Peanut allergy burden survey: Factors associated with health-related quality of life in adolescents.

Background: Patients with peanut allergy (PA) experience significant burden of illness, which impacts health-related quality of life (HRQoL), particularly in adolescence. There is a paucity of research evaluating drivers of HRQoL scores.

Methods: A prospective, online survey of adolescents with self-reported, provider-diagnosed PA completed from November 2018 to January 2019 was used to explore drivers of the real-world impact of PA on HRQoL using the Pediatric Quality of Life Inventory 4.

Health care utilization and costs associated with functional status in patients with psoriatic arthritis.

The Health Assessment Questionnaire Disability Index (HAQ-DI) has been validated and widely used in psoriatic arthritis (PsA) clinical trials for the assessment of patient functional status. Significant improvements in the HAQ-DI have been reported in response to therapeutic interventions; however, few US studies have evaluated the economic impact of functional disability in patients with PsA. To evaluate the association of functional status with health care resource utilization (HCRU) and total health care costs in US patients diagnosed with PsA.

Comparison of Clinical Manifestations in Rheumatoid Arthritis vs. Spondyloarthritis: A Systematic Literature Review.

Introduction: Misclassification of spondyloarthritis (SpA) as rheumatoid arthritis (RA) may lead to delayed SpA diagnosis and suboptimal therapeutic outcomes. Here, we evaluate the literature on clinical manifestations in patients with SpA and RA, particularly seronegative RA, to understand the potential overlap, distinctions, and most reliable approaches to accurate diagnosis.

Methods: In this systematic literature review, conducted according to PRISMA guidelines, we searched key biomedical databases for English-language publications of original research articles (up to July 23, 2020) and rheumatology conference abstracts (January 1, 2018-July 31, 2020) reporting key SpA clinical presentations in patients with SpA or RA.

Medical and nursing home costs: From cognitively unimpaired through dementia.

Introduction: Efforts to model the cost-effectiveness of managing/modifying cognitive impairment lack reliable, objective, baseline medical, and nursing-home (NH) costs.

Methods: A stratified-random sample of Olmsted County, MN, residents ages 70-89 years (N = 3545), well-characterized as cognitively unimpaired, mild cognitive impairment (MCI), or dementia, were followed forward ≤1 year in provider-linked billing data and the Centers for Medicare & Medicaid Services NH assessments. Direct medical/nursing home/medical + NH costs were estimated.

The Peanut Allergy Burden Study: Real-world impact of peanut allergy on resource utilization and productivity.

Background: There is limited research demonstrating the real-world economic burden of peanut allergy (PA) in the United States. The Peanut Allergy Burden Study (PABS) is a cross-sectional quantitative survey designed to determine the real-world experience of patients and caregivers with PA. The objective of the study was to understand the real-world utilization of PA-related healthcare resources and the impact of PA on productivity.

The Peanut Allergy Burden Study: Impact on the quality of life of patients and caregivers.

Background: Peanut allergy (PA) places significant burden on peanut-allergic individuals and their families, yet limited research in the United States has quantitatively examined the impact on peanut-allergic individuals and their families' health-related quality of life (HRQoL). The Peanut Allergy Burden Study (PABS) aimed to quantify the impact of PA on the general and disease-specific HRQoL of children, adolescents, and adults with PA, as well as caregivers of children with PA.

Methods: A cross-sectional survey design was employed to examine the real-world impact of PA in children, adolescents, and adults with PA, and caregivers of children with PA.

Patient Preferences for Device-Aided Treatments Indicated for Advanced Parkinson Disease.

Background: Effective treatment for advanced Parkinson disease (PD) uncontrolled with oral medication includes device-aided therapies such as deep brain stimulation (DBS) and continuous levodopa-carbidopa infusion to the duodenum via a portable pump.

Objective: Our objective was to quantify patient preferences for attributes of these device-aided treatments.

Methods: We administered a Web-enabled survey to 401 patients in the United States.

Nursing Home Use Across The Spectrum of Cognitive Decline: Merging Mayo Clinic Study of Aging With CMS MDS Assessments.

Background/objectives: Objective, complete estimates of nursing home (NH) use across the spectrum of cognitive decline are needed to help predict future care needs and inform economic models constructed to assess interventions to reduce care needs.

Design: Retrospective longitudinal study.

Setting: Olmsted County, MN.

Comparing outcomes from clinical studies of oral disease-modifying therapies (dimethyl fumarate, fingolimod, and teriflunomide) in relapsing MS: Assessing absolute differences using a number needed to treat analysis.

Dimethyl fumarate (DMF), fingolimod, and teriflunomide are oral disease-modifying therapies (DMTs) indicated for the treatment of relapsing-remitting multiple sclerosis. Despite well-established limitations of cross-trial comparisons, DMTs are still frequently compared in terms of relative reductions in specific endpoints, most commonly annualized relapse rate. Consideration of absolute risk reduction and number needed to treat (NNT) provides an alternative approach to assess the magnitude of treatment effect and can provide valuable additional information on therapeutic gain.

Measuring treatment satisfaction in MS: Is the Treatment Satisfaction Questionnaire for Medication fit for purpose?

Background: The Treatment Satisfaction Questionnaire for Medication (TSQM) was designed to assess patient treatment satisfaction in chronic diseases. Its performance has not been examined in multiple sclerosis (MS). The 14 items of the TSQM cover four domains: Effectiveness, Side Effects, Convenience, and Global Satisfaction.

Direct medical costs and source of cost differences across the spectrum of cognitive decline: a population-based study.

Background: Objective cost estimates and source of cost differences are needed across the spectrum of cognition, including cognitively normal (CN), mild cognitive impairment (MCI), newly discovered dementia, and prevalent dementia.

Methods: Subjects were a subset of the Mayo Clinic Study of Aging stratified-random sampling of Olmsted County, MN, residents aged 70 to 89 years. A neurologist reviewed provider-linked medical records to identify prevalent dementia (review date = index).

Elagolix, an Oral GnRH Antagonist for Endometriosis-Associated Pain: A Randomized Controlled Study.

Objective: The aim of this study was to estimate the efficacy of elagolix, an oral gonadotropin-releasing hormone (GnRH) antagonist, for the treatment of endometriosis-associated pelvic pain.

Methods: This was a phase II, randomized, placebo-controlled parallel group study conducted at 37 US centers, consisting of an 8-week double-blind period followed by a 16-week open-label period. Patients were 137 women aged 18 to 49, with laparoscopically confirmed endometriosis and moderate to severe nonmenstrual pelvic pain and dysmenorrhea, who were administered elagolix 150 mg daily or placebo.

Levodopa-carbidopa intestinal gel in advanced Parkinson's disease open-label study: interim results.

Levodopa-carbidopa intestinal gel (LCIG) delivered continuously via percutaneous endoscopic gastrojejunostomy (PEG-J) tube has been reported, mainly in small open-label studies, to significantly alleviate motor complications in Parkinson's disease (PD). A prospective open-label, 54-week, international study of LCIG is ongoing in advanced PD patients experiencing motor fluctuations despite optimized pharmacologic therapy. Pre-planned interim analyses were conducted on all enrolled patients (n = 192) who had their PEG-J tube inserted at least 12 weeks before data cutoff (July 30, 2010).

Are gastroenterologists less tolerant of treatment risks than patients? Benefit-risk preferences in Crohn's disease management.

Background: Crohn's disease is a serious and debilitating gastrointestinal disorder with a high, unmet need for new treatments. Biologic agents have the potential to alter the natural course of Crohn's disease but present known risks of potential serious adverse events (SAEs). Previous studies have shown that patients are willing to accept elevated SAE risks in exchange for clinical efficacy.

Are adult patients more tolerant of treatment risks than parents of juvenile patients?

Understanding patient-specific differences in risk tolerance for new treatments that offer improved efficacy can assist in making difficult regulatory and clinical decisions for new treatments that offer both the potential for greater effectiveness in relieving disease symptoms, but also risks of disabling or fatal side effects. The aim of this study is to elicit benefit-risk trade-off preferences for hypothetical treatments with varying efficacy and risk levels using a stated-choice (SC) survey. We derive estimates of "maximum acceptable risk" (MAR) that can help decisionmakers identify welfare-enhancing alternatives.

Health-related quality of life during natalizumab maintenance therapy for Crohn's disease.

Objectives: We evaluated the effects of treatment on health-related quality of life (HRQoL) during a randomized controlled trial of natalizumab maintenance therapy (ENACT-2) using both disease-specific and generic measures.

Methods: Crohn's disease patients who received natalizumab as induction therapy in ENACT-1 (N=724) and responded (N=339) were re-randomized to ENACT-2 in which they received natalizumab 300 mg (N=168) or placebo (N=171) every 4 wk for 48 additional wk. Outcome measures were the change from baseline on the inflammatory bowel disease questionnaire (IBDQ), the short form-36 (SF-36), the EuroQol-5D (EQ-5D), and a subject global assessment.

Crohn's disease patients' risk-benefit preferences: serious adverse event risks versus treatment efficacy.

Background & Aims: Regulatory assessments of drug risks do not routinely consider patient preferences, despite evidence that some patients are willing to accept increased side-effect risk in exchange for therapeutic benefits. The aim of this study is to estimate the willingness of Crohn's disease (CD) patients to accept life-threatening adverse event risks in exchange for CD symptom relief.

Methods: Patients with CD completed choice-format conjoint trade-off tasks involving hypothetical treatments with varying efficacy and risk levels.

The association of consumer expectations, experiences and satisfaction with newly prescribed medications.

Objective: To examine the association between medication expectations and subsequent experience on treatment satisfaction and intention to continue using the medication.

Methods: A longitudinal study with two surveys administered to each patient. Patients prescribed a new medication were recruited in pharmacies within Michigan.

Hierarchical construct validity of the treatment satisfaction questionnaire for medication (TSQM version II) among outpatient pharmacy consumers.

Objectives: The objectives of this study were twofold: 1) to evaluate the construct validity of the Treatment Satisfaction Questionnaire for Medication (TSQM v. II) using structural equation modeling (SEM); and 2) to assess its concurrent validity using medication adherence criteria.

Methods: Pharmacy patients filling a new medication prescription (n = 342) were recruited from 14 Michigan pharmacies to participate in a 4-week treatment satisfaction study.

The impact of depression on the academic productivity of university students.

Background: Depression is a common disorder that impacts an individual's ability to perform life activities, including those required by the workplace. Academic performance can be viewed as a direct parallel to workforce performance, with students belonging to a unique set of individuals whose ability to perform can be measured on criteria applied by an observer and by self-report. While the prevalence of depression for this group is high and preparation for entry into the workplace is critical for these individuals, this relationship has not been adequately investigated.

Incidence and characterization of diagnosed endometriosis in a geographically defined population.

Objective: We examined whether widespread use of laparoscopy was accompanied by increased diagnosis of asymptomatic endometriosis, inflated rates of diagnosis, or changes in the clinical spectrum of disease.

Design: Population-based cohort.

Setting: Olmsted County, Minnesota.

Validation of a general measure of treatment satisfaction, the Treatment Satisfaction Questionnaire for Medication (TSQM), using a national panel study of chronic disease.

Background: The objective of this study was to develop and psychometrically evaluate a general measure of patients' satisfaction with medication, the Treatment Satisfaction Questionnaire for Medication (TSQM).

Methods: The content and format of 55 initial questions were based on a formal conceptual framework, an extensive literature review, and the input from three patient focus groups. Patient interviews were used to select the most relevant questions for further evaluation (n = 31).

Validation of the Health-Related Productivity Questionnaire Diary (HRPQ-D) on a sample of patients with infectious mononucleosis: results from a phase 1 multicenter clinical trial.

The objective of this work was to assess the performance of the newly developed Health-Related Productivity Questionniare-Diary (HRPQ-D). Patients completed the HRPQ-D daily for 1-week periods during weeks 1, 2, 4, and 8 of a clinical trial for infectious mononucleosis. Productivity data were collected on a daily basis in terms of absenteeism, presenteeism, and combined lost productivity for three work venues (work outside home, housework, and classes/homework).