clinical study insights

Updates on Second-Line Therapies for Immune Thrombocytopenia

by Terry B. Gernsheimer, MD

Overview

Clinical Study Title:
Clinical updates in adult immune thrombocytopenia

Clinical Study Abstract:
Immune thrombocytopenia (ITP) occurs in 2 to 4/100 000 adults and results in variable bleeding symptoms and thrombocytopenia. In the last decade, changes in our understanding of the pathophysiology of the disorder have led to the publication of new guidelines for the diagnosis and management of ITP and standards for terminology. Current evidence supports alternatives to splenectomy for second-line management of patients with persistently low platelet counts and bleeding. Long-term follow-up data suggest both efficacy and safety, in particular, for the thrombopoietin receptor agonists and the occurrence of late remissions. Follow-up of patients who have undergone splenectomy for ITP reveals significant potential risks that should be discussed with patients and may influence clinician and patient choice of second-line therapy. Novel therapeutics are in development to address ongoing treatment gaps.

Reference:
Lambert MP, Gernsheimer TB. Clinical updates in adult immune thrombocytopenia. Blood. 2017;129(21):2829-2835.

Expert Commentary

Terry B. Gernsheimer, MD

Director of the University of Washington
Medical Transfusion Service
Professor of Medicine, Division of Hematology
University of Washington
Seattle, WA

“Although there have been many recent advances in the diagnosis and management of ITP, clinical guidelines have not yet been updated with the available data and treatments currently used in clinical practice as second-line therapy.”

Terry B. Gernsheimer, MD

Although there have been many recent advances in the diagnosis and management of ITP, clinical guidelines have not yet been updated with the available data and treatments currently used in clinical practice as second-line therapy. Over the past decade, the pathophysiology of ITP has become better understood. Studies have demonstrated that ITP is a platelet production defect as well as platelet destruction. Platelet production and thrombopoietin levels are only mildly increased if at all and may actually be lower than normal in some patients with ITP. Therefore, thrombopoietin receptor agonists should be considered early on as a second-line agent for the treatment of ITP, having been shown in clinical trials to be safe and effective for long-term use, enabling patients to postpone or avoid splenectomy. Understanding bleeding risk and the factors that increase this risk is important in determining which patients will require treatment of their ITP, even at higher platelet counts. Updated ITP treatment guidelines will need to include data on newer agents for therapy. Clinical trials of novel therapies continue to be needed to further improve patient outcomes.


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