Immune thrombocytopenia (ITP) was a major focus at the 2019 American Society of Hematology annual meeting in Orlando, as the society released new clinical practice guidelines in the journal Blood Advances). Developed in partnership with the University of Oklahoma Health Sciences Center, the guidelines are intended to support patients, clinicians, and other health care professionals in their decisions about the management of ITP.
In this exclusive Ƶ video, the chair of the ASH ITP guideline panel, , of the Cleveland Clinic, discusses the new recommendations.
Following is a transcript of her remarks:
We're very excited to announce that the ASH 2019 guidelines on the management of immune thrombocytopenia are published and available. These guidelines really should help to instruct clinicians by putting all of the evidence-based articles and papers in one place, and then assimilating that into answering clinical questions for providers. These guidelines really highlight a couple key things in the care of patients with ITP. Specifically for adults, there's a push to really move away from chronic, long-term corticosteroid use, recognizing the side effects of those corticosteroids when they are ongoing. There was also a lot of discussion about how to use second-line therapies in adult patients with ITP, particularly when we started to talk about the agents such as the TPO receptor agonists, rituximab, and then the role of splenectomy.
In these guidelines, really that came down to the fact that we don't have great evidence. We don't have any side-by-side randomized clinical trials for these. Really, patients and providers need to engage in shared decision-making, so a discussion about how long has the patient had ITP? What are their goals? What's their previous history of living with ITP and their bleeding events? What's their overall quality of life and what are they trying to achieve in their treatment? By using shared decision-making, that can fill in some of the gaps of what exists in the evidence.
It also helped us by doing this to identify the gaps that need to be addressed with future research studies. What we found was that really we need to start applying or using more rigorous patient-related outcomes. It used to be in the past that all we did was rely on the platelet count to tell us what was going on, but we've come to understand and appreciate that more and more we need to get at the patient's perspective of what's going on in terms of looking at their quality of life. Many patients with ITP suffer from fatigue, better capturing of medication side effects, so that we can use all of that to inform what we do for patients.
On the pediatric side, again, we really recommended and tried to push for observation. We've come to appreciate that our treatments have significant side effects that will impact patients, and therefore, sometimes we recommend just observation for a patient, a watch-and-wait approach, particularly for a child that has no or mild bleeding when they first present, recognizing that the likelihood that something more significant will happen to them from their ITP is about the same as the likelihood that they'll have an adverse event from medication.
With regards to the second-line agents in pediatric patients, it seems as though the favor is for TPO receptor agonists. These agents seem to have the lowest side effect profile for pediatric patients, and then also rituximab. Then lastly, we try to defer splenectomy as long as possible in children, and in adults, we try to defer it for at least a year. Again, though, even in pediatrics, there's not a lot of good data putting these things side by side, and there's no randomized clinical trials, and so therefore, again, the push is really to engage in shared decision-making and to conduct clinical trials that get at patient-related outcomes.