysis, 50 people with early-stage PD and nine relatives were interviewed. Study design and results interpretation were led by a multidisciplinary group of patient, clinical, regulatory, and outcome measurements experts, and patient organization representatives. Identification of the cardinal concepts was informed by the relative frequency of reported concepts combined with insights from patient experts and movement disorder specialists.
A conceptual model of the patient experience of early-stage PD was developed. Concept elicitation generated 145 unique concepts mapped across motor and non-motor symptoms, function, and impacts. Bradykinesia/slowness (notably in the form of "functional slowness"), tremor, rigidity/stiffness, mobility (particularly fine motor dexterity and subtle gait abnormalities), fatigue, depression, sleep/dreams, and pain were identified as cardinal in early-stage PD. "Functional slowness" (related to discrete tasks involving the upper limbs, complex mobility tasks, and general activities) was deemed to be more relevant than "difficulty" to patients with early-stage PD, who report being slower at completing tasks rather than encountering significant impairment with task completion.
Patient experiences in early-stage PD are complex and wide-ranging, and the currently available patient-reported outcome (PRO) instruments do not evaluate many early-stage PD concepts such as functional slowness, fine motor skills, and subtle gait abnormalities. The development of a new PRO instrument, created in conjunction with people with PD, that fully assesses symptoms and the experience of living with early-stage PD, is required.
关于帕金森病（PD）早期患者体验的定性研究有限。人们越来越认识到，试验中使用的临床结局评估并不能完全涵盖对早期PD患者有意义的症状/影响范围。我们旨在对早期PD患者的体验进行概念化，并从患者角度确定那些在临床试验中可能更有助于测量的主要症状/影响。
在一项混合方法分析中，对50名早期PD患者和9名亲属进行了访谈。研究设计和结果解释由患者、临床、监管和结局测量专家以及患者组织代表组成的多学科小组主导。主要概念的确定基于所报告概念的相对频率，并结合患者专家和运动障碍专家的见解。
建立了早期PD患者体验的概念模型。概念引出产生了145个独特的概念，涵盖运动和非运动症状、功能及影响。运动迟缓/缓慢（特别是“功能性缓慢”形式）、震颤、僵硬/强直、活动能力（尤其是精细运动灵活性和细微步态异常）、疲劳、抑郁、睡眠/梦境和疼痛被确定为早期PD的主要症状。“功能性缓慢”（与涉及上肢的离散任务、复杂活动能力任务和日常活动有关）被认为对早期PD患者比“困难”更相关，这些患者报告在完成任务时速度较慢，而非在任务完成方面遇到重大障碍。
早期PD患者的体验复杂且广泛，目前可用的患者报告结局（PRO）工具并未评估许多早期PD概念，如功能性缓慢、精细运动技能和细微步态异常。需要开发一种与PD患者共同创建的新PRO工具，以全面评估症状和早期PD患者的生活体验。
