Current medical research and opinion
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Observational Study
Outcomes and Resource Use of Patients with Large Hemispheric Infarction and Cerebral Edema: Analysis of Real-world Data.
Large hemispheric infarction (LHI) is associated with a high likelihood of the evolution of life-threatening edema. Few studies have assessed real-world clinical outcomes and management strategies among patients with LHI. The objective of this study was to describe the management, in-hospital outcomes, and direct healthcare resource burden of patients with LHI, as well as those of patients with subsequent cerebral edema. ⋯ Among patients with ischemic strokes, LHI was associated with increased clinical management and direct healthcare resource burden in real-world hospital settings. The burden was substantially increased among patients who developed cerebral edema.
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Sickle cell disease (SCD) is a lifelong blood disorder affecting approximately 100,000 individuals in the United States (US). A number of new treatments have recently become available to improve SCD clinical outcomes, but it is unclear how treatment innovations that reduce disease severity could affect patients' humanistic and economic outcomes. ⋯ In conclusion, disease severity, strongly predicted worse self-reported HRQoL, moderately predicted increased likelihood of collecting disability insurance, and weakly predicted lower household income levels.
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To critically review researchers' use of diagnosis codes to identify congenital cytomegalovirus (cCMV) infection or disease in healthcare administrative databases. Understanding the limitations of cCMV ascertainment in those databases can inform cCMV surveillance and health services research. ⋯ The administrative prevalence of cCMV cases reported in published analyses of administrative data from North America, Western Europe, Japan, and Australia (0.6-3.8 per 10,000 infants) is an order of magnitude lower than the estimates of the true birth prevalence of 3-7 per 1,000 newborns based on universal newborn screening pilot studies conducted in the same regions. Nonetheless, in the absence of systematic surveillance for cCMV, administrative data might be useful for assessing trends in testing and clinical diagnosis. To the extent that cCMV cases recorded in administrative databases are not representative of the full spectrum of cCMV infection or disease, per-child cost estimates generated from those data may not be generalizable. On the other hand, claims data may be useful for estimating patterns of healthcare use and expenditures associated with combinations of diagnoses for cCMV and known complications of cCMV.
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Plexiform neurofibroma (PN) is one of the most striking clinical features of neurofibromatosis 1. Growth of PN can occur at any stage of life but mostly in childhood and during hormonal changes. They arise from multiple nerve fascicles and may transform into malignant peripheral nerve sheath tumors. ⋯ Approval was based on one pivotal, single-arm, phase II trial. 70% of participants experienced confirmed partial response of tumor shrinkage, and 68% also had improvement of related complications, and other studies have also shown beneficial responses. The major limitation of this molecule regarding its mechanism of action is the dose-dependent effect of MEK inhibition in growth of neurofibroma. Long-term safety and efficacy studies are to be done in the future to establish selumetininb as a useful medicine.