Validation of the Portuguese MDS-UPDRS: Challenges to Obtain a Scale Applicable to Different Linguistic Cultures.

Background: The MDS-UPDRS has been available in English since 2008, showing satisfactory clinimetric results and being proposed as the new official benchmark scale for Parkinson's disease (PD), being cited as a core instrument for PD in the National Institutes of Neurological Disorders and Stroke Common Data Elements program. For this reason, the MDS created guidelines for development of MDS-UPDRS official, clinimetrically validated translations.

Objective: This study presents the formal process used to obtain the officially approved Portuguese version of the MDS-UPDRS.

Semiparametric regression modeling of the global percentile outcome.

When no single outcome is sufficient to capture the multidimensional impairments of a disease, investigators often rely on multiple outcomes for comprehensive assessment of global disease status. Methods for assessing covariate effects on global disease status include the composite outcome and global test procedures. One global test procedure is the O'Brien's rank-sum test, which combines information from multiple outcomes using a global rank-sum score.

Study in Parkinson's disease of exercise phase 3 (SPARX3): study protocol for a randomized controlled trial.

Background: To date, no medication has slowed the progression of Parkinson's disease (PD). Preclinical, epidemiological, and experimental data on humans all support many benefits of endurance exercise among persons with PD. The key question is whether there is a definitive additional benefit of exercising at high intensity, in terms of slowing disease progression, beyond the well-documented benefit of endurance training on a treadmill for fitness, gait, and functional mobility.

Using increased trust in medical researchers to increase minority recruitment: The RECRUIT cluster randomized clinical trial.

While extensive literature exists on barriers and strategies to increase minority participation in clinical trials, progress is limited. Few strategies were evaluated in randomized trials. We studied the impact of RECRUIT, a trust-based, cluster randomized minority recruitment trial layered on top of four traditional NIH-funded parent trials (BMT CTN, CABANA, PACES, STEADY-PD III; fifty specialty sites).

Intent-to-treat analysis of cluster randomized trials when clusters report unidentifiable outcome proportions.

Background: Cluster randomized trials are designed to evaluate interventions at the cluster or group level. When clusters are randomized but some clusters report no or non-analyzable data, intent-to-treat analysis, the gold standard for the analysis of randomized controlled trials, can be compromised. This article presents a very flexible statistical methodology for cluster randomized trials whose outcome is a cluster-level proportion (e.

Cost-effectiveness evaluation of the PROPPR trial transfusion protocols.

Background: There have been no prior investigations of the cost effectiveness of transfusion strategies for trauma resuscitation. The Pragmatic, Randomized, Optimal Platelet and Plasma Ratios (PROPPR) study was a Phase III multisite, randomized trial in 680 subjects comparing the efficacy of 1:1:1 transfusion ratios of plasma and platelets to red blood cells with the 1:1:2 ratio. We hypothesized that 1:1:1 transfusion results in an acceptable incremental cost-effectiveness ratio, when estimated using patients' age-specific life expectancy and cost of care during the 30-day PROPPR trial period.

Recruitment of racial and ethnic minorities to clinical trials conducted within specialty clinics: an intervention mapping approach.

Background: Despite efforts to increase diversity in clinical trials, racial/ethnic minority groups generally remain underrepresented, limiting researchers' ability to test the efficacy and safety of new interventions across diverse populations. We describe the use of a systematic framework, intervention mapping (IM), to develop an intervention to modify recruitment behaviors of coordinators and specialist investigators with the goal of increasing diversity in trials conducted within specialty clinics. To our knowledge IM has not been used in this setting.

Validation of the Hebrew version of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale.

Background: The Movement Disorders Society (MDS) published the English new Unified Parkinson's Disease Rating Scale (MDS-UPDRS) as the official benchmark scale for Parkinson's disease (PD) in 2008. We aimed to validate the Hebrew version of the MDS-UPDRS, explore its dimensionality and compare it to the original English one.

Methods: The MDS-UPDRS questionnaire was translated to Hebrew and was tested on 389 patients with PD, treated at the Movement Disorders Unit at Tel-Aviv Medical Center.

Benefits of stroke treatment delivered using a mobile stroke unit trial.

Rationale Mobile stroke units speed treatment for acute ischemic stroke, thereby possibly improving outcomes. Aim To compare mobile stroke unit and standard management clinical outcomes, healthcare utilization, and cost-effectiveness in tissue plasminogen activator-eligible acute ischemic stroke patients calling 911. Sample size 693.

Design of a cluster-randomized minority recruitment trial: RECRUIT.

Background: Racial/ethnic minority groups remain underrepresented in clinical trials. Many strategies to increase minority recruitment focus on minority communities and emphasize common diseases such as hypertension. Scant literature focuses on minority recruitment to trials of less common conditions, often conducted in specialty clinics and dependent on physician referrals.

Autonomic and electrocardiographic findings in Parkinson's disease.

Parkinson disease (PD) is a progressive neurodegenerative disorder characterized by motor and non-motor symptoms and signs. Many reports suggest that diminished heart rate variability occurs early, even prior to the cardinal signs of PD. In a longitudinal study of PD, we evaluated whether heart rate variability (HRV) obtained using a 10-second ECG tracing, and the electrocardiographic QT-interval would be associated with PD severity and progression.

Cross-Cultural Differences of the Non-Motor Symptoms Studied by the Traditional Chinese Version of the International Parkinson and Movement Disorder Society- Unified Parkinson's Disease Rating Scale.

Background: Given the importance of ethnic differences in the evaluation of various aspects of symptoms in patients with Parkinson's disease (PD), we present the formal procedure for completing the traditional Chinese translation of the International and Parkinson and Movement Disorder Society/UPDRS (MDS-UPDRS) and highlight the discrepancy in nonmotor symptoms (NMS) between patients in Eastern and Western countries.

Methods: A total of 350 native Chinese-speaking PD patients were recruited from multiple hospitals in Eastern countries; they completed the MDS-UPDRS. The translation process was executed and factor analysis was performed to determine the structure of the scale.

Caffeine, creatine, GRIN2A and Parkinson's disease progression.

Caffeine is neuroprotective in animal models of Parkinson's disease (PD) and caffeine intake is inversely associated with the risk of PD. This association may be influenced by the genotype of GRIN2A, which encodes an NMDA-glutamate-receptor subunit. In two placebo-controlled studies, we detected no association of caffeine intake with the rate of clinical progression of PD, except among subjects taking creatine, for whom higher caffeine intake was associated with more rapid progression.

Damage control laparotomy utilization rates are highly variable among Level I trauma centers: Pragmatic, Randomized Optimal Platelet and Plasma Ratios findings.

Background: Damage control laparotomy (DCL) is intended to limit deleterious effects from trauma-induced coagulopathy. DCL has been associated with mortality reduction, but may increase complications including sepsis, abscess, respiratory failure, hernia, and gastrointestinal fistula. We hypothesized that (1) DCL incidence would vary between institutions; (2) mortality rates would vary with DCL rates; (3) standard DCL criteria of pH, international normalized ratio, temperature and major intra-abdominal vascular injury would not adequately capture all patients.

Earlier Endpoints are Required for Hemorrhagic Shock Trials Among Severely Injured Patients.

Background: Choosing the appropriate endpoint for a trauma hemorrhage control trial can determine the likelihood of its success. Recent Phase 3 trials and observational studies have used 24-h and/or 30-day all-cause mortality as the primary endpoint and some have not used exception from informed consent (EFIC), resulting in multiple failed trials. Five recent high-quality prospective studies among 4,064 hemorrhaging trauma patients provide new evidence to support earlier primary endpoints.

Gender-, age-, and race/ethnicity-based differential item functioning analysis of the movement disorder society-sponsored revision of the Unified Parkinson's disease rating scale.

Objective: Assess MDS-UPDRS items for gender-, age-, and race/ethnicity-based differential item functioning.

Background: Assessing differential item functioning is a core rating scale validation step. For the MDS-UPDRS, differential item functioning occurs if item-score probability among people with similar levels of parkinsonism differ according to selected covariates (gender, age, race/ethnicity).

Longer Duration of MAO-B Inhibitor Exposure is Associated with Less Clinical Decline in Parkinson's Disease: An Analysis of NET-PD LS1.

Background: Monoamine oxidase type B (MAO-B) inhibitors exhibit neuroprotective effects in preclinical models of PD but clinical trials have failed to convincingly demonstrate disease modifying benefits in PD patients.

Objective: To perform a secondary analysis of NET-PD LS1 to determine if longer duration of MAO-B inhibitor exposure was associated with less clinical decline.

Methods: The primary outcome measure was the Global Outcome (GO), comprised of 5 measures: change from baseline in the Schwab and England (ADL) scale, the 39-item Parkinson's Disease Questionnaire (PDQ-39), the UPDRS Ambulatory Capacity Scale, the Symbol Digit Modalities Test, and the most recent Modified Rankin Scale.

Religiosity in black and white older Americans: Measure adaptation, psychometric validation, and racial difference.

Racial difference of religiosity in a heterogeneous older population had long been a focal point of gerontological research. However, most religiosity measures were developed from homogenous sample, few underwent rigorous psychometric validation, and studies on racial difference of religiosity had been obstructed. This cross-sectional study adapted a religiosity measure originally designed for blacks only to a heterogeneous older population of blacks and whites, validated its psychometric properties, and examined racial difference of religiosity.

Communicating with participants during the conduct of multi-center clinical trials.

Background: Communicating with trial participants is an important aspect of study conduct, relevant for informed consent and respect for participants. Group teleconferences are one means to convey information to trial participants. We used group teleconferences during an ongoing large-scale clinical trial to communicate important trial updates.

Assessing protocol adherence in a clinical trial with ordered treatment regimens: Quantifying the pragmatic, randomized optimal platelet and plasma ratios (PROPPR) trial experience.

Background: Medication dispensing errors are common in clinical trials, and have a significant impact on the quality and validity of a trial. Therefore, the definition, calculation and evaluation of such errors are important for supporting a trial's conclusions. A variety of medication dispensing errors can occur.

Exploring Willingness of Elder Chinese in Houston to Participate in Clinical Research.

Background And Objectives: Inadequate minority participation in clinical research can threaten the applicability and strength of scientific findings. Previous research suggests that trial participation rates are lowest among Asian Americans, compared to other groups. This study explored barriers to clinical research participation among elder Chinese living in Houston, Texas.

Continuous time Markov chain approaches for analyzing transtheoretical models of health behavioral change: A case study and comparison of model estimations.

Continuous time Markov chain models are frequently employed in medical research to study the disease progression but are rarely applied to the transtheoretical model, a psychosocial model widely used in the studies of health-related outcomes. The transtheoretical model often includes more than three states and conceptually allows for all possible instantaneous transitions (referred to as general continuous time Markov chain). This complicates the likelihood function because it involves calculating a matrix exponential that may not be simplified for general continuous time Markov chain models.