title: How I treat refractory immune thrombocytopenia.
abstract: This article summarizes our approach to the management of children and adults with primary immune thrombocytopenia (ITP) who do not respond to, cannot tolerate, or are unwilling to undergo splenectomy. We begin with a critical reassessment of the diagnosis and a deliberate attempt to exclude nonautoimmune causes of thrombocytopenia and secondary ITP. For patients in whom the diagnosis is affirmed, we consider observation without treatment. Observation is appropriate for most asymptomatic patients with a platelet count of 20 to 30 × 10(9)/L or higher. We use a tiered approach to treat patients who require therapy to increase the platelet count. Tier 1 options (rituximab, thrombopoietin receptor agonists, low-dose corticosteroids) have a relatively favorable therapeutic index. We exhaust all Tier 1 options before proceeding to Tier 2, which comprises a host of immunosuppressive agents with relatively lower response rates and/or greater toxicity. We often prescribe Tier 2 drugs not alone but in combination with a Tier 1 or a second Tier 2 drug with a different mechanism of action. We reserve Tier 3 strategies, which are of uncertain benefit and/or high toxicity with little supporting evidence, for the rare patient with serious bleeding who does not respond to Tier 1 and Tier 2 therapies.
authors.affiliation: Department of Medicine and Department of Pathology and Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA; and.
authors.forename: Adam
authors.initials: A
authors.lastname: Cuker
authors.affiliation: Department of Pediatrics, Columbia University, New York, NY.
authors.forename: Cindy E
authors.initials: CE
authors.lastname: Neunert
mesh_terms: D000305:Adrenal Cortex Hormones / Q000627:therapeutic use*; D002648:Child; D004351:Drug Resistance / Q000187:drug effects*; D006801:Humans; D007155:Immunologic Factors / Q000627:therapeutic use*; D008297:Male; D008875:Middle Aged; D011379:Prognosis; D016553:Purpura, Thrombocytopenic, Idiopathic / Q000188:drug therapy* / Q000276:immunology / Q000473:pathology; D000069283:Rituximab / Q000627:therapeutic use*
chemical_list: D000305:Adrenal Cortex Hormones; D007155:Immunologic Factors; D000069283:Rituximab