New care pathway improves treatment decisions for children with ITP
Multicenter effort boosts adherence to guidelines without more bleeds
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A group of U.S. treatment centers worked together to standardize care for children with immune thrombocytopenia (ITP), leading to more patients receiving treatment that follows current standard-of-care guidelines.
This initiative also reduced the use of medications that may cause side effects in children with ITP, without increasing bleeding events or hospitalizations.
Researchers described the results of the initiative in a study, “Multicenter initiative to standardize management of pediatric immune thrombocytopenia improves adherence to guidelines,” published in Blood Advances.
Understanding immune thrombocytopenia and how it affects children
ITP is an autoimmune disorder in which the immune system attacks and destroys the platelets, the cell fragments that help blood clot. Without enough platelets, people with ITP may develop symptoms such as unusually heavy or excessive bleeding.
ITP can occur in both children and adults. However, in children it often does not cause severe bleeding, and many cases resolve on their own within about six months.
The American Society of Hematology (ASH) publishes guidelines for managing pediatric ITP that are updated as scientific understanding evolves. The most recent guidelines, published in 2019, emphasize that treatment decisions should be guided primarily by a patient’s bleeding symptoms, rather than platelet levels.
For patients with no or only minor bleeding symptoms, the guidelines recommend observation rather than medical treatment, meaning children are monitored without immediate therapy. This approach helps avoid side effects from unnecessary medications or procedures.
However, if a newly diagnosed patient has at least mild bleeding, the guidelines recommend a short course of oral corticosteroids as first-line treatment. This differs from the previous guidelines published in 2011, which recommended either corticosteroids or intravenous immunoglobulin (IVIG).
This change was made to help reduce treatment burden and potential side effects. Available data suggest that short courses of corticosteroids and IVIG are similarly effective, but IVIG is generally considered more burdensome because it can involve additional side effects and medical visits.
Multicenter effort aimed to align care with updated ITP guidelines
After these guidelines were published, 12 treatment centers across the U.S. joined together in an initiative to standardize how children with ITP are managed, aiming to ensure that the care aligned with best-practice recommendations.
“Collaboration across multiple institutions is a key success factor in studying rare conditions and improving adherence to evidence-based guidelines,” the researchers wrote.
To assess how well the initiative worked, the scientists compared medical records from 284 children with newly diagnosed ITP treated before the initiative with those of 266 children treated after it was implemented.
Results showed that before the initiative, medical records from a first visit with a hematologist rarely included a formal score measuring bleeding severity, even though bleeding symptoms are meant to be the main factor guiding treatment decisions.
After the initiative was introduced, 95% of patients had a bleeding severity score documented at their first visit. The researchers noted that standardizing how bleeding severity is recorded helps guide treatment decisions and makes it easier to track how a patient’s condition changes over time.
Before the intervention, nearly two-thirds (62%) of patients with mild or no bleeding symptoms still received ITP-directed medications. After the intervention, that rate decreased to 31%. Additionally, use of IVIG fell from 51% of patients before the intervention to 15% afterward.
Initiative reduced unnecessary treatments without worse outcomes
“Through the adoption of the 2019 ASH ITP guidelines and the clinical care pathway, centers demonstrated a reduction in IVIG usage in patients with low-risk bleeding scores and an increased use of corticosteroids rather than IVIG for patients with moderate bleeding scores,” the researchers wrote. They noted that these shifts are expected to reduce treatment burden for patients and may also lower healthcare costs.
The scientists also noted that it was “expected and appropriate” that some patients received treatment that did not fully align with the recommendations, since the guidelines stress the importance of considering families’ individual preferences when making treatment decisions.
The researchers also highlighted that clinical outcomes, including rates of hospitalizations and bleeding events, were similar before and after the intervention, suggesting that following the updated guidelines may help reduce treatment burden without increasing the risk of uncontrolled disease.
