Neuromyelitis optica spectrum disorder (NMOSD) is a rare and disabling neuroinflammatory disorder. Previous studies of aquaporin-4 IgG-positive (AQP4+) NMOSD in Canada have been limited by small sample sizes and regional focus. This study aimed to characterize the sociodemographic and clinical profile of individuals living with AQP4+ NMOSD and the treatment landscape in a multicenter study.

Data were collected from the CAnadian Neuromyelitis OPTIca Spectrum Disorder and other atypical demyelinating diseases Cohort Study (CANOPTICS), a prospective longitudinal study involving seven Canadian centers. Participants were diagnosed with AQP4+ NMOSD using the 2015 International Panel for NMO Diagnostic criteria. We analyzed sociodemographic and clinical characteristics in addition to treatment utilization over time.

133 participants with AQP4+ NMOSD were included, with a mean (SD) age of 52.0 (15.0) years at enrollment and 45.9 (15.5) years at index attack. Most were female (84.9%), ethnically and racially varied (33.3% North American or European White, 25.2% African/Caribbean Black, 22.7% East/Southeast Asian) and born abroad (63.3%). Two-thirds had moderate or mild disability, as measured by an Expanded Disability Status Scale of 3.5 or less. Azathioprine was the most used first-line therapy (59.3%), followed by mycophenolate mofetil (12.5%) and rituximab (10.5%). Use of novel monoclonal antibodies (MAbs) was least common (6.3%); however, first-line use of rituximab and novel MAbs increased in recent years (2018–2023).

Canadians with AQP4+ NMOSD are predominantly female and ethnically and racially varied, with more favorable outcomes than in previous reports. Despite a trend toward increasing use of rituximab and novel MAbs, these higher-efficacy treatments remain underutilized for NMOSD in Canada.

We aimed to (1) report updated estimates of direct healthcare costs for people living with MS (pwMS), (2) contrast costs to a control population and (3) explore differences between disability levels among pwMS.

Administrative data were used to identify adult pwMS (MS cohort) and without (control cohort) in Alberta, Canada; disability level (based on the Expanded Disability Status Scale) among pwMS was estimated. One- and two-part generalized linear models with gamma distribution were used to estimate the incremental direct healthcare cost (2021 $CDN) of MS during a 1-year observation period.

Adjusting for confounders, the total healthcare cost ratio was higher in the MS cohort (n = 13,089) versus control (n = 150,080) (5.24 [95% CI: 5.08, 5.41]) with a predicted incremental cost of $15,016 (95% CI: $14,497, $15,535) per person-year. Among the MS cohort, total predicted direct healthcare costs were higher with greater disability, $14,430 (95% CI: $13,980, $14,880) to $58,697 ($51,514, $65,879) per person-year in mild and severe disability, respectively. The primary health resource cost component shifted from disease-modifying therapies in mild disability to supportive care in moderate and severe disability.

Adult pwMS had greater direct healthcare costs than those without. Extrapolating to the population level (where 14,485 adult pwMS were identified in the study), it is estimated that $218 million per year in healthcare costs may be attributable to MS in Alberta. The significantly larger economic impact associated with greater disability underscores the importance of preventing or delaying disease progression and functional impairment in MS.

Understanding disease-modifying therapy (DMT) use and healthcare resource utilization by different geographical areas among people living with multiple sclerosis (pwMS) may identify care gaps that can be used to inform policies and practice to ensure equitable care.

Administrative data was used to identify pwMS on April 1, 2017 (index date) in Alberta. DMT use and healthcare resource utilization were compared between those who resided in various geographical areas over a 2-year post-index period; simple logistic regression was applied.

Among the cohort (n = 12,338), a higher proportion of pwMS who resided in urban areas (versus rural) received ≥ 1 DMT dispensation (32.3% versus 27.4%), had a neurologist (67.7% versus 63.9%), non-neurologist specialist (88.3% versus 82.9%), ambulatory care visit (87.4% versus 85.3%), and MS tertiary clinic visit (59.2% versus 51.7%), and a lower proportion had an emergency department (ED) visit (46.3% versus 62.4%), and hospitalization (20.4% versus 23.0%). Across the provincial health zones, there were variations in DMT selection, and a higher proportion of pwMS who resided in the Calgary health zone, where care is managed by MS tertiary clinic neurologists, had an outpatient visit to a neurologist or MS tertiary clinic versus those who resided in other zones where delivery of MS-related care is more varied.

Urban/rural inequalities in DMT use and healthcare resource utilization appear to exist among pwMS in Alberta. Findings suggest the exploration of barriers with consequent strategies to increase access to DMTs and provide timely outpatient MS care management, particularly for those pwMS residing in rural areas.

Early and effective treatment of central nervous system (CNS) inflammatory disorders is vital to reduce neurologic morbidity and improve long-term outcomes in affected children. Rituximab is a B-cell-depleting monoclonal antibody whose off-label use for these disorders is funded in the province of Alberta, Canada, by the Short-Term Exceptional Drug Therapy (STEDT) program. This study describes the use of rituximab for pediatric CNS inflammatory disorders in Alberta.

Rituximab applications for CNS inflammatory indications in patients <18 years of age were identified from the STEDT database between January 1, 2012, and December 31, 2019. Patient information was linked to other provincial datasets including the Discharge Abstract Database, Pharmaceutical Information Network, and Provincial Laboratory data. Analysis was descriptive.

Fifty-one unique rituximab applications were identified, of which 50 were approved. New applications increased from one in 2012 to a high of 12 in 2018. The most common indication was autoimmune encephalitis without a specified antibody (n = 16, 31%). Most children were approved for a two-dose (n = 33, 66%) or four-dose (n = 16, 32%) induction regimen. Physician-reported outcomes were available for 24 patients, of whom 14 (58%) were felt to have fully met outcome targets.

The use of rituximab for pediatric CNS inflammatory disorders has increased, particularly for the indication of autoimmune encephalitis. This study identified significant heterogeneity in dosing practices and laboratory monitoring. Standardized protocols for the use of rituximab in these disorders and more robust outcome reporting will help better define the safety and efficacy of rituximab in this population

Intravenous rt-PA (IV rt-PA) for acute stroke has raised many concerns, including its inadvertent use in patients presenting with acute stroke-like symptoms as the expression of their somatoform disorder. Diagnosis of the somatoform disorder is often delayed, and thrombolytics in these patients for their stroke-like presentation subjects them to risk for hemorrhage.

The presentation, neurological findings, and the therapeutic decision making was audited in 85 patients who received IV rt-PA for a diagnosis of acute stroke. All the surviving patients were re-examined neurologically at least three months after IV rt-PA. Baseline and follow-up brain CT scans were re-reviewed by a neuroradiologist who was blinded to clinical presentation and outcome. Patients whose clinical presentation, brain CT and neurological outcome did not fit into known or expected anatomical and clinical patterns of stroke underwent psychological assessment using the Minnesota Multiphasic Personality Inventory-2.

In two patients three stroke-like presentations of somatoform disorder inadvertently were treated with IV rt-PA. This was primarily caused by abbreviated neurological examination and narrow differential diagnosis.

Patients with somatoform disorder may present with symptoms mimicking acute stroke. Under the time constraints of IV rt-PA use, a diagnosis of somatoform disorder can be missed, subjecting such patients to the potential complications of thrombolytics.