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.

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.