Carla DeMuro

Lower Urinary Tract Symptoms, Prostate Volume and Uroflow in Norwegian Community Men

Objective: To describe lower urinary tract symptoms, prostate volume and peak urinary flow rate, and investigate the relationships among urological variables in a community sample of Norwegian men. Materials and Methods: A cross–sectional study of 611 men, aged 55–70 years, who underwent a clinical urological examination including uroflowmetry, residual urine measurement, and transrectal ultrasonography of the prostate. All the men completed a questionnaire which included the International Prostate Symptom Score (IPSS). Results: Severe symptoms were reported by 5%, while 23.6% reported moderate symptoms, and the overall median IPSS was 4 (q1 = 25th percentile, 1; q3 = 75th percentile, 9). The median peak flow rate was 15 ml/s (q1 = 11; q2 = 22) while median prostate volume was 30 cm3 (q1 = 23; q3 = 38), with little variation evident across the narrow age range of 55–70 years. A positive modest correlation (r = 0.176) was found between IPSS and prostate volume, and a negative correlation between IPSS and peak flow rate (r = –0.278). There was a modest correlation between body mass index (BMI) and prostate size, but no significant correlation between BMI and IPSS. Conclusion: In this population–based study, moderate lower urinary tract symptoms were reported by 24% and severe symptoms by 5% of community men. The distribution of lower urinary tract symptoms, prostate volume and peak urinary flow rate in Norwegian men is comparable to that described in similar studies conducted in Spain, Holland and USA.

Diabetes Medication Satisfaction Tool A focus on treatment regimens

OBJECTIVE—To develop and test a patient questionnaire on treatment satisfaction with diabetes regimens. RESEARCH DESIGN AND METHODS—Survey items were developed from community clinic focus groups, pretested in patients with diabetes, and examined in two samples of treated patients. RESULTS—Sixteen items performed well in assessing treatment experiences: ease and convenience, lifestyle burdens, well-being, and medical control. Construct validity was supported by associations (P < 0.05) with treatment complexity, self-rated glucose control, health worries, and A1C. Internal consistency ranged from 0.89 to 0.95. CONCLUSIONS—The Diabetes Medication Satisfaction Tool offers a comprehensive assessment of patient acceptability, with diabetes therapy useful for individualizing therapeutic decision making.

The Diabetes Medication Satisfaction Tool (DMSAT): A Focus on Treatment Regimens

OBJECTIVE—To develop and test a patient questionnaire on treatment satisfaction with diabetes regimens. RESEARCH DESIGN AND METHODS—Survey items were developed from community clinic focus groups, pretested in patients with diabetes, and examined in two samples of treated patients. RESULTS—Sixteen items performed well in assessing treatment experiences: ease and convenience, lifestyle burdens, well-being, and medical control. Construct validity was supported by associations (P < 0.05) with treatment complexity, self-rated glucose control, health worries, and A1C. Internal consistency ranged from 0.89 to 0.95. CONCLUSIONS—The Diabetes Medication Satisfaction Tool offers a comprehensive assessment of patient acceptability, with diabetes therapy useful for individualizing therapeutic decision making.

Mortality and Causes of Death in Patients with Sporadic Inclusion Body Myositis: Survey Study Based on the Clinical Experience of Specialists in Australia, Europe and the USA

Background: There is a paucity of data on mortality and causes of death (CoDs) in patients with sporadic inclusion body myositis (sIBM), a rare, progressive, degenerative, inflammatory myopathy that typically affects those aged over 50 years. Objective: Based on patient records and expertise of clinical specialists, this study used questionnaires to evaluate physicians’ views on clinical characteristics of sIBM that may impact on premature mortality and CoDs in these patients. Methods: Thirteen physicians from seven countries completed two questionnaires online between December 20, 2012 and January 15, 2013. Responses to the first questionnaire were collated and presented in the second questionnaire to seek elaboration and identify consensus. Results: All 13 physicians completed both questionnaires, providing responses based on 585 living and 149 deceased patients under their care. Patients were reported to have experienced dysphagia (60.2%) and injurious falls (44.3%) during their disease. Over half of physicians reported that a subset of their patients with sIBM had a shortened lifespan (8/13), and agreed that bulbar dysfunction/dysphagia/oropharyngeal involvement (12/13), early-onset disease (8/13), severe symptoms (8/13), and falls (7/13) impacted lifespan. Factors related to sIBM were reported as CoDs in 40% of deceased patients. Oropharyngeal muscle dysfunction was ranked as the leading feature of sIBM that could contribute to death. The risk of premature mortality was higher than the age-matched comparison population. Conclusions: In the absence of data from traditional sources, this study suggests that features of sIBM may contribute to premature mortality and may be used to inform future studies.

Patient-Reported Outcomes Labeling for Products Approved by the Office of Hematology and Oncology Products of the US Food and Drug Administration (2010-2014)

PURPOSEnTo review the use of patient-reported outcome (PRO) data in medical product labeling granted by the US Food and Drug Administration (FDA) for new molecular entities and biologic license applications by the FDA Office of Hematology and Oncology Products (OHOP) between January 2010 and December 2014, to elucidate challenges faced by OHOP for approving PRO labeling, and to understand challenges faced by drug manufacturers to include PRO end points in oncology clinical trials.nnnMETHODSnFDA Drug Approval Reports by Month were reviewed to obtain the number of new molecular entities and biologic license applications approved from 2010 to 2014. Drugs approved by the FDA OHOP during this period were selected for further review, focusing on brand and generic name; approval date; applicant; indication; PRO labeling describing treatment benefit, measures, end point status, and significant results; FDA reviewer feedback on PRO end points; and study design of registration trials. First in class, priority review, fast track, orphan drug, or accelerated approval status was retrieved for selected oncology drugs from 2011 to 2014. Descriptive analyses were performed by using Microsoft Excel 2010.nnnRESULTSnOf 160 drugs approved by the FDA (2010-2014), 40 were approved by OHOP. Three (7.5%) of the 40 received PRO-related labeling (abiraterone acetate, ruxolitinib phosphate, and crizotinib). Compared with nononcology drugs (2011-2014), oncology drugs were more likely to be orphan and first in class. The majority of oncology drug reviews by FDA were fast track, priority, or accelerated.nnnCONCLUSIONnAlthough symptoms and functional decrements are common among patients with cancer, PRO labeling is rare in the United States, likely because of logistical hurdles and oncology study design. Recent developments within the FDA OHOP to capture PROs in oncology studies for the purpose of product labeling are encouraging.

Development of the sporadic inclusion body myositis physical functioning assessment.

Introduction: Sporadic inclusion body myositis (sIBM) is a progressive idiopathic inflammatory myopathy characterized by atrophy and weakness of proximal and distal muscle groups that results in a loss of independence and the need for assistive devices and supportive care. To assess treatment benefit of new therapies, a patient‐reported outcome measure of physical function was developed. Methods: The tool was rigorously developed in accordance with the United States Food and Drug Administration (FDA) patient‐reported outcomes (PRO) guidance. A single‐visit, observational study was conducted. Standard qualitative analytical methods were employed to analyze interview data and generate questionnaire items. Results: Twenty concept elicitation and 19 cognitive debriefing interviews were conducted, and 6 expert physicians were consulted. The tool consists of 11 items scored on a 0–10 numerical rating scale. Subjects completed the questionnaire utilizing either paper or electronic administration. Conclusion: We have developed a PRO tool in alignment with FDA PRO guidance for use in the functional assessment of treatment benefit in sIBM. Muscle Nerve, 2016 Muscle Nerve 54: –, 2016 Muscle Nerve 54: 653–657, 2016

5 Development of a Symptom Tracker for Use by Patients with Heart Failure

s / Heart & Lung 46 (2017) 215e219 216 activation, and heart failure knowledge with self-management behavior. The hypothesis was that in persons with heart failure, self-care behaviors are associated with health literacy, heart failure knowledge, and patient activation. Background: Current investigations of self-management by persons with HF have focused primarily on examining the effects of clinical interventions by health care providers to improve patients’ self-management behaviors. While clinician support of self-management is important, more information is needed to understand intrinsic patient characteristics that influence selfmanagement behaviors. Knowledge of these characteristics can better inform providers in delivering care to promote self-management and remove barriers. More research regarding determinants of self-care behavior in HF patients can enhance the knowledge base for nurses and other providers to design evidencebased, effective interventions. Methods: The study used a prospective, cross-sectional, correlational design to test the hypothesis. A convenience sample of 151 persons meeting eligibility criteria (18 years or older; diagnosis of heart failure, ability to read and respond to questionnaires in English) completed questionnaires containing the European Heart Failure Self-Care Behavior Scale, the Test of Functional Health Literacy in Adults, Short form (S-TOFHLA), the 13-Item Patient Activation Measure, the Dutch Heart Failure Knowledge Scale, the Stanford Self-Rated Health Measure, and a demographic data form. A member of the research team approached potential participants, who were present for their regularly-scheduled appointments at one of six chronic care outpatient clinics of a regional health system, informed them of the nature of the study, and invited them to participate. Those willing to participate completed questionnaires at the site. Data were entered using the double entry technique to reduce error, and analyzed using SAS software. Sample size was based on a power analysis for 4 variables and an effect size of .20. Scale scores were computed according to published recommendations, and all scale scores were computed so that higher scores represented higher levels of the attribute being measured. Means and frequencies were computed to describe the sample. To test the hypothesis, correlations and multiple regression were used, with alpha set at .05. Results: The sample was 57% male with a mean age of 68 (SD 13.0). Fifty-one percent were white and 44% African American. Highest education completed was 18% below high school, 27% high school, 31% some college, and 25% college degree or higher. Self-management (measured with the European Heart Failure Self-Care Behavior Scale) was significantly correlated with patient activation level (r 1⁄4 .266, p 1⁄4 .001) and age (r 1⁄4 .275, p 1⁄4 .002). Relationships between self-management and functional health literacy (r 1⁄4 -.096, p 1⁄4 .25), and self-management and heart failure knowledge (r 1⁄4 .019, p 1⁄4 .81) were not statistically significant. However, health literacy was correlated with educational level (r 1⁄4 .234, p < .05) and heart failure knowledge (r 1⁄4 .292, p < .05). Heart failure knowledge and educational level were also positively correlated (r 1⁄4 .339, p < .05). Conclusion: The findings of this study partially support the hypothesis, in that self-management behavior was positively associated with patient activation, but not with health literacy or heart failure knowledge, in persons with heart failure. Although further research is needed to confirm and further elucidate these findings, they suggest that persons with HF may better manage their condition if sufficiently activated, regardless of their level of health literacy or knowledge of their disease process. Caution in interpreting these results is warranted based on the potential for bias in self-report. Because of the significant correlations between heart failure knowledge, health literacy and educational level, this study’s findings suggest that interventions targeted at promoting knowledge in persons with heart failure should take into account patients’ educational level and health literacy. 4 Measures of Ventricular-Arterial Coupling and Incident Heart Failure Preserved Ejection Fraction (HFpEF): A Matched Case-Control Analysis