Retinitis Pigmentosa Clinical Measures and Repeatability Testing of Potential Outcome Measures

Author(s):  
2020 ◽  
Vol 17 (4) ◽  
pp. 437-445
Author(s):  
Irene Ciancarelli ◽  
Giovanni Morone ◽  
Marco Iosa ◽  
Stefano Paolucci ◽  
Loris Pignolo ◽  
...  

Background: Limited studies concern the influence of obesity-induced dysregulation of adipokines in functional recovery after stroke neurorehabilitation. Objective: To investigate the relationship between serum leptin, resistin, and adiponectin and functional recovery before and after neurorehabilitation of obese stroke patients. The adipokine potential significance as prognostic markers of rehabilitation outcomes was also verified. Methods: Twenty obese post-acute stroke patients before and after neurorehabilitation and thirteen obese volunteers without-stroke, as controls, were examined. Adipokines were determined by commercially available enzyme-linked immunosorbent assay (ELISA) kits. Functional deficits were assessed before and after neurorehabilitation with the Barthel Index (BI), modified Rankin Scale (mRS), and Functional Independence Measure (FIM). Results: Compared to controls, higher leptin and resistin values and lower adiponectin values were observed in stroke patients before neurorehabilitation and no correlations were found between adipokines and clinical outcome measures. Neurorehabilitation was associated with improved scores of BI, mRS, and FIM. After neurorehabilitation, decreased values of Body Mass Index (BMI) and resistin together increased adiponectin were detected in stroke patients, while leptin decreased but not statistically. Comparing adipokine values assessed before neurorehabilitation with the outcome measures after neurorehabilitation, correlations were observed for leptin with BI-score, mRS-score, and FIM-score. No other adipokine levels nor BMI assessed before neurorehabilitation correlated with the clinical measures after neurorehabilitation. The forward stepwise regression analysis identified leptin as prognostic factor for BI, mRS, and FIM. Conclusions: Our data show the effectiveness of neurorehabilitation in modulating adipokines levels and suggest that leptin could assume the significance of biomarker of functional recovery.


2002 ◽  
Vol 4 (3) ◽  
pp. 125-138 ◽  
Author(s):  
Jeffrey I. Greenstein

Historically, it has been difficult to demonstrate the effectiveness of treatments for multiple sclerosis (MS) because of the variability in the course of the disease, the lack of well-defined, reliable clinical measures, and the pervasiveness of poorly controlled clinical trials. Hence, to interpret the results of clinical trials in MS and make evidence-based decisions regarding treatment for their patients, neurologists should have a basic understanding of appropriate outcome measures and the necessary controls of a well-designed study. This paper reviews the controls required to test the efficacy of agents for the treatment of MS and offers examples of poorly controlled clinical trials to illustrate the problems in interpreting data without such controls. In addition, the outcome measures that should be used to assess the efficacy of treatments on the physical, inflammatory, and cognitive components of the disease are discussed. (Int J MS Care. 2002; 4: 125–131, 136–137)


Stroke ◽  
2021 ◽  
Author(s):  
Bruce Mason ◽  
Kirsty Boyd ◽  
Fergus Doubal ◽  
Mark Barber ◽  
Marian Brady ◽  
...  

Background and Purpose: Stroke is the second commonest cause of death worldwide and a leading cause of severe disability, yet there are no published trials of palliative care in stroke. To design and evaluate palliative care interventions for people with stroke, researchers need to know what measurable outcomes matter most to patients and families, stroke professionals, and other service providers. Methods: A multidisciplinary steering group of professionals and laypeople managed the study. We synthesized recommendations from respected United Kingdom and international consensus documents to generate a list of outcome domains and then performed a rapid scoping literature review to identify potential outcome measures for use in future trials of palliative care after stroke. We then completed a 3-round, online Delphi survey of professionals, and service users to build consensus about outcome domains and outcome measures. Finally, we held a stakeholder workshop to review and finalize this consensus. Results: We generated a list of 36 different outcome domains from 4 key policy documents. The rapid scoping review identified 43 potential outcome measures that were used to create a shortlist of 16 measures. The 36 outcome domains and 16 measures were presented to a Delphi panel of diverse healthcare professionals and lay service users. Of 48 panelists invited to take part, 28 completed all 3 rounds. Shared decision-making and quality of life were selected as the most important outcome domains for future trials of palliative care in stroke. Additional comments highlighted the need for outcomes to be feasible, measurable, and relevant beyond the initial, acute phase of stroke. The stakeholder workshop endorsed these results. Conclusions: Future trials of palliative care after stroke should include pragmatic outcome measures, applicable to the evolving patient and family experiences after stroke and be inclusive of shared decision-making and quality of life.


2019 ◽  
Vol 40 (4) ◽  
pp. 383-391 ◽  
Author(s):  
Daniel Waltho ◽  
Lucas Gallo ◽  
Matteo Gallo ◽  
Jessica Murphy ◽  
Andrea Copeland ◽  
...  

Abstract Background Reduction mammaplasty remains critical to the treatment of breast hypertrophy. No technique has been shown to be superior; however, comparison between studies is difficult due to variation in outcome reporting. Objectives The authors sought to identify a comprehensive list of outcomes and outcome measures in reduction mammaplasty. Methods A comprehensive computerized search was performed. Included studies were randomized or nonrandomized controlled trials involving at least 100 cases of female breast hypertrophy and patients of all ages who underwent 1 or more defined reduction mammaplasty technique. Outcomes and outcome measures were extracted and tabulated. Results A total 106 articles were eligible for inclusion; 57 unique outcomes and 16 outcome measures were identified. Frequency of patient-reported and author-reported outcomes were 44% and 88%, respectively. Postoperative complications were the most frequently reported outcome (82.2%). Quality-of-life outcomes were accounted for in 37.7% of studies. Outcome measures were either condition-specific or generic; frequencies were as low as 1% and as high as 5.6%. Five scales were formally assessed in the breast reduction populations. Clinical measures were defined in 15.1% of studies. Conclusions There is marked heterogeneity in reporting of outcomes and outcome measures in the literature. A standardized outcome set is needed to compare outcomes of various reduction mammaplasty techniques. Level of Evidence: 4


2016 ◽  
Vol 145 (4) ◽  
pp. 825-838 ◽  
Author(s):  
X. J. LEE ◽  
G. R. FULFORD ◽  
A. N. PETTITT ◽  
F. RUGGERI

SUMMARYMethicillin-resistant Staphylococcus aureus (MRSA) transmission in hospital wards is associated with adverse outcomes for patients and increased costs for hospitals. The transmission process is inherently stochastic and the randomness emphasized by the small population sizes involved. As such, a stochastic model was proposed to describe the MRSA transmission process, taking into account the related contribution and modelling of the associated microbiological environmental contamination. The model was used to evaluate the performance of five common interventions and their combinations on six potential outcome measures of interest under two hypothetical disease burden settings. The model showed that the optimal intervention combination varied depending on the outcome measure and burden setting. In particular, it was found that certain outcomes only required a small subset of targeted interventions to control the outcome measure, while other outcomes still reported reduction in the outcome distribution with up to all five interventions included. This study describes a new stochastic model for MRSA transmission within a ward and highlights the use of the generalized Mann–Whitney statistic to compare the distribution of the outcome measures under different intervention combinations to assist in planning future interventions in hospital wards under different potential outcome measures and disease burden.


2007 ◽  
Vol 22 (16) ◽  
pp. 2371-2377 ◽  
Author(s):  
Susanne May ◽  
Sid Gilman ◽  
B. Brooke Sowell ◽  
Ronald G. Thomas ◽  
Matthew B. Stern ◽  
...  

Lung Cancer ◽  
2012 ◽  
Vol 75 ◽  
pp. S62-S63
Author(s):  
A. Sharkey ◽  
S. Begum ◽  
D. Hopkinson ◽  
T. Locke ◽  
J. Rao ◽  
...  

2020 ◽  
Vol 8 ◽  
Author(s):  
Leigh-ann Onnis ◽  
Irina Kinchin ◽  
Josephine Pryce ◽  
Priscilla Ennals ◽  
Joe Petrucci ◽  
...  

There is increasing demand for mental health services to be accessible to diverse populations in flexible, yet, cost-effective ways. This article presents the findings from a study that evaluated the process of implementing Connect to Wellbeing (CTW), a new mental health intake, assessment and referral service in regional Australia, to determine how well it improved access to services, and to identify potential measures that could be used to evaluate value for money. The study used a hybrid study design to conduct a process evaluation to better understand: the process of implementing CTW; and the barriers and factors enabling implementation of CTW. In addition, to better understand how to measure the cost-effectiveness of such services, the hybrid study design included an assessment of potential outcome measures suitable for ascertaining both the effectiveness of CTW in client health outcomes, and conducting a value for money analysis. The process evaluation found evidence that by improving processes, and removing waitlists CTW had created an opportunity to broadened the scope and type of psychological services offered which improved accessibility. The assessment of potential outcome measures provided insight into suitable measures for future evaluation into service effectiveness, client health outcomes and value for money.


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