Evaluation and treatment of severe and prolonged thrombocytopenia in neonates

Thrombocytopenia is one of the most common hematologic problems in the neonatal intensive care unit (NICU). Despite its prevalence,several basic pathophysiologic questions remain unanswered. For instance, there is a lack of evidence-based guidelines for treatment,and the kinetic mechanisms (decreased platelet production,increased platelet consumption, or sequestration) responsible for most varieties of neonatal thrombocytopenia are not well defined.Moreover, a clear correlation between degree of thrombocytopenia and the resulting bleeding risk has not been demonstrated, and no transfusion-trigger studies have been conducted in neonates. As a consequence of these deficiencies in knowledge, there is great variability in platelet transfusion practices among NICUs. This article presents an overview of the evaluation of a neonate with severe thrombocytopenia and a review of current and projected therapeutic options.