Measurement of patient-centered outcomes in Parkinson’s disease: What do patients really want from their treatment?☆
Introduction
While motor impairment may be the hallmark of Parkinson’s disease, many other behavioral domains are drastically compromised. The 2002 Global Parkinson’s Disease Survey revealed that patients’ disease severity accounted for only 17.3% of their stated quality of life while psychosocial factors accounted for approximately 60% [1]. Research examining non-motor aspects of PD is comparatively limited, but has steadily gained greater clinical attention [2], [3]. However, treatment success in clinical practice is commonly determined by comparing pre- and post-treatment scores on standardized motor examinations or staging systems. Notably, patients’ goals following treatment are often different from their physicians’, in both magnitude of change and in functional outcome [4], [5], [6]. In terms of overall care, Fargel et al. [4] demonstrated that PD patients were significantly less satisfied with their treatment when compared to patients with other chronic illnesses, rating their care on average as 6.6 on a 10-point scale. Furthermore, PD patients are requesting a greater personal role in their care, which has subsequently been associated with increased self-reported patient satisfaction and treatment adherence, even in the absence of apparent changes in motor scores or activities of daily living [7].
In order to convert patients’ care perspectives into a focal point of treatment outcome research, certain considerations must be made in statistical methodology. Robinson and colleagues [8], [9] have argued that interpreting results of central tendency analyses in clinical studies has various caveats, such as the possibility of significance based on sample size or of the attribution of improvement to a few participants. These investigators have suggested a patient-defined perspective of treatment success rather than relying on metrics established by healthcare providers or third party payers. Additionally, PD patients’ expectations for treatment outcome are important to consider when assessing clinical outcome, as studies have suggested that expectations for motor symptom relief mediate placebo and nocebo responses following deep brain stimulation (DBS) manipulations [10], [11]. Furthermore, understanding the breadth of behavioral domains impacted by PD may facilitate discussion between patients and their providers in terms of treatment priorities.
The aim of our current study was to implement the Patient-Centered Outcomes Questionnaire (PCOQ-PD), originally developed by Robinson and colleagues [8], and modified for this study to better understand treatment success, expectations, and importance in both motor and non-motor domains from the PD patient’s perspective.
Section snippets
Patients and methods
Data were collected from 181 Parkinson’s disease patients receiving treatment from the University of Florida Movement Disorders Clinic during their routine care visit. Patients specifically referred to the clinic for DBS candidacy were not included in the study; thus, our sample consisted of a wide range of patients with varying levels of treatment experience. The study protocol was approved by the Institutional Review Board, and each participant provided informed consent. Data were carefully
Results
There were 102 men and 46 women in the final sample, reflecting known sex differences in PD rates. Demographic and disease characteristics are described in Table 1. Data for 19 subjects (10.5%) were unusable due to invalid questionnaire completion, and 14 subjects (7.7%) were excluded due to non-idiopathic PD diagnoses, leaving a total sample of 148 patients. ANOVAs or χ2 tests comparing the final sample with those who were excluded indicated no significant differences in demographic or disease
Discussion
The present study is a preliminary attempt to describe how PD patients define their treatment expectations and criteria for successful outcomes across several functional domains. Originally conceived for chronic pain patients, the PCOQ was modified to address common concerns for PD patients, including both motor and non-motor symptoms. While these issues are important to patients and their clinicians [4], multiple areas of concern have been minimally addressed from the patients’ perspective. We
Conclusions
Parkinson’s disease patients are a heterogeneous group, particularly when it comes to what they expect and desire in their treatment. Because treating PD is a complicated and ever-changing process, it is important to examine the multiple domains impacted by the disease from the patients’ perspective. In highlighting patients’ most debilitating symptoms and their expectations for care, we aim to encourage a dialogue between patients and providers, thereby creating an environment of greater
Acknowledgements
This project was funded by NIH Grant R01 NS047119 and with support from the National Parkinson Foundation Center of Excellence.
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The review of this paper was entirely handled by an Associate Editor, Robert Rodnitzky.