Translation of the Preference-Based Amyotrophic Lateral Sclerosis Scale into French.

The objective of this study was to translate the Preference-Based Amyotrophic Lateral Sclerosis Scale to French-Canadian. After the scale underwent forward and back translations, the expert committee examined the translated versions and found minor grammatical errors and suggested idioms to be changed to better represent French-Canadian language. Cognitive debriefing interviews were carried out to assess the pre-final version for clarity, and minor changes were made. Consensus from the expert committee and people with amyotrophic lateral sclerosis on the measure's clarity, word choice, and meaning were achieved, resulting in the final French version of the Preference-Based Amyotrophic Lateral Sclerosis Scale.

Cognitive Interviewing to Develop a New Health-Related Quality of Life Measure for Parkinson's Disease: The Preference-Based Parkinson's Disease Index (PB-PDI).

Preference-based measures (PBM) for health-related quality of life (HRQoL) are essential in assessing the cost-utility of different treatment options. The preference-based Parkinson's disease Index (PB-PDI) is being developed for people with Parkinson's disease (PD). The aim of this study was to refine the PB-PDI through cognitive interviews with people with PD. Cognitive debriefing was conducted to assess patients' interpretation of items, both in English and French. Participants' feedback guided the iterative modification of the PB-PDI and items were accepted for final inclusion if they were endorsed by three consecutive participants. A total of 16 participants provided feedback on the items, refined the response options, and discussed how to clarify questions. They selected a 2-week timeframe for the PB-PDI recall period. At the end of the cognitive interviews, all seven items and their response options were endorsed in both languages. The cognitive interview process allowed us to refine items and ensure that they were clear in terms of instructions and response options from the perspective of people with PD. The next step will be to elicit preference weights to develop a scoring algorithm and assess its measurement properties.

Assessing the Measurement Properties of the Self-Administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R): A Rasch Analysis.

OBJECTIVE: The self-administered version of the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) is used to monitor function and disease progression in individuals with amyotrophic lateral sclerosis (ALS). However, the performance of the self-administered ALSFRS-R has not been assessed using Rasch Measurement Theory. Therefore, the purpose of this study was to examine the psychometric properties of the self-administered ALSFRS-R using Rasch analysis. METHODS: Rasch analysis was performed on self-administered ALSFRS-R data from individuals with ALS across Canada. The following 6 aspects of Rasch analysis were examined using RUMM2030: fit via residuals and chi-square statistics, targeting via person-item threshold maps, dependency via item residual correlations, unidimensionality through principal components analysis of residuals, reliability via person separation index, and stability through differential item functioning analyses for sex, age, and language. RESULTS: Analysis was performed on 122 participants (mean age: 52.9 years; 62.8% men). The overall scale demonstrated good fit, reliability, and stability; however, multidimensionality was found. To address this issue, items were divided into 3 subscales (bulbar, motor, and respiratory function), and Rasch analysis was performed for each subscale. The subscales demonstrated good fit, reliability, stability, and unidimensionality. However, there were still issues with item dependency for all subscale and targeting for bulbar and respiratory subscales. CONCLUSIONS: The self-administered ALSFRS-R is reliable, internally valid, and stable across sex, age, and language subgroups; however, it is recommended that the ALSFRS-R be scored by subscale. Future studies can look at revising and/or adding items to tackle misfit, redundancy, and ceiling effects. IMPACT: Self-administered measures are simple to administer and inexpensive. The self-administered ALSFRS-R was found to be psychometrically sound and can be used as a tool to monitor disease progression and function in ALS.

Item Selection for a New Health-Related Quality of Life Measure for Parkinson's Disease: The Preference-Based Parkinson's Disease Index (PB-PDI).

Background: Parkinson's disease (PD) is a neurodegenerative condition, predominantly affecting older adults. Preference-based measures (PBMs) can be used to make decisions about the cost-utility of different treatments. There are currently no PBMs for health-related quality of life (HRQoL) for PD. A previous study identified important health domains for individuals with PD and developed an item pool from existing measures per domain. The current study aims to contribute to the development of a new disease-specific PBM of HRQoL for PD by reducing the current pool of items according to the preferences of individuals with PD. Methods: Fifty-three participants completed a visual analogue scale (VAS) of self-perceived health, the prototype PBM measure, and an item importance rating. To reduce the item pool, the following were calculated: (1) inter-item correlations; (2) impact of each item based on item performance and importance rating; (3) directionality of response options by comparing the VAS scores against each item. Results: Participants (male = 54.7%, age = 60.0 ± 10.2) had a median Hoehn and Yahr score of 2.5 (interquartile range = 1). Items supported for inclusion by this analysis were sleep, fatigue, tremor, mood, walking, memory, and dexterity. Items demonstrating a logical decrease in VAS score with each increasing severity level were sleep, memory, tremor, fatigue, and mood. Conclusion: This PBM will be critical for informing decisions about the cost-utility of PD treatments, guiding the resource allocation within our healthcare system. Future research will include cognitive debriefing with individuals with PD to refine item response options.

A critical review of the pathophysiology of thrombotic complications and clinical practice recommendations for thromboprophylaxis in pregnant patients with COVID-19.

Those who are infected with Severe Acute Respiratory Syndrome-related CoronaVirus-2 are theoretically at increased risk of venous thromboembolism during self-isolation if they have reduced mobility or are dehydrated. Should patients develop coronavirus disease (COVID-19) pneumonia requiring hospital admission for treatment of hypoxia, the risk for thromboembolic complications increases greatly. These thromboembolic events are the result of at least two distinct mechanisms - microvascular thrombosis in the pulmonary system (immunothrombosis) and hospital-associated venous thromboembolism. Since pregnancy is a prothrombotic state, there is concern regarding the potentially increased risk of thrombotic complications among pregnant women with COVID-19. To date, however, pregnant women do not appear to have a substantially increased risk of thrombotic complications related to COVID-19. Nevertheless, several organizations have vigilantly issued pregnancy-specific guidelines for thromboprophylaxis in COVID-19. Discrepancies between these guidelines reflect the altruistic wish to protect patients and lack of high-quality evidence available to inform clinical practice. Low molecular weight heparin (LMWH) is the drug of choice for thromboprophylaxis in pregnant women with COVID-19. However, its utility in non-pregnant patients is only established against venous thromboembolism, as LMWH may have little or no effect on immunothrombosis. Decisions about initiation and duration of prophylactic anticoagulation in the context of pregnancy and COVID-19 must take into consideration disease severity, outpatient vs inpatient status, temporal relation between disease occurrence and timing of childbirth, and the underlying prothrombotic risk conferred by additional comorbidities. There is currently no evidence to recommend the use of intermediate or therapeutic doses of LMWH in thromboprophylaxis, which may increase bleeding risk without reducing thrombotic risk in pregnant patients with COVID-19. Likewise, there is no evidence to comment on the role of low-dose aspirin in thromboprophylaxis or of anti-cytokine and antiviral agents in preventing immunothrombosis. These unanswered questions are being studied within the context of clinical trials.

Development of a core outcome set for studies on prevention and management of pregnancy-associated venous thromboembolism (COSPVenTE): a study protocol.

INTRODUCTION: Pregnancy and post partum are times of heightened risk for the development of venous thromboembolism (VTE), which in turn is one of the leading causes of maternal mortality and long-term morbidity. The current research aimed at improving health guidelines for women with pregnancy-associated VTE is limited by inconsistency in outcome reporting preventing comparison across studies, and lack of input from patients with respect to outcomes they propose are most important to measure. A suggested solution is the development of a core outcome set (COS) that defines the minimum criteria for outcome reporting in clinical trials and prospective studies. COSs function to facilitate data harmonisation and increase homogeneity in outcome reporting while incorporating the voice of women in this population in the planning of research to inform their ongoing care. METHODS AND ANALYSIS: The development of a COS for studies on pregnancy-associated VTE will comprise five steps. First, a systematic review of the published literature will identify currently reported outcomes, their definitions and measurements if applicable. This will be followed by in-person interviews with patients, clinicians, researchers, hospital administrators and policy-makers to identify outcomes they consider important. Third, the long list of outcomes obtained from steps I and II will be condensed through online Delphi surveys involving an international group of relevant stakeholders including patients. This will be followed by a face-to-face consensus meeting with representatives of all stakeholder groups to arrive at a consensus on the final COS. Lastly, to determine how the identified core outcomes should be measured, another literature review and Delphi process will be carried out as necessary. ETHICS AND DISSEMINATION: This study has been approved by the Mount Sinai Hospital Research Ethics Board (REB 18-0314-E). Study results will be published in open-access journals and presented at obstetrics, maternal-fetal medicine and haematology conferences. All progress will be documented on the international prospective register of systematic reviews (PROSPERO) and Core Outcome Measures in Effectiveness Trials databases. PROSPERO REGISTRATION NUMBER: CRD42019111479.