clinical topic updates

Future Goals for Advances in Chronic Immune Thrombocytopenia Management

by Adam Cuker, MD, MS

Overview

Immune thrombocytopenia (ITP) management has changed substantially over the last decade with the introduction of new medical therapies, including rituximab and the thrombopoietin receptor agonists (TPO-RAs) eltrombopag and romiplostim. A goal for the future is to determine whether intensive upfront therapy has the potential to ameliorate the long-term course of—or even cure—ITP. Currently, there are novel treatments being studied based on an improved understanding of ITP pathophysiology, including antibodies targeting the CD40-CD154 interaction between B cells and T cells, treatments targeting FcRn receptor, and additional TPO-RAs. Future well-designed, randomized clinical trials may improve treatment and overall patient outcomes. In addition to clinical trials of novel treatments, future research in ITP should focus on individualized therapy and predictive markers of treatment response.

Expert Commentary

Adam Cuker, MD, MS

Assistant Professor of Medicine
Director, Penn Comprehensive Hemophilia and Thrombosis Program
Perelman School of Medicine
University of Pennsylvania
Philadelphia, PA

“The goals for ITP for the next 10 years include identifying treatment regimens that will ameliorate the long-term course of, or even cure, the disease and identifying biomarkers that would allow us to match treatments with a patient’s underlying pathophysiologic mechanisms to enhance response rates.”

Adam Cuker, MD, MS

The landscape of ITP therapy, especially in adults, has changed dramatically over the past 10 to 20 years, with a decrease in splenectomy and growing use of medical therapies, particularly rituximab and the TPO-RAs. The goals for ITP for the next 10 years include identifying treatment regimens that will ameliorate the long-term course of, or even cure, the disease and identifying biomarkers that would allow us to match treatments with a patient’s underlying pathophysiologic mechanisms to enhance response rates. I do believe that in the future, whether this is 5 years, 10 years, or 20 years away, we will have tests that will help us to understand the pathophysiology of ITP in each individual patient. This increased understanding will allow us to be better able to match treatment with pathophysiology, which will, in turn, improve response rates and patient outcomes. We are not there yet, but I think that is a goal for the future.

References

Cuker A, Prak ET, Cines DB. Can immune thrombocytopenia be cured with medical therapy? Semin Thromb Hemost. 2015;41(4):395-404.

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

Neunert CE. Management of newly diagnosed immune thrombocytopenia: can we change outcomes? Blood Adv. 2017;1(24):2295-2301.

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