Measuring the economic burden of neuromyelitis optica spectrum disorder in Colombia.

OBJECTIVE: To assess the economic burden of neuromyelitis optica spectrum disorder (NMOSD) in the Colombian context. METHODS: Analyses were conducted from a societal perspective using the prevalence-based approach. Costs were expressed in 2022 US dollars (1 USD = $3,914.46 COP). Direct medical costs were assessed from a bottom-up approach. Indirect costs included loss of productivity of the patient and their caregivers. The economic burden of NMOSD in Colombia was estimated as the sum of direct and indirect costs. RESULTS: The direct cost of treating a patient with NMOSD was USD$ 8,149.74 per year. When projecting costs nationwide, NMOSD would cost USD$ 7.2 million per year. Of these costs, 53.5% would be attributed to relapses and 34.4% to pharmacological therapy. Indirect costs potentially attributed to NMOSD in Colombia were estimated at USD$ 1.5 million per year per cohort. Of these, 78% are attributable to loss of patient productivity, mainly due to reduced access to the labor market and premature mortality. CONCLUSIONS: The NMOSD has a representative economic burden at the patient level, with direct costs, particularly related to relapses and medicines, being the main component of total costs. These findings are useful evidence that requires attention from public policymakers in Colombia.

Barriers to access and unmet needs to neuromyelitis optica spectrum disorders care in an Argentinean cohort.

INTRODUCTION: Neuromyelitis optica spectrum disorder (NMOSD) is a rare but severe neuroimmunological condition associated with a significant financial burden. NMOSD is also associated with increased health care utilization, including neurology outpatient visits, magnetic resonance imaging (MRI) use, long-term medication, among others. We aimed to evaluate real-world patient experiences in access to care and NMOSD burden in an Argentinean cohort. METHODS: This cross-sectional study used a self-administered survey and was conducted in Argentina (2022). Patients with NMOSD were divided into three groups: private health insurance (PHI), social health insurance (SHI), and public health insurance (PHI, Ministry of Public Health). Differences in access and health care barriers were assessed. RESULTS: One hundred patients with NMOSD (74 women) with a mean age at diagnosis of 38.7 years were included. Their EDSS was 2.8 and they were followed for 5.2 years. Of them, 51%, 11%, and 13% were employed (full-time: 57.5%), currently unemployed and retired by NMOSD, respectively. 55% of them visited between 2-3 specialists before NMOSD diagnosis. Aquaporin-4-antibody and/or myelin oligodendrocyte glycoprotein-antibody testing was requested in 91% (health insurance covered this partially in 15.3% and 32.9% of the time the test was entirely paid by patient/family). Patients with NMOSD receiving private medical care reported greater access to MRI, outpatient visits, and fewer issues to obtain NMOSD medications compared to those treated at public institutions. A longer mean time to MRI and neurology visit was found in the PHI group when compared with the other two subgroups. Regression analysis showed that private insurance (OR=3.84, p=0.01) was the only independent factor associated with appropriate access to NMOSD medications in Argentina. CONCLUSION: These findings suggest that barriers to access and utilization of NMOSD care services in Argentina are common. NMOSD patients experienced problems to receive NMOSD medication properly, especially those from the public sector.

Social participation and quality of life among patients with neuromyelitis optica spectrum disorders: The mediating effects of depression.

BACKGROUND: Depression is one of the most common and important symptoms of patients with neuromyelitis optica spectrum disorders. Depression is an important aspect of mental health which would be expected to affect a person's social participation. Studies have shown that depression and social participation are independent predictors of health-related quality of life. Depression especially affects its mental component and social participation especially affects its physical aspects. This study was designed to explore to what extent depression may regulate the relationship between social participation and quality of life. METHODS: A convenience sample of 138 discharged patients with neuromyelitis optica spectrum disorders were surveyed. They were asked about their demographic characteristics and disease-related information. Their ability in the activities of daily living was quantified using the Barthel Index. The 36-item short-form health survey, the Beck Depression Inventory and the Impact on Participation and Autonomy questionnaire were also administered. Univariate analysis and Pearson correlations were used to test for any significant relationships between the variables and quality of life. The mediating effect of depression on the association between social participation and life quality was examined using structural equation modeling. RESULTS: The structural equation models provided an excellent fit for the data. Social participation and the physical aspects of life quality were found to be strongly associated. And depression was of course strongly related to the mental aspects. Depression was not a mediator in the significant relationship between social participation and the physical aspects of life quality, but depression was found to be a strong and significant meditator in the association between social participation and the mental aspects of life quality. CONCLUSION: These findings help to clarify the direct and indirect effects of depression and social participation on the life quality of patients with neuromyelitis optica spectrum disorders. While helping and encouraging patients to actively participate in social life should be supplemented by monitoring them for signs of depression and providing appropriate treatment.

Overcoming the challenges in diagnosis of AQP4-IgG positive neuromyelitis optica spectrum disorders in resource poor settings using an indigenized and cost effective cell based assay.

BACKGROUND: Diagnosis of neuromyelitis optica spectrum disorders (NMOSD) in India is hindered by limited access to cost effective and sensitive assays for detection of aquaporin-4 antibody (AQP4-IgG) in India. OBJECTIVE: To develop a cost effective, sensitive, cell based assay (CBA) for detection of AQP4-IgG and to evaluate the serological status in patients with NMOSD diagnosed by 2015 diagnostic criteria. METHOD: Stably transfected Chinese hamster ovary (CHO) cell line expressing aquaporin M23 isomer was established. A fixed CBA was developed and validated in 381 samples including clinically definite NMOSD (n = 87), high risk NMOSD (n = 51), other demyelinating disorders (n = 92), other neurological disorders (n = 51) and healthy volunteers (n = 100). We tested the same samples again using a commercially available CBA and compared the results. All assays were performed by 2 independent investigators blinded to clinical and serological status. RESULTS: Our "in house"(Mangalore) assay showed sensitivity of 81.6% (95% CI 71.86-89.11%) for clinically definite NMOSD and 29.41% (95% CI 17.50-43.8%) for high risk NMOSD. Specificity was 100% for both groups. Both assays showed similar results for 67/ 87 (77.01%) patients with definite NMOSD while 4 samples tested positive by our assay alone (Cohen's kappa coefficient [K] - 0.86). Among the high risk group 14/51 (27.5%) samples showed similar results, one patient additionally was positive by the Mangalore assay (K - 0.95).

Burden and cost of comorbidities in patients with neuromyelitis optica spectrum disorder.

BACKGROUND: Neuromyelitis optica spectrum disorder (NMOSD) is associated with various comorbidities, including non-autoimmune and autoimmune conditions. The burden and cost of illness for NMOSD are unclear, particularly in the context of comorbidities. METHODS: Claims data from IBM MarketScan Commercial and Medicare Supplemental Databases between 2014 and 2018 were analyzed. Patients with NMOSD were specified as having inpatient or outpatient claims for NMOSD diagnosis or specific NMOSD symptoms claims and no subsequent claims for multiple sclerosis (MS) or use of MS disease-modifying therapy (DMT). Continuous enrollment ≥ 6 months before and ≥ 1 year after the first claim (index date) was required for study inclusion. Total costs stratified by comorbidities within 12 months post-index date were calculated per patient and compared 1:5 with matched non-NMOSD controls. RESULTS: A total of 162 patients with NMOSD and 810 non-NMOSD controls were evaluated. A significantly higher proportion of NMOSD patients had comorbidities than non-NMOSD controls (66.7% vs 41.5%; P < 0.001). Concomitant autoimmune disease occurred in 19.1% vs 4.9% (P < 0.001) of patients with NMOSD vs non-NMOSD controls. NMOSD patients incurred significantly higher total median (interquartile range) healthcare costs per patient ($68,386.48 [$23,373.54-$160,862.70]) than matched non-NMOSD controls with autoimmune disease ($17,215.13 [$6715.48-$31,441.93]; P < 0.001) or patients with NMOSD without autoimmune comorbidity ($23,905.42 [$8632.82-$67,251.54]; P = 0.022). Similarly, patients with NMOSD and non-autoimmune comorbidities incurred higher median healthcare costs than matched controls. CONCLUSIONS: Patients with NMOSD experience significant disease burden and cost that are amplified by comorbidities. Effective therapies are needed, particularly for patients with concomitant autoimmune disease.

Identification and temporal trends of patients with neuromyelitis optica spectrum disorder in a US insurance claims database.

BACKGROUND: This US claims-based study aimed to identify and characterize temporal trends in diagnostic pathways for patients likely to have neuromyelitis optica spectrum disorder (NMOSD). METHODS: Patients were identified from IBM MarketScan Commercial Databases if, within 1 year, they had two NMOSD claims separated by ≥ 60 days; two transverse myelitis (TM) or optic neuritis (ON) claims separated by ≥ 60 days, and one additional symptom (TM, ON, or area postrema syndrome); or one NMOSD claim and one additional symptom. The first NMOSD or TM/ON claim was the index date, and the second claim was the diagnosis date. Similar methodology was used in temporal trend and incidence and prevalence analyses. RESULTS: Among 1,901 patients with NMOSD, 34.2% were identified by two NMO claims, 53.2% by ON or TM +1 symptom, and 12.6% by one NMOSD claim +1 symptom. Anti-aquaporin-4 immunoglobin G (AQP4-IgG) autoantibody tests and magnetic resonance imaging was used for 23.0% and 71.9% of cases, respectively. Across cohorts, 21.4-49.1% had multiple sclerosis (MS) diagnosis claims prior to index date, and 37.3-60.6% had an MS diagnosis, 14.9-31.0% had MS disease-modifying therapy (DMT) claims and 6.3-44.8% had immunosuppressive therapy (IST) claims <1 year after diagnosis. Over time, there were slight changes in MS diagnosis claims, AQP4-IgG autoantibody testing, and DMT and IST use before and after NMOSD diagnosis. LIMITATIONS: This study is limited by the information available in US claims databases, which included the potential for misclassification of NMOSD based solely on claims codes and lack of reimbursement for AQP4-IgG testing by insurance companies. CONCLUSIONS: Among patients likely to have NMOSD, low AQP4-IgG testing rates, IST use, frequent MS diagnosis claims, and DMT use highlight the need for a diagnostic algorithm and timely treatment of NMOSD.

Assessment of mental health, knowledge, and attitude of patients with multiple sclerosis and neuromyelitis optica spectrum disorder in response to 2019 novel coronavirus.

BACKGROUND: With the recent pandemic of the novel coronavirus disease (COVID-19), multiple sclerosis (MS) and neuromyelitis optica spectrum disorder (NMOSD) patients for their compromised immune system have been in significant concern. Awareness and attitude about this virus have an important impact on infection prevention and coping with stress and anxiety. So we conducted this study to assess knowledge, attitude, and mental health status in MS and NMOSD patients within the COVID-19 pandemic. METHOD: In this cross-sectional study, the link of Depression Anxiety Stress Scales-21 (DASS-21) and a self-administered structured questionnaire were sent through social media to MS and NMOSD patients and two control groups consisting of healthy and migraine individuals. RESULTS: A total of 223 MS patients, 41 NMOSD, 29 migraine, and 245 healthy subjects participated in this study. MS patients had higher total DASS scores compared to healthy participants (p = 0.012). There were no significant differences among the study groups regarding knowledge and attitude. In MS patients, physical comorbidity was associated with a total score of attitude (OR 1.59, 95% CI 0.53, 2.66, p = 0.004). We did not find association between other demographic and clinical variables with DASS scores, attitude, and knowledge in MS patients. CONCLUSION: The current data highlight the necessity of attitude, knowledge, and mental health assessment among MS and NMOSD patients. Further studies in other countries need to be carried to assess these points among MS and NMOSD patients.

Cost effectiveness of rituximab and mycophenolate mofetil for neuromyelitis optica spectrum disorder in Thailand: Economic evaluation and budget impact analysis.

Neuromyelitis optica spectrum disorder (NMOSD) is an inflammatory condition of the central nervous system. The extent of disability depends on the severity of the disease and the number of relapses. Although azathioprine is currently the main treatment for patients with NMOSD in Thailand, patients often relapse during its use. Hence, it is argued that there are other drugs that would be more effective. The purpose of this study is to evaluate, from a societal perspective and from the economic impact on Thailand's healthcare system, the cost utility of treatment with mycophenolate mofetil (MMF) and rituximab in patients resistant to azathioprine. The Markov model with a one-year cycle length was applied to predict the health and cost outcomes in patients with NMOSD over a lifetime. The results showed that rituximab exhibited the highest quality-adjusted life year (QALY) gains among all the options. Among the rituximab-based treatments, the administration of a rituximab biosimilar with CD27+ memory B cell monitoring proved to be the most cost-effective option. At the willingness-to-pay threshold of 160,000 Thai baht (THB), or 5,289 US dollar (USD), per QALY gained, the treatment exhibited the highest probability of being cost effective (48%). A sensitivity analysis based on the adjusted price of a generic MMF determined that the treatment was cost effective, exhibiting an incremental cost-effectiveness ratio of -164,653 THB (-5,443 USD) and a 32% probability of being cost effective. The calculated budget impact of treating patients resistant to conventional therapy was 1-6 million THB (33,000-198,000 USD) for the first three years, while after the third year, the budget impact stabilized at 3-4 million THB (99,000-132,000 USD). These data indicate that, in Thailand, treatment of drug resistant NMOSD with a rituximab biosimilar with CD27+ memory B cell monitoring or treatment with a generic MMF would be cost effective and would result in a low budget impact. Therefore, the inclusion of both the rituximab biosimilar and a generic MMF in the National Drug List of Essential Medicine for the treatment of NMOSD may be appropriate.

The prevalence and types of oral- and pharyngeal-stage dysphagia in patients with demyelinating diseases based on subjective assessment by the study subjects.

BACKGROUND: Studies show that dysphagia is a common problem in patients with demyelinating diseases. However, there are no published studies on dysphagia in this group of patients, which would include the individual phases or the safety and effectiveness of the swallowing process. OBJECTIVE: The main objective of this study was to assess the prevalence of swallowing disorders and to characterize them based on subjective assessment by the study subjects with multiple sclerosis and Devic's syndrome. METHOD: The study included 72 patients (47 F, 25 M). Patients at risk of dysphagia were identified using the DYMUS, EAT-10 and SDQ questionnaires. To assess the type of oral- and pharyngeal-stage dysphagia, questions in the questionnaires were classified into groups according to symptoms typical of each stage. RESULTS: The risk of dysphagia and the need for instrumental examination were identified in 37.5% of the study subjects. Pharyngeal-stage dysphagia (repeated swallowing, increased effort of swallowing, cough, a feeling of food sticking in the throat) was reported to occur at a significantly higher frequency. However, no differences were found between difficulty in swallowing liquids and difficulty in swallowing solid food. CONCLUSION: There is a need for further research, which should include a detailed dysphagia-oriented diagnosis, with a view to gaining a detailed insight into the pathophysiology of deglutition in this group of patients.

Evaluation of health-related quality of life, fatigue and depression in neuromyelitis optica.

BACKGROUND: The burden of multiple sclerosis (MS) includes fatigue, depression and worsening of health-related quality of life (HRQOL). These changes have not been yet measured in neuromyelitis optica (NMO). Our aim was to assess the HRQOL, fatigue and depression in NMO. METHODS: We administered French validated self-questionnaires on HRQOL (SEP-59), fatigue (EMIF-SEP) and depression (EHD) to 40 patients followed up in two centres. We assessed the relationship of these parameters with gender, age, disability, disease duration, visual acuity and NMO-antibody status and also compared our results with equivalent data in MS and normal subjects derived from previous studies. RESULTS: Health-related quality of life scores were lower (P < 0.01) in patients with NMO when compared to normal subjects. No significant difference was noted between patients with NMO and MS for most scores, the exceptions being HRQOL related to cognitive function (better in NMO than in MS), HRQOL related to sphincter dysfunction (worse in NMO than in MS) and the psychological dimension of fatigue (milder in NMO than in MS). Disability was the main predictive factor of an unfavourable evolution. DISCUSSION: This study reveals the strong impact of NMO on HRQOL, fatigue and depression and the importance of screening patients, especially the more disabled, so as to initiate suitable treatment.

Epidemiology, immunopathogenesis and management of pediatric central nervous system inflammatory demyelinating conditions.

PURPOSE OF REVIEW: Pediatric inflammatory demyelinating central nervous system diseases comprise monofocal and potentially monophasic disorders like optic neuritis and transverse myelitis, the multifocal, self-limiting disorder of acute disseminated encephalomyelitis, and multifocal chronic diseases like relapsing neuromyelitis optica and multiple sclerosis. This review discusses characteristics of these disorders with focus on epidemiology and treatment of acute disseminated encephalomyelitis, neuromyelitis optica and multiple sclerosis. RECENT FINDINGS: An international consensus group very recently defined diagnostic criteria for pediatric multiple sclerosis and acute disseminated encephalomyelitis. Immunological studies on pediatric inflammatory demyelinating disorders revealed possible disease-related humoral and T-cellular pathomechanisms. The recently identified biomarker for neuromyelitis optica, aquaporin-4-autoantibody, was detected in children with relapsing neuromyelitis optica with a similar frequency as in adult neuromyelitis optica patients. Clinically relevant, there is growing evidence that disease-modifying treatments are well tolerated and effective in the pediatric age group also. SUMMARY: Recent studies on pediatric inflammatory demyelinating central nervous system diseases have contributed to current awareness that these disorders are not restricted to the adult age group, and that some of them carry an unfavorable long-term prognosis. Growing knowledge will hopefully enable more timely diagnoses and more specifically tailored therapies in the near future, with the goal of improving outcomes in this young patient group.