Current medical research and opinion
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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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Cold agglutinin disease (CAD) is a rare form of autoimmune hemolytic anemia that may manifest in complement-mediated chronic hemolytic anemia, profound fatigue, and transient agglutination-mediated circulatory symptoms. This study compared the healthcare resource utilization (HRU) of patients with CAD with a matched non-CAD comparison cohort using national Danish health registry data. ⋯ Characterization of HRU among European patients with CAD has not previously been conducted. This study shows that patients with CAD utilize significant resources in Denmark. Increased HRU uses among patients with CAD before diagnosis presents opportunities for earlier diagnosis and management.
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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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Onasemnogene abeparvovec, a one-time intravenous gene replacement therapy, and nusinersen, an antisense oligonucleotide that requires ongoing intrathecal administration, have been evaluated as treatments for spinal muscular atrophy (SMA) type 1 in separate Phase III trials, but no head-to-head comparison studies have been conducted. Onasemnogene abeparvovec was compared with nusinersen using a matching-adjusted indirect comparison (MAIC) to estimate the treatment effect of onasemnogene abeparvovec relative to nusinersen for the treatment of symptomatic patients with SMA type 1 for up to 24 months of follow-up. ⋯ Despite limitations of the current MAIC analysis (mainly a small sample size for statistical testing, even for the pooled onasemnogene abeparvovec trials, and potential differences in prognostic and predictive factors between studies), the relative treatment effects in EFS, OS, and motor milestone achievement indicate that onasemnogene abeparvovec may offer continued benefit compared with nusinersen through 24 months of follow-up.