A qualitative study to understand the Duchenne muscular dystrophy experience from the parent/patient perspective.

Background: Duchenne muscular dystrophy (DMD) is a rare, severe, fatal neuromuscular disease characterized by progressive atrophy and muscle weakness, resulting in loss of ambulation, decreased upper body function, and impaired cardiorespiratory function. This study aimed to generate qualitative evidence to describe the primary symptoms and impacts of DMD in ambulatory and non-ambulatory patients as reported by patient/caregiver dyads. Information was also gathered on expectations for future DMD treatments.

Concept Elicitation Interviews and Conceptual Model to Understand the Patient Experience of Limb Girdle Muscular Dystrophy.

Introduction: Limb girdle muscular dystrophies (LGMDs) are a group of rare and heterogeneous disorders involving progressive wasting of shoulder and pelvic girdle musculature. This study aimed to generate qualitative evidence on patient and caregiver experiences with symptoms and impacts of LGMD on overall function and daily life for sarcoglycanopathy subtypes 2C/R5, 2D/R3, and 2E/R4.

Methods: Twenty-three individuals with LGMD with (n = 5) or without (n = 18) a caregiver participated in 60-minute semi-structured video interviews.

A randomized, controlled trial of ZYN002 cannabidiol transdermal gel in children and adolescents with fragile X syndrome (CONNECT-FX).

Background: Fragile X syndrome (FXS) is associated with dysregulated endocannabinoid signaling and may therefore respond to cannabidiol therapy.

Design: CONNECT-FX was a double-blind, randomized phase 3 trial assessing efficacy and safety of ZYN002, transdermal cannabidiol gel, for the treatment of behavioral symptoms in children and adolescents with FXS.

Methods: Patients were randomized to 12 weeks of ZYN002 (250 mg or 500 mg daily [weight-based]) or placebo, as add-on to standard of care.

Establishing the content validity of the Epworth Sleepiness Scale for Children and Adolescents in Prader-Willi syndrome.

Study Objectives: Excessive daytime sleepiness is common in Prader-Willi syndrome (PWS), with prevalence ranging from 52% to 100%. The goal of this study was to establish the content validity (ie, evidence that an instrument measures an intended concept of interest) of the parent/caregiver version of the Epworth Sleepiness Scale for Children and Adolescents (ESS-CHAD), a measure of daytime sleepiness, in PWS.

Methods: Qualitative, dyadic semistructured video interviews were conducted with 18 caregivers and their children with PWS from April to June 2020.

Determining clinically meaningful thresholds for innovative burn care products to reduce autograft: A US burn surgeon Delphi panel.

Reducing the amount of donor skin needed for definitive wound closure can improve outcomes in patients with severe burns. This Delphi Consensus Panel (DCP) aimed to achieve expert consensus on the percentage reduction in donor skin for autograft that constitutes a clinically meaningful benefit. A two-round DCP of fifteen US burn surgeons was conducted via a web-based survey platform.

Assessing the Relationship between Performance on the University of California Performance Skills Assessment (UPSA) and Outcomes in Schizophrenia: A Systematic Review and Evidence Synthesis.

Objective: To perform a systematic review of the published literature to evaluate how functional capacity, as measured by the University of California at San Diego (UCSD) Performance-based Skills Assessment (UPSA), relates to other functional measures and real-world outcomes among individuals with schizophrenia.

Methods: The MEDLINE® and Embase® databases were searched to identify joint evaluations with UPSA and key functional outcomes (functional scale measures; generic or disease-specific, health-related quality of life [HRQoL]; or real-world outcomes [residential status; employment status]) in patients with schizophrenia. Pearson correlations were estimated between UPSA scores, HRQoL, other functional scale measures, and real-world outcomes, for outcomes described in at least six studies.

Estimating Alzheimer's Disease Progression Rates from Normal Cognition Through Mild Cognitive Impairment and Stages of Dementia.

Background: Alzheimer's Disease (AD) can be conceptualized as a continuum: patients progress from normal cognition to mild cognitive impairment (MCI) due to AD, followed by increasing severity of AD dementia. Prior research has measured transition probabilities among later stages of AD, but not for the complete spectrum.

Objective: To estimate annual progression rates across the AD continuum and evaluate the impact of a delay in MCI due to AD on the trajectory of AD dementia and clinical outcomes.

Treatment patterns, healthcare resource utilization, and costs following first-line antidepressant treatment in major depressive disorder: a retrospective US claims database analysis.

Background: Although the symptoms of major depressive disorder (MDD) are often manageable with pharmacotherapy, response to first-line antidepressant treatment is often less than optimal. This study describes long-term treatment patterns in MDD patients in the United States and quantifies the economic burden associated with different treatment patterns following first-line antidepressant therapy.

Methods: MDD patients starting first-line antidepressant monotherapy and having continuous enrollment ≥12 months before and ≥24 months following the index date (i.

The effect of vortioxetine on overall patient functioning in patients with major depressive disorder.

Background: The objectives of this meta-analysis of data from randomized, placebo-controlled studies were to assess the effect of vortioxetine on overall functioning (primary) and functional remission (secondary) using the Sheehan Disability Scale (SDS) in adults with major depressive disorder (MDD).

Methods: Data from nine short-term (6/8 weeks) pivotal studies that included patient functioning assessments were included in this random-effects meta-analysis, which used aggregated study-level data for all therapeutic vortioxetine doses and a mixed-effect model for repeated measures using the full analysis set.

Results: A total of 4,216 patients received ≥1 dose of study treatment (1,522 placebo, 2,694 vortioxetine 5-20 mg/day).

Determination of a clinically important difference and definition of a responder threshold for the UCSD performance-based skills assessment (UPSA) in patients with major depressive disorder.

Background: This article reports an evaluation of the psychometric properties and clinically important difference (CID) threshold of the UCSD Performance-Based Skills Assessment (UPSA) in major depressive disorder (MDD), using data from a large-scale study of the effects of vortioxetine on cognitive functioning and functional capacity in MDD patients.

Methods: Adults with moderate-to-severe recurrent MDD and self-reported cognitive dysfunction were randomized to 8 weeks of double-blind treatment with vortioxetine 10/20mg QD (flexible), duloxetine 60mg QD, or placebo. Pearson correlation coefficients were calculated between UPSA composite score and demographic/disease characteristics at baseline to examine construct validity.

Physician preferences for medication attributes for the prophylactic treatment of patients with severe haemophilia A with inhibitors to factor VIII.

Prophylaxis may be beneficial for patients with severe haemophilia A who have developed inhibitors to factor VIII. The aim of this study was to determine physicians' preferences for medication attributes in the prophylactic treatment of this patient population. Haematologists from Europe (EU) and the United States (US) participated in a discrete choice exercise to explore their preferences for medication attributes (efficacy, cost, scientific evidence, dosing frequency and administration time) associated with prophylaxis for severe haemophilia A in patients with inhibitors to factor VIII.

Smoking, smoking cessation and smoking relapse patterns: a web-based survey of current and former smokers in the US.

Aim: The aim of this study was to characterise the natural course of smoking cessation behaviour in a web-based survey of current and former cigarette smokers (CS and FS) in the United States.

Methods: A web-based survey of CS and FS was conducted in April 2009; demographic and socioeconomic characteristics and smoking history (including the number of lifetime and length of latest quit attempts, aids used and time to relapse) were collated. The surveyed cohort was selected from prescreened CS and FS panellists and matched for age, race and education, to be representative of the US population.

Impact of a fixed combination of clindamycin phosphate 1.2%-benzoyl peroxide 2.5% aqueous gel on health-related quality of life in moderate to severe acne vulgaris.

The acne-specific quality of life (Acne-QoL) questionnaire was developed to measure the impact of facial acne across 4 domains (acne symptoms, role-emotional, self-perception, role-social) of health-related quality of life (HRQL). This analysis assessed the impact of clindamycin phosphate 1.2%-benzoyl peroxide 2.

Economic evaluation of high-dose (80 mg/day) atorvastatin treatment compared with standard-dose (20 mg/day to 40 mg/day) simvastatin treatment in Canada based on the Incremental Decrease in End-Points Through Aggressive Lipid-Lowering (IDEAL) trial.

Background: The Incremental Decrease in End-Points Through Aggressive Lipid-Lowering (IDEAL) trial demonstrated incremental cardiovascular benefit of treatment with high-dose atorvastatin (80 mg/ day) versus standard-dose simvastatin (20 mg/day to 40 mg/day) in 8888 patients with a previous myocardial infarction (MI) over a median follow-up period of 4.8 years.

Objectives: To assess the cost-effectiveness of high-dose atorvastatin versus standard-dose simvastatin treatment in patients with a history of MI from a Canadian societal perspective.

Cost-effectiveness of intensive lipid lowering therapy with 80 mg of atorvastatin, versus 10 mg of atorvastatin, for secondary prevention of cardiovascular disease in Canada.

Background: The TNT study compared high dose atorvastatin (80 mg) versus moderate atorvastatin (10 mg) treatment in 10,001 patients with stable coronary heart disease (CHD), over 4.9 years. Intensive lipid-lowering with atorvastatin (80 mg) reduced major cardiovascular events by 22%.

A retrospective analysis of the prevalence and treatment of hypertension and dyslipidemia in Southwestern Ontario, Canada.

Background: Previous evaluations of the Southwestern Ontario (SSWO) cohort have reported that hypertension (HTN) and dyslipidemia (DYS) are undertreated illnesses; however, concomitant treatment is unknown.

Objectives: The objectives of this study were to assess the prevalence and associated treatment of HTN and DYS in primary health care in SWO and to identify care gaps across subpopulations.

Methods: In this retrospective cohort analysis, chart-abstracted medical records of patients aged>or=118 years with a clinical diagnosis of HTN, DYS, or both and the clinical practice records of primary health care facilities in London, Ontario, Canada, and the surrounding area were conducted between April and December 2000; longitudinal updates were performed quarterly until December 2004.

Choices, persistence and adherence to antihypertensive agents: evidence from RAMQ data.

Background: Most treatment recommendations for hypertension are based on criteria that consider efficacy, safety and cost. Given the need for long-term use of antihypertensive agents, treatment compliance should also be taken into consideration in the selection process.

Objective: The purpose of the present study was to estimate persistence and adherence to antihypertensive agents in a real-life setting.

Prevalence and treatment of dyslipidemia in Canadian primary care: a retrospective cohort analysis.

Background: Dyslipidemia is an important modifiable risk factor for cardiovascular disease (CVD). Studies suggest that dyslipidemia is underdiagnosed and undertreated in Canada.

Objective: The objective of this study was to describe dyslipidemia prevalence, patient characteristics, and lipid-lowering treatment (LLT) patterns in a cohort representing Canadian primary care practice.

A model for assessing the cost-effectiveness of atorvastatin and simvastatin in achieving Canadian low-density lipoprotein cholesterol targets.

Background: Elevated low-density lipoprotein cholesterol (LDL-C) is an important modifiable risk factor for cardiovascular (CV) disease. Statins differ in their LDL-C-lowering effects and acquisition costs. Atorvastatin and simvastatin are the 2 most commonly used statins in Canada.

Multivariate framework of the Brief Questionnaire of Smoking Urges.

We conducted an investigation of the 10-item Brief Questionnaire of Smoking Urges (QSU-Brief) to confirm its hypothesized two-factor structure and to assess the validity and reliability of its total score and these factors. Data were obtained from a 7-week clinical trial on smoking cessation (N=626). The hypothesized two-factor structure of the QSU-Brief was supported by fit indexes (>0.

Prevalence, treatment, and control of hypertension in primary care: gaps, trends, and opportunities.

Since most cases of hypertension are managed in family practice, estimates of the prevalence, treatment, and control in the primary care population are needed to adequately address the burden of hypertension in Canada as it has in other countries. The authors used a large primary care research database to determine the prevalence of hypertension between 2000 and 2003. Blood pressure recordings were used to estimate the rates of prevalence, treatment, and control of hypertension for the overall population and for important subgroups.

Confirmatory factor analyses and reliability of the modified cigarette evaluation questionnaire.

We examined the validity and reliability of the modified Cigarette Evaluation Questionnaire (mCEQ) that assesses the degree to which subjects experience the reinforcing effects of smoking. Data came from three phase II clinical trials (n=626, n=627, n=312) on varenicline for smoking cessation. Comparative fit indexes and non-normed fit indexes from a confirmatory factor analysis exceeded 0.

Persistence and adherence to cholesterol lowering agents: evidence from Régie de l'Assurance Maladie du Québec data.

Background: There is a concern that poor compliance to cholesterol lowering agents (CLA) may compromise their potential benefits. The objective of this study was to estimate compliance to CLAs, in the context of clinical practice in Quebec.

Methods: This study was performed using data from the Régie de l'Assurance Maladie du Québec Persistence and adherence to treatment were estimated separately.