patient care perspectives

Use of Combination Therapy in the Management of Chronic Immune Thrombocytopenia

by Adam Cuker, MD, MS

Overview

Clinical studies have demonstrated that second-line treatment options for chronic immune thrombocytopenia (ITP), such as rituximab and the thrombopoietin receptor agonists (TPO-RAs) eltrombopag and romiplostim, can be used in combination with corticosteroids or other immunosuppressive agents in an effort to improve treatment outcomes in some patients. Studies have shown, for instance, that combination therapy with rituximab and dexamethasone can be an effective treatment in a small population of pediatric and adult patients with chronic ITP, with one study showing 30% of 33 pediatric patients studied maintaining a continued response at 60 months or last checkup. In addition, a combination treatment approach with an immunosuppressive agent added to the TPO-RA eltrombopag has been found to result in a higher complete remission in patients with chronic ITP compared with single-agent therapy alone. In patients with chronic ITP who do not respond to monotherapy, combination therapy targeting multiple mechanisms may result in a greater 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

“Combination therapies of a low-dose steroid with a second- or third-line treatment option may be useful in patients with chronic ITP.”

Adam Cuker, MD, MS

Combination therapies of a low-dose steroid with a second- or third-line treatment option may be useful in patients with chronic ITP. When we are talking about chronic ITP, we are not talking about rescue therapy. We are talking about what I call maintenance therapy, and trying to find a long-term strategy for maintaining a hemostatic platelet count that is both effective and tolerable for the patient. I would never want to use high doses of steroids over the long term because of cumulative toxicity. Therefore, one of my major treatment goals is to get patients either off of steroids entirely or to get them on very low doses. But, I do think that there is a role for using low-dose steroids, and by that I mean prednisone 5 mg per day or less in combination with other therapies to maintain a safe platelet count. I have had a number of patients, for example, who did not respond well to a TPO-RA alone, but, when we combined it with low-dose prednisone (maybe 5 mg per day or 5 mg every other day), that combination achieved a good and sustainable response. I do think that there’s a role for combination therapy with corticosteroids in select patients, but the key is that it has to be a low dose.

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