Myasthenia gravis-treatment and severity in nationwide cohort.

INTRODUCTION: Myasthenia gravis (MG) is a rare autoimmune disorder of neuromuscular junction. MG healthcare burden has not been studied in Poland before. METHODS: Data were drawn from the National Health Fund database; MG patient was defined as a person who received at least once medical service with ICD-10 code MG (G70) and at least two reimbursed prescriptions for pyridostigmine bromide or ambenonium chloride in two consecutive years. We have analyzed treatment: immunosuppression, intravenous immunoglobulins (IVIg), plasma exchange (PE), the number and length of hospitalizations (LOS), intensive care unit (ICU) care, and deaths between 2013 and 2018. RESULTS: In 2018, there were 9012 MG patients (F:M 1.62:1), and 30.6% had early -onset MG (<50 years). 66.3% received symptomatic treatment only, 33.7%-glucocorticoids (CS) and/or other immunosuppressants (IS), 64.6%-CS only, 17.5%-azathioprine plus CS, 11%-azathioprine only, 4.6%-CS plus other IS (methotrexate, mycophenolate mofetil, cyclosporine, or tacrolimus), and 2%-other IS only. In 2018, 42.3% of patients were hospitalized at least once (mean 2.05/year), 13.7% due to MG (1.47/year). In 2018, 1.63% patients received PE, 2.33% IVIg. In 2013-2018, 2.7%-3.2% of MG patients required hospitalization in ICU. ICU mean LOS 2013-2018 was 11.5-15.0 days/per patient/year. 2.1% of all MG patients had myasthenic crisis. Mean age at death was 75.7 years for MG and 73.9 for general population (p = .006). All-cause mortality was higher for men (4.1%-5.1%) than for women (2.5%-3.1%), p < .01. CONCLUSIONS: Our findings confirm significant healthcare burden of MG, comprising a tool to plan resources needed for MG patients.

Spinal muscular atrophy: epidemiology and health burden in children - a Polish national healthcare database perspective before introduction of SMA-specific treatment.

INTRODUCTION: Spinal muscular atrophy (SMA) is one of the most frequent autosomal recessive neuromuscular disorders. It leads to progressive muscle weakness, premature death or permanent ventilation. Significant disability, scoliosis, severe pulmonary infections and other problems require in- and outpatient medical care. Various approaches have been used to evaluate SMA epidemiology, healthcare burden and adherence to standard of care. The recent introduction of pharmacological treatment in a large SMA population will change the course of the disease and the healthcare requirements of patients. MATERIAL AND METHODS: We have used the National Health Fund database to identify children with SMA and the healthcare service they received in the pre-pharmacological treatment era. Pivotal phase II and III medical trials for nusinersen were conducted between 2013 and 2015. The National Treatment Programme of SMA patients with nusinersen in our country was started in January 2019. The year 2014 was used to evaluate incident cases. RESULTS: 51 new SMA cases (incidence 1:7,356) and 518 SMA patients younger than 18 were identified in 2014. 32 (6.2%) deaths were recorded, half in the first two years of life. 35 (6.8%) patients received palliative and 115 (22.2%) long-term care (including assisted ventilation). A total number of 3,057 days of hospital stay were reported. Only 65/518 (12.6%) patients did not receive publicly-funded healthcare service other than specialist or general practitioner's consultation. CONCLUSIONS: SMA caused significant mortality and morbidity in children. The National Health Fund database can be used to reliably record incident cases and track the care provided to paediatric SMA patients.

Myasthenia Gravis in Poland: National Healthcare Database Epidemiological Study.

INTRODUCTION: Myasthenia gravis (MG) is a rare autoimmune disorder of the neuromuscular junction. MG epidemiology has not been studied in Poland in a nationwide study before. METHODS: Our epidemiological data were drawn from the National Health Fund (Narodowy Fundusz Zdrowia, NFZ) database; an MG patient was defined as a person who received at least once medical service coded in ICD-10 as MG (G70) and at least 2 reimbursed prescriptions for pyridostigmine bromide (Mestinon®) or ambenonium chloride (Mytelase®) in 2 consecutive years. RESULTS: On 1st of January 2019, 8,702 patients with MG were receiving symptomatic treatment (female:male ratio: 1.65:1). MG incidence was 2.36/100,000. The mean age of incident cases in 2018 was 61.37 years, 59.17 years for women and 64.12 years for men. Incidence of early-onset MG (<50 years) was 0.80/100,000 and 4.98/100,000 for late-onset MG (LOMG), with male predominance in LOMG. Prevalence was 22.65/100,000. In women, there was a constant increase in prevalence of symptomatic MG from the first decade of life up to 80-89 years. In men, an increase in prevalence appeared in the 6th decade. The highest prevalence was observed in the age group of 80-89 years: 59.65/100,000 in women and 96.25/100,000 in men. CONCLUSIONS: Our findings provide information on epidemiology of MG in Poland and can serve as a tool to evaluate healthcare resources needed for MG patients.