Advances in the treatment of sickle cell disease in children
R. B. Scott
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.
