Diabetes, metformin and cancer risk in myotonic dystrophy type I.

Myotonic dystrophy type I (DM1) is an autosomal dominant multisystem disorder characterized by myotonia and muscle weakness. Type 2 diabetes (T2D) and cancer have been shown to be part of the DM1 phenotype. Metformin, a well-established agent for the management of T2D, is thought to have cancer-preventive effects in the general population. In our study, we aimed to assess the association between T2D, metformin use and the risk of cancer in DM1 patients. We identified a cohort of 913 DM1 patients and an age-, sex- and clinic-matched cohort of 12,318 DM1-free controls from the UK Clinical Practice Research Datalink, a large primary care records database. We used Cox regression models to assess cancer risk in T2D patients who were metformin users or nonusers compared to patients without T2D. Separate analyses were conducted for DM1 patients and controls. T2D was more prevalent in DM1 than in controls (8% vs. 3%, p < 0.0001). DM1 patients with T2D, compared to those without T2D, were more likely to develop cancer (hazard ratio [HR] = 3.60, 95% confidence interval [CI] = 1.18-10.97; p = 0.02), but not if they were treated with metformin (HR = 0.43, 95% CI = 0.06-3.35; p = 0.42). Among controls, we observed no significant associations between T2D and cancer risk in either users or nonusers of Metformin (HR = 1.28, 95% CI = 0.91-1.79; p = 0.16 and HR = 1.13, 95% CI = 0.72-1.79; p = 0.59, respectively). These results show an association between T2D and cancer risk in DM1 patients and may provide new insights into the possible benefits of Metformin use in DM1.

Training Teachers to Teach Epidemiology in Middle and High Schools.

Objective The purpose of this study was to evaluate the outcomes of a workshop which was designed to prepare teachers to teach epidemiology to middle and high school students. Methods The workshop introduced the fundamental enduring understandings of epidemiology as well as a pedagogical framework for teaching it. Using an online questionnaire and telephone interviews, we assessed post-workshop outcomes. Results The majority (80%) of workshop alumni had taught epidemiology with the most common approach (52%) being the inclusion of epidemiologic concepts into other courses. Teachers felt that learning epidemiology was a valuable experience for their students and reported that students found it engaging and relevant to their lives. Conclusions These preliminary findings suggest that there may be benefit to teaching epidemiology in middle and high schools. Further research should directly evaluate public health-related outcomes among students, such as improvements in health literacy and health behavior.

Examining Sexual and Dating Violence by Gender Identity Among High School Students.

PURPOSE: This study examined the reporting of sexual and dating violence among high school students using a standard gender identity question. METHODS: Using data from the 2017 and 2019 Youth Risk Behavior Surveys, multivariable regression models estimated the association between gender identity and four measures of sexual and dating violence, adjusted for confounding by race/ethnicity, grade, and reported sex. RESULTS: Of the 198,900 teenagers, 1.8% identified as transgender, 1.6% were not sure, and 1.9% responded that they did not know what question was being asked. Across all measures of violence, transgender, "unsure" and "don't understand" students were significantly more likely than cisgender students to report having been victimized. DISCUSSION: Standard analysis practices of eliminating unsure and/or don't understand responses to gender identity questions may underestimate at-risk youth. Researchers seeking to understand disparities in youth violence by gender identity will need to ensure that they employ inclusive measures.

Health Savings Account Ownership and Financial Barriers to Health Care: What Social Workers Should Know.

Over a decade ago, Health Savings Accounts (HSAs) were deemed contrary to social work values, leading to greater inequality in access to health care. Using data from the 2015 National Health Interview Survey (NHIS) (n= 12,265), we examine whether HSA ownership is associated with unmet need for health care due to cost (financial barrier). HSA ownership was significantly associated with reduced financial barriers to health care (p< .001) in the regression model. Owning an HSA may be related to reducing financial barriers to health care access, which could inform improvements in HSA policy provisions for social work practice.

Benign tumors in myotonic dystrophy type I target disease-related cancer sites.

OBJECTIVES: Recent evidence showed that myotonic dystrophy type I (DM1) patients are at increased risk of certain cancers, but the risk of benign tumors is unknown. We compared the risk of benign tumors in DM1 patients with matched DM1-free individuals and assessed the association between benign tumors and subsequent cancers. METHODS: We identified 927 DM1 patients and 13,085 DM1-free individuals matched on gender, birth-year, clinic, and clinic-registration year from the UK Clinical Practice Research Datalink, a primary care records database. We used Cox regression models for statistical analyses. RESULTS: DM1 patients had elevated risks of thyroid nodules (Hazard Ratio [HR] = 10.4; 95% Confidence Interval [CI] = 3.91-27.52; P < 0.001), benign tumors of the brain or nervous system (HR = 8.4; 95% CI = 2.48-28.47; P < 0.001), colorectal polyps (HR = 4.3; 95% CI = 1.76-10.41; P = 0.001), and possibly uterine fibroids (HR = 2.7; 95% CI = 1.22-5.88; P = 0.01). Pilomatricomas and salivary gland adenomas occurred almost exclusively in DM1 patients (Fisher's exact P < 0.001). The HR for colorectal polyps was elevated in DM1 males but not in females (HR = 8.2 vs. 1.3, respectively; P-heterogeneity < 0.001), whereas endocrine and brain tumors occurred exclusively in females. The data suggested an association between benign tumors and subsequent cancer in classic DM1 patients (HR = 2.7; 95% CI = 0.93-7.59; P = 0.07). INTERPRETATION: Our study showed a similar site-specific benign tumor profile to that previously reported for DM1-associated cancers. The possible association between benign tumors and subsequent cancer in classic DM1 patients warrants further investigation as it may guide identifying patients at elevated risk of cancer. Our findings underscore the importance of following population-based screening recommendations in DM1 patients, for example, for colorectal cancer.

Cancer Risk in Myotonic Dystrophy Type I: Evidence of a Role for Disease Severity.

BACKGROUND: Myotonic dystrophy type 1 (DM1) is an inherited trinucleotide repeat disorder in which specific cancers have been implicated as part of the disease phenotype. This study aimed to assess whether cancer risk in DM1 patients is modified by disease severity. METHODS: Using the United Kingdom Clinical Practice Research Datalink (primary care electronic medical records), we identified a cohort of 927 DM1 and a matched cohort of 13 085 DM1-free individuals between January 1, 1988 and February 29, 2016. We used Cox regression models to calculate the hazard ratios (HRs) and 95% confidence intervals (CIs) of organ-specific cancer risks. Analyses were stratified by age at DM1 diagnosis as a surrogate for disease severity. Statistical tests were two-sided. RESULTS: Patients with classic DM1 (age at diagnosis: 11-40 years) were at elevated risk of cancer overall (HR = 1.81; 95% CI = 1.12 to 2.93); cancers of the thyroid (HR = 15.93; 95% CI = 2.45 to 103.64), uterus (HR = 26.76; 95% CI = 2.32 to 309.26), and cutaneous melanoma (HR = 5.98; 95% CI = 1.24 to 28.79) accounted for the excess. In late-onset DM1 patients (age at diagnosis >40 years), a reduced overall cancer risk was observed (HR = 0.53; 95% CI = 0.32 to 0.85), possibly driven by the deficit in hematological malignancies (DM1 = 0 cases, DM1-free = 54 cases; P = .02). The difference between the observed HR for classic and late-onset DM1 was statistically significant (P < .001). CONCLUSIONS: The observed difference in relative cancer risk between classic and late-onset DM1 patients compared with their DM1-free counterparts provides the first evidence that disease severity modifies DM1-related cancer susceptibility. This novel finding may guide clinical management and scientific investigations for the underlying molecular mechanisms in DM-related carcinogenesis.

Participation in research studies: factors associated with failing to meet minority recruitment goals.

PURPOSE: To determine the recruitment goals that investigators set for racial/ethnic minorities and the factors associated with failure to meet those goals. METHODS: Four hundred forty principal investigators (PIs) conducting clinical research funded by the National Heart, Lung, and Blood Institute (NHLBI) in 2001 completed a mailed survey providing their minority recruitment goals and enrollment data for their most recent NHLBI-funded study. RESULTS: Ninety-two percent of PIs set goals for African Americans, 68% for Hispanics, 55% for Asian Americans, 35% for Native Hawaiians/Pacific Islanders, and 23% of PIs set recruitment goals for American Indians/Native Alaskans. Among those PIs who did set minority recruitment goals, the mean goal for the recruitment of African Americans was 31%, 16% for Hispanics, and 9% for Asian Americans. Twenty-seven percent of PIs failed to meet their recruitment goals for African Americans, 23% for Asian Americans, and 23% for Hispanics. After adjusting for multiple investigator and trial characteristics, the type of study (odds ratio [OR] 1.8; 95% confidence interval [CI] 1.2, 3.4 for observational vs. phase III trial) completion of study enrollment (OR 2.0; 95% CI 1.2, 3.4), and PI identification of a larger number of major barriers to participation (OR 1.8; 95% CI 1.1, 3.0) were all associated with failure to meet recruitment goals for African Americans. However, no factors were consistently associated with failure to meet recruitment goals across different racial/ethnic groups. CONCLUSIONS: Investigators often do not set recruitment goals for some racial/ethnic groups. Factors associated with failure to meet recruitment goals vary in the recruitment of different minority groups.

Investigators' assessment of NIH mandated inclusion of women and minorities in research.

BACKGROUND: While investigators were concerned about the impact of mandated inclusion of minorities and women on the conduct of research, there has been little subsequent assessment of investigator views on the effectiveness of the mandate in achieving gender and racial/ethnic research diversity. We describe investigators' assessment of the impact of the mandate on inclusion of women and minorities in research. METHODS: 440 principal investigators (PIs) surveyed between August 2002 and December 2002. RESULTS: Most PIs (69%) felt the mandate has been successful in increasing gender diversity, 7% felt that the mandate had failed to increase females and 24% were unsure. Fifty-five percent of PIs felt the mandate had been successful in increasing minorities; 12% felt it had failed and 32% were unsure. Those who felt that failure to include women makes it hard to draw conclusions about treatment/prevention strategies (P=0.05), white PIs (P=0.05), and those who ascribed importance to female inclusion (P=0.03) were more positive about the mandate's success for women. Similarly, those who felt more strongly that diversity in study samples ensures generalizability (P=0.03), white PIs (P=0.01), and those who ascribed importance to minority inclusion (P<0.01) were more positive about the mandate's success for minorities. CONCLUSION: While the majority of PIs were positive in their assessment of the mandate on increasing diversity in research, many were ambivalent. Greater clarity on the criteria the scientific community should use in assessing the effectiveness of the mandate would allow us to reach the mandate goals.

Distrust, race, and research.

BACKGROUND: Investigators have voiced concerns that distrust of research and the medical community impedes successful recruitment of African Americans into clinical research. OBJECTIVES: To examine possible differences in distrust by race and to determine to what extent other sociodemographic factors explain any racial differences in distrust. METHODS: We analyzed data from 527 African American and 382 white respondents of a national telephone survey on participation in clinical research. Our main outcome measure was a 7-item index of distrust. RESULTS: African American respondents were more likely than white respondents not to trust that their physicians would fully explain research participation (41.7% vs 23.4%, P<.01) and to state that they believed their physicians exposed them to unnecessary risks (45.5% vs 34.8%, P<.01). African American respondents had a significantly higher mean distrust index score than white respondents (3.1 vs 1.8, P<.01). After controlling for other sociodemographic variables in a logistic regression model, race remained strongly associated with a higher distrust score (prevalence odds ratio, 4.7; 95% confidence interval, 2.9-7.7). CONCLUSIONS: Even after controlling for markers of social class, African Americans were less trusting than white Americans. Racial differences in distrust have important implications for investigators as they engage African Americans in research.

Adequacy of reporting race/ethnicity in clinical trials in areas of health disparities.

Although federal initiatives have mandated broader inclusion of minorities in clinical research on diseases that have disparities in health by race and ethnicity, it is not clear whether these initiatives have affected reporting of trial results. The objective of this study was to examine the reporting of race/ethnicity in clinical trials reports in areas of known disparities in health (i.e., diabetes, cardiovascular disease, HIV/AIDS, and cancer) and to determine what factors were associated with reporting of race/ethnicity in results. We performed a Medline search covering the period January 1989 to Oct 2000 to identify clinical trials of diabetes, cardiovascular disease, HIV/AIDS, and cancer published in the Annals of Internal Medicine, JAMA, and New England Journal of Medicine. The main outcome measure was the reporting of participation and of results by race/ethnicity of trial participants. Of 253 eligible trials, 40% (n=102) were non race-focused yet did not report race, while 2% (n=4) were non gender-focused and did not report gender. Forty-six percent of trials that reported the race/ethnicity of the sample reported only one or two racial/ethnic categories, and in 43% of these trials the total number of individuals reported in each race/ethnicity category did not equal the total reported sample size. Analysis of results by race/ethnicity was reported in only two trials, and by gender in only three trials. In diseases with known racial and ethnic disparities, many clinical trials do not report the race/ethnicity of the study participants, and almost none report analyses by race/ethnicity. Although federal initiatives mandate inclusion of minority groups in research, that inclusion has not translated to reporting of results that might guide therapeutic decisions.

Research expectations among African American church leaders in the PRAISE! project: a randomized trial guided by community-based participatory research.

OBJECTIVES: This study sought to examine the expectations and satisfaction of pastors and lay leaders regarding a research partnership in a randomized trial guided by community-based participatory research (CBPR) methods. METHODS: Telephone and self-administered print surveys were administered to 78 pastors and lay leaders. In-depth interviews were conducted with 4 pastors after study completion. RESULTS: The combined survey response rate was 65%. Research expectations included honest and frequent communication, sensitivity to the church environment, interaction as partners, and results provided to the churches. Satisfaction with the research partnership was high, but so was concern about the need for all research teams to establish trust with church partners. CONCLUSIONS: Pastors and lay leaders have high expectations regarding university obligations in research partnerships. An intervention study based on CBPR methods was able to meet most of these expectations.

Trust, benefit, satisfaction, and burden: a randomized controlled trial to reduce cancer risk through African-American churches.

BACKGROUND: Community-based participatory research (CBPR) approaches that actively engage communities in a study are assumed to lead to relevant findings, trusting relationships, and greater satisfaction with the research process. OBJECTIVE: To examine community members' perceptions of trust, benefit, satisfaction, and burden associated with their participation. DESIGN, SETTING, AND PARTICIPANTS: A randomized controlled trial tested a cancer prevention intervention in members of African-American churches. Data were collected at baseline and 1-year follow-up. MEASUREMENTS: Subscales measured perception of trust in the research project and the project team, benefit from involvement with the project, satisfaction with the project and the team, and perception of burden associated with participation. MAIN RESULTS: Overall, we found high levels of trust, perceived benefit, and satisfaction, and low perceived burden among community members in Partnership to Reach African Americans to Increase Smart Eating. In bivariate analyses, participants in the intervention group reported more perceived benefit and trust (P <.05). Participants in smaller churches reported more benefit, satisfaction and trust, while participants from churches without recent health activities perceived greater benefit, greater satisfaction, and lower burden with the project and the team (P <.05). Participants whose pastors had less educational attainment noted higher benefit and satisfaction; those whose pastors were making personal lifestyle changes noted higher benefit and satisfaction, but also reported higher burden (P <.05). CONCLUSIONS: A randomized clinical trial designed with a CBPR approach was associated with high levels of trust and a perceived benefit of satisfaction with the research process. Understanding variations in responses to a research partnership will be helpful in guiding the design and implementation of future CBPR efforts.