patient care perspectives

Achieving Safe Platelet Counts in Immune Thrombocytopenia With Thrombopoietin Receptor Agonists

by Howard A. Liebman, MD


The main goal of treatment in patients with chronic immune thrombocytopenia (ITP) is to minimize the bleeding risk, and this should be done by achieving safe platelet counts with minimal side effects. After initial treatment, commonly with corticosteroids or intravenous immunoglobulin (IVIg), many patients with chronic ITP will require treatment with a second-line therapy, which commonly includes a thrombopoietin receptor agonist (TPO-RA), rituximab, or splenectomy. The TPO-RAs and splenectomy have been associated with positive long-term effects on platelet counts, with similar rates of response of 85% to 95% observed. Rituximab is associated with a lower (approximately 62%) platelet count response rate in adult patients with ITP, and it is associated with treatment toxicity and morbidity in some individuals. In addition, rituximab has been found to have a 38% response rate at 1 year and only a 21% response rate at 5 years in adult patients. Although effective, a risk of thrombosis and severe sepsis have been reported with splenectomy, but the TPO-RAs have been reported to have good tolerability, low toxicity rates, and long-term efficacy.

Expert Commentary

Howard A. Liebman, MD

Donald I Feinstein Chair in Medicine
Professor of Medicine and Pathology
Jane Anne Nohl Division of Hematology
USC Norris Cancer Hospital
Los Angeles, CA

“Due to treatment with the TPO-RAs, we rarely see true refractory ITP anymore. It is very rare nowadays because we can get safe platelet counts in patients for a long period of time with TPO-RA treatment.”

Howard A. Liebman, MD

Patients will respond to a TPO-RA even if they do not have ITP. Therefore, I love to see an initial response to treatment with corticosteroids in patients with ITP. I know that I am not going to cure the patient or provide long-term platelet control with corticosteroids 90% of the time, but an initial treatment response to corticosteroids or IVIg tells me that this is an immune-mediated process. That is very important. You have to use some immune modulation agent to maintain the platelet count because you do not want to keep patients on IVIg or corticosteroids long-term. Although patients improve their platelet count on corticosteroids or IVIg, you cannot wean them off of them and have patients unmaintained. As second-line therapy, platelet response with rituximab is only maintained in 21% of adult patients at 5 years, and thus is simply not a cure for approximately 80% of individuals. Therefore, you can use a TPO-RA at this point as second-line therapy to achieve safe platelet counts long-term. Due to treatment with the TPO-RAs, we rarely see true refractory ITP anymore. It is very rare nowadays because we can get safe platelet counts in patients for a long period of time with TPO-RA treatment.


Ahmed R, Devasia AJ, Viswabandya A, et al. Long-term outcome following splenectomy for chronic and persistent immune thrombocytopenia (ITP) in adults and children. Ann Hematol. 2016;95(9):1429-1434.

Arnold DM, Dentali F, Crowther MA, et al. Systematic review: efficacy and safety of rituximab for adults with idiopathic thrombocytopenic purpura. Ann Intern Med. 2007;146(1):25-33.

Khan AM, Mydra H, Nevarez A. Clinical practice updates in the management of immune thrombocytopenia. P T. 2017;42(12):756-763.

Kuter DJ, Bussel JB, Newland A, et al. Long-term treatment with romiplostim in patients with chronic immune thrombocytopenia: safety and efficacy. Br J Haematol. 2013;161(3):411-423.

Neunert C, Lim W, Crowther M, Cohen A, Solberg L Jr, Crowther MA; American Society of Hematology. The American Society of Hematology 2011 evidence-based practice guideline for immune thrombocytopenia. Blood. 2011;117(16):4190-4207.

Patel VL, Mahévas M, Lee SY, et al. Outcomes 5 years after response to rituximab therapy in children and adults with immune thrombocytopenia. Blood. 2012;119(25):5989-5995.

Provan D, Stasi R, Newland AC, et al. International consensus report on the investigation and management of primary immune thrombocytopenia. Blood. 2010;115(2):168-186.

Saleh MN, Bussel JB, Cheng G, et al; EXTEND Study Group. Safety and efficacy of eltrombopag for treatment of chronic immune thrombocytopenia: results of the long-term, open-label EXTEND study. Blood. 2013;121(3):537-545.

Thai LH, Mahévas M, Roudot-Thoraval F, et al. Long-term complications of splenectomy in adult immune thrombocytopenia. Medicine (Baltimore). 2016;95(48):e5098.

Wong RSM, Saleh MN, Khelif A, et al. Safety and efficacy of long-term treatment of chronic/persistent ITP with eltrombopag: final results of the EXTEND study [published correction appears in Blood. 2018;131(6):709]. Blood. 2017;130(23):2527-2536.

More in cITP



Long-Term Efficacy and Safety of the Thrombopoietin Receptor Agonist Eltrombopag

Clinical Study Insights by Howard A. Liebman, MD

Clinical Study Title:Safety and Efficacy of Long-term Treatment of Chronic/Persistent ITP With Eltrombopag: Final Results of the EXTEND StudyClinic...READ MORE



Future Goals for Advances in Chronic Immune Thrombocytopenia Management

Clinical Topic Updates by Adam Cuker, MD, MS

Immune thrombocytopenia (ITP) management has changed substantially over the last decade with the introduction of new medical therapies, including r...READ MORE



Proper Clinical Diagnosis Is Essential in Primary Immune Thrombocytopenia

Clinical Topic Updates by Howard A. Liebman, MD

A diagnosis of immune thrombocytopenia (ITP) is commonly made by a thorough patient and family history, physical examination, complete blood count,...READ MORE

More In Hematology

Sickle Cell Disease

Sickle Cell Disease: Managing Pain Without Encouraging Opioid Dependence

Expert Roundtables
by Stella T. Chou, MD; Michael Rutledge DeBaun, MD, MPH; and Julie Kanter, MD

Sickle Cell Disease

Sickle Cell Disease: Updates on Newer Therapeutic Options

Expert Roundtables
by Stella T. Chou, MD; Michael Rutledge DeBaun, MD, MPH; and Julie Kanter, MD

Sickle Cell Disease

Acute Management of Ischemic Priapism in Sickle Cell Disease

Patient Care Perspectives
by Michael Rutledge DeBaun, MD, MPH