Beyond Efficacy

Perhaps the largest need is to translate the results of research that has been performed in laboratories and academic centers into community-based practice tools and clinical protocols. Such translational studies should lead to effectiveness trials. “Efficacy” does not always translate to “effectiveness.” One of the best examples is hydroxyurea. Although hydroxyurea has proven efficacious in RCTs, the majority of eligible persons with SCA in the United States do not yet receive this agent. Well-designed effectiveness and translational studies are needed to overcome the identified barriers that result in underutilization of hydroxyurea. These barriers include the limited number of physicians with knowledge and experience with the agent, and patients’ misconceptions and fears about side effects. Studies are also needed to examine the role of hydroxyurea in people with genotypes and clinical manifestations other than those in the MSH study. The SCD research community can make a real difference through comparative effectiveness research and other investigative strategies, as well as strengthening clinical and public health SCD collaborative efforts at the State and local levels. Finally, all of the research addressed in these guidelines cannot be successful for the approximately 70,000 to 100,000 individuals with SCD in the United States until sickle cell centers, practicing hematologists, and primary care providers in particular are fully willing and capable of taking on the challenges of serving these individuals and their families.

The expert panel realizes that these guidelines leave many uncertainties for health professionals caring for or planning to begin caring for individuals with SCD. However, we hope that these guidelines begin to facilitate improved and more accessible care for all individuals with SCD, and that the discrepancies in the data will trigger new research programs and processes that will provide the evidence necessary to expand upon evidence­-based SCD guidelines in the future.