Adult Immune Thrombocytopenia Purpura - Episode 5
Ivy Altomare, MD: Not considering someone an acutely ill patient but, rather, a regular patient that does need treatment, who you’re treating in the outpatient setting—we are using steroids as appropriate first-line treatment. Are there data to add other agents to steroids in the up-front setting?
Ralph V. Boccia, MD, FACP, LLC: So at ASH this year, there’s a paper presented now giving 14 days of eltrombopag with pulsed-dose steroids compared with pulsed-dose steroids alone, and response rates were significantly higher—about 90%—in the combination arm compared with about 60%, 65% on the dex [dexamethasone] alone arm. And, at 6 months—this was a 12-week study, so 12 weeks of eltrombopag, and 3 months or three 4-day courses of high-dose dexamethasone—at 6 months, over 50% of the patients—I think it was 56% of the patients—were still responding with platelet counts in the…I think the medium was like 150,000.
Ivy Altomare, MD: Wow. So do you think that this will become a new approach? Is it too soon to use this approach?
Ralph V. Boccia, MD, FACP, LLC: I think what will be nice is that at next year’s ASH, we can see what the durability of that is beyond 6 months. But I think that…we do a lot of combination therapies in many of the diseases that we as hematologists-oncologists treat, and there’s no reason not to expect the best response. And so 14 days of eltrombopag is not a lot of eltrombopag.
Ivy Altomare, MD: Absolutely.
Amit Mehta, MD: Yeah, and I’ll just add to that a little bit…that, just thinking from a mechanism perspective, especially given that ITP [idiopathic thrombocytopenic purpura] is a clinical diagnosis and we’re trying to think of these patients, in this heterogeneous population of patients that we face with ITP…, the response rate was nice to see with eltrombopag, given in conjunction with dexamethasone. But, just as I think Ralph is alluding to…, the issue is: What is the durability of the benefit, especially in an acute ITP setting, where your goal is, hopefully, to induce a long-term remission in the patients? So that will be a key thing. I think given the fact that you give a thrombopoietin-stimulating agent in addition to a steroidal agent, it’s not surprising, clinically, intuitively, that we’ll have a nice response rate benefit, but is that response rate translating longer term, especially when it’s just 14 days of eltrombopag? So I think, like you said, stay tuned for what the update may be next year.
Ralph V. Boccia, MD, FACP, LLC: And historically, we know that the dex data would suggest maybe 20%, 25% 6-month relapse-free.
Ivy Altomare, MD: Right.
Ralph V. Boccia, MD, FACP, LLC: So that’s really more than doubled.
Ivy Altomare, MD: Well, that will be exciting to look forward to. Speaking of looking forward to, we are looking forward to the new version of the ASH guidelines. I understand you have looked at this already?
Amit Mehta, MD: Yes. So, I had a chance to look at it. The ASH guidelines currently, of this filming, are still in a comment period, so they’re not finalized yet. So we may see further changes. A lot of the update—this is the first update in several years.
Ivy Altomare, MD: Yes.
Amit Mehta, MD: For the ASH guidelines for ITP, so it’s nice.
Ivy Altomare, MD: At least 5, right?
Amit Mehta, MD: At least 5 years, yes. So I think it’s nice to see that there is an update.…Some of the key points are the same that I have seen thus far, so the platelet count of 30,000 is still the recommended threshold to consider treatment. Again, as we discussed earlier, the individual threshold for considering treatment can depend on, hinge on so many different factors. But that’s what the ASH guidelines still say in the preliminary one right now. And they still mentioned…some of the issues that we’ll get to shortly, that rituximab is recommended over splenectomy for second-line or later ITP treatment in the chronic phase. And those are some of the major points.
And the other point—that, hopefully, we’ll see in the guidelines but we’ll wait for the final version to come out—is, how much do they inform clinicians about newer agents that we have available currently, FDA agents that we have currently available, that were totally not in the picture when the last guidelines were released several years ago?
Ivy Altomare, MD: Right. That’s why we need the update.
Amit Mehta, MD: That’s why we need the update. But I haven’t seen much of that section yet, so I think we’ll be interested to see if they, hopefully, include some of those features in the ASH guideline, because clinicians are definitely quite inquisitive about how…they use these new medications.
Ivy Altomare, MD: Well, it’s exactly what you said very astutely: that first-line treatment is not a difficult decision because we know really what to do. But second-line treatment, or treatment of refractory disease, that’s where guidance is needed, because we had several effective therapies. We don’t have randomized head-to-head trials of any of these modalities to help, and treatment is so individualized to the patient.
Transcript Edited for Clarity