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[Severe poor nutrition following stomach: the need for lifelong follow-up].
A further transformation with a Tox3-Homology Directed Repair cassette harbouring a selectable marker with 50 bp micro-homology flanks was also achieved with 25% homologous recombination efficiency. The success of these homology directed repair approaches demonstrate that CRISPR/Cas9 is amenable to other in vivo DNA manipulation approaches such as the insertion of DNA and generating point mutations. Conclusion These data highlight the significant potential that CRISPR/Cas9 has in expediting transgene-free gene knockouts in Parastagonospora nodorum and also in facilitating other gene manipulation approaches. Access to these tools will significantly decrease the time required to assess the requirement of gene for disease and to undertake functional studies to determine its role. © The Author(s) 2020.Background Leptomeningeal dissemination (LD) in adults is an exceedingly rare complication of low-grade neuroepithelial CNS tumors (LGNs). We aimed to determine relative incidence, clinical presentation, and predictors of outcome. Methods We searched the quality control database of the Section of Neuro-Oncology, Yale Cancer Center, for patients with LGN (WHO grade I/II) seen between 2002 and 2017. For cases complicated by LD, we recorded demographics, clinical signs, histopathological diagnosis, and imaging findings. A comprehensive literature review was performed. Results Eleven consecutive patients with LD were identified, representing 2.3% of individuals with LGN seen at our institution between 2002 and 2017 (n = 475). Ependymoma was the predominant histological entity. Mean time interval from diagnosis of LGN to LD was 38.6 ± 10 months. Symptoms were mostly attributed to communicating hydrocephalus. Tumor deposits of LD were either nodular or linear with variable enhancement (nonenhancing lesions in 4 of 11 patients). Localized (surgery, radiosurgery, involved-field, or craniospinal radiation therapy) or systemic treatments (chemotherapy) were provided. All patients progressed radiographically. Median overall survival after LD was 102 months. Survival was prolonged when a combination of localized and systemic therapies was administered (188.5 vs 25.5 months; P = .03). Demographics and tumor spectrum reported in the literature were similar to our cohort. Conclusions LD is a rare complication of LGNs. A high level of suspicion is required for timely diagnosis as early symptoms are nonspecific and commonly do not occur until years after initial tumor diagnosis. Repeated aggressive treatment appears to be beneficial in improving survival. © The Author(s) 2019. Published by Oxford University Press on behalf of the Society for Neuro-Oncology and the European Association of Neuro-Oncology. All rights reserved. For permissions, please e-mail [email protected] Few supportive and palliative care interventions have been described for people with high-grade glioma (HGG) and their caregivers. A structured intervention, I-CoPE, which includes regular screening of patients' and caregivers' needs, was delivered as a pilot study in a tertiary neuro-oncological unit. This study prospectively described the severity and content of key concerns raised by patients and their caregivers in the 3 months following a diagnosis of HGG. Methods Consecutive patients and caregivers were screened for distress longitudinally using the Distress Thermometer at 3 time points. Exploration of the issues raised during screening was undertaken by the care coordinator and the issues documented. This documentation was subsequently audited to map patterns and nature of distress. Quizartinib mouse The key reasons underlying distress and responses offered were grouped thematically and summarized using descriptive statistics. Results Thirty-two patients and 31 caregivers were recruited and underwent [email protected] The incidence of symptomatic radiation necrosis (RN) has risen as radiotherapy is increasingly used to control brain tumor progression. Traditionally managed with steroids, symptomatic RN can remain refractory to medical treatment, requiring surgical intervention for control. The purpose of our study was to assess a single institution's experience with craniotomy for steroid-refractory pure RN. Methods The medical records of all tumor patients who underwent craniotomies at our institution from 2011 to 2016 were retrospectively reviewed for a history of preoperative radiotherapy or radiosurgery. RN was confirmed histopathologically and patients with active tumor were excluded. Preoperative, intraoperative, and outcome information was collected. Primary outcomes measured were postoperative KPS and time to steroid freedom. Results Twenty-four patients with symptomatic RN were identified. Gross total resection was achieved for all patients. Patients with metastases experienced an increase in KPS (80 vs 100, P less then .001) and required a shortened course of dexamethasone vs patients with high-grade gliomas (3.4 vs 22.2 weeks, P = .003). RN control and neurological improvement at 13.3 months' follow-up were 100% and 66.7%, respectively. Adrenal insufficiency after rapidly tapering dexamethasone was the only morbidity (n = 1). Overall survival was 93.3% (14/15) at 1 year. Conclusion In cases of treatment-refractory symptomatic RN, resection can lead to an overall improvement in postoperative health status and neurological outcomes with minimal RN recurrence. Craniotomy for surgically accessible RN can safely manage symptomatic patients, and future studies assessing the efficacy of resection vs bevacizumab may be warranted. © The Author(s) 2019. Published by Oxford University Press on behalf of the Society for Neuro-Oncology and the European Association of Neuro-Oncology. All rights reserved. For permissions, please e-mail [email protected] Though conformal partial-brain irradiation is the standard adjuvant treatment for glioblastoma, there is no consensus regarding the optimal volume that needs to be irradiated. European Organisation for Research and Treatment of Cancer (EORTC) and The University of Texas MD Anderson Cancer Center (MDACC) guidelines differ from the Radiation Therapy Oncology Group (RTOG) in their approach toward peritumoral edema, whereas RTOG and MDACC guidelines differ from EORTC in the concept of boost phase. A scarcity of randomized comparisons has resulted in remarkable variance in practice among institutions. Methods Fifty glioblastoma patients were randomized to receive adjuvant radiotherapy using RTOG or MDACC protocols. Apart from dosimetric and volumetric analysis, acute toxicities, recurrence patterns, progression-free survival (PFS), overall survival (OS), and quality of life (QoL) were compared using appropriate statistical tests. Results Both groups were comparable with respect to demographic characteristics.
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