Current medical research and opinion
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Congenital hemophilia B is a rare bleeding disorder caused by defects in the gene encoding factor IX (FIX) leading to coagulation deficiency. Recurrent bleeds may cause chronic pain, disability, and reduced quality of life. Phase 2 b and 3 single-arm, open-label, single-dose trials assessing etranacogene dezaparvovec gene therapy for hemophilia B have demonstrated sustained FIX activity levels over observed periods, but long-term durability of the treatment effect has not been established. Using statistical modeling, we estimate long-term durability of FIX activity levels after receiving etranacogene dezaparvovec. ⋯ People with hemophilia B receiving etranacogene dezaparvovec would likely achieve durable FIX activity levels and remain free of prophylactic FIX replacement products for up to 25.5 years following single administration. The long-term factor IX durability predictions are based on statistical methods and results in vivo may differ.
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Observational Study
Psychiatric healthcare resource utilization following initiation of aripiprazole once-monthly: a retrospective real-world study.
This observational retrospective real-world study examined changes in healthcare resource utilization (HCRU) pre- and post-initiation of aripiprazole once-monthly (AOM 400) in patients with schizophrenia or bipolar I disorder. ⋯ A reduction in the rate of hospitalizations, emergency department visits, 30-day readmissions, and average length-of-stay was observed for patients diagnosed with either schizophrenia or bipolar I disorder, which suggests a positive effect of AOM 400 treatment on HCRU outcomes and is supportive of earlier analyses from different data sources.
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Review Meta Analysis
Effectiveness and safety of baricitinib in patients with alopecia areata: a systematic review and meta-analysis of randomized controlled trials.
Since there is now no medication available that has been approved by the US Food and Drug Administration, alopecia areata (AA) is an autoimmune condition that has a detrimental impact on individuals. Recent clinical trials using baricitinib demonstrated that it may be effective in treating AA. This meta-analysis was done to evaluate the effectiveness and safety of baricitinib in comparison to placebo. ⋯ When compared to placebo, baricitinib is an effective and well-tolerated medication for the treatment of AA.
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Review
Referral criteria to palliative care for patients with Parkinson's disease: a systematic review.
This systematic review aimed to identify the referral criteria for palliative care in patients with Parkinson's disease. ⋯ The 14 themes identified in this systematic review were categorized into disease- and needs-based criteria. These themes show the wide range of referral timing and procedures. Further studies should be conducted to reveal standardized referral criteria.