scholarly journals A Canadian Adult Spinal Muscular Atrophy Outcome Measures Toolkit: Results of a National Consensus using a Modified Delphi Method

2021 ◽  
pp. 1-10
Author(s):  
Jeremy Slayter ◽  
Victoria Hodgkinson ◽  
Josh Lounsberry ◽  
Bernard Brais ◽  
Kristine Chapman ◽  
...  
Keyword(s):  
Spinal Muscular Atrophy ◽  
Outcome Measures ◽  
Delphi Method ◽  
Muscular Atrophy ◽  
Quality Data ◽  
High Quality ◽  
High Quality Data ◽  
Modified Delphi Method ◽  
Modified Delphi ◽  
Disease Modifying

Background: Spinal Muscular Atrophy (SMA) is a rare disease that affects 1 in 11 000 live births. Recent developments in SMA treatments have included new disease-modifying therapies that require high quality data to inform decisions around initiation and continuation of therapy. In Canada, there are no nationally agreed upon outcome measures (OM) used in adult SMA. Standardization of OM is essential to obtain high quality data that is comparable among neuromuscular clinics. Objective: To develop a recommended toolkit and timing of OM for assessment of adults with SMA. Methods: A modified delphi method consisting of 2 virtual voting rounds followed by a virtual conference was utilized with a panel of expert clinicians treating adult SMA across Canada. Results: A consensus-derived toolkit of 8 OM was developed across three domains of function, with an additional 3 optional measures. Optimal assessment frequency is 12 months for most patients regardless of therapeutic access, while patients in their first year of receiving disease-modifying therapy should be assessed more frequently. Conclusions: The implementation of the consensus-derived OM toolkit will improve monitoring and assessment of adult SMA patients, and enrich the quality of real-world evidence. Regular updates to the toolkit must be considered as new evidence becomes available.

Collecting High-Quality Data

2018 ◽  
Author(s):  
Mary Kay Gugerty ◽  
Dean Karlan
Keyword(s):  
Data Collection ◽  
Social Desirability ◽  
Measurement Errors ◽  
Monitoring And Evaluation ◽  
Quality Data ◽  
Social Desirability Bias ◽  
High Quality ◽  
High Quality Data ◽  
Evaluation Systems ◽  
Demand Effect

Without high-quality data, even the best-designed monitoring and evaluation systems will collapse. Chapter 7 introduces some the basics of collecting high-quality data and discusses how to address challenges that frequently arise. High-quality data must be clearly defined and have an indicator that validly and reliably measures the intended concept. The chapter then explains how to avoid common biases and measurement errors like anchoring, social desirability bias, the experimenter demand effect, unclear wording, long recall periods, and translation context. It then guides organizations on how to find indicators, test data collection instruments, manage surveys, and train staff appropriately for data collection and entry.

scholarly journals Treatment satisfaction in 5q-spinal muscular atrophy under nusinersen therapy

2021 ◽  
Vol 14 ◽  
pp. 175628642199890 ◽  
Author(s):  
Alma Osmanovic ◽  
Gresa Ranxha ◽  
Mareike Kumpe ◽  
Claudia D. Wurster ◽  
Benjamin Stolte ◽  
...  
Keyword(s):  
Side Effects ◽  
Spinal Muscular Atrophy ◽  
Outcome Measures ◽  
Treatment Satisfaction ◽  
Treatment Duration ◽  
Treatment Effectiveness ◽  
Muscular Atrophy ◽  
Patients Satisfaction ◽  
Motor Outcome ◽  
Ambulatory Patients

Background: Nusinersen was the first approved disease-modifying therapy for all 5q-spinal muscular atrophy (SMA) patients regardless of age or disease severity. Its efficacy in adults has recently been demonstrated in a large cohort by motor outcome measures, which were only partially suitable to detect changes in very mildly or severely affected patients. Patient-reported outcome measures (PROs) have been suggested as a valuable addition. Here, we aimed to assess treatment satisfaction and investigate whether it may be a useful PRO to monitor SMA patients. Methods: We enrolled 91 mainly adult 5q-SMA patients treated with nusinersen in a national, multicenter, cross-sectional observational study. 21 patients underwent longitudinal follow up. Patients’ satisfaction with treatment in four dimensions (global, effectiveness, convenience, side effects) was assessed by the Treatment Satisfaction Questionnaire for Medication German version 1.4 (TSQM-1.4©) and related to clinical parameters, motor scores, and treatment duration. Results: More than 90% of SMA patients were consistently satisfied over a median treatment duration of 10 months. Highest mean scores were observed in the dimensions ‘side effects,’ ‘global satisfaction,’ and ‘effectiveness’ (93.5 ± 14.8 versus 73.1 ± 21.0 and 64.8 ± 20.6, respectively). Patients’ satisfaction with the convenience of treatment was considerably lower (43.6 ± 20.2). Interestingly, satisfaction with the effectiveness was higher in ambulatory ( p = 0.014) compared with non-ambulatory patients and directly correlated to motor outcome measures. Five non-ambulatory patients withdrew from therapy. All of them presented with a deterioration of motor outcome measures and reported dissatisfaction with treatment effectiveness and convenience. Conclusion: Most patients were satisfied with nusinersen treatment effectiveness. Less severely affected patients indicated higher satisfaction. The TSQM-1.4© helped to identify therapy non-responders, who mainly addressed dissatisfaction with effectiveness and convenience. We suggest introducing the TSQM-1.4© as an additional PRO in SMA into clinical practice.

Derivation of a procedural performance checklist for ultrasound-guided femoral arterial line placement using the modified Delphi method

2020 ◽  
Vol 21 (5) ◽  
pp. 715-722
Author(s):  
Jessica Baez ◽  
Elizabeth Powell ◽  
Megan Leo ◽  
Uwe Stolz ◽  
Lori Stolz
Keyword(s):  
Delphi Method ◽  
Competency Assessment ◽  
Arterial Line ◽  
Ultrasound Guided ◽  
Arterial Access ◽  
Modified Delphi Method ◽  
Modified Delphi ◽  
Item Checklist ◽  
Standard Quality ◽  
Femoral Arterial Access

Background: Many specialties utilize procedural performance checklists as an aid to teach residents and other learners. Procedural checklists ensure that the critical steps of the desired procedure are performed in a specified manner every time. Valid measures of competency are needed to evaluate learners and ensure a standard quality of care. The objective of this study was to employ the modified Delphi method to derive a procedural checklist for use during placement of ultrasound-guided femoral arterial access. Methods: A 27-item procedural checklist was provided to 14 experts from three acute care specialties. Using the modified Delphi method, the checklist was serially modified based on expert feedback. Results: Three rounds of the study were performed resulting in a final 23-item checklist. Each item on the checklist received at least 70% expert agreement on its inclusion in the final checklist. Conclusion: A procedural performance checklist was created for ultrasound-guided femoral arterial access using the modified Delphi method. This is an objective tool to assist procedural training and competency assessment in a variety of clinical and educational settings.

Developmental Impacts of Remittances on Migrant-Sending Households: Micro-Level Evidence from Punjab, Pakistan

2019 ◽  
Vol 14 (3) ◽  
pp. 338-366
Author(s):  
Kashif Imran ◽  
Evelyn S. Devadason ◽  
Cheong Kee Cheok
Keyword(s):  
Human Development ◽  
Human Development Index ◽  
Quality Data ◽  
High Quality ◽  
High Quality Data ◽  
Development Index ◽  
Policy Interventions ◽  
Education And Health ◽  
Micro Level ◽  
The Government

This article analyzes the overall and type of developmental impacts of remittances for migrant-sending households (HHs) in districts of Punjab, Pakistan. For this purpose, an HH-based human development index is constructed based on the dimensions of education, health and housing, with a view to enrich insights into interactions between remittances and HH development. Using high-quality data from a HH micro-survey for Punjab, the study finds that most migrant-sending HHs are better off than the HHs without this stream of income. More importantly, migrant HHs have significantly higher development in terms of housing in most districts of Punjab relative to non-migrant HHs. Thus, the government would need policy interventions focusing on housing to address inequalities in human development at the district-HH level, and subsequently balance its current focus on the provision of education and health.

Capturing sexual assault data: An information system designed by forensic clinicians and healthcare researchers

2017 ◽  
Vol 47 (1) ◽  
pp. 46-55 ◽  
Author(s):  
S Aqif Mukhtar ◽  
Debbie A Smith ◽  
Maureen A Phillips ◽  
Maire C Kelly ◽  
Renate R Zilkens ◽  
...  
Keyword(s):  
Information System ◽  
Sexual Assault ◽  
Data Management ◽  
Sexual Violence ◽  
Western Australia ◽  
Management System ◽  
Data Management System ◽  
Quality Data ◽  
High Quality ◽  
High Quality Data

Background: The Sexual Assault Resource Center (SARC) in Perth, Western Australia provides free 24-hour medical, forensic, and counseling services to persons aged over 13 years following sexual assault. Objective: The aim of this research was to design a data management system that maintains accurate quality information on all sexual assault cases referred to SARC, facilitating audit and peer-reviewed research. Methods: The work to develop SARC Medical Services Clinical Information System (SARC-MSCIS) took place during 2007–2009 as a collaboration between SARC and Curtin University, Perth, Western Australia. Patient demographics, assault details, including injury documentation, and counseling sessions were identified as core data sections. A user authentication system was set up for data security. Data quality checks were incorporated to ensure high-quality data. Results: An SARC-MSCIS was developed containing three core data sections having 427 data elements to capture patient’s data. Development of the SARC-MSCIS has resulted in comprehensive capacity to support sexual assault research. Four additional projects are underway to explore both the public health and criminal justice considerations in responding to sexual violence. The data showed that 1,933 sexual assault episodes had occurred among 1881 patients between January 1, 2009 and December 31, 2015. Sexual assault patients knew the assailant as a friend, carer, acquaintance, relative, partner, or ex-partner in 70% of cases, with 16% assailants being a stranger to the patient. Conclusion: This project has resulted in the development of a high-quality data management system to maintain information for medical and forensic services offered by SARC. This system has also proven to be a reliable resource enabling research in the area of sexual violence.

scholarly journals Fooled by Performance Randomness: Overrewarding Luck

2019 ◽  
Vol 101 (4) ◽  
pp. 658-666 ◽  
Author(s):  
Romain Gauriot ◽  
Lionel Page
Keyword(s):  
Causal Effect ◽  
Experimental Situation ◽  
Quality Data ◽  
High Quality ◽  
High Quality Data ◽  
Economic Organizations ◽  
Quasi Experimental ◽  
Similar Location ◽  
Do So

We provide evidence of a violation of the informativeness principle whereby lucky successes are overly rewarded. We isolate a quasi-experimental situation where the success of an agent is as good as random. To do so, we use high-quality data on football (soccer) matches and select shots on goal that landed on the goal posts. Using nonscoring shots, taken from a similar location on the pitch, as counterfactuals to scoring shots, we estimate the causal effect of a lucky success (goal) on the evaluation of the player's performance. We find clear evidence that luck is overly influencing managers' decisions and evaluators' ratings. Our results suggest that this phenomenon is likely to be widespread in economic organizations.

scholarly journals R factors in Rietveld analysis: How good is good enough?

2006 ◽  
Vol 21 (1) ◽  
pp. 67-70 ◽  
Author(s):  
Brian H. Toby
Keyword(s):  
Rietveld Analysis ◽  
Poor Quality ◽  
Quality Data ◽  
High Quality ◽  
High Quality Data ◽  
Poor Quality Data ◽  
Error Index ◽  
R Factors ◽  
Very High

The definitions for important Rietveld error indices are defined and discussed. It is shown that while smaller error index values indicate a better fit of a model to the data, wrong models with poor quality data may exhibit smaller values error index values than some superb models with very high quality data.

Sign in / Sign up

Export Citation Format

Share Document