Patient-driven N-of-1 in Parkinson’s Disease

Abstract

KeywordsParkinson's disease, N-of-1, levodopa induced dyskinesia, patient-driven, Quantified Self Summary 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 selfmanagement 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.

The Value Equation: Three complementary propositions for reconciling fidelity and adaptation in evidence-based practice implementation

“…Used in this way, the learning healthcare system [92] will provide the most precise version of “what works for whom, when” we can think of: personalized value equations in patient- or provider-driven n = 1 studies [93]. Aggregation of all n = 1 studies will then provide the basis for accumulation of knowledge of “what works for whom, when,” thereby bridging personalized medicine and the ideas for systemizing knowledge about adaptations as outlined in the Adaptome [26].…”

The Importance of Starting With Goals in N-of-1 Studies

“…N-of-1 designs have received attention for their potential to facilitate understanding and management of health conditions that require individualized insights and approaches (1,2). N-of-1 studies come in a variety of designs [e.g., Heyvaert and Onghena (3) and Daskalova et al, (4)], including self-monitoring, in which people track data related to their condition to examine progress toward goals or changes over time [e.g., Mishra et al, (5), Ayobi et al, (6), and Consolvo et al, (7)]; correlational analyses, in which people investigate what factors may affect their symptoms [e.g., behavioral, environmental, medical; (8)]; and self-experiments, in which people determine causality between factors and symptoms [e.g., Karkar et al, (9) and Riggare et al, (10)]. Each of these designs can support a range of goals.…”

E-patients hold key to the future of healthcare

“…Together with other patients, I have published articles about how we can contribute to our health and care when our healthcare systems have not yet responded to the latest research. One article describes an approach to multidisciplinary teams10; another our patient driven study 11…”