New Research is Needed

Cure is always the most desirable outcome for any chronic disease. Therefore, research that increases the evidence for and availability of a cure for SCD is a high priority. Hematopoietic stem cell transplantation (HSCT, formerly called bone marrow transplantation) is a treatment option for an increasing but still small number of people with SCD. The procedure involves “conditioning” therapy, utilizing myelosuppressive and/or immune-modifying drugs, followed by infusion of histocompatible stem cells (derived from bone marrow, peripheral blood, or umbilical cord blood). Substantial risks are involved with the procedure, and it is not yet feasible in the majority of people with SCD. Although clinical trials have provided promising results, and cure appears to be possible in a large proportion of patients receiving HSCT, additional research is still needed that addresses the potential risks of this therapy (e.g., failure of engraftment and chronic graft-versus-host disease) before HSCT can become a widely used therapy.

Additional research is also required to address the many other areas with little or no evidence that were identified during the development and writing of these guidelines. The needed studies include observational work to better describe the utility of screening asymptomatic individuals with SCD for commonly occurring chronic diseases; studies to better describe the clinical course of the occurrence and treatment results of all the acute and chronic complications of SCD; comparative effectiveness studies to provide clear outcomes on best approaches to SCD and its complications;clinical trials for new therapeutic approaches or to improve on current therapeutic approaches such as examining the role of hydroxyurea in people with genotypes and clinical manifestations other than those in the MSH study or transfusion goals in chronic conditions. A few of the other larger research agenda issues, in addition to the need for an SCD cure, are summarized below.