Current medical research and opinion
-
To evaluate real-world annualized bleeding rates (ABRs), dosing frequency, and factor consumption of four recombinant FVIII (rFVIII) products using pooled data from centers in the US, Germany, and Italy. ⋯ rVIII-SingleChain prophylaxis may provide improved bleed protection, less frequent dosing, and lower consumption compared with standard-acting FVIII products, and comparable protection and consumption to the other long-acting FVIII product, in patients with hemophilia A.
-
To describe the trends in epidemiology, healthcare resource use (HCRU), and costs associated with Lewy body dementia (LBD), dementia with Lewy bodies (DLB), and Parkinson's disease dementia (PDD) in the United States. ⋯ Our findings highlight the substantial epidemiological and economic burden across the LBD spectrum and underscore a high unmet need for effective treatments to improve patient outcomes.
-
Aromatic L-amino acid decarboxylase deficiency (AADCd) is an ultra-rare genetic neurometabolic disorder caused by mutations in the DDC gene. ⋯ In terms of symptoms and healthcare resource utilization, the burden of illness of AADCd is substantial. This study provides insights into several aspects of the disease that are difficult to ascertain from published case reports.
-
To describe the design of the CLARION post-approval safety study (EU PAS Register number, EUPAS24484) and provide a status update, including characteristics of patients included up to 1 May 2021. ⋯ By providing further information on adverse events of special interest during long-term follow-up, CLARION will assist neurologists and patients regarding treatment decision-making for management of relapsing MS.
-
The role of personalized treatment approaches, including those based on genetic testing, are increasingly enabling informed decision-making to improve health outcomes. Research involving Indigenous Australians has been lagging behind, although this population experiences a higher prevalence of chronic disease and mental health disorders. ⋯ Indigenous Australians are under-represented in pharmacogenomics research despite well-documented epidemiological research demonstrating that Indigenous people globally experience greater risk of developing certain chronic diseases and more severe disease progression. Positive outcomes from this study highlight the importance of not only involving Indigenous participants, but providing leadership and governance opportunities for future genetics research.