Recombinant human
erythropoietin represents a potential therapeutic alternative to
red blood cell transfusions in a number of pediatric
anemias. It is effective in correcting
anemia associated with
chronic renal failure and may significantly reduce the morbidity associated with childhood CRF. Most exposures to allogeneic blood products in pediatrics for treatment of
anemia with
blood transfusions occur in neonatal intensive care units. If proven effective in treating
anemia in premature babies, r-HuEPO will be responsible for a major reduction in the use of
blood transfusions in clinical neonatology. Carefully designed, placebo-controlled clinical trials will be required to establish the role of r-HuEPO in
anemia of prematurity. Recombinant human
erythropoietin also may be useful to increase the amount of blood that can be collected before
elective surgical procedures. Another potential indication is to raise the hematocrits of infants with large intracardiac shunts who develop
congestive heart failure coincident with the developmental fall in
hemoglobin concentration after birth. Finally, r-HuEPO may one day play a role in modifying the expression of
globin genes and, thereby, ameliorate the course of
sickle cell disease and
beta thalassemia. Many questions surrounding the use of r-HuEPO in infancy and childhood are being addressed in ongoing clinical trials.