How I treat refractory immune thrombocytopenia

Adam Cuker, Cindy E Neunert

Research output: Contribution to journalArticle

38 Citations (Scopus)

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 × 109/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.

Original languageEnglish (US)
Pages (from-to)1547-1554
Number of pages8
JournalBlood
Volume128
Issue number12
DOIs
StatePublished - Sep 22 2016

Fingerprint

Idiopathic Thrombocytopenic Purpura
Platelets
Refractory materials
Toxicity
Thrombopoietin Receptors
Immunosuppressive Agents
Platelet Count
Pharmaceutical Preparations
Adrenal Cortex Hormones
Observation
Splenectomy
Therapeutics
Thrombocytopenia
Hemorrhage
Rituximab

ASJC Scopus subject areas

  • Biochemistry
  • Immunology
  • Hematology
  • Cell Biology

Cite this

How I treat refractory immune thrombocytopenia. / Cuker, Adam; Neunert, Cindy E.

In: Blood, Vol. 128, No. 12, 22.09.2016, p. 1547-1554.

Research output: Contribution to journalArticle

Cuker, A & Neunert, CE 2016, 'How I treat refractory immune thrombocytopenia', Blood, vol. 128, no. 12, pp. 1547-1554. https://doi.org/10.1182/blood-2016-03-603365
Cuker, Adam ; Neunert, Cindy E. / How I treat refractory immune thrombocytopenia. In: Blood. 2016 ; Vol. 128, No. 12. pp. 1547-1554.
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