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  • Lancaster Hunt posted an update 1 week, 3 days ago

    The AMBUVASC trial evaluated the cost effectiveness of outpatient vs. inpatient hospitalisation for endovascular repair of lower extremity arterial disease (LEAD).

    AMBUVASC was a national multicentre, prospective, randomised controlled trial conducted in nine public and two private French centres. The primary endpoint was the incremental cost effectiveness ratio (ICER), defined by cost per quality adjusted life year (QALY). Analysis was conducted from a societal perspective, excluding indirect costs, and considering a one month time horizon.

    From 16 February 2016 to 29 May 2017, 160 patients were randomised (80 per group). A modified intention to treat analysis was performed with 153 patients (outpatient hospitalisation n=76; inpatient hospitalisation n=77). The patients mainly presented intermittent claudication (outpatient arm 97%; inpatient arm 92%). Rates of peri-operative complications were 20% (15 events) and 18% (14 events) for the outpatient and inpatient arms respectively (p=.81). Overall costs (difference €187.83; 95% confidence interval [CI]-275.68-651.34) and QALYs (difference 0.00277; 95% CI -0.00237 – 0.00791) were higher for outpatients due to more re-admissions than the inpatient arm. The mean ICER was €67 741 per QALY gained for the base case analysis with missing data imputed using multiple imputation by predictive mean matching. The outpatient procedure was not cost effective for a willingness to pay of €50 000 per QALY and the probability of being cost effective was only 59% for a €100 000/QALY threshold.

    Outpatient hospitalisation is not cost effective compared with inpatient hospitalisation for endovascular repair of patients with claudication at a €50 000/QALY threshold.

    Outpatient hospitalisation is not cost effective compared with inpatient hospitalisation for endovascular repair of patients with claudication at a €50 000/QALY threshold.

    Outcomes of newly diagnosed multiple myeloma (NDMM) in developing regions have not paralleled those in developed settings. Economic disadvantage, comorbidities, and aggressive disease behavior play competing roles on defining outcomes. Our aim was to analyze the impact of socioeconomic characteristics and comorbidities on therapy initiation, drug selection, and survival outcomes of NDMM in a resource-constrained setting.

    This retrospective single-center cohort included≥ 18-year-old NDMM patients from January 2006 to December2018.

    A total of 245 patients were included with a median age of 62 years, Eastern Cooperative Oncology Group performance status≤ 2 in 70.2%, International Staging System score≥ 2 in 89.4%, and high-risk disease in 31.6%. Comorbidities were reported in 69.4%, and Charlson comorbidity index (CCI) was≥ 2 in 64.1%. A total of 87.4% (n= 214) received thalidomide-, alkylating-, and bortezomib-based induction in 67.8%, 18.2%, and 13.1%. selleck Patient-related factors including performance status,eatment-related factors are the main determinants of survival in NDMM induction-eligible patients. Patient-related factors play a pivotal role determining access to therapy and survival outcomes. Comorbidity index and performance status were determinant on defining therapy initiation in this real-world population, which emphasizes the need to improve health baseline conditions in resource-constrained settings.Patients affected by pulmonary arterial hypertension (PAH) benefit from intensive, continuous clinical monitoring to guide escalation of treatments that carry the potential to improve survival and quality of life. During the coronavirus disease 2019 pandemic, the need for physical distancing has fueled the expeditious expansion of various telehealth modalities, which may apply in a unique manner to individuals with PAH. Performance of objective risk assessments in patients with PAH remotely via telemedical visits and other telehealth mechanisms is unprecedented and not yet rigorously validated. The uniquely high risk for rapid deterioration in patients with PAH demands a high degree of sensitivity to detect changes in functional assessments. In this review, several telehealth modalities for potential utilization in risk assessment and treatment titration in patients with PAH are explored, yet additional study is needed for their validation with the pre-pandemic care paradigm.Demyelinating disorders of the central white matter are among the most prevalent and disabling conditions in neurology. Since myelin-producing oligodendrocytes comprise the principal cell type deficient or lost in these conditions, their replacement by new cells generated from transplanted bipotential oligodendrocyte-astrocyte progenitor cells has emerged as a therapeutic strategy for a variety of primary dysmyelinating diseases. In this review, we summarize the research and clinical considerations supporting current efforts to bring this treatment approach to patients.

    This study aimed to compare the absolute numbers of various types of lymphocytes in the peripheral blood before and after chemotherapy following radio-chemotherapy in patients with cervical cancer, so as to explore the correlation between the changes in the absolute numbers of peripheral various types of lymphocytes and the overall survival rate of patients.

    Data of 205 patients with cervical cancer admitted to the hospital from June 2014 to August 2016 were retrospectively analyzed. These patients underwent concurrent radio-chemotherapy, followed by chemotherapy. The absolute numbers of peripheral blood lymphocytes and subtypes were compared before and after re-chemotherapy.

    For patients with a good prognosis, the number of lymphocytes, T cells, and cytotoxic T cells (Tc) significantly decreased (P<0.05) after re-chemotherapy, while this phenomenon was not observed in patients with poor prognosis. Kaplan-Meier univariate analysis showed that patients with cervical cancer who had an advanced FIGO stan be considered as a predictor of the survival of patients with advanced cervical cancer.

    Nasopharyngeal carcinoma (NPC) radiotherapy (RT) irradiates parts of the brain which may cause cerebral tissue changes. This study aimed to systematically review the brain microstructure changes using MRI-based measures, diffusion tensor imaging (DTI), diffusion kurtosis imaging (DKI) and voxel-based morphometry (VBM) and the impact of dose and latency following RT.

    PubMed and Scopus databases were searched based on PRISMA guideline to determine studies focusing on changes following NPC RT.

    Eleven studies fulfilled the inclusion criteria. Microstructural changes occur most consistently in the temporal region. The changes were correlated with latency in seven studies; fractional anisotropy (FA) and gray matter (GM) volume remained low even after a longer period following RT and areas beyond irradiation site with reduced FA and GM measures. For dosage, only one study showed correlation, thus requiring further investigations.

    DTI, DKI and VBM may be used as a surveillance tool in detecting brain microstructural changes of NPC patients which correlates to latency and brain areas following RT.

    DTI, DKI and VBM may be used as a surveillance tool in detecting brain microstructural changes of NPC patients which correlates to latency and brain areas following RT.More than 80 genes are known to be associated with Charcot-Marie-Tooth disease (CMT). Mutations of LRSAM1 were identified as a rare cause and define the subgroup of axonal neuropathy CMT2P. We identified additional 14 patients out of 12 families. Clinical and electrophysiological data confirm a late-onset axonal neuropathy with a predominance of sensorimotor impairment. The patients harbored ten different variants in LRSAM1, seven of which were novel. Due to variable inheritance patterns and clustering of pathogenic variants in 3´-prime exons, interpretation of genetic variants in LRSAM1 is challenging. The majority follows dominant inheritance, whereas recessive inheritance has been described for one variant. Variants at the 3`end may or may not escape from nonsense-mediated decay, thereby defining the pattern of inheritance. Our data emphasize the importance of the C-terminal RING domain, which exerts a dominant-negative effect on protein function, whenever affected by an altered or truncated protein. In conclusion, CMT2P is a rare, but nevertheless relevant cause of adult-onset axonal and painful neuropathy. ACMG (American College of Medical Genetics and genomics) criteria should be carefully applied in variant interpretation, with special attention to premature termination codon-introducing variants and their location within the gene.

    Thymic carcinoma is a rare mediastinal neoplasm, and little is known about its genetic variability, which has hampered the development of targeted therapies.

    We tested a next-generation sequencing panel containing 50 common cancer-related genes in 48 cases of thymic carcinoma and 6 cases of thymic neuroendocrine tumor.

    We detected 42 variant calls in 21 of 54 cases. There was no significant difference in mutation frequency between thymic carcinoma and thymic neuroendocrine tumors. Among these, TP53 was the most frequently mutated gene (18.5%), followed by KIT (7.4%) and PDGFRA (5.6%). According to the gene pathways and groups, the p53 pathway, including TP53 and ATM, was most frequently affected (20.4%), followed by the receptor tyrosine kinase (RTK)/RAS pathway (18.5%) and PI3K pathway (5.6%). According to the OncoKB, an expert-guided precision oncology knowledge base, 7 genes among 10 cases (18.5%) were annotated with level 1 evidence, suggesting potentially therapeutic targets. Prognostic analyses, conducted in thymic squamous cell carcinomas, revealed that tumor cases harboring gene mutations in RTKs, including KIT (7.4%), PDGFRA (5.6%) and EGFR (3.7%), were significantly associated with a worse overall survival time (P= .0481). Among clinicopathologic factors, the advanced Masaoka stage was marginally associated with a worse overall survival (P= .0757). In the subsequent multivariate analysis, neither of the factors achieved statistical significance.

    In this preliminary next-generation sequencing study, we unexpectedly found evidence suggesting that several gene mutations might be therapeutic targets. The gene mutations in RTKs may be a valuable prognostic factor in thymic squamous cell carcinoma.

    In this preliminary next-generation sequencing study, we unexpectedly found evidence suggesting that several gene mutations might be therapeutic targets. The gene mutations in RTKs may be a valuable prognostic factor in thymic squamous cell carcinoma.

    We analyzed the survival trends for patients with metastatic lung cancer in California.

    We identified patients first diagnosed with primary lung cancer at distant (metastatic) stage in the California Cancer Registry between 1990 and 2014, with follow-up through end of 2015. link2 Race/ethnicity was categorized into non-Hispanic white, non-Hispanic black, Hispanic, and Asian/Pacific Islander. One-year and 5-year relative survival rates were calculated overall and by age at diagnosis, gender, race/ethnicity, and histology during the study period. Joinpoint regression was used to evaluate the trends and to calculate the annual percentage changes (APCs).

    A total of 186,156 adults were identified for analysis. link3 Between 1990 and 2014, 1-year relative survival significantly improved from 18.4% to 29.4%, with most improvement observed between 1993 and 2012 (APC, 2.60%; 95% confidence interval, 2.41-2.79; P< .01). Five-year relative survival significantly improved from 2.2% to 5.0%, with an APC of 4.05% (95% confidence interval, 3.