Recombinant hematopoietic growth factors have emerged as valuable treatments for a variety of medical conditions. Recently, their applications have reached the neonatal intensive care unit, where they offer new therapeutic options for problems as common as anemia of prematurity, or as catastrophic as neonatal sepsis. When facing bacterial infection, it is known that newborn infants are capable of increasing their serum G-CSF concentrations. However, their response does not reach the concentrations that adults are able to achieve, and frequently neutropenia complicates the picture of neonatal sepsis. Although Phase III clinical trials are still in progress, published animal studies, case reports, and Phase I trials suggest that neonates with a variety of neutropenias experience a rapid elevation in their blood neutrophil concentration following administration of rG-CSF, without significant adverse effects. Although many factors contribute to the development of the “anemia of prematurity,” one of the major factors is the inability of preterm infants to generate an erythropoietin (Epo) response appropriate to their degree of anemia. On the basis of this fact, administration of rEpo to preterm neonates to treat or to prevent the anemia of prematurity has been the subject of multiple clinical studies, and it is now clear that rEpo administration to this population can indeed result in lower transfusion requirements, with only occasional and mild adverse effects. Neonatal thrombocytopenia is also a frequent clinical problem, which in most patients develops without a clear underlying cause. Recent studies, quantifying circulating megakaryocyte progenitors in the peripheral blood of thrombocytopenic neonates, suggest that impaired megakaryocytopoiesis may be the main underlying mechanism of many cases of thrombocytopenia. On the basis of this finding, it is tempting to speculate that recombinant thrombopoietin, the newly discovered physiological stimulator of platelet production, will be of clinical relevance in the treatment of thrombocytopenic neonates.
