Understanding Perspectives on Presbyopia and Use of Pilocarpine HCl 1.25% Twice Daily from Participants of the Phase 3 VIRGO Study.

INTRODUCTION: The phase 3, randomized, vehicle-controlled, 14-day VIRGO study evaluated the efficacy and safety of twice-daily dosing of pilocarpine hydrochloride ophthalmic solution 1.25% (Pilo) in presbyopia. On VIRGO exit, a companion study was conducted to assess the patient experience with presbyopia and satisfaction with Pilo. METHODS: Recruited individuals completed the Presbyopia Patient Satisfaction Questionnaire (PPSQ) plus a three-part exit survey, or a live interview. The PPSQ evaluated respondents' experience with Pilo. Survey parts 1 and 2 evaluated experience managing presbyopia before and during VIRGO, respectively; part 3 assessed future possibilities of using Pilo in real-world situations. The interview further informed the interviewees' experience with presbyopia and Pilo. The primary endpoint was responders (%) in each rating category of the PPSQ items 1-7; the secondary endpoints were summary of categorical (survey) and qualitative (interviews) responses. RESULTS: The PPSQ and survey included 62 participants who received Pilo (N = 28) or vehicle (N = 34) in VIRGO; the interview included ten participants (Pilo, N = 4; vehicle, N = 6). Per the PPSQ, 64.3% of Pilo users reported vision improvement, including 17.9% with complete improvement; ≥ 46.4% were satisfied/very satisfied with their ability to perform daily activities, see up close unaided, and read in dim light. Among vehicle users, these percentages were 35.3%, 0%, and ≤ 23.5%, respectively. In both subgroups, ≥ 67.9% were interested in using Pilo or Pilo and eyeglasses/contact lenses in the future. Per the interview, vehicle users (n = 6/6) found the eyedrop easy to use but none experienced meaningful near-vision improvements, stopped using other correction method(s) part of the day, were satisfied with the eyedrop, preferred it over their previous correction method(s), or would continue using it if prescribed. Conversely, 75% (n = 3/4) of Pilo users responded positively to each of these six criteria. CONCLUSIONS: Findings validate the VIRGO results and improve our understanding of the patient experience, demonstrating improved vision and satisfaction with Pilo (vs. vehicle) when performing daily activities.

Glycogen storage disease type III: a mixed-methods study to assess the burden of disease.

Background: Glycogen storage disease type III (GSD III) is a rare inherited disorder that results from a glycogen debranching enzyme deficiency. Objectives: The purpose of this research was to collect data on the signs, symptoms, and impacts of GSD III from the perspective of adult patients and caregivers of individuals with GSD III. Design: Online survey and qualitative interviews. Methods: Following institutional review board approval, adult patients and caregivers of children with GSD III were recruited through advocacy networks and clinical sites. If eligible, participants were consented, screened, and sent a survey and/or participated in a 60-min interview. The survey and interview included questions about family history, diagnosis, signs and symptoms, impacts, and management of GSD III. Conceptual models were developed following the analysis of results. Results: In all, 29 adults and 46 caregivers completed the online survey and/or the interviews with 73 survey and 19 interview respondents. Adults and caregivers reported digestive, musculoskeletal, growth and physical appearance, and cardiac signs and symptoms. Liver conditions were reported by most respondents (83%). Adults and caregivers frequently reported impacts such as difficulty keeping up with peers (77%) and difficulty exercising/difficulty with physical activity (53%). Hypoglycemia was frequently reported in both adults and children, with more than half reporting hospitalizations due to hypoglycemia. Caregivers focused on hypoglycemia when reporting signs/symptoms that most interfere with their child's life and prevention of hypoglycemia as a desired outcome for an effective therapy. Adults most often reported muscle weakness as a top interfering symptom and the most important goal of a potential therapy. Impacts were also reported in activities of daily living, cognitive, emotional, work/school, and sleep domains. Conclusion: Individuals with GSD III experience a broad spectrum of symptoms and disease impacts. There is an unmet need for therapies that improve metabolic control, reduce the burden of dietary management, reduce fatigue and liver problems, and improve muscle strength and function.

The Vulvodynia Experience Questionnaire: Qualitative Development of a New Patient-Reported Outcome Measure for Vulvodynia.

INTRODUCTION: Vulvodynia is defined as vulvar pain of at least 3 months duration without a clear identifiable cause. There are currently no validated questionnaires that assess the experience of women with localized vulvodynia of the vestibule (vestibulodynia, previously known as vulvar vestibulitis) that meet the requirements of the Food and Drug Administration's Patient Reported Outcome (PRO) Guidance. AIM: To develop a new content-valid PRO assessment in accordance with the Food and Drug Administration's PRO guidance to assess the symptoms and impacts of localized vulvodynia. MATERIAL AND METHODS: Participants were recruited for concept elicitation interviews (ie, interviews with open-ended questions with the goal of eliciting volunteered data about the symptoms and impacts of vulvodynia). Participants were identified as having localized vulvodynia by clinicians who were experts in treating vulvar disorders. Eligibility was confirmed by the recruiting clinician, and informed consent was obtained; participants were then scheduled for in-person interviews. 25 participants were interviewed from United States (US). After concept elicitation interviews, the draft Vulvodynia Experience Questionnaire (VEQ) was developed based on the results. Cognitive interviews were conducted with 20 participants from US sites to assess the content validity of the VEQ (eg, interpretation and clarity of the items, relevance of concepts). The VEQ was further revised after cognitive interviews. All interviews were conducted face-to-face, audio-recorded, transcribed verbatim, anonymized, and analyzed using a qualitative data analysis software program. RESULTS: 17 unique symptoms and 32 unique impacts were reported during concept elicitation interviews. Pain (n = 25, 100%) and burning (n = 24, 96%) were the most frequently reported symptoms of localized vulvodynia, and negative impact on emotional well-being (n = 25, 100%) was the most frequently reported impact. After analysis, item generation, and cognitive interviews, the resulting VEQ v2.0 contains 3 parts (part 1, pain; part 2, associated symptoms; part 3, impacts) with a total of 25 items that measure the most frequently reported symptoms and impacts of localized vulvodynia. STRENGTH AND LIMITATIONS: The VEQ is a multidimensional assessment of the core symptoms and impacts of localized vulvodynia that, after additional psychometric testing including the ability to detect change, may be used in clinical trials to characterize the benefits of novel treatments. The VEQ requires additional testing to establish its cultural relevance and linguistic validity in other countries. CONCLUSION: The VEQ is a novel method of collecting information on localized vulvodynia symptoms and impacts that may be suitable for use in clinical trials after psychometric testing. Goldstein AT, Diez PMQ, Kapanadze S, et al. The Vulvodynia Experience Questionnaire: Qualitative Development of a New Patient-Reported Outcome Measure for Vulvodynia. J Sex Med 2020;17:2055-2066.

Navigating a 'Perfect Storm' on the Path to Prevention of Type 2 Diabetes Mellitus After Gestational Diabetes: Lessons from Patient and Provider Narratives.

Objectives Complications of pregnancy such as gestational diabetes mellitus (GDM) forewarn future chronic illness and disability, and demonstrate the need for a life course approach to prevention. Our study had two aims: (1) to elucidate how experiences reported by patients and providers converge to facilitate or impede follow-up care after GDM, and (2) to elicit recommendations for system-level changes to enhance prevention across key care transitions. Methods We conducted in-depth interviews with 30 GDM patients and 29 providers of maternity, specialty and primary care in an urban safety hospital network, and used a three-tiered thematic analysis to interpret their narratives. Results Findings reveal that a 'perfect storm' gathers on the path to prevention across stages of care. At diagnosis, patients feel profound anxiety about the debilitating effects of type 2 diabetes mellitus in their communities, providers choose reassurance over risk communication, and both focus primarily on the birth of a healthy baby. Providers report that clinical teams often lack coordination, and confuse patients with a barrage of often-inconsistent advice. In the postpartum period, providers juggle competing clinical priorities and mothers juggle overwhelming demands; for both, the recommended 2-h oral glucose tolerance test is too arduous for women and providers to do as prescribed. Finally, the transition from maternity to primary care is complicated by communication barriers between clinicians and patients, and between maternity and primary care providers. Conclusions for Practice Respondents propose systems innovations to open communication between provider specialties in order to bridge the chasm between reproductive care and life course prevention.

Design and methods for a community-based intervention to reduce sugar-sweetened beverage consumption among youth: H2GO! study.

BACKGROUND: Reducing sugar-sweetened beverage (SSB) intake is an important dietary target among underserved children at high risk for obesity and associated morbidities. Community-based approaches to reduce SSB intake are needed. The use of narrative-based approaches (presenting messages within the context of a story) can facilitate connection with target health messages and empower children as behavior change agents within their families. The H2GO! program is a community-based behavioral intervention that integrates narrative-based strategies to reduce SSB consumption and promote water intake among school-age youth and parents. METHODS: Guided by the Social Cognitive Theory and the Social Ecological Model, the H2GO! intervention consists of 6 weekly sessions that target beverage knowledge, attitudes, and behaviors through youth-produced messages and narratives to reduce SSB intake and encourage water intake and parent-child activities. To reach underserved youth and families, we identified Boys & Girls Clubs (B&GC) (youth-based community centers that serve an ethnically diverse and predominantly low socioeconomic status population) as a community partner and study setting. Participants (children ages 9-12 years and their parents) will be recruited from B&GC sites in Massachusetts, USA. Intervention efficacy will be assessed through a site-randomized trial (N = 2 youth-based community sites, pair-matched for size and racial/ethnic composition) with 54 parent-child pairs (N = 108) enrolled per site (N = 216 total). The comparison site will carry on with usual practice. Child and parental SSB and water consumption (primary outcomes) and parent and child beverage knowledge and attitudes (secondary outcomes) will be measured via self-report surveys. Additional outcomes include children's anthropometric data, additional dietary behaviors, and physical activity. Measures will be collected at baseline, 2 and 6 months follow-up. With an estimated 20 % dropout rate, the study will have 80 % power to detect a group difference of 3.9 servings of SSBs per week. DISCUSSION: Community-based approaches hold potential for decreasing SSB consumption among youth and families, particularly among underserved populations who are at greater obesity risk. This article describes the design and methods of a community-based behavioral intervention designed to reduce SSB consumption among youth and parents/caregivers. TRIAL REGISTRATION: ClinicalTrials.gov NCT02890056 . Date of Registration: August 31, 2016.

The phenol red thread test for lacrimal volume--does it matter if the eyes are open or closed?

OBJECT: To assess the results from using a commercially available phenol red thread (PRT) test with the eyes being kept open (as recommended by the company) vs a closed eye protocol (as used by some investigators). METHODS: A single PRT (Zone-Quick) test was carried out on 97 young adults (average age 21 years, range: 17-36) for 15 s with either the eyes open or closed. The right eye was assessed with 52 subjects undertaking the open eye test first, with the test being repeated 1 h later in the same indoor environment (temperature 21 degrees C and 40% humidity). RESULTS: The mean ( +/- S.D.) PRT value for the open eye protocol was marginally higher (at 20.5 +/- 7.0 mm) than for the closed eye (19.7 +/- 5.9 mm), but the overall outcome from the two protocols could range from being 18 mm greater in the open eye to 15 mm greater in the closed eye. Overall, the difference was not statistically different, neither were any substantial differences noted according to age, gender, iris colour, prior contact lens wear or according to which protocol was used first (p >or= 0.2). CONCLUSION: These studies indicate that there can be a small difference in single PRT test data according to whether the eyes are kept open or closed, and it would be useful if the protocol used was clearly stated.

One year prospective survey of Candida bloodstream infections in Scotland.

A 12 month survey of candidaemia in Scotland, UK, in which every Scottish hospital laboratory submitted all blood isolates of yeasts for identification, strain typing and susceptibility testing, provided 300 isolates from 242 patients, generating incidence data of 4.8 cases per 100,000 population per year and 5.9 cases per 100,000 acute occupied bed days; 27.9 % of cases occurred in intensive care units. More than half the patients with candidaemia had an underlying disease involving the abdomen, 78 % had an indwelling intravenous catheter, 62 % had suffered a bacterial infection within the 2 weeks prior to candidaemia and 37 % had undergone a laparotomy. Candida albicans was the infecting species in 50 % of cases, followed by Candida glabrata (21 %) and Candida parapsilosis (12 %). Seven cases of candidaemia were caused by Candida dubliniensis, which was more prevalent even than Candida lusitaniae and Candida tropicalis (six cases each). Among C. glabrata isolates, 55 % showed reduced susceptibility to fluconazole, but azole resistance among other species was extremely low. Multilocus sequence typing showed isolates with high similarity came from different hospitals across the country, and many different types came from the hospitals that submitted the most isolates, indicating no tendency towards hospital-specific endemic strains. Multiple isolates of C. albicans and C. glabrata from individual patients were of the same strain type with single exceptions for each species. The high prevalence of candidaemia in Scotland, relative to other population-based European studies, and the high level of reduced fluconazole susceptibility of Scottish C. glabrata isolates warrant continued future surveillance of invasive Candida infections.

Candida albicans and Candida dubliniensis respond differently to echinocandin antifungal agents in vitro.

Candida dubliniensis isolates tested for susceptibility to anidulafungin, caspofungin, and micafungin commonly showed artifactual regrowth and/or trailing effects with MIC tests done under conditions involving a high initial yeast concentration. The artifacts were less common with Candida albicans and seldom seen for either species under Clinical and Laboratory Standards Institute method M27-A test conditions.

Efficacy of caspofungin and voriconazole combinations in experimental aspergillosis.

Guinea pigs were infected with Aspergillus fumigatus at two challenge doses and treated for 7 days with a placebo, intraperitoneal caspofungin (1 mg/kg daily), oral voriconazole (1 mg/kg twice a day), or a combination of the caspofungin and voriconazole treatments. The combination therapy statistically significantly prolonged survival over that with the control at both challenge doses and achieved a statistically significant reduction in kidney burdens as measured by quantitative PCR. The same was true for animals given caspofungin alone at both levels of challenge and for animals treated with voriconazole alone at the lower challenge dose. However, the effects of combination therapy on prolongation of survival were greater than those of either monotherapy at both challenge doses, and the reduction in kidney burdens with combination therapy was significantly greater than that with caspofungin alone in the animals given the lower challenge dose. No synergistic interactive effects were seen for the two agents in checkerboard titration experiments in vitro. We conclude that therapy of experimental aspergillosis with caspofungin and voriconazole combined offers slight additional improvements in efficacy rather than effects of a clearly synergistic nature.