Current medical research and opinion
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Randomized Controlled Trial
Duration and onset of effect of incobotulinumtoxina for the treatment of blepharospasm in botulinum toxin-naïve subjects.
Blepharospasm is a focal dystonia whereby excessive eyelid muscle contractions cause involuntary eye closure. Botulinum neurotoxin type A (BoNT-A) injections are an approved treatment. This randomized placebo-controlled trial (NCT01896895; EudraCT number 2012-004821-26) assessed the efficacy, safety, and treatment effect duration of incobotulinumtoxinA (Xeomin, Merz Pharmaceuticals GmbH), a BoNT-A formulation without complexing proteins, in BoNT-A-naïve adults with blepharospasm. ⋯ Subjects reported an effect onset from 5 days after injection lasting up to 20 weeks (maximum observation period). Data indicate that incobotulinumtoxinA re-treatment of blepharospasm may not be required at fixed 12-week intervals and provide evidence for a patient-tailored approach.
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To estimate the prevalence, incidence and economic burden of schizophrenia among Medicaid beneficiaries. ⋯ Annual prevalence and incidence of schizophrenia varied by state but remained stable over time. Adults with schizophrenia incurred greater HRU and costs relative to adults without schizophrenia; the burden appeared comparable among young adults.
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Randomized Controlled Trial
Clinical development and evaluation of a VEGF-D assay in plasma from patients with metastatic colorectal cancer in the RAISE study.
Vascular endothelial growth factor (VEGF)-D was identified as a potential predictive biomarker for ramucirumab efficacy in second-line metastatic colorectal cancer using a research use only (RUO) assay. We describe results with a new assay for detecting VEGF-D in human plasma. ⋯ In patients with high VEGF-D, ramucirumab demonstrated a greater improvement in OS and PFS vs placebo; however, baseline VEGF-D level was not predictive of ramucirumab OS benefit using VEGF-D assay for IUO. The RAISE intent-to-treat results remain valid.
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After fecal microbiota transplantation (FMT) to treat Clostridioides difficile infection (CDI), cognitive improvement is noticeable, suggesting an essential association between the gut microbiome and neural function. Although it is known that the gut microbiome is linked with cognitive function, whether FMT may lead to cognitive improvement in patients with neurodegenerative disorders remains to be elucidated. We present the case of a 90-year-old woman with Alzheimer's dementia and severe CDI who underwent FMT. ⋯ In addition, short-chain fatty acids were found to be significantly different between before and after FMT. This finding suggests the presence of an association between the gut microbiome and cognitive function. Further, it emphasizes the need for clinical awareness regarding the effect of FMT on the brain-gut-microbiome axis and its potential as a therapy for patients with dementia.
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Several novel treatments have been approved for relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL) since chimeric antigen receptor T-cell (CAR-T) therapy became available. The objective of this study was to describe characteristics and treatment patterns in patients with R/R DLBCL post-CAR-T approval. ⋯ Post-CAR-T approval, the majority of patients were treated with CT/CIT or targeted therapies in 3 L and 4 L, though most of the targeted therapies prescribed are not indicated for DLBCL. Treatment duration was short. A high proportion of patients moved to the next line of therapy (LOT) during a short follow-up period. This study highlights the unmet need for more effective treatments for patients with R/R DLBCL in 3 L+.