Patient-driven N-of-1 in Parkinson's Disease. Lessons Learned from a Placebo-controlled Study of the Effect of Nicotine on Dyskinesia.

ABSTRACT

BACKGROUND: New insights and knowledge in biomedical science often come from observation and experimentation. Methods traditionally used include self-experimentation, case reports, randomised controlled trials, and N-of-1 studies. Technological advances have lead to an increasing number of individuals and patients engaging in self-tracking. We use the term patient-driven N-of-1 for self-tracking performed with the explicit intention to disseminate the results by academic publishing. OBJECTIVES: The aim of the study was to 1) explore the potential role for patient-driven N-of-1 studies as a tool for improving self-management in Parkinson's disease (PD) using the example of managing levodopa-induced dyskinesia (LID) with nicotine, and 2) based on this example; identify some specific challenges of patient-driven N-of-1 studies. METHODS: We used a placebo controlled patient-driven N-of-1 study with nicotine administered via e-cigarette to treat LID. The first author initiated and conducted the experiment on herself and noted her observations. The evaluations of the potential of N-of-1 for improving self-management of PD as well as the effects of nicotine on dyskinesia were based on the perception of the subject. During the planning and undertaking of the experiment, notes were made to identify challenges specific to patient-driven N-of-1 studies. RESULTS: The subject was able to distinguish a decrease of her LID from nicotine but no effect from placebo. The main challenges of patient-driven N-of-1 studies were identified to be associated with planning of the study, recruiting a suitable research team, making sure the data collection is optimal, analysis of data, and publication of results. CONCLUSIONS: Our study indicates that nicotine administered via e-cigarette may have an effect on levodopa-induced dyskinesia in individual patients with PD. The main contribution is however highlighting the work done by patients on a daily basis for understanding their conditions and conducting self-tracking experiments. More work is needed to further develop methods around patient-driven N-of-1 studies for PD.