The Use of Patient-Reported Outcomes to Assess Symptom Burden in Sickle Cell Disease
In this podcast, Nirmish Shah, MD, discusses patient involvement and the use of patient-reported outcomes in the development of sickle cell disease interventions. Dr Shah’s team presented research on this topic titled “Development of a Patient Reported Outcome Daily Diary to Assess Symptom Burden in Sickle Cell Disease” at the American Society of Hematology’s 2023 annual meeting in San Diego, CA.
Additional Resource:
For more coverage of ASH 2023, visit the newsroom.
TRANSCRIPTION:
Jessica Bard: Hello everyone, and welcome to another installment of Podcasts360—your go-to resource for medical education and clinical updates. I’m your moderator Jessica Bard with Consultant360, a multidisciplinary medical information network.
Dr Nirmish Shah is here to speak with us today about his team’s research presented at ASH 2023 titled “Development of a Patient Reported Outcome Daily Diary to Assess Symptom Burden in Sickle Cell Disease.”
Dr Shah is the director of the sickle cell transition program and the director of clinical research in benign hematology at Duke University School of Medicine in Durham, North Carolina.
Thank you for joining us today, Dr Shah. Please provide an overview of this research.
Dr Nirmish Shah:
Yeah, so I think that one effort that we've all realized is that there's a lot of clinical resource studies and what is the best method to show that we're making a benefit to our patients who are being treated with these different new novel therapies? So, for example, we have lots of different new drugs in development. We have lots of new interventions we're considering. But what we've learned over the years is that because sickle cell is a complicated disease, because there are lots of different aspects to the pathophysiology, the outcome of just saying, "Did I get better with pain?" is really oversimplifying what the problem is within sickle cell. We can't just look at how many times have they been in the hospital. How many times are they having pain crisis? Because the severity of sickle cell varies. The definitions all vary. And so what we're moving to, and I think rightfully so, is trying to have a big-picture understanding of all the aspects that patients with sickle cell can have issues with.
And therefore, if they're having issues with it, I can potentially make an improvement. And that's where patient-reported outcomes come in. We ask patients through validated questions how they're doing in regards to their sickle cell, and therefore if we make an intervention, then do we make a benefit in those aspects of their lives? And so the overview really essentially is can I develop a patient-reported outcome that meets FDA guidance? The FDA has actually outlined how they want these patient-reported outcomes to be developed and then leveraged and used for clinical studies. And that was really our intention with this research project.
Jessica Bard:
What were the results of this study?
Dr Nirmish Shah:
As a very quick summary, we looked at both adults and adolescents to go through interviews in what we call concept elicitation and then cognitive interviews. And what that really means is we want the patients to go through a series of concept elicitations, basically is what are the hot topics for patients in the sense? What is it that's meaningful for us to know about their disease? And of course, when you go through the first level of interviews, you're going to keep repeating these until you don't have any new concept elicitation, new topics that are really meaningful to them. And then you really want to dive deep into which aspects of those also are seen within adolescents. And interestingly, the ones that make a lot of sense, of course, are pain and fatigue. But there are a lot of other aspects that were brought up as well. So, the results included for symptoms, for meaningful aspects of their lives, interference in school and productivity, and then really just an overall kind of assessment of their severity and how patients feel the severity of their diseases.
So, the end result is not only coming up with these topics that are important to patients through, again, these concept elicitation interviews and these cognitive interviews, but now we have this list where we can have patients grade themselves and we can further validate. So, that's where we go from here. And kind of leading into the next question that often comes up is, okay, well now we have these results. I know what are the most important topics to our patients, which again, pain, fatigue, stiffness, not being able to do the things they want to do in school and activities, and rating of severity, and use that question that we just developed and validate it. So, we want to now take the next step as validation of that patient-reported outcome. And once we go through that process, then we now have a nice patient-reported outcome that can then be used in clinical trials.
Jessica Bard:
So, you certainly maybe just covered the next question of the importance. Is there anything else that you wanted to add about why this study, why now, and the importance of it?
Dr Nirmish Shah:
Yeah. I would again want to just really emphasize that the experience of patients with sickle cell is very complex and I think we really need to make sure that the patients are involved in our conversations about what is meaningful. What are the outcomes that really make a difference to you? So, when we make an intervention, when we design a drug trial, we're not trying to oversimplify and have results that are not reflective of what benefit that we are hoping to get. So, I think that's why I'm really, again, optimistic in what we're doing because we're doing it in a way that not only the FDA has guided us to say that this is the way we want you to develop these patient-reported outcomes, but we're involving the patients through these different steps. And I think that that's really, I think, an important aspect moving forward.
Jessica Bard:
Moving forward. I've been listening to your answers here. You might've already covered this one too. I mean, just what's next for research on this topic? Or is there anything else that you'd like to share about what's next?
Dr Nirmish Shah:
Yeah. I think what's next in addition to the validation that we're mentioning, is our next step of making sure that the different topics that we have really do fall out as being a good measure of each of those topics. But I think that the next step is to actually use it in a clinical trial. So, once we've validated this, then the hope is that this patient-reported outcome could be leveraged by different sponsors, investigators, and clinical studies, to then be a good reflection of how the patient experience is really hopefully improving from each of those interventions.
Jessica Bard:
And the overall take-home messages from this study.
Dr Nirmish Shah:
So, the take-home is that number one, we really do need to involve the patients. The patients are really, of course, the whole purpose of why we're developing these interventions, and the patient experience is extremely important in that. Number two, we can't just ask the patients how they're doing at one point in time in regards to developing these surveys. We need to make sure we continue to circle back to them and show them in the community, that this is what product we came up with. This is the patient outcome that we validated. And third, as we do clinical studies, I actually think it's just as important to make sure that they're involved. I think that effort has resonated in the sickle cell community to make sure patients, advocates, and families are all involved in the clinical studies because they, I think, have really insightful aspects to give guidance to the developers of these clinical trials.
Jessica Bard: For more coverage of ASH 2023, visit Consultant360.com