scholarly journals A snapshot of “real world” current neuro-oncology practice in ten UK centres participating in the Tessa Jowell BRAIN MATRIX platform study

2021 ◽  
Vol 23 (Supplement_4) ◽  
pp. iv17-iv18
Author(s):  
Victoria Wykes ◽  
John R Apps ◽  
Joshua Savage ◽  
Sara Meade ◽  
Ute Pohl ◽  
...  

Abstract Aims The TJBM Platform Study (https://www.birmingham.ac.uk/research/crctu/trials/brain-matrix) is a programme of work aimed at improving the knowledge of, and treatment for, glioma. The study will develop a backbone infrastructure for a molecular diagnostic pathway, particularly epigenetic classification and whole genome sequencing (WGS), and a data-repository of disease imaging, including real-time centralised Response Assessment in Neuro-Oncology (RANO) review, treatment and outcome data. Developed with the involvement of patients and relatives, patient reported outcome measures (PROMs), particularly quality of life assessment will be collected for patients. We present the feasibility data collected from the initial ten UK centres selected to participate in the TJBM study. This presents an opportunity to understand current local neuro-oncology practice, identify differences between services and chances to optimise these, bearing in mind the heterogeneity in our patient populations, staff and hospital facilities. Method Ten UK centres were selected to participate in the TJBM platform study, and each centre completed a multi-disciplinary feasibility questionnaire to facilitate participation and collaboration across the centres. Data were collected from hospital electronic MDT (Multi-Disciplinary Team) records, clinic letters, operative and imaging notes, MDT or personal experience. Continuous variables were reported using medians and ranges due to the non-normality of the data and categorical variables were reported as numbers and percentages. Tables and bar charts were generated to display relevant data. Analysis and plots were generated using Microsoft Excel sheets and SPSS (IBM) version 26. Results Work load Between 2016-2018 service provision redistribution reflects a trend towards higher volume centres. Overall, glioma workload within ten TJBM centres has remained stable (Figure 1). Imaging All TJBM centres have good access to imaging techniques and neuroradiology expertise, including relevant ‘advanced’ imaging. All have RANO capability, although not widely used clinically. Neurosurgery All centres have access to 5 ALA, perform awake craniotomy for language assessment and motor/sensory mapping are typically performed asleep, with subtle variation in techniques. Pathology Despite molecular analysis advances, current practice is limited to the evaluation of formalin embedded tissue by traditional morphology/ immuno-histochemical staining, with limited targeted testing of specific genetic changes (Figure 2). Clinical oncology Oncology treatments for glioma were as per NICE guidance with some minor local variation. The data has informed the development of the TJBM protocol, an overview of which will be presented (Figure 3). Conclusion Through systematic real-world data collection the TJBM platform study will provide a detailed understanding of practice within the UK, linked to molecular tumour genotype, treatment response outcome measures, and also regular quality of life assessments. Use of the platform infrastructure, will facilitate trials and add-on observational and biological studies to obtain rapid, efficient, and cost-effective data collection, and integrate findings with comprehensive molecular biological profiling and radiological features, thereby reducing the time and administrative burdens in trial delivery. This infrastructure will help establish a trial-competent network on which future research and collaborations can be based. Academic and industry partners will be able to use the TJBM platform through collaboration, overseen by a strong governance framework. This will maximise the opportunities and abilities to translate advances into trials and patient benefit.

Author(s):  
Alexandra N Nowbar ◽  
Darrel P Francis ◽  
Rasha K Al-Lamee

AbstractThe main aims of therapy in chronic stable angina are to reduce the risk of myocardial infarction and death and improve symptoms and quality of life (QoL). Unblinded trials have shown that revascularization does not reduce the risk of myocardial infarction or death but does appear to improve symptoms. However, symptoms are susceptible to the placebo effect which can bias therapies to appear more effective than they are. To assess the true physical impact of a treatment on symptoms, placebo-controlled trials with patients and medical and research teams blinded to treatment allocation are necessary. Symptoms and QoL can be reported directly by the patient or indirectly by the physician. Patient-reported outcome measures in angina trials can include angina frequency, frequency of nitrate use, exercise capacity, and questionnaires such as the Seattle Angina Questionnaire (SAQ) and the generic EuroQOL-5D-5L (EQ-5D-5L) QoL questionnaire. Physician-assessed outcome measures include Canadian Cardiovascular Society Class. The Objective Randomised Blinded Investigation with Optimal Medical Therapy of Angioplasty in Stable Angina (ORBITA) trial was the first blinded placebo-controlled study investigating the role of percutaneous coronary intervention (PCI) in chronic stable angina. The trial showed a smaller than expected and not statistically significant effect of placebo-controlled PCI on the primary endpoint of change in exercise time at 6 weeks follow-up in single-vessel coronary artery disease. There was also no significant placebo-controlled treatment effect of PCI for the prespecified secondary endpoints of SAQ or EQ-5D-5L, although PCI did result in 20% more patients becoming free from angina than placebo in a non-prespecified secondary analysis. ORBITA has demonstrated the need for symptomatic and QoL effects of PCI to be studied using placebo control. Here, we describe ways of measuring QoL, the impact of the unblinded and blinded trials to date, what we have learned from ORBITA, and what is next for this common and complex condition.


Author(s):  
Cecilia Durán ◽  
María del Carmen Abreu ◽  
Juan J. Dapueto

PURPOSE: The aim of the study was to evaluate the Assessment and Orientation Treatment Program for children and adolescents with disabilities treated at the Center Teletón Uruguay from the perspective of the patients and their parents based on several patient reported outcome measures: health related quality of life (HRQOL), treatment satisfaction, family impact, and emotional distress. METHODS: The sample consisted of all the 126 dyads of children/adolescents with ages ranging from 2 to 18 years and their primary caregivers who entered the program from April to October 2012. A set of instruments were filled out by children and parents before and six months after the program. T test for paired samples for continuous variables and McNemar's test for categorical variables were used to assess changes pre and post intervention. RESULTS: There was a high prevalence of depression and anxiety in the caregivers. In the second evaluation, improvements that were statistically significant only for the caregivers were observed in the HRQOL of children, adolescents and caregivers. CONCLUSIONS: The patient reported outcome measures used were effective in detecting changes in several areas of the quality of life of children observed by their caregivers and in their own quality of life after the intervention.


2021 ◽  
Vol 264 ◽  
pp. 394-401
Author(s):  
Mary Kate Luddy ◽  
Rachel Vetter ◽  
Jessica Shank ◽  
Whitney Goldner ◽  
Anery Patel ◽  
...  

2021 ◽  
Author(s):  
Jalal Maghfour ◽  
Torunn Elise Sivesind ◽  
Cory A. Dunnick ◽  
Robert Paul Dellavalle

BACKGROUND While there has been an increase in the number of randomized, controlled trials (RCTs) evaluating treatment efficacy for HS, instrument measurements of disease severity and quality of life (QoL) are varied, making compilation of data and comparison between studies a challenge for clinicians. OBJECTIVE The aim of this review is to evaluate trends in disease severity scales and patient reported outcome measures used in RCTs assessing treatment interventions among HS patients. METHODS A primary systematic literature review was conducted in August 2020. PubMed/MEDLINE, Embase, Web of Science, and Cochrane databases were used to identify all articles published from January 1964 to July 2020. The study was prospectively registered with PROSPERO (ID: 209582). Twenty-five articles were included in the systematic review. RESULTS Sartorius and modified Sartorius scores (n=8), and Hidradenitis Suppurativa Clinical score (HiSCs) (n=8) were the most commonly used instruments for disease severity. Participants’ pain, followed by Dermatology Life Quality Index (DLQI), were the most common QoL measures used in the reviewed studies. CONCLUSIONS Heterogeneity of data characterizing both the validity and reliability of existing outcome measures hinders interpretation and translation of the results from RCTs into clinical practice. Many of the QoL measures identified were not specific to HS and may not be representative of all factors impacting patients.


Author(s):  
Diego Gómez Herrero ◽  
Rafael Sanjuan-Cerveró ◽  
Pedro Vazquez-Ferreiro ◽  
Francisco Javier Carrera-Hueso ◽  
Marina Sáez-Belló ◽  
...  

Objective: The objective of this study is to carry out a systematic review of the outcome measures reported by the patient that are used to measure the quality of life of patients with Dupuytren´s disease (DD), assessing their relevance and effectiveness. Methods: A systematic literature search was carried out in the PubMed®, Web of Sciencie®, SciELO®, EMBASE®, Google Scholar® and Cochrane® databases. We searched for peer-reviewed articles evaluating health related quality of life (HR-QoL) in patients with DD diagnosed and/or treated until April 1, 2017, for English or Spanish language. The following keywords were used: “Dupuytren´s disease (MeSH)” AND “health related quality of life (MeSH)”. The documents were eligible for inclusion if they described data on the HR-QoL domains in relation to diagnosis or treatment of DD after a revision process by two independent authors. The checklist (STROBE) was used to evaluate the quality of the works. Results: From 352 identified articles were finally selected 26 studies in the systematic review, mostly European. A total of nine outcomes measures specifically reported by the patient were identified: DASH (used in 13 of the 26 selected studies), Quick-DASH (8/26), MHQ (7/26), briefMHQ (1/26), URAM (4/26), POS-HAND/ARM (1/26), SDSS (1/26), DDSP (1/26) and CHFS (1/26) questionnaires. We analyze their quantitative results to evaluate the effectiveness and evaluate the methodological quality of the studies on the measurement properties of the results reported by patients related to health. Conclusion: More work is urgently needed in these areas before we can reach a consensus on which instrument is the best to assess functional deterioration and improvement in patients with DD.


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