Best-worst scaling preferences among patients with well-controlled epilepsy: Pilot results

March Best-worst scaling preferences among patients with well-controlled epilepsy: Pilot results Samuel W. TermanID 0 2 3 He´ lène E. Aschmann 1 2 3 David W. Hutton 2 3 James F. Burke 2 3 0 Department of Neurology, University of Michigan , Ann Arbor, Michigan , United States of America 1 Department of Epidemiology and Biostatistics, University of California, San Francisco , San Francisco , California, United States of America, 3 Epidemiology Biostatistics and Prevention Institute, University of Zurich , Zurich , Switzerland , 4 Department of Health Management and Policy, School of Public Health, University of Michigan , Ann Arbor , Michigan, United States of America, 5 Department of Neurology, the Ohio State University , Columbus, Ohio , United States of America 2 Editor: Francesco Deleo, Foundation IRCCS Carlo Besta Neurological Institute: Fondazione IRCCS Istituto Neurologico Carlo Besta , ITALY 3 Data Availability Statement: The University of Michigan's Data Office for Clinical and Translational Research (https://research.medicine.umich.edu/ our-units/data-office-clinical-translational-research) requires a data use agreement be completed before sharing any patient data, even if deidentified. Should investigators wish to request the dataset, they may contact this study's , USA Epilepsy is a common, serious condition. Fortunately, seizure risk decreases with increasing seizure-free time on antiseizure medications (ASMs). Eventually, patients may consider whether to stop ASMs, which requires weighing treatment benefit versus burden. We developed a questionnaire to quantify patient preferences relevant to ASM decision-making. Respondents rated how concerning they would finding relevant items (e.g., seizure risks, side effects, cost) on a Visual Analogue Scale (VAS, 0-100) and then repeatedly chose the most and least concerning item from subsets (best-worst scaling, BWS). We pretested with neurologists, then recruited adults with epilepsy who were seizure-free at least one year. Primary outcomes were recruitment rate, and qualitative and Likert-based feedback. Secondary outcomes included VAS ratings and best-minus-worst scores. Thirty-one of 60 (52%) contacted patients completed the study. Most patients felt VAS questions were clear (28; 90%), easy to use (27; 87%), and assessed preferences well (25; 83%). Corresponding results for BWS questions were 27 (87%), 29 (97%), and 23 (77%). Physicians suggested adding a 'warmup' question showing a completed example and simplifying terminology. Patients suggested ways to clarify instructions. Cost, inconvenience of taking medication, and laboratory monitoring were the least concerning items. Cognitive side effects and a 50% seizure risk in the next year were the most concerning items. Twelve (39%) of patients made at least one 'inconsistent choice' for example ranking a higher seizure risk as lower concern compared with a lower seizure risk, though 'inconsistent choices' represented only 3% of all question blocks. Our recruitment rate was favorable, most patients agreed the survey was clear, and we describe areas for improvement. 'Inconsistent' responses may lead us to collapse seizure probability items into a single 'seizure' category. Evidence regarding how patients weigh benefits and harms may inform care and guideline development. - OPEN ACCESS Funding: SWT: Susan S Spencer Clinical Research Training Scholarship (https://www.aan.com/ research/susan-s-spencer-md-clinical-trainingscholarship) and the Michigan Institute for Clinical and Health Research J Award UL1TR002240 (https://michr.umich.edu/). HEA: Early Postdoc. Mobility Fellowship by the Swiss National Science Foundation (https://www.snf.ch/en/ XIZpfY3iVS5KRRoD/funding/careers/postdocmobility). JFB: National Institute of Minority Health and Health Disparities R01 MD008879 (https:// www.nimhd.nih.gov/). The funders had no role in study design, data collection and analysis, decision to publish, or preparation of the manuscript. Introduction Fifty million people have epilepsy [ 1 ]. Fortunately, antiseizure medications (ASMs) render two-thirds of patients with epilepsy seizure-free [ 2 ] and may reduce mortality by preventing seizures [ 3 ]. However, side effects are common (ranging from 10–40%, even as high as 60– 90%) [ 4, 5 ], and explain up to one-quarter of variance in quality of life after seizure remission [6]. ASMs also may exert drug-drug interactions, are costly [ 7 ], and require monitoring. Thus, clinicians and patients must balance the pros and cons of treatment [ 8 ]. Particularly for patients who become seizure-free on ASMs, clinical guidelines suggest that seizure risk may eventually drop low enough to consider ASM discontinuation rather than lifelong treatment [ 9 ]. This recommendation is supported by an average post-discontinuation relapse risk (30– 40%) [ 10, 11 ] falling below the usual threshold to start an ASM of 60% [12]. Existing literature poorly informs how to weigh disparate considerations. Qualitative work found seizure risk, side effects, and driving restrictions to be among the most important factors relevant to ASM discontinuation [ 13 ]. Surveys have tabulated the most important factors relevant to ASM discontinuation such as driving, seizures, and side effects or fear of long-term negative ASM consequences [ 14, 15 ]. However, neither qualitative interviews nor tabulations inform the relative importance of any factor, which would be useful to inform guidelines and policy-making [16]. Work correlating factors such as seizures or side effects with global quality of life scales has been performed [ 17 ], but such work does not directly inform how patients weigh tradeoffs inherent to the pros and cons of treatment decisions. However, existing preference studies have omitted numerous important items (e.g., driving restrictions related to seizures, lab monitoring), lumped disparate items together (e.g., fatigue and moodiness as a single category [ 18 ]), treated seizure risk in terms of relative rather than absolute seizure risks, did not study physician-confirmed epilepsy, and did not study patients with well-controlled epilepsy. Thus, major gaps remain which could be filled by developing a new survey instrument. Particularly, discrete choice experiments and more recently best-worst scaling (BWS) are frequently used to quantitatively measure patient preferences [ 19, 20 ]. In BWS, health outcomes or treatment options are ranked by choosing the best (or least concerning) and the worst (or most concerning) item repeatedly among subsets [21]. A core strength is that respondents are best at identifying preference extremes from a limited set of options, and both the most and least important factors inform decision processes. BWS has been used across medical conditions to clarify patient values [ 19, 22–26 ]. Here, we describe the development and pilot testing of our novel instrument eliciting patient preference (...truncated)