ITP Treatment Selection Criteria and Treatment Goals


Ivy Altomare, MD: That brings us to the features that you take into consideration when you’re initiating treatment, and bleeding is 1 of those key components. What are your goals when you’re initiating treatment for patients?

Amit Mehta, MD: Yeah. When I’m initiating treatment for a patient with ITP…the 2 fundamental pieces really are, what is the clinical symptomatology for the patient as far as the severity of their bleeding symptoms, as they’re presenting with? And secondly, what is the absolute platelet count? So on the latter point, there is often significant debate about if there is even a platelet count, at which a hematologist will say, is the threshold over what you want or under which you might treat or not treat? So the ASH [American Society of Hematology] guidelines, for example, have suggested 30,000 as a threshold to consider treatment. However, I think in practice, many of us will feel comfortable with a platelet count that’s even lower for patients, as long as their bleeding is under control beyond simple transient epistaxis or very mild—what do you call it—mucocutaneous bleeding, then we might be OK with those kind of very mild symptoms that might happen.

Ivy Altomare, MD: Sure, yeah. There are so many components—you know, what the degree of bleeding is, what the lifestyle of the patient is, their age, if they’re active, if they have access to care. So you know as well as I do, in North Carolina, that many patients have to drive 2 hours away to see their hematologist, so you’re going to probably keep that patient at a different platelet count level than another patient who’s more plugged into the healthcare system.

Amit Mehta, MD: Yeah. And I believe comorbid medications—so, people on antiplatelet therapy, anticoagulant for any reason—these are definitely important features that you might be more likely to treat than not treat because of those competing risks for the patient. Are they a fall risk, which happens, especially with the elderly like we’re talking, about who have a higher bleeding risk anyway—plus if they’re a fall risk and plus issues like vascular fragility in that same patient, and suddenly you have more of an argument for treating a particular patient. But I think the thing that really would clinch my decision to treat…a patient I’m treating is, are they having bleeding symptoms? And if they’re clinically significant bleeding symptoms by whatever measure you feel is relevant, then that usually will justify treatment for that patient.

Ivy Altomare, MD: Yup, good point. And so once a physician starts to initiate treatment, Dr Boccia, can you describe what your goals are? So you decided to initiate therapy—what are your short- and long-term goals?

Ralph V. Boccia, MD, FACP, LLC: So obviously stopping either the bleeding or the risk of bleeding is the primary goal, and doing that in as safe and convenient and nontoxic a way as possible. So, initiating therapy, as we’ve already discussed, and the trigger points and whatnot, is going to be choosing something that will get the patient out of the risk period and get rid of the bleeding that they have acutely, and then trying to choose a therapy that will, hopefully, maintain that response as long as possible with as few drug-related adverse effects as possible. And we know with all the therapies that we have today, there’s no free ride, and so the selection of drug is very patient specific, lifestyle specific, and risk of bleeding specific.

Ivy Altomare, MD: I absolutely think that the goal of reducing lifetime exposure to steroids is one of the most prominent things, as you mentioned. And fortunately, we have other agents now to be able to do that.

Ralph V. Boccia, MD, FACP, LLC: And I think that the steroids are also probably the most hated drug that we prescribe.

Ivy Altomare, MD: I have a patient that blamed her divorce on steroids, so, yeah, I think so very hated. And that, actually we should talk about the patients. So, Dr Mehta, what do patients tell you in terms …what their goals are for getting management?

Amit Mehta, MD: One thing that’s very prominent, from the patient perspective, is the more constitutional type of symptoms that they’ll bring up during their presentation. So a classic situation I think we all face is that the patient will come in and say they’re more fatigued than they used to be, and they…almost I believe…in my view, they’re more concerned about those kind of constitutional symptoms than they perhaps are with the petechiae they’re experiencing or the nosebleeds. The nosebleeds might be annoying, but they’ll often say…, “Well, I can put up with a nosebleed doctor, but I want to feel better in a more general sense.” So I think that definitely is a very prominent goal from the patient perspective, and definitely it’s a feature of ITP that patients do have these kind of other, more constitutional nebulous type symptoms that appear to be connected by some mechanism that we don’t understand.

Ivy Altomare, MD: Absolutely.

Amit Mehta, MD: But it definitely is happening. Now, of course, for the patient who is bleeding, especially if they’re having any kind of significant bleeding like hematuria or rectal bleeding, that definitely will be the patient’s concern, as well as the clinician’s, for sure. So those would be some of, I think, the biggest things they want to do. And…like Ralph mentioned about the duration of steroid therapy and so forth and the potential adverse effects of steroids some patients, even if they haven’t been on steroids will say, “Oh, well, I’ve heard all these bad things.”

Ivy Altomare, MD: Yeah, that’s true.

Amit Mehta, MD: This kind of perspective is out there. I have patients who have never taken steroids, and they’ll say, “I’ve heard bad things about prednisone,” you know? And that’s also where, fortunately, we have some options about how to minimize a duration of treatment, and, therefore, just like Dr Boccia was saying about maximizing the benefit, minimizing the adverse effect risk of the medication hopefully achieve a stable response in the patient.

Ivy Altomare, MD: I was nodding my head vigorously when you were discussing the fatigue because I really feel that this is another underrecognized feature of this disease. And if you look, it’s in 40% of patients where when their platelet counts are low, they will be extremely tired. And you’re right, we don’t know how that happens, what the mechanism of that is, but I have noticed and patients have told me that when their platelet count is in a better range they really do have more energy. So this is an interesting phenomenon and you’re right, I absolutely agree with you, it can really affect patients’ lives. So, thank you for bringing that up.

Ralph V. Boccia, MD, FACP, LLC: I think it’s a little bit like iron deficiency. Patients who have had iron deficiency anemia, been treated for iron deficiency anemia effectively, and gained that benefit of iron replacement know when they’re becoming iron deficient even before they’re anemic again. And I think it’s the same thing. These patients can feel what their platelet count is and know, “I think it’s low.”

Transcript Edited for Clarity

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