New therapies and approaches to transfusion in sickle cell disease in children.

Until recently, transfusion was the only effective therapy for children with sickle cell disease (SCD). Although transfusion can be used to prevent or treat many of the complications of SCD, it has often been used indiscriminantly and with considerable risk to the patient. Recent studies attempted to define those clinical situations in which transfusion is effective as well as to optimize its delivery while minimizing complications. Other therapies are emerging, and in the future many will likely play an important role in the treatment of children with SCD. These therapies include pharmacologic interventions, such as hydroxyurea; bone marrow and cord blood transplantation; and improvements in supportive and preventive care. The future for new therapies looks promising, but the appropriate role for many of these therapeutic interventions remains to be determined.