Refractory immune thrombocytopenic purpura is infrequent in children, but carries substantial morbidity and even mortality. Management of these children is controversial despite the availability of several treatment options as these strategies have not been directly compared and there are many definitions for refractory immune thrombocytopenic purpura. This review will provide an update on the pathogenesis, diagnosis and treatment options for children with severe, acute and chronic refractory immune thrombocytopenic purpura.
Recent studies have demonstrated a number of immunologic alterations (dominant proinflammatory state, inadequate thrombopoiesis, and various B and T lymphocyte disturbances) in the pathogenesis of chronic immune thrombocytopenic purpura. New agents that target certain of these mechanisms (e.g. anti-CD20 monoclonal antibody, thrombopoietic agents) have shown promising results in recent clinical trials, primarily but not solely in adults.
Management of refractory immune thrombocytopenic purpura often requires multiple agents that may provide only short-term benefit. Lack of clear views about the use of these medications, their unwanted side effects and an inability to specifically target a particular patient's disease all lead to frustration among patients, family and the physicians. Better understanding of pathogenesis with the availability of newer therapies with different mechanisms of effect should, however, allow improved management of these patients.
aDivision of Pediatric Hematology and Oncology, Medical University of South Carolina, Charleston, South Carolina
bDivision of Pediatric Hematology and Oncology, Weill Medical College of Cornell University, New York, New York, USA
Correspondence to James B. Bussel, MD, Weill Medical College of Cornell University, New York Presbyterian Hospital, Payson 695, 525 East 68th Street, New York, NY 10021-4885, USA Tel: +1 212 746 3494; fax: +1 212 746 5121; e-mail: firstname.lastname@example.org