A randomized recruitment intervention trial in Parkinson's disease to increase participant diversity: early stopping for lack of efficacy.

BACKGROUND: Failure to include participants of diverse race and ethnicity (i.e. those other than European Caucasian, non-Hispanic) in clinical trials impedes the safe development of new therapies given the potential for racial/ethnicity-related variations in treatment response. Increasing diversity is problematic for low prevalence diseases, where most community-based approaches do not reach those with the disease. PURPOSE: Increase racial/ethnic diversity of participants in a Parkinson's disease therapeutic trial. METHODS: We incorporated a randomized Ancillary Trial into the multisite National Institute of Neurologic Disorders and Stroke Exploratory Trials in Parkinson's Disease Long-Term Study 1. Movement disorders clinics already participating in long-term trial 1 were eligible and were the unit of randomization and analysis. At least 14% of adult residents over age 55 and living within 30 miles of the eligible site were from a diverse population, or there was a near-by zip code with a highly diverse population. Eligible sites also agreed to be randomized. The intervention was designed to increase community physicians' trust in long-term trial 1 investigators and address recruitment barriers in diverse populations. Primary outcomes included percentage of participants from diverse racial/ethnic groups enrolled in long-term trial 1, and qualitative findings from key informant interviews of the Ancillary Trial investigators and coordinators at the end of the trial. RESULTS: The Ancillary Trial stopped early for lack of efficacy, conditional power less than 1%. The 17 intervention sites had 12.6% diverse participants compared to 15.6% in 15 control clinics; odds ratio 0.82 (95% confidence interval = 0.32-2.16). In key informant interviews, high enrollers of diverse participants reported more use of existing physician relationships, untargeted community outreach, and extensive efforts to overcome participants' barriers. Low enrollers reported more use of patients in their practices and placed more responsibility for low enrollment on prospective participants. LIMITATIONS: The Ancillary Trial included only those with Parkinson's disease. Whether our findings generalize to trials in other low prevalence diseases is unknown. CONCLUSIONS: Increasing diversity in Parkinson's disease clinical trials requires new paradigms for trial investigator and coordinator interactions with community physicians and prospective trial participants.

Statewide investigation of medically attended adverse health conditions of persons with spinal cord injury.

BACKGROUND/OBJECTIVE: To report over a 10-year period the statewide prevalence and incidence of medically attended adverse health conditions in people with new traumatic spinal cord injury (TSCI). DESIGN: Retrospective cohort study. METHODS: (a) Identified all new TSCI cases discharged alive from statewide acute care hospitals, 1996 to 2000, using ICD-9-CM methodology. (b) Followed cases from 1996 to 2005 to quantify medically attended health conditions documented during emergency department visits, acute care hospitalizations, and outpatient hospital visits. (c) Used the life table method to calculate the prevalence and incidence of health conditions. (d) Examined Cox proportional hazard ratio of mortality by gender controlling for age and TSCI severity. RESULTS: Nine hundred eighty-eight residents (257 women, 731 men) with TSCI were alive 90 days after discharge from acute care hospitalization from 1996 to 2000. Nine hundred twenty-three (251 female, 672 male) (93.4%) residents had an observed medically attended adverse health condition in the 10-year followup period. The most prevalent classes of diseases and disorders were (a) muscle and connective tissue, (b) renal and urinary, (c) digestive, (d) circulatory, (e) respiratory, (f) endocrine/nutritional/metabolic, and (g) infectious. Incidence of new injury was 29.0% for males and 26.9% for females. During the follow-up period, 49 women (19%) and 104 men (14%) died. CONCLUSIONS: People with TSCI experience diverse adverse health conditions in the 10 years after initial injury. An interdisciplinary health care provider team approach to allocating resources and implementing countermeasures to prevent or limit occurrence of these conditions is vital to these patient's continuum of care.

Behavioral risk factors of mortality after spinal cord injury.

OBJECTIVE: To test hypothesized relationships between multiple behavioral indicators and mortality among persons with spinal cord injury (SCI), while controlling for biographic and injury characteristics. DESIGN: Prospective cohort study with behavioral data collected by mailed survey in late 1997 and early 1998. Mortality status was ascertained as of December 31, 2005. SETTING: A large rehabilitation hospital in the southeastern United States. PARTICIPANTS: Adults (N=1386) with traumatic SCI, at least 1 year postinjury. INTERVENTIONS: Not applicable. MAIN OUTCOME MEASURES: Primary outcome was time from survey to mortality or censoring. Mortality status was determined using the National Death Index and the Social Security Death Index. There were 224 deaths (16.2%) in the full sample, and due to missing data, 188 deaths were observed in the 1251 participants included in the final statistical model. RESULTS: Cox proportional hazards modeling identified several significant behavioral predictors of mortality. In the first set of analyses, the significance of a single behavioral variable was assessed while controlling for biographic and injury predictors. We subsequently built a comprehensive model based on an optimal group of behaviors. The best set of behavioral predictors included: smoking, binge drinking (number of episodes with 5 or more drinks), prescription medication use, and number of hours out of bed per day. Inclusion of these variables improved prediction of survival compared with biographic and injury variables alone, as the pseudo-R2 increased from .121 to .164 and the concordance from .730 to .769. CONCLUSIONS: The results affirm the importance of avoiding basic risk behaviors, such as smoking and alcohol misuse, and affirm their importance as targets of intervention in association with SCI rehabilitation.

A latent variable structural path model of health behaviors after spinal cord injury.

BACKGROUND/OBJECTIVE: To develop a latent behavioral model by identifying and confirming the factor structure of health behaviors of people with spinal cord injury (SCI) and their relationships with biographic, injury, and educational characteristics. RESEARCH DESIGN: Survey data were collected from 1388 adults with traumatic SCI of at least 1 year duration. MAIN OUTCOME MEASURES: Selection of health behaviors was based on a bidimensional behavioral risk model. Behaviors were measured by core item sets from the Behavioral Risk Factor Surveillance System and supplemented by an alcohol screening measure, select fitness proxies, and the SCI Health Survey. RESULTS: Latent variable structural equation modeling was used to identify underlying factors and their relationship with participant characteristics. Seven specific factors were identified by exploratory factor analysis and were cross-validated using confirmatory factor analysis. They included: (a) healthy nutrition, (b) unhealthy nutrition, (c) fitness, (d) smoking, (e) alcohol use, (f) psychotropic prescription medications, and (g) SCI healthy activities. Two higher-order dimensions were also identified, including a risk dimension (b, d, e) and a protective dimension (a, c, g). Participant characteristics were associated with the domains. For instance, participants with the most severe injuries scored lower on smoking and alcohol but higher on psychotropic medications; age was positively correlated with healthy nutrition and negatively correlated with alcohol and tobacco use but also negatively correlated with fitness. CONCLUSION: Behaviors can be meaningfully combined into underlying dimensions to more efficiently use them as predictors of secondary conditions.

Unequal burden of disease, unequal participation in clinical trials: solutions from African American and Latino community members.

African Americans and Latinos are underrepresented in clinical trials. The purpose of this study was to elicit solutions to participation barriers from African Americans and Latinos. Fifty-seven adults (32 African Americans, 25 Latinos) ages 50 years and older participated. The Institute of Medicine's Unequal Treatment conceptual framework was used. Six racially/ ethnically homogenous focus groups were conducted at five sites in three counties. Themes within groups and cross-cutting themes were identified. The NVIVO program was used for data classification. The data were reviewed for final coding and consensus. Shared solutions included addressing costs, recruiting in community contexts, conducting community and individualized patient education, and sharing patient safety information. Participants were unanimously in favor of clinical trials navigation recruitment interventions. Solutions specific to African Americans included diversifying research teams, recognizing past research abuses, and increasing community trust. Solutions specific to Latinos included providing low-literacy materials, providing Spanish-speaking clinicians and advocates, and clarifying that immigration status would neither be documented nor prevent participation. Solutions from African Americans and Latinos reflect their cultural backgrounds and historical experiences. The results suggest the importance of developing a tailored, barriers-focused navigation intervention to improve participation among diverse racial and ethnic populations.