Current medical research and opinion
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Advanced-stage biliary tract cancers (BTC) are rare malignancies with poor prognosis. There are few prospective trials, but several retrospective studies regarding treatment options. In this study, we aimed to investigate the role of systemic inflammatory parameters (SIP) and other possible independent factors that may affect survival and treatment approaches and to determine the benefit of later-line treatments in these patients. ⋯ SII and NLR are useful prognostic factors and may be helpful in making treatment decisions. Additionally, second and later-line treatments in advanced BTC have a significant impact on survival under real-life conditions.
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As a residual risk factor for coronary heart disease, lipoprotein(a) [Lp(a)] is associated with the occurrence of cardiovascular events after percutaneous coronary intervention (PCI). The revascularization rate after PCI is high among diabetic patients. However, the relationship between Lp(a) and revascularization after PCI in type 2 diabetes mellitus (T2DM) patients with acute coronary syndrome (ACS) remains unclear. ⋯ Lp(a) was an independent predictor of revascularization in patients with ACS complicated with T2DM, regardless of LDL-C levels. This suggests that Lp(a) measurement may help identify high-risk diabetic patients with ACS.
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Factor VIII (FVIII) replacement and emicizumab have demonstrated efficacy for prevention of bleeds among patients with hemophilia A (PwHA) compared to on-demand (OD) use. Evidence investigating clinical outcomes and healthcare costs of non-inhibitor PwHA switching from prophylaxis with FVIII concentrates to emicizumab has not been well-established within large real-world datasets. This study aimed to investigate billed annualized bleed rates (ABRb) and total cost of care (TCC) among non-inhibitor PwHA switching from FVIII-prophylaxis to emicizumab-prophylaxis. ⋯ This study found that in male non-inhibitor PwHA, switching from FVIII prophylaxis to emicizumab incurs substantial cost increase with no significant benefit in ABRb. This evidence may help guide providers, payers, and patients in shared decision-making conversations around best treatment options.
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Friedreich ataxia (FA) is a rare, inherited neuromuscular disease characterized by an early onset and progressive limb and gait ataxia. Currently, there are no approved treatments for FA. It is important to understand the burden of FA, including its extent and the most salient elements. The objective of this study is therefore to systematically review the literature regarding the aspects of prevalence, health-related quality of life (HRQoL), and economic outcomes that are associated with FA, and to subsequently identify relevant knowledge gaps. ⋯ Findings from this systematic review revealed several knowledge gaps that would preclude the conduct of a robust assessment of the benefits and outcomes associated with a disease-modifying FA therapy. Additional understanding regarding patient and caregiver HRQoL and costs is required.
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Approximately one-third of patients with sepsis-associated acute kidney injury (AKI) progress to acute kidney disease (AKD) with higher short-term mortality. We aimed to identify the clinical characteristics that influence in-hospital death in sepsis-associated AKD and develop a nomogram to facilitate early warning. ⋯ We identified seven predictors of in-hospital death in patients with sepsis-associated AKD. In addition, we developed an online dynamic nomogram to accurately and conveniently predict short-term outcomes, which performed well in the external dataset.