How Real-World Data Can Address Problems in MM

Rafael Fonseca, MD: Can I put I a plug in for 1 of my favorite topics, which I think is going to evolve? It’s going to explode: it’s real-world data. Real-world data will address some of these problems. Because even with large clinical trials. Look at ASPIRE, which is a massive clinical trial. We still don’t have the experience of thousands of patients being treated, and we’re learning, I’m engaged in several of those projects. For instance, you can cross-reference this with, you know, admissions for heart failure, etc, and you actually get a much better picture that is reflective also of the real world because of the eligibility concerns you mentioned before. I think we’ll learn a lot more. Organizations like the MMRF [Multiple Myeloma Research Foundation] [are] working on this. I’ve been working with Myeloma Crowd, with HealthTree as well. So there [are] a number of opportunities [in which] data will be collected, and I think our wisdom will be greater.

Mary E. DeRome: We are going to be starting a very large patient registry in the near future, probably in the fall, and we’ll be collecting a lot of data from patients in that effort.

Keith Stewart, MB, ChB: I worry a bit now that these regimens are becoming so complex that, for all the reasons you just spoke about, what’s the right [dosage] of carfilzomib to use? Should you use it once or twice weekly? What about infection prophylaxis when we start combining them together? Will the community still be using AR infusions or split [dosage] instead of 90 minutes? I worry that the bad things will be amplified in those real-world things, and I feel less and less comfortable with having a lot of inexperienced physicians looking after these patients.

Andrzej Jakubowiak, MD, PhD: We definitely need to help simplify it. But I want to bring patients’ perspective very quickly, because we have a trial where we had to use per trial in 2 years, 24 months of KRd [carfilzomib, pomalidomide, dexamethasone] treatment. Median duration of treatment on this trial was 24 months, which means that most of the patients complete the treatment as planned. I spoke with those patients later on and of course during the treatment. And I’m hearing unanimously the same. The patients [are] always awaiting convenience versus ultimate benefits. And there was such a high level of achieving complete responses and durable responses. And for most patients, fairly good tolerability because KRd [carfilzomib, pomalidomide, dexamethasone] for the most part with few exception is very well tolerated. There was no question about it that they didn’t regret that commitment for their freedom of being in remission for years to come.

Keith Stewart, MB, ChB: You know, 1 of the things we sort of alluded to earlier is overall survival, and certainly the only 2 trials today that are showing overall survival advantage in relapse are the carfilzomib-containing trials, ENDEAVOR and ASPIRE. Although I’m sure we’ll see the POLLUX trial showing—if it hasn’t already, I may have missed it—the overall survival...

Rafael Fonseca, MD: I think the ELOQUENT [trial] had overall survival update, too. I believe so. Even though it’s not a common regimen.

Keith Stewart, MB, ChB: And then the other thing that we did in some of these trials was the health-related call-to-life assessment. I think [with] those things—together with the economics, with the ASPIRE trial, and I think in ENDEAVOR too, but particularly the ASPIRE, which I know better—the health-related quality of life globally improved during treatment, as well. Even though they were spending more money on 3 drugs, the overall survival was better, progression-free was better, and quality of life was better. So I think all the things you need for value were there.

Rafael Fonseca, MD: You know, [there is] one important point for patients who may read very dated literature. You know they’ll come to the office and sometimes say, listen, “I’m more about quality of life than quantity of life.” The good news is if you are mindful of toxicity [in] myeloma, both are 1 and the same. Because as you improve their disease...gets controlled, the quality of life comes up and certainly all the clinical trials [to] suggest improvements in overall survival.

Again, real-world data, which can show this when trials [don’t], there [are] massive improvements in survival of myeloma patients. We’re not there yet. We’re not celebrating that we have reached our goal. But there is no denying that survival has significantly improved.

Keith Stewart, MB, ChB: Well, thank you for a great discussion. It’s been extremely informative. Before we sign off today, I’d like to get final thoughts from each of our panelists. Mary, what do you want to leave as your parting gesture here, your parting thought?

Mary E. DeRome: One of the biggest areas of need, I feel, is education of the community health care providers. I mean, that’s where the vast majority of our patients are treated. And these community doctors need a lot of education and a lot of help keeping up with all the developments in myeloma over the years. And I’m hoping that collectively the community can come up with ways of really keeping the community physicians on track, which will totally benefit our patients.

Keith Stewart, MB, ChB: I would 100% agree with that. And I don’t know how they [do it]. I can keep track of [only] 1 disease, I don’t know how my colleagues in the community do it. It’s amazing. Rafael?

Rafael Fonseca, MD: [Even though] at first glance [it] may seem [like an] incredibly complex field for drug selection, I think it’s going to get simpler. I think there [are] 2 or 3 common pathways that are going to convert, thankfully. But the support and the care of myeloma patients are going to get intimately more complex, as you just alluded to. So we have that conundrum. I think our treatment selection is going to be simpler. Prophylaxis decision making—when do you estimate, when do you change? Incredibly complex. So I think the participation—you know, partnership with community doctors—is critical.

The second point is [that] this is all possible because of clinical trials, and I have to say, Andrzej, I’m very impressed because of your reference to your participation in clinical trials, because that’s what we should all be doing: active participation in trials to move the needle forward, so [we] actually we have the right answers for our patients.

Keith Stewart, MB, ChB: Last word to you.

Andrzej Jakubowiak, MD, PhD: We do help patients by providing them with the opportunity. There’s a right trial for a given patient. But I want to say that thanks to that particular experience, and thanks to the trials [that] have generated the data [that] we have shown, I think that we are at the stage [at which] we can start very seriously revising goals of our therapy. And that eventually will also affect our treatment choices. Even if we cannot declare at this point that we are curing myeloma, many of us believe that some patients are indeed getting cured. If we set up our discussion, our treatment plan, with that goal as [the] ultimate goal, even if we don’t achieve it, in the process we are achieving good outcomes for the patient because they stay at least a lot in remission. So I think that will eventually help us simplify our choices and make it easier.

Keith Stewart, MB, ChB: Well, thank you all for your contributions to this discussion. On behalf of our panel, we thank you for joining us, and we hope you have found this AJMC® Peer Exchange discussion on multiple myeloma both useful and informative.