Elliott Vichinsky, Carlo Brugnara, Lori Styles, John Wagner
American Society of Hemotology
In the near future, therapy may be available that will cure or markedly alter the natural history of sickle cell disease. With better understanding of the pathology of sickle cell disease, coordination of multiple therapies that attack different pathological mechanisms of the disease seems likely.
The long-term safety and efficacy of these novel therapies have not all been well studied, yet there is good potential for them to reach clinical trials in the near future. Pilot studies of the ability of magneslum and clotrimazole to decrease intracellular dehydration have been successful, and nitric oxide therapy for changing oxygen affinity looks promising.
Extra corpuscular therapy aimed at altering red cell adhesion to the endothelium and hemostatic abnormalities appear possible with novel therapies such as antibodies to adhesive molecules and new anticoagulants. Hemoglobin F (HbF) modulation with combination chemotherapy early in life offers the opportunity to prevent organ disease.
Finally, unrelated or HLA-mismatched transplants open the door for a cure for a majority of affected individuals. Improved HLA typing techniques expanding stem cell sources and safer conditioning regimens suggest that this unrelated transplantation may be a possibility.