Eliciting Exploratory Patient Preference Data: A Case Study in a Rare Disease.

INTRODUCTION: Qualitative and quantitative methods provide different and complementary insights into patients' preferences for treatment. OBJECTIVE: The aim of this study was to use a novel, mixed-methods approach employing qualitative and quantitative approaches to generate preliminary insights into patient preferences for the treatment of a rare disease-generalized myasthenia gravis (gMG). METHODS: We conducted a mixed-methods study to collect exploratory qualitative and quantitative patient preference information and generate informative results within a condensed timeline (about 4 months). Recruitment was facilitated by an international health research firm. Study participants first reviewed a brief document describing six treatment attributes (to facilitate more efficient review of the material during the focus groups) and were then provided a link to complete an online quantitative survey with a single risk threshold task. They then participated in online focus groups, during which they discussed qualitative questions about their experience with gMG treatment and completed up to three quantitative threshold tasks, the first of which repeated the threshold task from the online survey. RESULTS: The study elicited both quantitative data on 18 participants' risk tolerance and qualitative data on their treatment experience, additional treatment attributes of importance, the reasoning behind their preferences, and the trade-offs they were willing to make. Most participants (n = 15) chose the same hypothetical treatment in the first threshold task in the online survey and the focus groups. Focus group discussions provided insights into participants' choices in the threshold tasks, confirmed that all the attributes were relevant, and helped clarify what was important about the attributes. CONCLUSIONS: Patient preference information can be collected using a variety of approaches, both qualitative and quantitative, tailored to fit the research needs of a study. The novel mixed-methods approach employed in this study efficiently captured patient preference data that were informative for exploratory research, internal decision making, and future research.

Real-world healthcare resource utilization related to migraine treatment failure: a panel-based chart review in France, Germany, Italy, and Spain.

Aims: This retrospective chart review examined the six-month migraine-related healthcare resource use (HRU) among European patients who had ≥4 migraine days per month and previously failed at least two prophylactic migraine treatments. Methods: Neurologists, headache specialists, and pain specialists in France, Germany, Italy, and Spain who treated ≥10 patients with migraine in 2017 were recruited (April-June 2018) to extract anonymized patient-level data. Eligible physicians randomly selected charts of up to five adult patients with clinically-confirmed migraine, ≥4 migraine days in the month prior to the index date, and had previously failed at least two prophylactic migraine treatments. Treatment failure was defined as discontinuation due to lack of efficacy and/or tolerability. Demographic and disease characteristics as of the index date, and migraine-related HRU incurred during the 6-month study period, were recorded. Results: A total of 104 physicians contributed 168 charts for patients (63% female). On average, patients were 38 years old and failed 2.3 prophylactic treatments as of the index date. During the study period, 83% of patients had ≥1 outpatient visit for migraine in the physician's office, and 27% went to the ER/A&E. Approximately 5% of patients were hospitalized for migraine, with an average of one hospitalization and an average length of stay of 3 days. Approximately 39% of patients had ≥1 blood test, 22% had ≥1 magnetic resonance imaging, 17% had ≥1 electroencephalogram, and 13% had ≥1 computerized tomography scan. Visits to other healthcare providers were common. Limitations: This study is subject to the limitations of chart review studies, such as errors in data entry. Conclusions: Across four European countries, the HRU burden of migraine among patients who previously failed at least two prophylactic treatments was high, indicating a need for more effective prophylactic treatments to appropriately manage migraine and reduce the HRU burden attributable to this common disorder.

Migraine-related healthcare resource use in the emergency department setting: a panel-based chart review in France, Germany, Italy, and Spain.

Objective: Migraine is a common, disabling condition typically characterized by severe headache, nausea, and/or light and sound sensitivity. This study assessed migraine-related health resource utilization (HRU) occurring in the emergency room/accident & emergency department (ER/A&E) setting among European patients with 4 or more migraine days per month. Methods: Patient-level clinical and HRU data were collected via chart extraction by ER/A&E physicians in France, Germany, Italy, and Spain. Eligible patients had 4 or more migraine days in the month prior to a migraine-related ER/A&E visit and a history of migraine, among other criteria. The index date for each patient was defined as the date of an ER/A&E visit for migraine on or after January 1, 2013. Physician and ER/A&E characteristics, patient and disease characteristics, treatment history, migraine-medication used, and migraine-related HRU (i.e. procedures) during the ER/A&E visit were assessed. Descriptive analyses were conducted in the pooled population, and a sensitivity analysis was performed by country. Results: A total of 467 eligible patient's charts (120 in France, 120 in Germany, 107 in Italy, and 120 in Spain) were provided by 136 physicians (36 in France, 36 in Germany, 28 in Italy, and 36 in Spain). On average, patients spent nearly 8 hours in the ER/A&E. Approximately 82% of patients received a blood test, 62% received an electrocardiography, and 46% received a cranial computerized tomography scan. Despite the majority of patients already using acute or prophylactic treatment upon visiting the ER/A&E, almost all patients were administered or prescribed migraine treatment during the visit. Approximately 21% of patients were admitted to the hospital, and over half of patients were referred to a neurologist or headache specialist. Conclusions: European patients who had four or more migraine days in the month prior to a migraine-related ER/A&E visit had high HRU associated with the visit.

Auditory event-related potentials (P3) and cognitive performance in recreational ecstasy polydrug users: evidence from a 12-month longitudinal study.

RATIONALE: There is important preclinical evidence of the long-lasting neurotoxic and selective effects of ecstasy (MDMA) on serotonin systems in nonhuman primates. In humans, long-term recreational use of ecstasy has been mainly associated with memory impairment. OBJECTIVE: The first aim of our study was to evaluate the cognitive and electrophysiological long-term alterations associated with lifetime ecstasy use within a sample of ecstasy polydrug users along a 1-year follow-up. Our second aim was to explore the relationship between specific cognitive functions and P300 (P3) event-related potentials (ERPs) in ecstasy users. MATERIALS AND METHODS: We conducted auditory P3 latency and amplitude and administered a battery of cognitive tests to three groups of subjects: 14 current ecstasy polydrug users, 13 current cannabis users, and 22 controls free of illicit drugs in two evaluations during 1 year. RESULTS: We found significant differences between ecstasy users and controls on cognitive measures of word fluency, processing speed, and memory recognition after 1-year follow-up. We found no significant differences between ecstasy and cannabis users or cannabis users and controls on cognitive tests. Lifetime ecstasy use was associated with poorer memory recognition. No group differences were shown on P3 latency or amplitude. Significant correlations emerged between P3 latency and cannabis lifetime use (higher cannabis use was related to faster latency, showing a paradoxical effect) but not with ecstasy exposure. CONCLUSIONS: Our findings provide evidence of mild long-term cognitive deficits among ecstasy polydrug users. Both ecstasy use and the dynamic interaction between ecstasy and cannabis effects may account for these deficits. No significant P3 alterations were found in ecstasy users.

Simultaneous magnetoencephalography and intracranial EEG registration: technical and clinical aspects.

The purpose of this study was to describe the methodology necessary for simultaneous recording of intracranial EEG (ICEEG) and magnetoencephalography (MEG) and to assess the sensitivity of whole-head MEG versus depth electrode EEG in the detection and localization of epileptic spikes. Interictal MEG and depth electrode activities from the temporal mesial and occipital lobes were simultaneously recorded from four candidates for epilepsy surgery. Implanted depth electrodes identified neocortical and mesial structures of ictal onset. Interictal spikes detected by these same depth electrodes were compared with simultaneous MEG events. MEG detections of ICEEG spikes, ICEEG versus MEG spike amplitudes, number of ICEEG contacts involved in the spike, and anatomic locations of MEG equivalent current dipoles were analyzed. MEG detected and localized 95% of the neocortical spikes, but only 25% to 60% of spikes from mesial structures. Mesial temporal spikes resulted in lower MEG spike amplitudes, when compared with neocortical spikes. Equivalent current dipoles of MEG spikes localized to the ictal onset zones in all four patients. MEG can detect and localize interictal epileptiform spikes that are recorded from depth electrodes in both neocortical and mesial structures, despite the lesser amplitude of spikes of mesial origin.