Publication of data collection forms from NHLBI funded sickle cell disease implementation consortium (SCDIC) registry

Background: Sickle cell disease (SCD) is an autosomal recessive blood disorder affecting approximately 100,000 Americans and 3.1 million people globally. The scarcity of relevant knowledge and experience with rare diseases creates a unique need for cooperation and infrastructure to overcome challenges in translating basic research advances into clinical advances. Despite registry initiatives in SCD, the unavailability of descriptions of the selection process and copies of final data collection tools, coupled with incomplete representation of the SCD population hampers further research progress. This manuscript describes the SCDIC (Sickle Cell Disease Implementation Consortium) Registry development and makes the SCDIC Registry baseline and first follow-up data collection forms available for other SCD research efforts. Results: Study data on 2400 enrolled patients across eight sites was stored and managed using Research Electronic Data Capture (REDCap). Standardized data collection instruments, recruitment and enrollment were refined through consensus of consortium sites. Data points included measures taken from a variety of validated sources (PHENX, PROMIS and others). Surveys were directly administered by research staff and longitudinal follow-up was coordinated through the DCC. Appended registry forms track medical records, event-related patient invalidation, pregnancy, lab reporting, cardiopulmonary and renal functions. Conclusions: The SCDIC Registry strives to provide an accurate, updated characterization of the adult and adolescent SCD population as well as standardized, validated data collecting tools to guide evidence-based research and practice

The risks and benefits of long-term use of hydroxyurea in sickle cell anemia: A 17.5 year follow-up.

A randomized, controlled clinical trial established the efficacy and safety of short-term use of hydroxyurea in adult sickle cell anemia. To examine the risks and benefits of long-term hydroxyurea usage, patients in this trial were followed for 17.5 years during which they could start or stop hydroxyurea. The purpose of this follow-up was to search for adverse outcomes and estimate mortality. For each outcome and for mortality, exact 95% confidence intervals were calculated, or tests were conducted at alpha = 0.05 level (P-value \u3c0.05 for statistical significance). Although the death rate in the overall study cohort was high (43.1%; 4.4 per 100 person-years), mortality was reduced in individuals with long-term exposure to hydroxyurea. Survival curves demonstrated a significant reduction in deaths with long-term exposure. Twenty-four percent of deaths were due to pulmonary complications; 87.1% occurred in patients who never took hydroxyurea or took it for \u3c5 years. Stroke, organ dysfunction, infection, and malignancy were similar in all groups. Our results, while no longer the product of a randomized study because of the ethical concerns of withholding an efficacious treatment, suggest that long-term use of hydroxyurea is safe and might decrease mortality

Implicit Bias and Healthy Equity

Presentation given at Medical Educator’s Institute Seminar, American Society of Hematology (ASH)

It All Goes Back to the Services : A Rural/Urban Comparison of Barriers and Facilitators to Disability Services

Background: Children with disabilities and their families face a variety of barriers in accessing services throughout their lifespan. The purpose of this study was to explore rural/urban differences in barriers and facilitators of services for youth with disabilities and their families in southeast Georgia. Methods: Twenty in-depth interviews (N=11 urban; N=9 rural) were conducted with organizational representatives who provide disability services. Two researchers coded the data until 100% consensus was reached. Convergence and divergence across rural and urban perspectives were examined. Major themes were identified and illustrative quotes selected. Results: Multiple barriers cutting across both communities (e.g., lack of health insurance, high out of pocket expenses) emerged. Access to public transportation and a range of services were major barriers in rural communities but not urban. Key services (e.g., dentists) are absent in rural areas or have long waiting lists. Socio-economic status is the determining factor in overcoming barriers to service provision, with many low income families in rural areas lack resources (e.g., transportation) to overcome barriers. Several facilitators of services emerged (e.g., training parents to deliver services on their own). Conclusion: Whereas there are range of services available in urban communities, low income, rural families of children with disabilities face unique barriers, particularly when their child transitions out of the school system. Service providers reported extensive efforts to work with parents to overcome barriers; however, community-based strategies (e.g., opportunities for community engagement) are recommended to overcome service barriers in rural communities

“It All Goes Back to the Services”: A Rural/Urban Comparison of Service Providers’ Perceptions of Challenges and Facilitators to Disability Services

The purpose of this qualitative study was to explore rural/urban differences in service providers’ perceptions of challenges and facilitators to services for children with special health care needs (CSHN) and their families in southeast Georgia. Methodology: A total of 23 individual interviews were conducted with service providers in one rural county in southeast Georgia (N = 12) and in one urban county in north central Georgia (N = 11). Interviews were based on the National Survey of Children with Special Health Care Needs 2009-2010 survey. All interviews were audio recorded, transcribed and coded with the intent of identifying themes and illustrative quotes. Findings: Service providers discussed a variety of challenges to accessing services related to socioeconomic status and the transition out of the school system. Urban and rural service providers’ perceptions were similar overall, with the exception of lack of transportation and access to a variety of services in rural area. Service providers overcome challenges through identifying parents’ needs and limitations and adapting service provision. Some of the facilitators mentioned by both urban and rural service providers are availability of transportation and resources. Practical Implications: The results of this study provide public health practice and policy implications that could guide the pathways of targeted interventions to improve the access and availability of variety of services for CSHCN. Originality/value of paper: This paper represents an in-depth exploration of service provider’s perceptions of challenges and facilitators related to service provision for children with special healthcare needs

Service Provider\u27s Perspectives on Barriers and Facilitators to the School-Community Transition Among Youth with Disabilities

Background: Schools are required to provide students with disabilities a free and appropriate education. At the age of 22, youth with disabilities must transition out of the school system into their community. The purpose of this study was to explore service provider’s perspectives of transition in rural and urban communities in Georgia. Methods: Twenty in-depth interviews (N=11 urban; N=9 rural) were conducted with organizational representatives who provide disability services. Two researchers coded the data until 100% consensus was reached. Convergence and divergence across rural and urban perspectives were examined. Major themes were identified, and illustrative quotes selected. Results: Whereas transition into the school system is easy, transition out is challenging. Service providers suggested schools begin the process too late, resulting in youth being faced with “nothing to do” and “nowhere to go,” especially those who live in rural communities. Many youth with disabilities are unsuccessful in gaining employment due to a lack of social skills – despite teachers’ instructional efforts. Overall, the main barrier to effective transition is a cycle of dependence that is created by government and school interaction with families and mediated by changing policy and lack of information. Conclusion: Schools provide an important support system that ends with transition. Discussions with youth and families about transition must start earlier (e.g., middle school), with additional focus needed on health and social services. Future research should investigate ways to enhance social skills required for future employment. Finally, schools should partner with service providers to facilitate parents’ transition

When Everything Changes: Parent Perspectives on the Challenges of Accessing care for a Child with a Disability

Background: Health disparities exist among individuals living in rural and urban contexts in terms of access to healthcare and overall mortality. These disparities are typically greater for youth with disabilities living in rural areas, who face additional barriers in receiving health and support services specific to their disability. Parents are typically the ones responsible for coordinating the care needed by children with a disability; however, with numerous barriers present families are not provided adequate support to care for a child with disabilities. Objective: The purpose of this study was to examine barriers and facilitators to accessing health and support services among urban and rural families of children with disabilities. Methods: In depth interviews were conducted with parents who provide care for an adolescent with a disability. The sample comprised of parents from one rural county (N=9) and one urban county (N=10) in Georgia. Parental interviews were conducted face to face by a trained researcher. Each interview was audio-recorded. The recordings were transcribed and content analysis used to create codes and identify emerging themes. Results: The common themes found during the analysis include accessibility of health and support resources, transitions, and social isolation. Conclusions: When comparing urban and rural areas, barriers to access do differ in terms of availability, but analysis revealed more similarities exist among parents from both contexts. Efforts must be made to increase opportunities for youth with disabilities to become connected with the local community in order to improve quality of life for families

Identifying the Impact of Social Determinants of Health on Disease Rates Using Correlation Analysis of Area-Based Summary Information

Area-based measures of socioeconomic variables can be used to identify risk factors associated with a disease of interest. When correlation analysis is used to identify risk factors, potential confounding from other variables must be taken into account