“Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP

“Impatient” Therapy: Physicians Too Aggressive in Treatment of ITP 150 150 Brianne Moore

Despite guidelines that advocate watching and waiting, physicians are still treating most patients with immune thrombocytopenia.

When a child presents with easy bruising or bleeding, red skin spots and fatigue – symptoms similar to those for leukemia – families seek evaluation immediately. However, more often these cases are pediatric immune thrombocytopenia (ITP), an acquired disorder that destroys blood platelets and is most common in 2- to 5-year-olds. Historically, the disorder has been treated pharmacologically to increase platelet count until the illness resolves.

In 2011, the American Society of Hematology (ASH) published new treatment guidelines for pediatric ITP that recommended a watchful waiting strategy in children with mild or no bleeding, regardless of platelet count. A multicenter cohort study recently published in Pediatric Blood and Cancer shows that the changes may not have been widely implemented.

“Given the guidelines, patients newly diagnosed with ITP should only be admitted to the hospital and given pharmacological treatment for clinically significant bleeding,” says Sarah O’Brien, MD, principal investigator in the Center for Innovation in Pediatric Practice in The Research Institute at Nationwide Children’s Hospital and an author of the retrospective study. “But we found that even after the guidelines, admission frequency was about the same, and 86 percent of patients admitted did not have any diagnostic codes consistent with bleeding. Most of these patients received treatment.”

The team looked at data from almost 5,000 ITP primary admission patients across the United States between the ages of 6 months and 18 years between 2008 and 2014 to determine whether pharmacologic therapy for pediatric patients without significant bleeding symptoms had decreased.

The study found that in the three years following the publication of the new guidelines, only about 14 percent of patients had documented bleeding complications due to ITP, but 93 percent of patients received pharmacological treatment. These treatments, including intravenous immunoglobulin and corticosteroids, can be expensive and have side effects such as fever, nausea and aseptic meningitis.

“We’re currently working to determine whether new ITP patients admitted to the hospital and patients that leave the emergency department and go home for observation are significantly different symptomatically from each other. The retrospective study focused exclusively on patients that have already been admitted to the hospital, not necessarily those seen in the ED or in a hematology clinic,” adds Dr. O’Brien, also a physician in the Division of Hematology, Oncology & Blood and Marrow Transplant.

Dr. O’Brien and her colleagues still have a lot of questions about the trends observed in the data.

“What are we missing in this drive to treat? Clearly, treatment is not associated with reducing bleeding, as most patients receiving treatment have little to no bleeding at all. To understand the discrepancy between what the guidelines say and what we practice, we need to understand what’s causing the variation.”

This article appeared in the Fall/Winter 2017 print issue. Download a PDF copy.


Witmer CM, Lambert MP, O’Brien SH, Neunert C. Multicenter cohort study comparing U.S. management of inpatient pediatric immune thrombocytopenia to current treatment guidelinesPediatric Blood & Cancer. 2016 Jul; 63(7):1227-1231.

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About the author

Brianne is interning as a science writer and editor for Clinical & Research Communications at Nationwide Children's Hospital. She contributes to a variety of publications, including Pediatrics Nationwide and PediatricsOnline.