On June 13-14, 2002, the National Heart, Lung, and Blood Institute (NHLBI), in collaboration with the Health Resources and Services Administration (HRSA), and the NIH Office of Behavioral and Social Science Research (OBSSR), convened a two-day Workshop on Adults with Sickle Cell Disease: Meeting Unmet Needs. The workshop was the first attempt to examine the spectrum of issues that affect adults who live with sickle cell disease. It brought together more than 100 researchers, sickle cell consumers and family members, physicians, nurses, social workers, and other experts who have a stake in addressing the needs of adults with sickle cell disease.
The workshop opened with presentations on the current scope of problems from the perspectives of consumers, treating physicians, and researchers. The keynote address framed the problems in terms of antecedents that begin in childhood, and the potential for intervening at various points during the lifespan to optimize health, educational attainment and employment, social activities and functioning, and quality of life (QOL). Consumers discussed their personal experiences, coping strategies, encounters with health care providers, and challenges in living with sickle cell disease. NHLBI- and HRSA-funded scientists provided overviews of the course of sickle cell disease and its complications among adults; economic and social barriers; information and educational issues for patients, their providers, and their families; QOL issues in general and specific to sickle cell disease; and health care availability and quality.
Following presentations about the problems faced by adult sickle cell patients, physicians, researchers, community nurses, and a social worker talked about pilot and model programs. Their presentations gave workshop participants an opportunity to focus on the results of programs that can be modified, transferred to other settings, or disseminated on a larger scale. The programs reflected the themes from the keynote address, including the antecedents of problems in childhood, transitions during adolescence, and the effects on adult patients’ health, health care, and QOL.
In the afternoon of the first day, workshop participants convened in facilitated discussion groups devoted to one of the following topics:
Discussion groups were charged with summarizing the problems and issues related to their topics, and with formulating recommendations to the NHLBI and HRSA for research and research-related priorities to address the problems.
In the evening, participants heard from a physician researcher who talked of his personal experiences and observations treating sickle cell disease patients, and the importance of continuity of care from childhood through adolescence to adulthood . The first day paved the way for workshop participants to reconvene on the second day when they concentrated on their lists of recommendations.
Each of the five discussion groups generated a list of recommendations to increase knowledge about sickle cell disease and management strategies for adults, expand the scope of clinical and community-based studies to include QOL and psychosocial issues, promote best practices in sickle cell disease care, and determine outcomes of patients served by knowledgeable, multidisciplinary teams. Each discussion group presented its recommendations in the form of an action plan during the closing session of the workshop.
Also available: Table of Consolidated Recommendations
Health Care Needs Discussion Group
1. Multidisciplinary Comprehensive Center Model for children should be developed
Centers should include community providers, community-based organizations, and researchers. Elements of care should include multidisciplinary teams, pain management, psychosocial programs, and day units. Day units should be transportable to communities with smaller populations and to rural areas. Studies should examine the health care utilization patterns of patients who use comprehensive centers and day units; costs; knowledge of sickle cell disease among providers and patients; health outcomes; effectiveness of educational interventions; and effects on patient-physician interactions, attitudes, and communication. Studies should evaluate methods for adopting day units in smaller communities, and the effectiveness of programs for transitioning children and adolescents to adult care.
2. Comparative studies of pain management should be funded.
Studies should compare outcomes of different pain management strategies in emergency rooms,
and outcomes of self-management programs for pain control.
Studies should include effects on work and family roles, in addition to health outcomes.
3. Community support programs should be studied.
Life skills, self-sufficiency, and independent living and training programs should be evaluated in terms of impact on QOL, work, and family roles.
4. Off-site clinics in remote/rural areas, physician education, and telemedicine should be evaluated for feasibility, effects on health outcomes, and effectiveness.
5. Development of a “Smart Card” for emergency room use should be encouraged.
The quality of patient information and treatment protocols, and effectiveness of the technology should be addressed, and regional and urban-rural differences should be assessed.
6. Family studies of psychosocial barriers to “wellness” should be conducted to answer questions about adoption of the “sick role,” family attitudes, functioning and support.
7. The effects of medical student education interventions should be studied in terms of patient outcomes, “bedside manner” (i.e., compassionate care), and physician career choices.
8. The effect of sickle cell disease on patients’ educational levels and employment status, and programs to improve education and work should be studied. Literacy, including health literacy, should be addressed.
9. Barriers to care and to compliance with treatment should be studied, including economic factors such as cost of care and insurance coverage.
10. Clinical studies are critical to determine best practices in sickle cell disease care.
Clinical trials should continue to investigate prevention of sickle cell disease complications and end-organ damage. Data should be collected and analyzed to determine the long-term effects of chronic transfusions in children, the prevalence and indicators of unnecessary transfusions, and best practices for managing iron overload and alloimmunization. Clinical studies in complementary therapies to assess their risks and outcomes should be pursued.
11. Best practices of information dissemination to primary care physicians, health care teams, patients and families, and other stakeholders (e.g., schools, employers) should be implemented.
Social and Economic Needs Discussion Group
1. Data are lacking that could otherwise help us define the social, economic, and health care utilization issues in the sickle cell disease population. Deficits in data preclude an accurate description of social and economic issues, including: demographics; poverty; educational and work opportunities and attainment and resulting economic status; stereotypes and stigmatization; family and other social support; availability (or lack) of support services such as social work and psychological services; availability of and barriers to emergency care, day hospitals, and health care providers; health insurance, reimbursement, social security income and disability insurance; and community characteristics.
Successful people who have lived and learned to cope with sickle cell disease should be included in efforts to characterize the sickle cell disease population. Their "lessons learned" can be valuable for modeling successful coping and pathways to productive social and work roles.
2. The discussion group encourages the development of a Social Epidemiological
Study to collect baseline and longitudinal data on these issues, and to link
the study with existing data systems such as health care utilization. This Social
Epidemiological Study would enable researchers to fully describe the adult
sickle cell population and its demographic, social-environmental, economic,
educational, and health care and health status characteristics; and to track
changes over time.
The study should:
Information and Education Discussion Group
1. This discussion group recommends promoting research in patient, family, provider, and public health education.
2. Sickle cell disease education should be a priority in health professional training
activities of the NIH.
Sickle cell disease and pain management curricula should be included in hematology training grants.
Educational research focused on improving compassion, humanity, and cultural competence among health care providers should be developed for use in training activities.
3. Research in new methods of improving access to information about
sickle disease should be encouraged.
Consumer and provider-based evaluations of effective approaches (focus groups, satisfaction surveys, etc.) should be used.
Outcomes research stressing results, not process, should be a priority.
4. A central repository of sickle cell information should be developed.
A Needs Assessment will help assure that educational materials are available for all interested audiences.
An evaluation of existing materials will prevent duplication of effort.
Information in multiple formats for patients, families, lay persons, and providers should be made available.
5. Research to assess the effectiveness of new and existing
approaches to health professional education should be conducted.
A primary outcome of this research should be improved patient and family functioning.
6. NHLBI should partner with other governmental and private agencies to develop
positive incentives for health care providers to give compassionate care to
adults with sickle cell disease.
There must be incentives for the health professionals to seek additional education and training in sickle cell disease.
7. Because industry has extensive experience in these areas, small
business grant initiatives should be used to promote sickle cell disease education and information
A diffusion of innovation model, or other effective model of health communication, should be used to stimulate and evaluate health education efforts.
8. Research in public health education that focuses on impact should be encouraged. Aims of the research should be to improve adult medical, social, vocational, and psychological functioning.
9. Educational initiatives should be included in a research agenda for sickle cell disease. Studies should evaluate and demonstrate the effectiveness
Multidisciplinary partnerships with expertise in outcomes research, marketing, social science, psychology, education, and anthropology will provide important perspectives.
Outcomes of the educational initiatives should be:
Quality of Life (QOL) Discussion Group
1. Assessment of QOL is necessary to fully understand the needs of adults with sickle cell disease.
2. Health care professionals should be able to use effective measures to evaluate QOL in the management of sickle cell disease.
3. There are no published studies on QOL in sickle cell disease. QOL has been studied in other diseases.
4. QOL measures and studies will be useful for patient management, clinical trials and improving effectiveness of the physician-patient relationship.
5. QOL measurements should be age-specific.
6. Definition of QOL: an individual’s perception of his/her happiness, well-being and life satisfaction.
7. Definition of Health-related QOL: a multidimensional assessment of the patient’s perception of his/her functioning in society, of the ability to function without pain, of being accepted, and of having intact relationships.
8. Sickle cell disease-specific assessment of QOL should include: pain, mental health, financial status, access to care, educational attainment, employment opportunities, burden of treatment, support systems, living arrangements, discrimination, stigmatization, use of spirituality, social relationships, coping strategies and skills.
9. Mental health impacts QOL through self-esteem, depression, and feeling credible (e.g., in encounters with the health care system).
10. NHLBI should convene 2 working groups: one a group of experts in QOL and the other, a group of experts in sickle cell disease which includes health care providers and individuals with the disease. These working groups should develop a framework and measures for studies of QOL in sickle cell disease.
Fill-in-the-Gaps Discussion Group
1. This group recommends developing Health Objectives for adults with sickle cell disease.
Children’s measures are largely applicable to adults.
A Task Force or Workgroup should be convened to develop principles, and to identify specific objectives for adults, and the data necessary to track progress in meeting the objectives.
2. Funding for research should reflect the needs of adults, deficits in data,
and informational needs of physicians.
The number of Comprehensive Sickle Cell Centers should be increased (currently there are 10).
A separate sickle cell-specific clinical research network should be established.
Grants for educational programs on mental health, pain and treatment, psychological counseling, and patient support interventions are needed.
Training programs should be expanded.
Genetic education and counseling should be part of the centers and networks.
3. A social marketing campaign is warranted.
The goal should be “accentuate the positive, eliminate the negative.”
“Face” of sickle cell disease: the perceptions about the face of sickle cell disease must be changed.
A media campaign should be conducted.
Activities for Sickle Cell Month (September) and sustained activities throughout the year should be included.
Tele/Video/Web Conference for health care providers and consumers and “Radio Healthbeat” should be some of the communication channels that are used.
4. Standards of care for adults with sickle cell disease should be developed.
The NIH should convene a task force of providers, consumers/patients and community representatives to develop a set of standards.
The NIH should organize and centralize information on standards of care.
The NIH should develop and implement a mechanism to disseminate “best practices in sickle cell disease” to all interested parties.
Education--getting the information out there, for example on a web page--is key.
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