By George Buchanan, MD, and Marilyn Telen, MD
2008-01-01
Dr. Buchanan is Children's Cancer Fund Distinguished Chair in
Pediatric Oncology and Hematology, and Director of Pediatric
Hematology-Oncology at the University of Texas Southwestern Medical
Center.
Dr. Telen is Wellcome Clinical Professor in Medicine, Division
of Hematology, at the Duke Comprehensive Cancer Center, and Chief of
the Hematology Division.
At the end of 2007, the Society released a summary of recommendations titled Toward a New Research Paradigm: Building A New Sickle Cell Disease (SCD) Research Agenda.
The document captures the discussions of the ASH Workshop on Sickle
Cell Disease in May 2007. The Workshop's goal was to explore new SCD
research objectives and create a real-world model to achieve them. The
Workshop document presents a synopsis of the new research priorities
identified and a blueprint for execution of the new sickle cell
research strategy.
Five research areas were identified and prioritized by workshop
participants. In rank order, they are: 1) development of improved and
new treatments, 2) identification of predictors of SCD outcomes, 3)
improved pain management and quality of life, 4) elucidation of the
pathophysiology of organ complications (kidneys, brain, heart, and
lungs), and 5) initiation of international strategies for collaborative
SCD research.
The document identifies barriers to a new research agenda,
acknowledging the psychosocial, economic, and clinical care delivery
problems that affect people with SCD, especially adults. The document
also recognizes that NIH's funding and organizational structure does
not optimally support multi-disciplinary and longitudinal approaches to
SCD, and notes that the lack of cohesive interaction among the many SCD
stakeholders hampers new research initiatives.
In an effort to avoid fragmentation and disengagement, workshop
participants recommended that NIH assemble a single national
DHHS-funded, multi-center collaborative research group. Children and
adults with SCD residing in areas of the United States not served by
current government-funded programs or who do not have access to care
for financial or other reasons must be included. The research agenda
describes two models for the NHLBI and other agencies to consider.
New research should focus on multi-disciplinary approaches to
characterize the genetic and environmental factors that contribute to
the heterogeneity of disease phenotype and response to therapy.
Continued improvement in drug therapies and pain management techniques
remains a high priority. Additional study must also focus on preventing
and reducing organ complications for those with SCD.
To maximize quality SCD treatment, ASH emphasizes the need to align
the efforts of SCD stakeholders, including government entities,
physicians, scientists, foundations, community advocates, and SCD
patients and their families. ASH recommends the creation of a new
Sickle Cell Research Group. This group would be composed of clinicians
and scientists at academic medical centers that serve a large number of
SCD patients.
ASH has responded to the NHLBI's Request for Information (RFI): Defining a Research Agenda for Sickle Cell Disease and Other Hemoglobinopathies by submitting this document and has also distributed it to other Federal Agencies and the sickle cell community.
back to top
