A qualitative study of perceptions of meaningful change in spinal muscular atrophy.

BACKGROUND: This qualitative study examined how individuals with Spinal Muscular Atrophy (SMA), their caregivers, and clinicians defined meaningful change, primarily in the Type II and non-ambulant type III patient populations, associated with treatment of this condition. In addition, we explored participants' views about two measures of motor function routinely used in clinical trials for these SMA subtypes, namely the expanded version of the Hammersmith Functional Motor Scale (HFMSE) and the Upper Limb Module (ULM). METHODS: The 123 participants (21 with SMA, 64 parents, and 11 clinicians), recruited through SMA advocacy organizations, participated in one of 16 focus groups or 37 interviews. The sessions were audio-recorded, and verbatim transcripts were analyzed using a grounded theory approach. RESULTS: For the participants, meaningful change was relative to functional ability, and small changes in motor function could have an important impact on quality of life. Because patients and families feared progressive loss of functional ability, the participants saw maintenance of abilities as a meaningful outcome. They believed that measures of motor function covered important items, but worried that the HFMSE and ULM might not be sensitive enough to capture small changes. In addition, they felt that outcome measures should assess other important features of life with SMA, including the ability to perform daily activities, respiratory function, swallowing, fatigue, and endurance. CONCLUSIONS: Given the heterogeneity of SMA, it is important to expand the assessment of treatment effects to a broader range of outcomes using measures sensitive enough to detect small changes.

Understanding the experiences and needs of individuals with Spinal Muscular Atrophy and their parents: a qualitative study.

BACKGROUND: The clinical features of SMA, which range along a spectrum of severity, are relatively well described. In contrast, the literature on how individuals with SMA and their families experience this condition is limited. To address this gap, we undertook a qualitative study with individuals affected by SMA Types I, II and III, parents of those affected, and clinicians. METHODS: We completed 16 focus group sessions and 37 interviews in the US with 96 participants including: 21 with individuals with SMA; 64 parents of individuals affected by SMA; and 11 clinicians who specialize in the care of SMA patients. RESULTS: The Diagnostic Journey: Families reported substantial diagnostic delays owing to: 1) lack of awareness and knowledge about SMA; 2) the difficulty of distinguishing normal from abnormal development; and 3) the challenge of differential diagnosis. Lack of sensitivity in how clinicians communicated this potentially devastating diagnosis compounded parents' negative impressions. Newborn Screening: Parents generally held positive views about adding SMA to newborn screening panels. For example, it would: 1) enable earlier access to care; 2) shorten the diagnostic journey; and 3) give families more time to prepare to care for a disabled child. Some noted negative outcomes such as prematurely affecting a parent's relationship with a child before symptoms are evident. The Psychosocial Impact of Living with SMA: Ten thematic areas characterized the impact: 1) confronting premature death; 2) making difficult treatment choices; 3) fearing the loss of functional ability; 4) coming to terms with lost expectations; 5) loss of sleep and stress; 6) stigma; 7) limitations on social activities; 8) independence; 9) uncertainty and helplessness; and 10) family finances. CONCLUSIONS: The results of this study suggest high levels of burden experienced by individuals with SMA and their families. The difficulties of living with SMA begin with the long and often arduous process of finding a diagnosis for their child. Newborn screening for SMA is seen as an important step toward shortening this journey. The psychosocial effects of coping with SMA are substantial and wide ranging both for the individual living with this condition and family members of affected individuals.

Sexual Behavior Change Among Gay and Bisexual Men During the First COVID-19 Pandemic Wave in the United States.

Background: After decades of navigating HIV and other sexually transmitted infections, gay and bisexual men are responding to new and uncertain risks presented by the coronavirus (COVID-19) pandemic by adapting their sexual behavior. Methods: This paper uses data from a national sample of 728 gay and bisexual men collected from April 10 to May 10, 2020, to examine changes to sexual behavior in response to the first wave of the pandemic in the USA. We also assess whether behavior modifications are associated with exposure to statewide public health measures, including Stay-at-Home orders. Results: Sexual minority men report significant changes to their sexual behavior and partner selection during the first wave. Nine out of 10 men reported having either one sexual partner or no sexual partner in the last 30 days at the time of interview, a decrease compared to just before the pandemic for nearly half of men surveyed. Reporting no sexual partners in the last 30 days was significantly predicted by increased exposure to a Stay-at-Home order. Sexual minority men also reduced interactions with casual partners, increased no-contact sexual behaviors (e.g., masturbation and virtual sex), and engaged in new strategies to reduce their risks of infection from partners. HIV-positive men were particularly likely to adopt strategies including avoiding casual partners and avoiding public transportation to meet sexual partners. Conclusion: Sexual minority men's behavior changes during the first wave may have reduced the impact of the coronavirus pandemic on their communities. Despite substantial changes in sexual behavior for most men in our sample during the initial first wave, we identify some concerns around the sustainability of certain behavioral changes over time and nondisclosure of COVID-19 symptoms to partners.

Using survey results regarding hepatitis B knowledge, community awareness and testing behavior among Asians to improve the San Francisco Hep B Free campaign.

Asians are disproportionately affected by chronic hepatitis B (HBV) infection and its fatal consequences. The Hep B Free campaign was launched to eliminate HBV in San Francisco by increasing awareness, testing, vaccination and linkage to care. The campaign conducted 306 street intercept and telephone interviews of San Francisco Asians to assess current levels of HBV knowledge, testing behaviors and effectiveness of existing campaign media materials. One-third of respondents ranked HBV as a key health issue in the Asian community, second to diabetes. General HBV awareness is high (85%); however, a majority could not name an effective prevention method. Sixty percent reported having been tested for HBV; provider recommendation was the most often cited reason for testing. Respondents reported a high level of trust in their providers to correctly assess which health issues they may be at risk for developing and test accordingly, confirming that efforts to increase HBV testing among Asians must simultaneously mobilize the public to request testing and compel providers to test high-risk patients. Regarding community awareness, more than half reported hearing more about HBV recently; younger respondents were more likely to have encountered campaign materials and recall correct HBV facts. Assessment of specific campaign materials found that while upbeat images and taglines captured attention and destigmatized HBV, messages that emphasize the pervasiveness and deadly consequence of infection were more likely to drive respondents to seek education and testing. The campaign used survey results to focus efforts on more intensive provider outreach and to create messages for a new public outreach media campaign.

Treatment Goals for Achondroplasia: A Qualitative Study with Parents and Adults.

INTRODUCTION: Achondroplasia is characterized by disproportionate short stature accompanied by other changes to the musculoskeletal system. Individuals with this condition typically experience a variety of medical complications. As pharmacologic treatments continue to be developed for the treatment of achondroplasia, it is important to understand treatment goals among those affected by achondroplasia and the factors that shape their goals. METHODS: This qualitative study is based on semi-structured interviews with 19 parents of children with achondroplasia and five adults with achondroplasia in the USA. We employed thematic analysis using an iterative process to identify themes across the interviews. RESULTS: Participants had two goals for pharmacologic treatment of achondroplasia: ameliorating complications associated with the condition and increasing stature to overcome functional limitations and psychosocial challenges. Complications of particular concern were chronic pain and surgeries to repair spinal, ear, nose, and throat (ENT) problems, and neurological sequelae. Increased height would enhance independence, help individuals to fit in socially, and avoid social stigma. Countervailing factors included the importance of stature to their identity and the concern that the condition would remain despite treatment. CONCLUSIONS: This study offers evidence about how individuals affected by achondroplasia think about the pharmacologic treatment of this condition, including both the benefits of ameliorating complications and increasing height. The findings can offer practical insights for parents of children considering treatment, treating physicians, and decision-makers evaluating coverage decisions for treatment of achondroplasia.

Design of a population-based longitudinal cohort study of SARS-CoV-2 incidence and prevalence among adults in the San Francisco Bay Area.

PURPOSE: We describe the design of a longitudinal cohort study to determine SARS-CoV-2 incidence and prevalence among a population-based sample of adults living in six San Francisco Bay Area counties. METHODS: Using an address-based sample, we stratified households by county and by census-tract risk. Risk strata were determined by using regression models to predict infections by geographic area using census-level sociodemographic and health characteristics. We disproportionately sampled high and medium risk strata, which had smaller population sizes, to improve precision of estimates, and calculated a desired sample size of 3400. Participants were primarily recruited by mail and were followed monthly with PCR testing of nasopharyngeal swabs, testing of venous blood samples for antibodies to SARS-CoV-2 spike and nucleocapsid antigens, and testing of the presence of neutralizing antibodies, with completion of questionnaires about socio-demographics and behavior. Estimates of incidence and prevalence will be weighted by county, risk strata and sociodemographic characteristics of non-responders, and will take into account laboratory test performance. RESULTS: We enrolled 3842 adults from August to December 2020, and completed follow-up March 31, 2021. We reached target sample sizes within most strata. CONCLUSIONS: Our stratified random sampling design will allow us to recruit a robust general population cohort of adults to determine the incidence of SARS-CoV-2 infection. Identifying risk strata was unique to the design and will help ensure precise estimates, and high-performance testing for presence of virus and antibodies will enable accurate ascertainment of infections.

Impact of Peyronie's disease on sexual and psychosocial functioning: qualitative findings in patients and controls.

INTRODUCTION: There are no validated scales for assessing the psychosocial impact of Peyronie's disease (PD), which affects approximately 5-10% of men over age 50. AIM: To develop a psychometrically valid outcome measure for assessing psychosocial and sexual consequences of PD. To conduct a qualitative study of men with PD and age-matched controls, and design a new patient-reported outcome measure of PD. METHODS: An expert advisory panel identified relevant topics and conceptual areas to be addressed based on clinical experience and literature reviews. A conceptual model was developed to serve as a discussion guide for qualitative interviews with geographically and ethnically diverse PD subjects and controls. Interviews were conducted in a focus-group format by a trained interviewer and were recorded and transcribed for qualitative analysis according to grounded theory concepts. MAIN OUTCOME MEASURE: Focus-group interviews. RESULTS: Focus-group interviews were conducted with 64 men (28 PD patients, 36 controls) in 13 separate focus groups over a 3-month period. Blinded analysis of the interview transcripts identified four core domains: (i) physical appearance and self-image; (ii) sexual function and performance; (iii) PD-related pain and discomfort; and (iv) social stigmatization and isolation. Based on feedback from participants and experts, a new outcome questionnaire was developed to assess core domain responses in a structured, self-report format. CONCLUSIONS: This qualitative study helped to refine and broaden the focus of the conceptual model for further assessment. It also confirmed that PD has a major impact on sexual and psychological function in these patients.

Survey of U.S. Boards that Review Mental Health-related Research.

WE OBTAINED DATA ON INSTITUTIONAL Review Boards (IRBs) that review mental health-related applications (MHRAs) in a national survey of institutions with federally assured human research protection programs. Approximately 57% of IRBs review MHRAs, and among these a small percentage may not have mental health experts on their committees (5%). Moreover, mental health experts on IRB committees at high research volume institutions are carrying substantially greater workloads than their lower volume counterparts. In terms of committee demographics, more women (36%) are serving as IRB Chairs on committees that review MHRAs than expected from their representation on medical or university faculties; ethnic minority faculty have lower representation among Chairs than might be expected from their overall faculty representation. Our findings suggest the need for additional studies to (a) examine if the number of mental health experts on IRBs should be increased particularly among IRBs reviewing a high volume of MHRAs, (b) determine if the breadth of expertise among IRB mental health experts corresponds to the range of substantive and methodological approaches represented by the mental health protocols under review, and (c) examine if recruiting IRB scientific expertise from outside an institution, a more common practice among smaller research entities, impacts review quality.

Survey of u.s. Human research protection organizations: workload and membership.

IN A NATIONAL SURVEY OF INSTITUTIONS with federally assured human research protection programs, we obtained workload and other relevant data on their Institutional Review Boards (IRBs) and management organizations. The number of IRBs increased substantially from 1995 to 2005/06 (491 to 2,728 IRBs) with a further increase in 2008 to 3,853 IRBs. Nationally, IRBs reviewed over a quarter million research applications in the year prior to our survey, of which 35% were new applications requiring full committee review. Compared to estimates from 1995, current IRBs review more new and full committee review applications, but the relative percentage of new and full committee applications remained about the same. High volume research institutions have IRBs with a substantially larger per person workload, relative to smaller volume IRBs (i.e., members spent nearly seven times more hours reviewing new applications outside formal committee meetings). Virtually all IRBs included community representatives as members (92%); however, a small number may not be compliant with federal regulations. The present findings suggest the need for research to (a) examine workload and its effects on review quality, research costs, and faculty morale, (b) develop methods for determining optimal fit between IRB workload demands and institutional labor and financing requirements, (c) construct benchmarks for judging reasonable workload for individual IRB members, and (d) examine if the need to recruit IRB scientific expertise from outside the institution, particularly true for smaller research institutions, causes delays in IRB review, and if a more effective way of locating and recruiting experts would improve quality and time to completion.

Research Participants' Perceptions of the Certificate of Confidentiality's Assurances and Limitations.

THE CERTIFICATE OF CONFIDENTIALITY (COC) provides additional protections to personal and sensitive research data. COC guarantees are not absolute and investigators are obligated to inform potential participants of COC limitations. The present study utilized qualitative and partnership methodology to examine participants' (N = 24) perceptions of COC assurances and limitations in the context of a hypothetical study on depression. Although some participants were comforted by COC assurances, a majority of participants had confidentiality/privacy concerns specifically with COC passages concerning federal audits and legal reporting requirements. As one respondent noted, "Why is it that you guys don't have to turn the records over to the court unless I say so . . . but you have to give them over to the government? . . . I don't know about what is goin' on." Our findings underscore the need for larger quantitative investigations to examine the negative and positive impact of COCs on research participation and response bias.

Viagra use in a community-recruited sample of men who have sex with men, San Francisco.

The authors conducted a community-based anonymous survey of 837 men who have sex with men (MSM) to gauge the extent of Viagra (sildenafil citrate) use, its association with high-risk sexual behavior, and its combination with other drugs. Participants' mean age was 35 years, 67% were white, and 66% had a college degree. The majority (76%) reported anal sex in the past 6 months, with 49% reporting always using condoms. Overall, 32% had ever used Viagra (CI: 29%-36%). Significant independent predictors of Viagra use were white race, older age, HIV positivity, illicit drug use, and having had unprotected anal sex with potentially serodiscordant partners. Over one third of Viagra users had combined Viagra with other drugs, 18% with amyl nitrate. Only a minority (44%) obtained Viagra under the care of a physician. For some MSM, Viagra appears to be an emerging contributing factor to unsafe sex, potentially increasing HIV transmission. HIV care and prevention providers should target Viagra users for enhanced education on safer sex and potentially harmful drug interactions.