Cost-effectiveness of eculizumab and efgartigimod for the treatment of anti-acetylcholine receptor antibody-positive generalized myasthenia gravis.

BACKGROUND: Eculizumab and efgartigimod were approved to treat anti-acetylcholine receptor antibody-positive generalized myasthenia gravis (anti-AChR Ab-positive gMG). These relatively new biological treatments provide a more rapid onset of action and improved efficacy compared with conventional immunosuppressive treatments, but at a higher cost. OBJECTIVE: To assess the cost-effectiveness of eculizumab and, separately, efgartigimod, each added to conventional therapy vs conventional therapy alone, among patients with refractory anti-AChR Ab-positive gMG and those with anti-AChR Ab-positive gMG, respectively. METHODS: A Markov model with 4 health states was developed, evaluating costs and utility with a 4-week cycle length and lifetime time horizon from a health care system perspective and a modified societal perspective including productivity losses from patients and caregiver burden. Model inputs were informed by key clinical trials and relevant publications identified from targeted literature reviews, and drug costs were identified from Micromedex Red Book. Costs and outcomes were discounted at 3% per year. Incremental cost-effectiveness ratios (ICERs; cost per quality-adjusted life-year [QALY] gained) were calculated for each comparison. RESULTS: Among the corresponding populations, lifetime costs and QALYs, respectively, for eculizumab were $5,515,000 and 11.85, and for conventional therapy, $308,000 and 10.29, resulting in an ICER of $3,338,000/QALY gained. For efgartigimod, lifetime costs and QALYs, respectively, were $6,773,000 and 13.22, and for conventional therapy, $322,000 and 9.98, yielding an ICER of $1,987,000/QALY gained. After applying indirect costs in a modified societal perspective, the ICERs were reduced to $3,310,000/QALY gained for eculizumab and $1,959,000/QALY gained for efgartigimod. CONCLUSIONS: Eculizumab and efgartigimod are rapidly acting and effective treatments for myasthenia gravis. However, at their current price, both therapies greatly exceeded common cost-effectiveness thresholds, likely limiting patient access to these therapies.

Economic burden of generalized myasthenia gravis (MG) in the United States and the impact of common comorbidities and acute MG-events.

BACKGROUND: This study assessed the incremental healthcare costs and resource utilization (HRU) associated with generalized myasthenia gravis (gMG), as well as variability in these outcomes among patients with gMG and common comorbidities and acute MG-related events. METHODS: Adults with gMG and without MG were identified from a large US database (2017-2021). The index date was the first MG diagnosis (gMG cohort) or random date (non-MG cohort). Cohorts were propensity score matched 1:1. The gMG cohort included subgroups of patients with a 12-month pre-index (baseline) cardiometabolic or psychiatric comorbidity, or a post-index MG exacerbation/crisis. Monthly healthcare costs (2021 USD) and HRU were compared post-index between gMG and non-MG cohorts. RESULTS: The gMG and matched non-MG cohorts each contained 2,739 patients. Mean incremental healthcare costs associated with MG were $4,155 (gMG: $5,567; non-MG: $1,411), with differences driven by incremental inpatient costs of $2,166 (gMG: $2,617; non-MG: $452); all p < 0.001. The gMG versus non-MG cohort had 4.36 times more inpatient admissions and 2.26 times more outpatient visits; all p < 0.001. Among patients with gMG in cardiometabolic (n = 1,859), psychiatric (n = 1,308), and exacerbation/crisis (n = 419) subgroups, mean monthly healthcare costs were $6,660, $7,443, and $17,330, respectively. CONCLUSIONS: gMG is associated with substantial incremental costs and HRU, with inpatient costs driving the total incremental costs. Costs increased by 20% and 34% among patients with cardiometabolic and psychiatric conditions, respectively, and over three times among those with acute MG-related events. gMG is a complex disease requiring management of comorbidities and treatment options that can prevent acute symptomatic events.

Generalized myasthenia gravis (gMG) is a rare long-standing condition that affects the junctions between nerves and muscles, causing them to be weak. In a serious case, the diaphragm ­ a muscle that helps with breathing ­ becomes so weak that a patient will need a machine to breathe for them. This is called MG exacerbation or crisis. In this study, we used a large insurance database in the United States to look at how much money healthcare payers paid for gMG patients on average and what healthcare resources patients with gMG used. We compared these findings with patients without gMG. Also, among patients with gMG, we reported these findings specifically for patients who also had heart, blood, or blood vessel disease; patients who had a mental illness; and patients who had MG exacerbation or crisis later on. We found that patients with gMG used $5,567 per month on average ($4,155 more than patients without gMG), mostly from overnight hospital stays. Patients with gMG also had four times more overnight hospital stays and two times more hospital day visits when we compared them to patients without gMG. Patients with gMG and other health conditions used even more money and resources per month. Patients with MG exacerbation or crisis used $17,330 per month on average. Our results showed that gMG led to higher healthcare cost and resource use. In order to reduce cost and resources, doctors also need to control for other health conditions as they treat patients with gMG, and to prevent patients from having MG exacerbation or crisis later on.

Economic Costs of Myasthenia Gravis: A Systematic Review.

OBJECTIVES: The objective of our study was to conduct a systematic literature review of economic costs (henceforth costs) associated with myasthenia gravis (MG). METHODS: We searched MEDLINE (through PubMed), CINAHL, Embase, PsycINFO, and Web of Science for studies reporting costs of MG published from inception up until March 18, 2020, without language restrictions. Two reviewers independently screened records for eligibility, extracted the data, and assessed included studies for risk of bias using the Newcastle-Ottawa Scale. Costs were inflated and converted to 2018 United States dollars ($). RESULTS: The search identified 16 articles for data extraction and synthesis. Estimates of costs of MG were found for samples from eight countries spanning four continents (Europe, North America, South America, and Asia). Across studies, the mean per-patient annual direct medical cost of illness was estimated at between $760 and $28,780, and cost per hospitalization between $2550 and $164,730. The indirect cost of illness was estimated at $80 and $3550. Costs varied considerably by patient characteristics, and drivers of the direct medical cost of illness included intravenous immunoglobulin and plasma exchange, myasthenic crisis, mechanical ventilatory support, and hospitalizations. CONCLUSIONS: We show that the current body of literature of costs of MG is sparse, limited to a few geographical settings and resource categories, mostly dated, and subject to non-trivial variability, both within and between countries. Our synthesis will help researchers and decision-makers identify gaps in the local health economic context of MG and inform future cost studies and economic evaluations in this patient population.

Hospital and healthcare insurance system record-based epidemiological study of myasthenia gravis in southern and northern China.

OBJECTIVE: This is the first cross-region epidemiological study of myasthenia gravis (MG) in China. We estimated the incidence, prevalence, and medical costs of MG in southern China and explored the differences between the southern and northern Chinese populations. METHODS: We collected and analyzed records from 20 hospitals in the southern city, Guangzhou, 13 hospitals in the northern city, Harbin, and two healthcare insurance systems: job based and residence based in Guangzhou during 2000-2017. RESULTS: (1) The estimated annual incidence of MG was 1.55-3.66 per 100,000, and the estimated prevalence of MG was 2.19-11.07 per 100,000 in southern China based on insurance records. (2) The proportion of hospitalized MG patients in the south-based hospital records was three times as high as that in the north-based hospital records. (3) Female MG prevalence was significantly higher than male MG prevalence in Guangzhou, while the similar gender difference in Harbin was not statistically significant due to higher variation in earlier years. (4) The average expense was $35-42 for each outpatient service and $2526-2673 for each hospitalization expense in the south. (5) Contrary to the increase of insurance-based estimate of MG prevalence, the proportion of hospitalized MG patients did not increase over the years, suggesting rising awareness and utilization of health insurance. CONCLUSIONS: The southern MG population had a significantly higher prevalence and a lower response threshold to medication than the northern MG population. These results are calling for further investigations on the genetic, cultural, and environmental variations of the Chinese MG populations between north and south.

Out-of-pocket expenses for myasthenia gravis patients in China: a study on patients insured by basic medical insurance in China, 2013-2015.

BACKGROUND: Myasthenia gravis is a rare autoimmune neuromuscular disorder. The disorder requires long-term use of expensive medication to control clinical symptoms. This study analyzed the change in trends of total medical expenses and out-of-pocket expenses for patients with myasthenia gravis and explored the factors influencing them. METHODS: In this retrospective study, data were derived from a survey of medical service utilization for patients insured by the Urban Basic Medical Insurance in China from 2013 to 2015. The cost data of 3347 patients with myasthenia gravis were included in this study. The baseline characteristics and medical expenses for patients with myasthenia gravis were analyzed using a descriptive method. The difference and influencing factors of the out-of-pocket ratio were analyzed from both outpatient and inpatient expenses by using the quantile regression method. RESULTS: The total expenses reimbursed by the Urban Basic Medicine Insurance for all patients with myasthenia gravis fell progressively from 73.1 to 58.7% during the study period. Patients' out-of-pocket expenses increased gradually, of which expenses within the scope of Basic Medicine Insurance increased from 14.7 to 22.6% and expenses outside of the Basic Medicine Insurance scope increased from 12.6 to 18.7%. Moreover, the panel quantile results showed a positive correlation between the year of receiving treatment and the out-of-pocket ratio. In addition to the 25th quantile of the out-of-pocket ratio among outpatients with myasthenia gravis, there were significant differences in medical insurance and medical institution among all the other quantiles. Significant regional differences were found in all quantiles of the out-of-pocket ratio, except for the 75th quantile among inpatients. Lastly, age had a negative effect on inpatients with myasthenia gravis across all quantiles, but not on outpatients. CONCLUSIONS: From 2013 to 2015, patients with myasthenia gravis's out-of-pocket expenses increased progressively. Moreover, the individual out-of-pocket ratio was affected by the year, medical insurance, medical institution, region, and age. The current medical insurance policy for the general public has a low ability to cater for patients with myasthenia gravis.

Healthcare resource utilization by patients with treatment-refractory myasthenia gravis in England.

Aims: To examine healthcare resource utilization associated with refractory myasthenia gravis (MG) in England. Materials and methods: This was a retrospective cohort study of linked data from the Clinical Practice Research Datalink and the Hospital Episode Statistics database collected between 1997 and 2016. Included patients were ≥18 years of age at the index MG diagnosis. Patients with refractory MG were identified using an algorithm based on treatments received. Healthcare resource utilization since the index date was compared between refractory and non-refractory cohorts. Results: The study included 1149 patients with MG, of whom 66 (5.7%) were refractory. Sex and age at diagnosis did not significantly differ between the refractory and non-refractory cohorts. Rates of healthcare resource utilization per person-year were significantly higher (p < .05) for patients with refractory compared to non-refractory MG for GP visits, visits to other healthcare professionals, outpatient visits and inpatient hospitalization. Patients in the refractory cohort spent more total days hospitalized since the index visit than patients in the non-refractory cohort (median, 33 vs. 16 days [p < .0001]). Limitations: The algorithm for identifying refractory patients did not include clinical criteria. Also, treatments administered in hospitals or by specialists were not available in the databases. Conclusions: Patients in England with refractory MG more often visit healthcare providers, are hospitalized and visit an emergency room than patients with non-refractory MG.

10-year-outcomes after rituximab for myasthenia gravis: Efficacy, safety, costs of inhospital care, and impact on childbearing potential.

Rituximab (RTX) has emerged as an attractive off-label treatment option for patients with myasthenia gravis (MG) refractory to other immune therapies. However, data on long-term outcome after RTX for MG are still scarce. Here we present the 10-year outcomes [median (range) 10.1 (6.7-11.2) years] with respect to efficacy, safety, costs of inhospital care, and impact on childbearing potential in all four MG patients treated by one of the authors with RTX. In all patients, RTX led to sustained clinical improvement and eventual tapering of other immune therapies. RTX was well tolerated, and complications were not observed. After the start of RTX, annual costs for hospital admissions were markedly reduced compared to costs in the year preceding RTX. Under close clinical observation, two patients had uncomplicated pregnancies giving birth to a healthy child. With regard to its efficacy, excellent tolerance, lack of complications, low frequency of repeat infusions and pending patent expiry in many countries, RTX appears to compare favourably with other immune therapies used for MG. Multicentre trials and registries are urgently needed to further address long-term safety issues and clarify the efficacy and role of RTX in managing MG.

Inpatient cost analysis for treatment of myasthenia gravis.

INTRODUCTION: We explore trends in U.S. inpatient costs of care over a 10-year period. METHODS: We compare myasthenia gravis (MG) with multiple sclerosis (MS) and overall U.S. hospital admissions using the Agency for Healthcare Research and Quality Nationwide Inpatient Sample database for 2003-2013. RESULTS: Total costs of MG inpatient care rose 13-fold from 2003 to 2013. This was accounted for by a greater than sixfold increase in discharges and a greater than twofold increase in cost per discharge. The < 17 and >85 years age groups experienced the greatest increases in discharges. Medicare and Medicaid use increased. Regional variations in cost were apparent. There were greater rises in the Midwestern and Southern United States, which is dissimilar to MS and all hospital admissions. DISCUSSION: There was a dramatic and disproportionate rise in the number of MG discharges, most likely because of changes in practice patterns. Muscle Nerve 56: 1114-1118, 2017.

Direct and indirect cost of myasthenia gravis: A prospective study from a tertiary care teaching hospital in India.

Myasthenia gravis (MG) requires lifelong treatment. The cost of management MG is very high in developed countries but there is no information on the cost of management of MG in the developing countries. This study reports the direct and indirect cost and predictors of cost of MG in a tertiary care teaching hospital in India. In a prospective hospital based study, from a tertiary hospital in India 66 consecutive patient during 2014-2015 were included. The age of the patients ranged between 6 and 75years. The severity of MG was assessed by myasthenia gravis foundation association (MGFA) class (MGFA) I-V. The patient data was collected s and their direct cost was calculated from the computerized Hospital information system. The indirect cost was calculated from patient's memory, checking the bills of transportation and wages loss by the patient or the care giver. Total annual cost of MG ranged between INR (4560-532227) with median INR 61390.5 (US$911.64). The median cost of outpatient department (OPD) consultation of 16 patients was INR 20439.9 (US$303.53), of 50 admitted patients was INR 44311.8 (US$658.03) and 21 intensive care unit (ICU) patients was INR 59574.3 (US$ 884.6) and the direct cost of thymectomy was INR 45000 (US$ 668.25). Direct cost was related to indirect cost (r=0.55; p=0.0001). Predictors of patient outcome were severity of MG, ICU admission, and thymectomy. The total median cost for management of myasthenia gravis was INR 61390.5 (4560-532227, US$911.64) per year, and the cost was mainly determined by the severity of MG.

Cost-minimization analysis comparing intravenous immunoglobulin with plasma exchange in the management of patients with myasthenia gravis.

INTRODUCTION: Myasthenia gravis (MG) exacerbations may be treated with intravenous immunoglobulin (IVIg) or plasma exchange (PLEX), which have equivalent effectiveness. This cost-minimization analysis compared IVIg with PLEX for treatment of patients with MG exacerbation. METHODS: We combined the Ontario-based health cost data with clinical data from a randomized clinical trial. Analyses were undertaken from the perspective of a public healthcare insurer and from the perspective of a tertiary university hospital payer. RESULTS: PLEX was less costly than IVIg among patients with a body mass index (BMI) > 15.7 kg/m(2) , from the perspective of the public healthcare insurer (P < 0.0001). However, PLEX was more costly than IVIg from the perspective of the hospital payer when the costs of blood products were excluded (P < 0.0001). CONCLUSIONS: PLEX can be considered a short-term cost-minimizing therapy when compared with IVIg for treatment of MG exacerbation among patients with BMI >15.7 kg/m(2) , from the perspective of a public healthcare insurer. Muscle Nerve 53: 872-876, 2016.

Prevalence of factors associated with poor outcomes of hospitalized myasthenia gravis patients in Thailand.

OBJECTIVE: To examine the prevalence of hospitalized myasthenia gravis (MG), and to determine the factors associated with poor outcomes of hospitalized MG patients at a national level. METHODS: This study was based on a retrospective design. We collected data of hospitalized MG adults recorded by the National Health Security Office, Bangkok, Thailand between October 2009 and September 2010. Clinical data and treatment outcomes were examined. RESULTS: The total number of hospitalized MG patients was 936 cases. The prevalence rate of hospitalized MG patients was 2.17/100,000 population. The average age (SD) was 44.93 (14.16) years. Regarding the discharge status of MG patients, 845 cases (90.3%) had improved. The total hospital charge of MG patients was 64,332,806 baht (USD 2,144,426.87) or an average of 68,731.63 baht/admission (USD 2,291.05), with an average length of stay of 10.45 days. There were 3 significant factors associated with poor outcomes in hospitalized MG patients; namely, hospital category, pneumonia, and respiratory failure. CONCLUSION: The prevalence of admission in MG patients was 2.17 persons/100,000 population. Hospital category, pneumonia, and respiratory failure were significant factors associated with poor outcomes.

The cost-effectiveness of open or thoracoscopic thymectomy compared to medical treatment in managing Myasthenia gravis without thymomas.

OBJECTIVE: Assessing the cost-effectiveness of open or thoracoscopic thymectomy compared to medical therapy in managing myasthenia gravis not associated with thymoma. METHODS: A Markov model was designed for evaluating three strategies' cost-effectiveness. Transition probabilities were taken from the pertinent literature; the costs were estimated from official tariff manuals. Incremental cost-effectiveness ratios were estimated and probabilistic and deterministic sensitivity analysis was used for clinical variables, costs and the model's assumptions. RESULTS: Thoracoscopic thymectomy was the most effective and least costly strategy and dominated the other two alternatives. The cost per life year gained was Col $ 1 129 531 without discount and Col $ 805 179 with discount. Univariate sensitivity analysis showed that the main variables affecting the results were the effects' discount rate, the cost of a myasthenic crisis and the probability of complete remission. Thoracoscopy thymectomy was the most cost-effective strategy for different thresholds of willingness to pay in probabilistic analysis. CONCLUSIONS: Thoracoscopic thymectomy is a cost-effective strategy in the treatment of MG without thymoma.

Estimated cost of treating myasthenia gravis in an insured U.S. population.

INTRODUCTION: In this study we estimated the costs paid by U.S. health plans for treating myasthenia gravis (MG) in 2009 and determined the major cost drivers. METHODS: One hundred thirteen MG patients were matched by propensity scores with 339 non-MG patients from a comprehensive health-care insurance database. The mean annual costs paid by the health plan for treating MG, costs by place of service, and costs for intravenous immunoglobulin (IVIg) and plasma exchange were determined. RESULTS: Mean annual costs paid by the health plan per MG patient were $20,190 (SEM $4,763) and costs attributable to treating MG were $15,675. Home health services accounted for 23% of MG patient costs and represented almost exclusively IVIg infusion costs. Six MG patients had a total of 136 outpatient IVIg infusions at an average annual cost of $109,463 ± $57,303. CONCLUSIONS: The estimated annual health plan paid costs for treating MG were $15,675. Home health services represented 23% of MG patient costs, largely driven by IVIg administration.

A standardized protocol for the perioperative management of myasthenia gravis patients. Experience with 110 patients.

BACKGROUND: Video-assisted thoracoscopic extended thymectomy (VATET) is well established in the treatment of myasthenia gravis; however, patient selection remains controversial. Perioperative management protocol is lacking, and concerns regarding post-operative myasthenic crisis still remain. We performed a retrospective observational study evaluating the impact of the introduction of a protocol in the perioperative management of patients with myasthenia gravis who underwent VATET. METHODS: The perioperative management protocol was developed by a team of neurologists and anesthesiologists who reviewed the literature and their previous experience on myasthenia gravis patients. Respiratory, clinical, and neurological patient features were included in the protocol evaluation. A retrospective review of patients who underwent VATET before and after introduction to the protocol was finally performed. RESULTS: The medical records of 66 patients (pre-protocol group) and 44 patients (protocol group) were available for the study. In the pre-protocol group, 17 patients (26%) were admitted to intensive care unit (ICU) during the post-operative period, while three patients (6.8%) of the protocol group met the criteria for ICU post-operative admission. This resulted in a reduction of 73.5% of patients admitted to ICU (P = 0.023) and in an 80% (P = 0.002) reduction of the use neuromuscular blocking agents. Two post-operative myasthenic crises preceded by bulbar symptoms (1.8%) were identified in the pre-protocol group patients. CONCLUSIONS: Although the application of our protocol results in a substantial reduction in the recovery of patients in the ICU and in hospital costs, there was no substantial difference in mortality and morbidity between patients admitted to the surgical ward or to ICU.

Cost analysis of myasthenia gravis from a large U.S. insurance database.

INTRODUCTION: Little is known about the costs of managing rare diseases, and comprehensive healthcare costs have not been reported for myasthenia gravis (MG). We evaluated the direct costs and healthcare resource utilization in insured MG patients. METHODS: Costs were obtained from 1288 patients diagnosed with MG who were identified from the Accordant Health Services nationwide medical and pharmacy claims database. RESULTS: Average annual medical/pharmacy claims costs per patient were: $6710/$1196 (age 0-19 years); $17,949/$19,573 (20-39 years); $15,112/$12,498 (40-64 years); and $12,597/$8,089 (65(+) years). Total annual MG-related pharmacy costs were $9.4 million; IVIg accounted for 85% of all MG-related pharmacy costs. Non-steroidal immunosuppressives, cholinesterase inhibitors, and corticosteroids accounted for 9.3%, 5.7%, and 0.2% of pharmacy costs, respectively. CONCLUSIONS: Costs related to the treatment of MG are higher than those of many other chronic neurological diseases. A large percentage of costs result from IVIg use, particularly among a subset of patients who receive frequent IVIg infusions.

Plasma exchange versus intravenous immunoglobulin for myasthenia gravis crisis: an acute hospital cost comparison study.

OBJECTIVE: To compare the short-term financial costs of treating a patient in myasthenia gravis crisis with intravenous immunoglobulin (IVIG) versus plasma exchange. METHODS: An itemized comparative cost-minimization analysis of IVIG versus plasma exchange for myasthenia gravis crisis was performed. Calculations were based on each therapy's implementation cost, associated hospitalization times, and predicted cost to treat known complications. A cost superiority determination was proposed based on the total cost profile of each therapy. RESULTS: The difference in total cost favored IVIG over plasma exchange with an average savings of $22,326 per patient. Sensitivity analysis demonstrated that overall costs are highly dependent on IVIG dosing, hospital lengths of stay, and the number of plasma exchange days required. CONCLUSIONS: The use of IVIG for myasthenia gravis crisis may be a short-term cost minimizing therapy compared with plasma exchange. Additional prospective studies are required to evaluate the extended cost profile and efficacy of these therapies.

Comparative analysis of therapeutic options used for myasthenia gravis.

OBJECTIVE: To compare clinical and economic outcomes following plasma exchange (PLEX) and intravenous immunoglobulin (IVIG) in U.S. patients with primary diagnoses of myasthenia gravis (MG). METHODS: Our cohort was identified from the Nationwide Inpatient Sample database for years 2000-2005 using codes from the International Classification of Diseases, 9th edition. Multivariate regression analyses were used to identify predictors of mortality, complications, length of stay, and total inpatient cost. RESULTS: Among 1,606 hospitalized patients, the unadjusted mortality rate of MG crisis remained higher than those without crisis (0.44% vs 4.44%, p < 0.001), as well as the unadjusted complication rate (26.36% vs 11.23%, p < 0.001). MG crisis patients receiving PLEX had significantly more complications than those receiving IVIG (30.06% vs 14.79%, p < 0.001). Among the whole cohort, adjusted mortality and complication rates were not significantly different between the treatment groups (p > 0.05). Acute respiratory failure, major cardiac complications, and acute renal failure were associated with an increased mortality rate (p < 0.001). Age and respiratory failure were associated with an increased complication rate (p < 0.001). Length of stay was significantly longer for MG (6 vs 4 days, p < 0.001) and MG crisis (10 vs 5 days, p < 0.001) patients receiving PLEX. Inpatient costs were higher for MG ($26,662 vs $21,124, p < 0.01) and MG crisis ($53,801 vs $33,924, p < 0.001) patients receiving PLEX. INTERPRETATION: Compared to PLEX, IVIG appears of similar clinical (mortality and complications) and perhaps of superior economic (length of stay and total inpatient charges) outcomes in the treatment of MG. Elderly and those with complex comorbid diseases including acute respiratory failure may be better treated with IVIG.

Nationwide population-based epidemiological study of myasthenia gravis in taiwan.

BACKGROUND: The purpose of this study is to investigate the epidemiology and medical expenses of myasthenia gravis (MG) in Taiwan. METHODS: Cases of MG were identified from the National Health Insurance Research Database with corresponding codes of the International Classification of Diseases, ninth revision (ICD-9), from January 2000 to December 2007. Age- and sex-specific incidences were estimated by dividing the incidence number by population data obtained from the Department of Statistics, Ministry of the Interior. RESULTS: During the study period, 5,211 cases were identified. The incidence ratio of males to females was 0.68. The average annual incidence rate was 2.1/100,000. MG occurred in all age groups with a higher incidence in older individuals and the lowest incidence in the 10- to 14-year-olds for both sexes. Among the 5,211 cases, 615 (12%) had a neoplasm of the thymus. The prevalence increased steadily during the study period from 8.4/100,000 in 2000 to 14.0/ 100,000 in 2007. CONCLUSIONS: This is the first population-based epidemiological study of MG in Taiwan. The incidence rate and prevalence were higher than in most published studies, especially in old age groups.

Socioeconomic burden of amyotrophic lateral sclerosis, myasthenia gravis and facioscapulohumeral muscular dystrophy.

Neuromuscular disorders (NMD) are chronic devastating diseases. The aim of this multicenter cross-sectional study was to evaluate the socioeconomic impact of three NMDs in Germany. Patients (n = 107) with amyotrophic lateral sclerosis (ALS), myasthenia gravis (MG) or facioscapulohumeral muscular dystrophy (FSHD) were recruited consecutively in seven centers in Germany. The health-economic data were collected using a "bottom-up" approach consisting of comprehensive questionnaires and patient diaries. Costs were evaluated from the societal perspective in 2009 Euros (EUR). Total annual costs from the societal perspective were EUR 36,380 (95% CI 27,090-47,970) per patient in ALS, EUR 26,240 (95% CI 17,770-37,940) in FSHD and EUR 14,950 (95% CI 10,470-21,730) in MG. The main components of costs were the expenditures of health insurance and the loss of productivity of patients and their caregivers. The following independent cost-driving factors were identified: disease severity, assistance in activities of daily living (ADL), dementia and younger age in ALS, disease severity in FSHD and assistance in ADL, disease severity and assistance in ADL in MG. The socioeconomic burden of NMDs in Germany is considerable. Further studies evaluating both the health-economic and clinical effects of NMD treatment as well as disease management programs and benchmarking activities are necessary.

The cost-effectiveness of open or thoracoscopic thymectomy compared to medical treatment in managing Myasthenia gravis without thymomas / Costo efectividad de timectomía abierta y toracoscópica frente al tratamiento médico, en el manejo de Miastenia gravis sin timoma

Objective Assessing the cost-effectiveness of open or thoracoscopic thymectomy compared to medical therapy in managing myasthenia gravis not associated with thymoma. Methods A Markov model was designed for evaluating three strategies' cost-effectiveness. Transition probabilities were taken from the pertinent literature; the costs were estimated from official tariff manuals. Incremental cost-effectiveness ratios were estimated and probabilistic and deterministic sensitivity analysis was used for clinical variables, costs and the model's assumptions. Results Thoracoscopic thymectomy was the most effective and least costly strategy and dominated the other two alternatives. The cost per life year gained was Col § 1 129 531 without discount and Col § 805 179 with discount. Univariate sensitivity analysis showed that the main variables affecting the results were the effects' discount rate, the cost of a myasthenic crisis and the probability of complete remission. Thoracoscopy thymectomy was the most cost-effective strategy for different thresholds of willingness to pay in probabilistic analysis. Conclusions Thoracoscopic thymectomy is a cost-effective strategy in the treatment of MG without thymoma.

Objetivo Evaluar la costo efectividad de las timectomía abierta y toracoscópica frente a la terapia mé,dica en el manejo de miastenia gravis sin timoma. Mé,todo Se construyó un modelo de Markov para evaluar la costo efectividad de las 3 estrategias. Las probabilidades de transición se obtuvieron de la literatura. Los costos se estimaron a partir de las tarifas oficiales. Se calculó la costo-efectividad incremental. Se realizaron análisis de sensibilidad probabilísticos y determinísticos para las variables clínicas, los costos supuestos del modelo. Resultados La timectomía toracoscópica es la estrategia más efectiva y menos costosa, y domina a las otras dos alternativas. El costo por año de vida ganado fue de § 1 129 531 y § 805 179 pesos colombianos, con y sin descuento. El análisis de sensibilidad univariado mostró que las principales variables que afectan los resultados son la tasa de descuento, el costo de una crisis miasté,nica y la probabilidad de remisión completa. En el análisis de sensibilidad probabilístico, la timectomía toracoscópica es la estrategia costo-efectiva para los diferentes umbrales de disponibilidad a pagar. Conclusiones La timectomía toracoscópica es una estrategia costo-efectiva en el tratamiento de miastenia gravis sin timoma.