Abstract Background Research priorities are best defined through engagement with communities who will be impacted by the research and have lived experience of the topics to be studied. We aimed to establish a pediatric rare disease community stakeholder group and empower them in (1) eliciting perspectives from affected families in the wider region and (2) synthesizing collective ideas into a research agenda focused on shared ethical, legal, and social implications (ELSI) across rare disease. Methods This two-year project utilized a community-centered approach to engage rare disease community members as equal partners in developing a research agenda for ELSI in rare disease. We established “Rare Voices” (RV), a 22-member stakeholder group of patients, parents, clinicians and researchers. Following capacity-building trainings, RV designed and conducted listening sessions with teen patients and parents of children with rare diseases to explore challenges, positive experiences, and ethical concerns. Listening session findings were synthesized and contextualized into research topics, which RV members further refined and prioritized. We used established measures to assess RV member engagement and satisfaction. Results From 14 listening sessions with parents (n = 52) and teen patients (n = 13), RV identified eight core research topics as most important for future rare disease research: coordinating care, communication, accessing resources and care, impact on family unit, community and support in society, mental health and identity, ethical aspects of care, and uncertainty. RV members were highly engaged throughout the two-year project and reported high levels of satisfaction with the experience and research agenda. Conclusions Through capacity-building and authentic engagement, this project resulted in a community-led rare disease research agenda to guide future rare disease ELSI research that aligns with patients’ and families’ priorities. An environment of equal partnership and respect created a space for mutual learning where community members were empowered to shape the research agenda based on their collective experiences. The agenda recognizes the shared psychosocial and healthcare experiences of rare disease and offers practical areas of research to address patient and family needs.
Suicide rates among adolescents in the United States continue to climb and many at-risk youths are undetected. Screening for suicidal thoughts has become the primary approach to identify those at risk, but no studies have assessed reactions to its deployment in pediatric outpatient settings. This mixed-method study assessed parents' and adolescents' thoughts about suicide risk screening in non-psychiatric, pediatric outpatient specialty settings.As part of a multi-site measurement validation study, adolescents (n = 269; ages 10–21) and parents (n = 246) at pediatric specialty clinics in the Midwest completed a survey regarding thoughts about suicide risk screening. Data were collected on tablet computers and transcribed verbatim. Three study team members independently coded transcripts of open-ended responses to identify major themes, and frequency data were analyzed using StataSE 15.1. Inter-rater agreement was substantial (Fleiss' Kappa ranged 75–86%).Parents (55% 41–50 years of age, 20% male, 80% White) and adolescents (Mean age = 14.3, 50% male, 77% White) agreed medical providers should screen adolescents for suicide risk (93% and 88%, respectively). Majority of parents indicated that the pediatric outpatient setting is appropriate for suicide risk screening. Major themes included the important role of providers in identifying at-risk youth, the potential for screening to prevent suicides, and concerns about iatrogenic risk and misdiagnosis.Most parents and adolescents support screening for suicide risk in pediatric outpatient settings. Nevertheless, some have concerns about the screening process and implications. As suicide risk screening becomes standard practice in adolescent care, it's critical to develop screening processes that maximize comfort and address concerns.
Social epigenomics research investigates links between social experiences and epigenetic modifications, which may ultimately impact health. Such research holds promise for precision medicine and addressing health disparities based on social conditions, but also brings unique ethical challenges. The linking of social experiences to biological changes risks pathologizing experiences, potentially leading individuals and communities to be seen as 'damaged.' This stigmatization or stereotyping based on experiences also risks placing disproportionate personal responsibility for health. These risks are likely to be amplified in historically marginalized communities already facing discrimination. It is therefore essential to engage members of historically marginalized communities to explore attitudes about social epigenomics research. This study focuses on the Black and African American (B/AA) population in the USA, studying perceptions of social epigenomic research participants, research decliners, and broadly representative community members to identify perceived benefits and risks of social epigenomic research as well as strategies to maximize benefits and lower risks for both participants and communities. Both research participants and community members perceived potential benefit of social epigenomic research for the B/AA population. While most research participants did not perceive research related risks, community members identified risks both specific to social epigenomic research and more generalized to medical research. Several of the risks identified, and a belief that the likelihood of harms was greater than the likelihood of benefits, were based on past research injustices to B/AA research participants and mistrust in the medical and research enterprise. However, community members provided concrete strategies for maximizing the chance of benefits and lowering risk of harms including acknowledging and addressing biases and past injustices, ensuring transparency and understanding, positive framing of research, thorough research and dissemination, and engaging with communities before, throughout, and beyond the research process. While B/AA community members identified risk of both individual and community harm from social epigenomic research, they also perceived potential health benefits for the B/AA community. Through concerted efforts to apply community recommendations to lower risks and enhance benefits, researchers can conduct ethical and valid epigenomic research that aims to address health disparities with historically marginalized communities.
Objective: To determine if Motivational Interviewing (MI) versus health education (HE) elicited different types of client language and whether these differences were associated with outcomes in a randomized clinical trial (RCT) for cessation induction among people who smoke with low motivation to quit. Methods: A secondary data analysis was conducted using data from the MI and HE arms of a trial in which people who smoke (N = 202) with low desire to quit were randomly assigned to four sessions of MI, HE or brief advice. Mediation analyses examined two types of client language: change talk (CT) and a novel form of client speech called "learning talk" (LT). Outcomes were assessed at baseline, 3 and 6 months. Results: With HE as the reference group, MI resulted in greater CT (OR = 3.0, 95% CI: 1.7-5.5) which was associated with better outcomes (average d = .34, SD = .13) and HE resulted in greater LT (OR = .05, 95% CI: .02-.10) which was also associated with better outcomes (average d = .42, SD = .08). Indirect parallel mediation effects on quit attempts were significant for both MI-CT (OR = 1.4, 95% CI: 1.1-1.7) and HE-LT (OR = .4, 95% CI: .2-.7). Conclusions: MI and HE were both efficacious via different pathways to change, confirming the utility of MI in this RCT as well as highlighting the potential of HE based on the "5R's" for smoking cessation. These findings emphasize the value of exploring theorized mechanisms of action of interventions evaluated in RCTs. (PsycInfo Database Record (c) 2021 APA, all rights reserved).
The ability to apply results from a study to a broader population remains a primary objective in translational science. Distinct from intrinsic elements of scientific rigor, the extrinsic concept of generalization requires there be alignment between a study cohort and population in which results are expected to be applied. Widespread efforts have been made to quantify representativeness of study cohorts. These techniques, however, often consider the study and target cohorts as monolithic collections that can be directly compared. Overlooking known impacts to health from socio-demographic and environmental factors tied to individual's geographical location, and potentially obfuscating misalignment in underrepresented population subgroups. This manuscript introduces several measures to account for geographic information in the assessment of cohort representation.
Interstage outcomes for babies with single ventricle remain suboptimal. We have developed a tablet PC-based platform (CHAMP) for remote monitoring. This provides immediate access to data and instant alerts to the team. This study evaluates the caregiver experience with CHAMP, and its impact on interstage mortality, morbidity and resource utilization. Methods: All neonates with single ventricle who were discharged from 5/2014 to 5/2015 were prospectively enrolled. For 1 month after discharge, they were all monitored using a notebook. They were randomized to receive CHAMP at either 1 or 2 months post discharge. A month after randomization, caregivers had to choose either the notebook or CHAMP for the remainder of the inter-stage period. Charts were reviewed for mortality, unplanned readmissions and hospital charges. Caregiver experience was assessed through an exit survey. Results: We enrolled 24 babies (Norwood, n=11; BT shunt, n=9; no stage I at discharge (balanced circulation), n=3; hybrid, n=1). They were interstage for 3143 days. There was no interstage mortality in either group. While using CHAMP, families transmitted data on 77% of days. Resource utilization is summarized in the Table. CHAMP instant alerts and scheduled daily alerts led to 10 readmissions for issues that were not recognized by caregivers (low saturations (n=6) and poor feeding / weight gain (n=4). When given the option after randomization, 23 of 24 families chose CHAMP. At the end of monitoring, 23 completed an exit survey; when asked what form of monitoring they would choose if they had to do this over, 19 (82%) stated they would choose CHAMP, 3 would choose either, and 1 would choose the notebook. Conclusions: CHAMP monitoring was associated with significant decreases in unplanned readmission days, ICU days and hospital charges. CHAMP was well-accepted by caregivers, and would appear to facilitate outpatient care for the fragile population of interstage babies with single ventricle.
Little is known about effective methods to recruit unmotivated smokers into cessation induction trials, the reasons unmotivated smokers agree to participate, and the impact of those reasons on study outcomes. A mixed-method approach was used to examine recruitment data from a randomized controlled cessation induction trial that enrolled 255 adult smokers with low motivation to quit. Over 15 months, 33% of smokers who inquired about the study were enrolled. Common recruitment methods included word-of-mouth, print advertisements and clinic referrals. Frequently mentioned reasons for participating included to: gain financial incentives (44.7%), learn about research or help others quit (43%), learn about smoking and risks (40%) and help with future quits (i.e. Quit Assistance, 23.9%). Separate regression models predicting study outcomes at 26 weeks indicated that smokers who said they participated for Quit Assistance reported higher motivation to quit (B 1.26) and were more likely to have made a quit attempt (OR 2.03) compared to those not mentioning this reason, when baseline characteristics were controlled. Understanding reasons for unmotivated smokers' interest in treatment can help practitioners and researchers design effective strategies to engage this population.
