scholarly journals Clinical Scales for the Evaluation of Myopathy Patients

2021 ◽  
Vol 39 (2 Suppl) ◽  
pp. 15-23
Author(s):  
Hyung Jun Park ◽  
Young-Chul Choi
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Strength Function ◽  
Clinical Status ◽  
Validity And Reliability ◽  
Detailed Consideration ◽  
Clinical Scales ◽  
The Past ◽  
Mathematical Properties ◽  
Key Characteristics

With the rapid increase in the number of clinical trials in myopathy over the past decade, there is an increasing need for clinical scales to reflect patient’s clinical status. This article outlines the process of identifying possible measures. Detailed consideration has been given to key measures of muscle strength, function, and disability. As well as the usual assessment of the validity and reliability of the measures, three key characteristics were identified as necessary to the assessment of clinical scales used in health care: 1) the type of scale; 2) the clinical significance of the property being measured; and 3) the mathematical properties of the data. Consideration of such aspects facilitates the choice of clinical scales and the interpretation of data.

scholarly journals The inherent tensions and ambiguities of hope: Towards a post-formal analysis of experiences of advanced-cancer patients

2014 ◽  
Vol 19 (2) ◽  
pp. 207-225 ◽  
Author(s):  
Patrick Brown ◽  
Sabine de Graaf ◽  
Marij Hillen
Keyword(s):  
Health Care ◽  
Advanced Cancer ◽  
Formal Analysis ◽  
Social Processes ◽  
Power Dynamics ◽  
Advanced Cancer Patients ◽  
Detailed Consideration ◽  
Institutional Processes ◽  
Key Characteristics ◽  
Political Economies

Sociological and anthropological analyses of hope in health-care contexts have tended to address institutional processes, especially the power dynamics that function through such systems or political economies of hope, which in turn shape interactions through which hopes are managed. This article extends this approach through a more detailed consideration of the experience of hoping itself. Our post-formal analysis denotes the tensions that are intrinsic and defining features of lifeworlds around hope, emphasising the dissonance and fragility of hoping. Drawing upon interview and observational data involving patients with advanced-cancer diagnoses who were taking part in clinical trials, we explore three main tensions which emerged within the analysis: tensions involving time and liminality between future and present; ontological tensions involving the concrete and the possible, the ‘realistic’ and the positive; and tensions in taken-for-grantedness between the reflective and the mundane, the specific and the ambiguous. Rather than three separate sets of tensions, those involving time, ontology and taken-for-grantedness are very much interwoven. In denoting the influence of social processes in engendering tensions, we bridge sociological and anthropological approaches with a more definition-oriented literature, developing understandings of hoping and its key characteristics in relation to other processes of coping amidst vulnerability and uncertainty.

scholarly journals Oncolytic virotherapy for pancreatic cancer

2011 ◽  
Vol 13 ◽  
Author(s):  
Sonia Wennier ◽  
Shoudong Li ◽  
Grant McFadden
Keyword(s):  
Pancreatic Cancer ◽  
Clinical Trials ◽  
Molecular Biology ◽  
Cancer Biology ◽  
Oncolytic Viruses ◽  
Oncolytic Virotherapy ◽  
The Past ◽  
Unique Biology ◽  
Key Characteristics ◽  
Genetically Manipulated

Within the past decade, many oncolytic viruses (OVs) have been studied as potential treatments for pancreatic cancer and some of these are currently under clinical trials. The applicability of certain OVs, such as adenoviruses, herpesviruses and reoviruses, for the treatment of pancreatic cancer has been intensively studied for several years, whereas the applicability of other more recently investigated OVs, such as poxviruses and parvoviruses, is only starting to be determined. At the same time, studies have identified key characteristics of pancreatic cancer biology that provide a better understanding of the important factors or pathways involved in this disease. This review aims to summarise the different replication-competent OVs proposed as therapeutics for pancreatic cancer. It also focuses on the unique biology of these viruses that makes them exciting candidate virotherapies for pancreatic cancer and discusses how they could be genetically manipulated or combined with other drugs to improve their efficacy based on what is currently known about the molecular biology of pancreatic cancer.

scholarly journals Outcome Measures in Clinical Trials of Patients With Myasthenia Gravis

2020 ◽  
Vol 11 ◽  
Author(s):  
Jan Lykke Scheel Thomsen ◽  
Henning Andersen
Keyword(s):  
Clinical Trials ◽  
Myasthenia Gravis ◽  
Outcome Measures ◽  
Patient Reported Outcomes ◽  
Clinical Scales ◽  
The Past ◽  
Patient Reported ◽  
Specific Outcome ◽  
The Individual ◽  
Clinical Deficits

Myasthenia gravis (MG) is a heterogeneous disorder whose clinical presentation ranges from mild ocular deficits to severe widespread weakness. This variance poses a challenge when quantifying clinical deficits. Deficits and symptoms are quantified using standardized clinical scales and questionnaires which are often used as outcome measures. The past decades have seen the development of several validated outcome measures in MG, which are used in clinical trials to obtain regulatory approval. In recent years, emphasis has moved from objective assessments to patient-reported outcomes. Despite a growing body of literature on the validity of the MG-specific outcome measures, several unresolved factors remain. As several novel therapeutics are currently in clinical development, knowledge about capabilities and limitations of outcome measures is needed. In the present paper, we describe the most widely used clinical classifications and scales in MG. We highlight the choice of outcome measures in published and ongoing trials, and we denote whether trial efficacy was reached on these outcomes. We discuss advantages and limitations of the individual scales, and discuss some of the unresolved factors relating to outcome assessments in MG.

scholarly journals An analysis of certification processes for Good Clinical Practice and project management competencies

2021 ◽  
Vol 43 ◽  
pp. 179-205
Author(s):  
Marek Zawada ◽  
◽  
Dorota Gągała ◽  
Keyword(s):  
Clinical Trial ◽  
Clinical Trials ◽  
Clinical Practice ◽  
Project Management ◽  
Clinical Trial Data ◽  
Well Being ◽  
Good Clinical Practice ◽  
Validity And Reliability ◽  
Certification Process ◽  
Key Characteristics

Aim/purpose–The purpose of this paper is to clarify the certification process of the Good Clinical Practice (GCP) competencies based on a comparison with the project management (PM) certification process. This aim was accomplished by model development.Design/methodology/approach–The study was divided into three main phases: 1st phase –the identification of key characteristics of PM and GCP certification processes, 2nd phase –the development of certification models for PM and GCP, 3rd phase –the conclusions from in-depth interviews.Findings–As a result of the research, key characteristics of PM and GCP certification processes were identified, certification models for PM and GCP were developed. In addition, based on conclusions from in-depth interviews, solutions for organizing the way of confirming knowledge of GCP guidelines were proposed.Research implications/limitations–The proposed rules may be too complex and may exceed the needs and expectations of the clinical trial environment. The models focus on stakeholder relations, without the rules of certification granting procedures, to enable broader contextualization of the issues discussed in the paper. The analysis might be fragmented as it regards the sole certification process. The research refers to Poland only, hence it will be valuable to identify whether foreign authorities have the same attitude to the GCP competency certification model. It is recommended to increase the number of respondents’ interviews to obtain results of higher validity and reliability. Originality/value/contribution–The paper raises research topics at the crossroads of project management, clinical trials, and GCP, topics that have been underdeveloped so far. The results might be significant for all organizations involved in conducting clinical research projects. The findings contribute to the quality of clinical trials and provide public assurance that the rights, safety, and well-being of trial subjects are protected and the clinical trial data are credible.Keywords:project management, clinical trials, certification, Good Clinical Practice, GCP.JEL Classification:M10, O32, I11.

The Impact of War on Population Health

2014 ◽  
Vol 1 (1) ◽  
Author(s):  
Turkan Ahmet
Keyword(s):  
Health Care ◽  
Health Status ◽  
Health Care Services ◽  
Negative Effects ◽  
The Past ◽  
Care Services ◽  
Physical Environments ◽  
The Social ◽  
Personal Experiences ◽  
The Impact

The past few decades of ongoing war in Iraq has had a dramatic impact on the health of Iraq’s population. Wars are known to have negative effects on the social and physical environments of individuals, as well as limit their access to the available health care services. This paper explores the personal experiences of my family members, who were exposed to war, as well as includes information that has been reviewed form many academic sources. The data aided in providing recommendations and developing strategies, on both local and international levels, to improve the health status of the populations exposed to war.

scholarly journals What Has Clinical Research in Suicide Prevention Done for You Lately?

CNS Spectrums ◽  
2006 ◽  
Vol 11 (6) ◽  
pp. 440-441 ◽  
Author(s):  
Jan Fawcett
Keyword(s):  
Suicidal Ideation ◽  
Suicide Risk ◽  
Clinical Status ◽  
Panic Attacks ◽  
Current Evidence ◽  
Suicide Risk Assessment ◽  
Short Term ◽  
The Past ◽  
Standard Risk ◽  
Suicide Plan

What have you heard or read over the past 10 years that has improved you ability to assess and manage suicide risk in your patients?There has been a paucity of data. What little data there is reviewed in this month's articles.They highlight findings that you should know about. Clinicians seem to cling to the familiar, unless some intense marketing is done.For instance, are you aware that the current evidence shows that a denial of suicide thoughts, plans, or intent—even a contract for safety—means absolutely nothing in the absence of a full suicide risk assessment?Yet clinicians seem to rely on these ’reassurances“ from their patients and are shocked when the patient later commits suicide. Why should a patient who is deciding that life is too painful to live tell you the truth? Robert I. Simon, MD, and Daniel W. Shuman, JD, review these facts.Are you aware that severe psychic anxiety, panic attacks, agitation, and severe insomnia often precede suicide within hours, days, or weeks and can be rapidly modified with treatment?On the other hand, standard risk factors for suicide such as suicidal ideation, hopelessness, and past suicidal attempts are not good predictors of suicide in the short term. A suicide plan, recent high intent attempt, or refusal to contract for safety may well indicate immediate risk, but a denial of suicidal ideation or intent and a contract for no harm mean absolutely nothing without a full suicide assessment that takes current clinical status, past suicidal tendencies, social support, and willingness to accept help into account.

scholarly journals Creating Price Indexes for Measuring Productivity in Mental Health Care

2001 ◽  
Vol 4 (1) ◽  
Author(s):  
Susan H. Busch ◽  
Ernst R. Berndt ◽  
Richard G. Frank
Keyword(s):  
Mental Health ◽  
Health Care ◽  
Major Depression ◽  
Price Index ◽  
Time Varying ◽  
Measurement Issues ◽  
The Past ◽  
Price Indexes ◽  
Treatment Technologies ◽  
New Treatment

Economists have long suggested that to be reliable, a preferred medical care price index should employ time-varying weights to measure outcomes-adjusted changes in the price of treating an episode of illness. In this article, we report on several years of research developing alternative indexes for the treatment of the acute phase of major depression, for the period 1991–1996. The introduction of new treatment technologies in the past two decades suggests well-known measurement issues may be prominent in constructing such a price index.We report on the results of four successively re

scholarly journals ANCA Status or Clinical Phenotype — What Counts More?

2021 ◽  
Vol 23 (6) ◽  
Author(s):  
Martin Windpessl ◽  
Erica L. Bettac ◽  
Philipp Gauckler ◽  
Jae Il Shin ◽  
Duvuru Geetha ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Granulomatosis With Polyangiitis ◽  
Clinical Phenotype ◽  
Antineutrophil Cytoplasmic Antibody ◽  
Cardiovascular Death ◽  
Genetic Associations ◽  
Ongoing Debate ◽  
The Past ◽  
Anca Associated Vasculitis

Abstract Purpose of Review There is ongoing debate concerning the classification of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. That is, whether classification should be based on the serotype (proteinase 3 (PR3)- or myeloperoxidase (MPO)-ANCA) or on the clinical phenotype (granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)). To add clarity, this review focused on integration of the most recent literature. Recent Findings Large clinical trials have provided evidence that a serology-based risk assessment for relapses is more predictive than distinction based on the phenotype. Research conducted in the past decade indicated that a serology-based approach more closely resembles the genetic associations, the clinical presentation (i.e., lung involvement), biomarker biology, treatment response, and is also predicting comorbidities (such as cardiovascular death). Summary Our review highlights that a serology-based approach could replace a phenotype-based approach to classify ANCA-associated vasculitides. In future, clinical trials and observational studies will presumably focus on this distinction and, as such, translate into a “personalized medicine.”

scholarly journals Science for the Next Century: Deep Phenotyping

2021 ◽  
pp. 002203452110018
Author(s):  
J.T. Wright ◽  
M.C. Herzberg
Keyword(s):  
Health Care ◽  
Human Health ◽  
Care Delivery ◽  
The Past ◽  
Large Populations ◽  
Health And Disease ◽  
Methodological Approaches ◽  
Omic Data ◽  
Precision Health ◽  
Deep Phenotyping

Our ability to unravel the mysteries of human health and disease have changed dramatically over the past 2 decades. Decoding health and disease has been facilitated by the recent availability of high-throughput genomics and multi-omics analyses and the companion tools of advanced informatics and computational science. Understanding of the human genome and its influence on phenotype continues to advance through genotyping large populations and using “light phenotyping” approaches in combination with smaller subsets of the population being evaluated using “deep phenotyping” approaches. Using our capability to integrate and jointly analyze genomic data with other multi-omic data, the knowledge of genotype-phenotype relationships and associated genetic pathways and functions is being advanced. Understanding genotype-phenotype relationships that discriminate human health from disease is speculated to facilitate predictive, precision health care and change modes of health care delivery. The American Association for Dental Research Fall Focused Symposium assembled experts to discuss how studies of genotype-phenotype relationships are illuminating the pathophysiology of craniofacial diseases and developmental biology. Although the breadth of the topic did not allow all areas of dental, oral, and craniofacial research to be addressed (e.g., cancer), the importance and power of integrating genomic, phenomic, and other -omic data are illustrated using a variety of examples. The 8 Fall Focused talks presented different methodological approaches for ascertaining study populations and evaluating population variance and phenotyping approaches. These advances are reviewed in this summary.

scholarly journals A Roadmap to Inform Development, Validation and Approval of Digital Mobility Outcomes: The Mobilise-D Approach

2020 ◽  
Vol 4 (1) ◽  
pp. 13-27 ◽  
Author(s):  
Lynn Rochester ◽  
Claudia Mazzà ◽  
Arne Mueller ◽  
Brian Caulfield ◽  
Marie McCarthy ◽  
...  
Keyword(s):  
Health Care ◽  
Clinical Trials ◽  
Health Care Professionals ◽  
Digital Health ◽  
Disease Status ◽  
Proximal Femoral Fracture ◽  
Chronic Obstructive ◽  
Obstructive Pulmonary Disease ◽  
Physical Mobility ◽  
High Level

Health care has had to adapt rapidly to COVID-19, and this in turn has highlighted a pressing need for tools to facilitate remote visits and monitoring. Digital health technology, including body-worn devices, offers a solution using digital outcomes to measure and monitor disease status and provide outcomes meaningful to both patients and health care professionals. Remote monitoring of physical mobility is a prime example, because mobility is among the most advanced modalities that can be assessed digitally and remotely. Loss of mobility is also an important feature of many health conditions, providing a read-out of health as well as a target for intervention. Real-world, continuous digital measures of mobility (digital mobility outcomes or DMOs) provide an opportunity for novel insights into health care conditions complementing existing mobility measures. Accepted and approved DMOs are not yet widely available. The need for large collaborative efforts to tackle the critical steps to adoption is widely recognised. Mobilise-D is an example. It is a multidisciplinary consortium of 34 institutions from academia and industry funded through the European Innovative Medicines Initiative 2 Joint Undertaking. Members of Mobilise-D are collaborating to address the critical steps for DMOs to be adopted in clinical trials and ultimately health care. To achieve this, the consortium has developed a roadmap to inform the development, validation and approval of DMOs in Parkinson’s disease, multiple sclerosis, chronic obstructive pulmonary disease and recovery from proximal femoral fracture. Here we aim to describe the proposed approach and provide a high-level view of the ongoing and planned work of the Mobilise-D consortium. Ultimately, Mobilise-D aims to stimulate widespread adoption of DMOs through the provision of device agnostic software, standards and robust validation in order to bring digital outcomes from concept to use in clinical trials and health care.

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