A Study to Collect Information of People With Narcolepsy in Spain

Sociodemographic variables categorized by age, gender (male/female), ethnicity, educational level ongoing or completed level of education (without studies, primary studies, secondary studies, university studies, other superior studies, not available); occupational status and occupation (student, employed, self-employed, employed but on sick leave due to the study disease, permanent incapacity to work due to the study disease, permanent incapacity to work due to other reasons, unemployed, retired, domestic work, other); civil status (married/with partner, divorced/separated, unmarried, widow/er, unknown); living conditions (alone, wife/husband and/or sons, family caregivers, other caregivers, unknown). Clinical variables categorized by smoking status, alcohol intake, exercise status and family history of narcolepsy in first-degree and second-degree relatives].

BMI = weight(kg)/height(m)^2

Specialist who diagnosed narcolepsy were categorized as general practitioners, neurologist, neuropediatrician, neurophysiologist, pneumologist, somnologist, somnologist-unit and specialist (Not specified). Percentage of participants are rounded-off to the nearest decimal point.

Different procedures or tests for diagnosis included clinical history, Clinical Assessment: Epworth sleepiness scale (ESS) [ESS consists of 8 items, on a 4-point scale (0-3) ranging from "0=No chance of dozing" to "3=High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are within the normal range], Neurological Assessment, Multiple sleep latency test (MSLT), Apnea-hypopnea index (AHI), Other procedures, Human leucocyte antigen (HLA) typing, Hypocretin-1 cerebrospinal fluid (CSF) or Orexin. Percentages are rounded-off to the nearest decimal point.

Time from first symptom in participant to diagnosis is the time when the first symptom of narcolepsy was identified until its diagnosis during retrospective visit. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.

Time from diagnosis to first treatment is the time required to diagnose the first symptom of narcolepsy until the first treatment is provided after diagnosis retrospectively. As the first symptom and the diagnosis at retrospective visit could be any time in the past up to end of enrolment in this study. Thus, as pre-planned data for this outcome measure may exceed the duration of the study.

Pharmacological Treatments were categorized as 1st treatment after diagnosis (monotherapy and combinations), treatments used from diagnosis to study visit and treatments at study visit (monotherapy and combinations). Percentages are rounded off to the nearest decimal point.

Behavioral measure of working memory, attention, and impulse/inhibitory control - The Sustained Attention to Response Task (SART) is a computer-based go/no-go task that requires participants to withhold behavioral response to a single, infrequent target (often the digit 3) presented amongst a background of frequent non-targets (0-2, 4-9). After each block, two probe questions are presented in succession. The first asks, "Where was your attention focused during this block of trials?" Participants respond on a 6-point Likert scale, where 1 represents, "on task," and 6, "off task." A second question asks, "How aware were you of where your attention was during this block of trials?" Participants respond on a similar scale, where 1 represents, "aware," and 6, "unaware."

The MWT is a validated objective measure that evaluates a person's ability to remain awake under soporific conditions for a defined period of time. This tendency to fall asleep is measured via electroencephalography-derived sleep latency. Sleep onset is defined as the first epoch of greater than 15 seconds of cumulative sleep in a 30-second epoch. During each MWT session (1 session = 40 minutes), participants will be instructed to sit quietly and remain awake for as long as possible. If no sleep has been observed according to these rules, then the latency is defined as 40 minutes. MWT sleep latency ranges from 0 to 40 minutes, with higher scores indicating greater ability to stay awake.

ESS is a subjective, self-administered questionnaire to assess daytime sleepiness and consists of 8 items, on a 4-point scale (0-3) ranging from "No chance of dozing" to "High chance of dozing" while engaged in eight different activities. The score can range from 0 to 24. Higher scores indicate stronger subjective daytime sleepiness, and scores below 10 are considered to be within the normal range.

Non-pharmacological treatment approach was categorized as take short naps, maintain a regular sleep schedule, avoid caffeine or alcohol before bedtime, avoid smoking, especially at night, exercise daily, avoid large, heavy meals right before bedtime, and other. Percentage of participants are rounded-off to the nearest decimal point.

Direct healthcare resource utilized per year were categorized as treatments for narcolepsy, routine monitoring visits, tests, emergency visits, hospitalizations and complications associated to narcolepsy. Percentage of participants are rounded-off to the nearest decimal point.

Duration of hospitalization is the total number of days at which participants were admitted in hospital.

The WPAI is a questionnaire that assesses the impact of an intervention on work productivity, evaluating 4 areas including absenteeism [Q2/(Q2+Q4)], presenteeism [Q5/10], work productivity loss {Q2/(Q2+Q4)+[(1-(Q2/(Q2+Q4))x(Q5/10)]}, and activity impairment [Q6/10], calculated based on three items: (Q2) the number of hours missed from work due to health problems in the past seven days; (Q4) the number of actual work hours in the past seven days; and (Q5) to what degree did the disease impair the productivity while working past seven days. The data calculated for each area is then converted to percent. Each of the areas is scored separately as a percentage, ranging from 0 to 100, with higher scores indicating greater impairment and less productivity.

Cost of direct healthcare resources utilized by participants per year based on costs of following direct healthcare resources was to be included: treatment received, routine monitoring visits (specialists and GPs), tests performed, emergency rooms visits, hospitalizations and complications derived from narcolepsy, and unitary costs.

The EQ-5D questionnaire is a generic instrument for describing and valuing health. It is based on a descriptive system that defines health in terms of 5 dimensions: Mobility, Self-Care, Usual Activities, Pain/Discomfort, and Anxiety/Depression. Each dimension has 3 response categories corresponding to no problems, some problems, and extreme problems. The instrument is designed for self-completion, and respondents also rate their overall health on the day of the interview on a 0-100 hash-marked, vertical visual analogue scale (EQ-VAS). Higher score indicates better health state. Percentage of participants are rounded-off to the nearest decimal point.

SSCl-8 is an 8 items scale (2 items measuring internalized stigma, 5 items measuring enacted stigma, and 1 item, which exhibited split loading, measuring either or both internalized and enacted stigma) developed to assess internalized and enacted/experienced stigma across neurological conditions. It uses a 5-point Likert scale ranging from 1 (never) to 5 (always) for scoring each item. The total score is calculated by summing individual items, ranging from 8 to 40. Higher scores indicate worsening of condition.

TSQM-9 measures participant satisfaction with the medication in 3 domains: Effectiveness, convenience, and global satisfaction. The scores were computed by adding items for each domain, i.e., 1 to 3 for effectiveness, 4 to 6 for convenience, and 7 to 9 for global satisfaction. The lowest possible score (1 for each item and 3 for all 3 subscales) was subtracted from the composite score and divided by the greatest possible score range. The greatest range was (7-1) x 3 items = 18 for effectiveness and convenience, and (5-1) x 3 items = 12 for global satisfaction. This provided a transformed score between 0 and 1 that was then multiplied by 100. TSQM-9 domain scores range from 0 to 100, with higher scores indicating greater satisfaction for that domain.

Percentage of participants are rounded-off to the nearest decimal point.

CCI is a scale to estimate 10-year mortality based on a score from a range of 12 comorbidities, the comorbidity score ranges from 0 to a maximum of 24 points. Higher scores indicate the higher chances of mortality.

An adverse event (AE) is any untoward medical occurrence in a participant administered a medicinal product and which does not necessarily have to have a causal relationship with this treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, a new disease or worsening in severity or frequency of a concomitant disease, temporally associated with the use of a medicinal product, whether or not the event is considered causally related to the use of the product.