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Non-factor vs. Factor Prophylaxis of Hemophilia A: A Comparison of Cost and Treatment Effectiveness Based on Real-World Data from the USA
30. 6. 2023 Source: HemophiliaIn the prophylactic treatment of patients with hemophilia A, particularly its severe form, concentrates of clotting factor VIII (FVIII) with extended half-life (EHL) are increasingly administered, or non-factor treatment, represented in clinical practice by FVIII mimetic antibody emicizumab. However, insights into the direct comparison of effectiveness and economic burden arising from prophylaxis with these modalities are limited, including data from real-world practice. One recently published study therefore attempted to conduct such a…
When to Suspect Dravet Syndrome?
In 2022, an international consensus panel of experts on the diagnosis and treatment of Dravet syndrome was published. This was created due to the recently approved specific treatments for this…29. 6. 2023 Source: Rare Diseases in Neurology
Medication in Patients with Myasthenia Gravis – What to Watch Out For?
According to current recommendations, the treatment of myasthenia gravis (MG) should be individualized, considering the severity of the disease, the type of autoantibodies, thymus pathology, and,…29. 6. 2023 Source: Rare Diseases in Neurology
Characteristics of Patients with Alpha-1 Antitrypsin Deficiency – Two-Year Data from the EARCO Registry
The international registry of individuals with alpha-1 antitrypsin deficiency (AATD) named EARCO has collected data on 1,044 patients from 15 countries, including the Czech Republic, over its first 2…28. 6. 2023 Source: Deficiency of Alpha-1-Antitrypsin
Nucleic Acid Amplification Tests in the Diagnosis of Sexually Transmitted Diseases
Based on 81 scientific papers published between 2010 and 2021, experts from Johns Hopkins University in Baltimore have prepared an overview of the current epidemiology, diagnosis, and treatment of…28. 6. 2023 Source: Genetics
How to Reduce the Risk of Venous Thromboembolism in Users of Combined Hormonal Contraceptives?
The use of combined oral contraceptives (COCs) is a known risk factor for thromboembolism. Therefore, we should consider the individual risk of each patient before prescribing COCs. Women with a…27. 6. 2023 Source: Genetics
What is new in the treatment of atopic dermatitis and how are comorbidities affecting it?
Allergic rhinitis, conjunctivitis, bronchial asthma, or autoimmune diseases like alopecia, vitiligo, celiac disease, and idiopathic inflammatory bowel diseases – these are comorbidities that often…
Tofacitinib in the Treatment of Ulcerative Colitis in Real-World Practice in the USA
A recently published study summarizes the characteristics of patients newly treated with tofacitinib for ulcerative colitis (UC), the impact of initiating tofacitinib on corticosteroid use, and…22. 6. 2023 Source: Intestinal Inflammations
Expanded Vaccination Option Against Pneumococci Thanks to 15valent Vaccine
Our arsenal for preventing pneumococcal diseases has been expanded with a 15-valent conjugate vaccine that includes important serotypes causing invasive pneumococcal diseases (IPD). The following…20. 6. 2023 Source: Regular Vaccination
Sports in People with Hemophilia – Possible and Safe
Active involvement in sports and an active lifestyle are important aspects of daily functioning for people with hemophilia. Modern treatment of this disease, including the prophylactic administration…19. 6. 2023 Source: Hemophilia with Movement
Analysis of Data from the EMPEROR-Preserved Study Revealed Whether Blood Pressure Affects the Effectiveness of Empagliflozin in Patients with Heart Failure with Preserved Ejection Fraction
Hypertension is among the most common etiological factors of heart failure with preserved ejection fraction (HFpEF), where pressure overload of the left ventricle leads to its eccentric hypertrophy…19. 6. 2023 Source: Heart Failure
Treatment for Transthyretin Cardiac Amyloidosis is Now More Accessible to Czech Patients
Transthyretin cardiac amyloidosis is a serious progressive disease that was until recently considered untreatable. In recent years, however, new treatment options targeting the underlying pathology…15. 6. 2023 Source: Amyloidosis
TTR amyloidosis – at the crossroads of cardiology and orthopedics
In wild-type transthyretin amyloid cardiomyopathy, extra-cardiac symptoms occur in addition to cardiac symptoms. Common and characteristic orthopedic manifestations include bilateral carpal tunnel…14. 6. 2023 Source: Amyloidosis
Clinical Profile and Outcomes of Patients with Cardiac Amyloidosis – Spanish Experience from Real Practice
Cardiac amyloidosis is caused by the deposition of amyloid fibrils in the myocardium and is among the diseases with significant diagnostic delay. Physicians from the University Hospital in Madrid…14. 6. 2023 Source: Amyloidosis
Meta-analysis of the efficacy and safety of dupilumab in the treatment of atopic dermatitis in children and adults
Moderate to severe atopic dermatitis (AD) is accompanied by strong inflammatory processes that can occur anywhere on the skin, intense itching, and pain. The biologic dupilumab is a promising…
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