How Can SCD Warriors and Families Help in Novel Therapy Research?
Dr. Marwan Shaikh, Maya Bloomberg, and Carla Lewis explain ways that sickle cell patients and care partners can help advance sickle cell disease care, resources for locating clinical trials, and how to advocate for improved sickle cell care.
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In this program:
Advancements in sickle cell disease treatment rely on clinical trial participation. Dr. Marwan Shaikh, hematology nurse practitioner Maya Bloomberg, and Carla Lewis explain ways that sickle cell patients and care partners can help advance sickle cell disease care, resources for locating clinical trials, and how to advocate for improved sickle cell care.
Transcript
Ariqa Everett:
So I did want to talk about the American Society of Hematology. So they have set priorities for sickle cell disease research for the next five years, and a few of those priorities are focused on disease severity as well as developing some novel therapies. So how can sickle cell disease patients and their families be a part and help you to speed up this process?
Maya Bloomberg, APRN:
So this is where recruitment is important because ultimately the faster we can recruit for clinical trials, the more fast, we'll get data and act on the information if something is going to actually be beneficial or not. So I think recruitment is important, continuing to bring awareness, and you can piggy-back and say so much from your experience.
Carla Lewis:
Definitely having a conversation with your doctor. I know that even from my son, we had to talk about does the risk outweigh the benefit? And that's a conversation that's necessary to also have, but understanding where you can go for those trials. He did mention on healthcare.gov, definitely has a list of trials and see what can I qualify for, and then maybe talk with your doctor about that. I think sometimes, even myself as a caregiver, we kind of wait for information. I remember trying to wait for information at times, but sometimes we have to actually be prompted even for patients, be prompted to look at information and then have that...start that conversation with our health decisions, that we can also know that there are resources out there. But also this might be for me, I will always remember the great NIH. They always put out information out to...they have a newsletter, great resources to be connected to, but also understanding what is a print clinical trial, sometimes that needs to be broken down in different ways to a particular patient, so understanding that you can feel comfortable to talk to not only a caregiver, not only another I would say peer-to-peer that have experienced being in a clinical trial that there is, seeing that what was the complications, what were the benefits and how was the staff that was also participating in the trial, how did they pretty much break everything down to understanding the waivers that you sign all that stuff, because I think a lot of times patients and even caregivers are very hesitant even to such things as clinical trials because of lack of knowledge so we're going back to that education, which is key.
So I definitely would like to say that again, education is key, and the information is there for clinical trials. You have to continue to have that discussion.
Maya Bloomberg, APRN:
And just one thing in the clinical trial, it's important for patients to realize, because we're going to be doing the Bluebird at our center, and there are so many patients who are interested in doing gene therapy that what makes a good participant of study participants. So if you have a patient who's not taking their medications regularly or they're not me showing up to their clinic appointments, even if you need all this inclusion and exclusion criteria, you probably won't be included in that. Research those. So if clinical trial is something that's of interest of you or trying to find a center that you can get gene therapy because who knows how it's going to be commercialized and how are you going to get access to it when it is commercially available, then doing the clinical trial really is a good opportunity, especially if there's a Phase III one, I would be jumping on that if I could to or if I needed to, I should say, but I think understanding what makes a good study participants is really important for a patient to know because it's another reason why you should take your hydroxyurea (Hydrea) regularly, get your labs, meet your doctors’ appointments, and all of that information getting taken care of.
Carla Lewis:
And I'll add to clinical trials, because I know some patients are pretty much hesitant because of resources. So being able to ask the question when there is the clinical trial available, even if they meet the criteria, they have to understand what resources, what support do you have transportation that will get you to that appointment, financial, being able to cover other things that may not be covered and ask, don't be scared to ask, what can they assist with? And there are assistance programs that clinical trials you have, so definitely being able to ask questions and understanding what your commitment will also be, and being able to communicate that also with you, be able to talk with that, what's your physician that's looking over that trial and having that discussion with your family.
Maya Bloomberg, APRN:
And it's such a good point, because you don't want a patient to be like, “Well, I can't participate, because I don't have a way to get to those appointments,” because a lot of times those clinical trials be working to the budget transportation, usually, you get compensated for the research visits and things of that nature.
So definitely have a conversation. So before you completely rule something in or out, you have all that information.
Dr. Marwan Shaikh:
And the one thing to mention about the American Society of Hematology, so this is the largest organization when it comes to hematology so that's one of the biggest memberships. But they are one of the leaders when it comes to all the hematologic, the disorders, including sickle cell, and so they actually just published earlier this year, a couple of months ago, they're repeat guidelines or their national guidelines for sickle cell, and so through in there and with what they're going to discuss at the meeting is going over all of these new therapies and the science being so that how can we develop new methods to manage pain, and so one of the new things that came out in the guidelines is that. For example, if someone goes to the ER, they're recommending that patients get their first dose of pain medication within 60 minutes, because sometimes we show hours in and all of us can know that, Okay, trying to get that to happen is going to be difficult, but it's about educating.
And so anyone can search these guidelines on home, if you're just type in A-S-H, ASH sickle cell guidelines 2020, you'll pull up the guidelines and you can read them or summary versions, so that they're going to be long, they are long. So getting to patients within 60 minutes in the ER, for example, reassessing them every 30 to 60 minutes, looking at other therapies and reminding patients like, yes, narcotics are one option. But obviously we have an epidemic on our hands, so we have to be able to incorporate other therapies as well to try to reduce the number of the narcotics the patient uses whether it's acupuncture or yoga or whatever it is, those kinds of things along with treatment for psychiatric, we're treating anxiety and depression along with family support along all of this kind of helps us provide a comprehensive program.
Maya Bloomberg, APRN:
And I think part of what they're doing as well as with the sickle cell trait research, which I think is so important because sickle cell trait we always say it's a benign condition, but the reality is, is under certain circumstances patients can have sickle cell complications and crises. I mean Ryan Clark was the Steelers football player who developed splenic sequestration while he was playing in Denver, so I don't even think he knew he had sickle cell trait, but he's playing in Denver with high altitudes over exerting himself playing NFL football, develops acute pain and ends up having this sickle cell complication. So I think we need to kind of change how we frame sickle cell trait, because I think it's so often referred to as a benign condition. And just from social media and just connecting with other people, there are people with sickle cell trait really are experiencing a lot of complications and they're not believed because we have this mentality that sickle cell trait is really just benign so I'm hoping with ASH's effort to bring more awareness and research for the sickle cell trait, you can get more answers and try and see why do some patients with trait have more symptoms, is it an oxygenation issue or underlying asthma or sleep apnea that contributes to it. But I think that's another area of unmet needs that we haven't had a lot of funding or trying to give attention to, so I'm excited to see where that goes.
Dr. Marwan Shaikh:
And I'm glad you brought up the trait, because one of the things that really gets lost in addition to what you mentioned is also kidney disease. People can have recurrent hematuria or blood in the , and they can't concentrate the urine as well because it changes in sodium and there's also a rare complication, medullary renal carcinoma. So these are very rare number one, but this stuff can kind of linger in the bottom there and go under the radar for a long time, and so it is important that you know that.
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