Advances in the Treatment of Sickle Cell Disease in Children

Support from the national sickle cell disease program in the United States is resulting in significant advances in health care. Ten regional comprehensive sickle cell centers provide a variety of management strategies. An example is the inauguration of neonatal diagnosis for sickle hemoglobinopathies, with parental education and the utilization of special follow-up clinics for affected infants. The administration of prophylactic antibiotics and improved vaccines for control of life-threatening infection is enhancing survival in infants and children. A number of antisickling agents are under preliminary clinical investigation in adult patients. Bone marrow transplantation represents another potential method for management of selected types of sickle cell disease. The results of a national cooperative study on the clinical course of the disease, which was inaugurated in 1978, is providing new information that will be helpful to clinicians and health planners. The federally funded sickle cell centers have effectively utilized interdisciplinary personnel to provide comprehensive medical care, psychosocial support, and patient education. These centers serve as models or bridges whereby the fruits of research activity can be more readily applied to the care of patients. This comprehensive approach, no doubt, can contribute to improvement in the survival of and quality of life for patients. There is, however, a need to continue national support for research efforts to attain a definitive cure for this serious, painful, and disabling illness, which affects about 50,000 people in the United States and many more in other countries. Currently, many families of affected patients are unable to cope personally with socioeconomic problems imposed by the long-term nature of this illness, insufficient income, inadequate insurance coverage, and escalating costs of health care. Clearly, there is a need for additional state and/or federal programs to provide supplements for the medical expenses incurred by persons with long-term handicapping diseases of genetic origin. © 1985, American Medical Association. All rights reserved