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Transforming Pediatric ITP Research: Inside the Pediatric ITP Consortium of North America (ICON)
35 Plays4 days ago
If treating pediatric ITP can sometimes feel like trial and error, this episode helps explain why—and what’s being done to change that. Host Barbara Pruitt talks with Dr. Kristin Shimano about the growing role of collaboration in advancing research, improving treatment decisions, and focusing not just on platelet counts, but on quality of life for patients. You’ll get an inside look at how specialists are working together across North America to better understand this rare disease and what that means for families searching for answers and hope.
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Transcript
Therapies for ITP: Trial and Error?
00:00:00
Speaker
One of the difficulties that we have in treating ITP is that there are a number of different therapies we can choose, but they haven't necessarily been compared directly against one another. And often, i think for families, often it feels like it's a little bit just trial and error and people who are just you know trying one thing and maybe it's going to work, maybe it's not.
00:00:24
Speaker
Pediatric ITP is a relatively rare disease and To learn more about it, to advance the field, we really have to do things in a collaborative way.
Introduction to 'Bruised But Not Broken'
00:00:38
Speaker
Welcome to the PDSA podcast, Bruised But Not Broken, Living with ITP. The diagnosis of a bleeding disorder like immune thrombocytopenia may leave you wondering, how can I really live my life with ITP?
00:00:52
Speaker
PDSA's podcast, Bruised But Not Broken, Living With ITP, brings empowering stories, the latest research and treatment updates, lifestyle tips, and answers to the real-life questions the ITP community is asking.
00:01:07
Speaker
Here's your host for this episode, Barbara Pruitt.
00:01:19
Speaker
I can only imagine the anxiety that a parent feels when their child is diagnosed with ITP. Aside from seeing your pediatrician frequently and your child's hematologist, I'm sure when you're home in the quiet of your room, you're wondering, what else is going on out there?
00:01:42
Speaker
Are there any new findings? Is there any new research? are How about new advancements? or in understanding and treating ITP? Well, today we might have some answers for you, and at the very least, give you some hope for the future.
Guest: Dr. Kristen Shimano and Her Role
00:01:58
Speaker
Because today's guest is Dr. Kristen Shimano.
00:02:03
Speaker
She is a pediatric hematologist and a bone marrow transplant physician at the UCSF Benioff Children's Hospital in San Francisco, California.
00:02:15
Speaker
Dr. Shimano has a particular interest in immune site cytopenias, including ITP. She currently is the operations committee chair of the ITP Consortium of North America.
00:02:31
Speaker
I know that's a mouthful, and we call the ITP Consortium of North America ICON, I-C-O-N. So thank you for joining us today, Dr. Shimano.
00:02:43
Speaker
Thank you for having me. I'm looking forward to learning more about ICON. And um could you tell us
What is ICON and Its Mission?
00:02:53
Speaker
about it? I'll let you start and just explain however you want to about what you are and what all ICON does.
00:03:02
Speaker
Sure. So ICON is the Pediatric ITP Consortium of North America. It's a group of pediatric hematologists from around the u S and Canada and Mexico, um who have a particular interest and expertise in treating patients with it.
00:03:25
Speaker
It was established in 2012 and has um grown quite a bit since then. We now have over 50 sites that participate. Um, and the mission of icon is um to be a collaborative research effort dedicated to improving understanding, treatment, and quality of life in pediatric patients with ITP.
ICON's Structure and Collaboration
00:03:51
Speaker
So when you say you have 50 sites, what what is considered a site? So a pediatric hospital or pediatric hematologist within a hospital that wants to participate as a part of ICON.
00:04:07
Speaker
So do hematologists join? Is there a membership process or who makes up the membership? Yeah. So members are pediatric hematologists from a variety of different ah children's hospitals and pediatric program, pediatric hematology programs around North America.
00:04:28
Speaker
Okay. So we're talking about the United States and Canada. Any other countries involved? It's Canada and Mexico. Great. and And how many members do you have now?
00:04:39
Speaker
So there's over 50 sites and then multiple ah members from each site. So ah quite a ah large number. That's great. How often do you meet?
00:04:51
Speaker
I'm sure you meet virtually since you're spread out all over, correct? Right. Well, we have one annual in-person. It's actually a hybrid in-person virtual meeting once a year, which is really nice to get to see everyone.
00:05:04
Speaker
together in one spot. um But most of our meetings are virtual and um the consortium has several different committees. And so each committee meets um you know once a month or once every other month.
00:05:21
Speaker
um And then within each committee, they're usually working on several different projects. And so the different projects groups may meet also on a regular basis, depending on what's going on with the projects.
00:05:33
Speaker
How are the committees determined? Is it by disease or disorder or what is it that makes up the committee and how many are there?
00:05:45
Speaker
So there's four main committees within ICON. The Biology Committee, the Quality Committee, the Interventional Committee, and the Patient Outcomes Committee.
00:05:56
Speaker
um And so all members of ICON are required to participate actively in one of the committees. And then within the committees, they work on research projects related to that specific area. So the interventional committee works on projects related to interventions for ITP, whereas the biology committee works on projects related to understanding the underlying biology and why ITP happens.
00:06:26
Speaker
Oh, that's interesting. So they can overlap then, I would think. Findings from one committee is shared with another committee, and then they may choose to go further with that.
00:06:36
Speaker
Does that make sense? Yeah, absolutely. There's a lot of overlap, and some projects may span multiple committees. so And now we are actually, this year, just started a database working group that has participants from all of the different committees because As we're establishing a database, those are all important things that we want to make sure that we're capturing. So um there's a lot of crosstalk. People can be on multiple different committees and there's crosstalk between the committees, projects that overlap.
00:07:08
Speaker
The main idea of having those four large areas is that those are all important aspects to cover when we're thinking about learning more about ITP.
00:07:19
Speaker
Well, i I think it's wonderful to hear you talk about this because that's telling us that you've got a lot of collaboration out there when it comes to research and findings, that the people dealing with it are actually communicating with each other, which is really such an important part. and And I think as a patient, you sometimes wonder, well, are they finding something out over here and not telling over there? But it sounds like you've got a wonderful collection of people that coordinate what their findings are.
00:07:53
Speaker
Yeah. i mean, I think that was really the genesis behind the consortium is that pediatric ITP is a relatively rare disease. And To learn more about it, to advance the field, we really have to do things in a collaborative way and um work with other pediatric hematologists around the country because one person may have one patient with a very severe course and they can learn from a patient, you know, from a hematologist elsewhere about
00:08:27
Speaker
how they've treated that patient. And to really understand the biology of the disease, you need to study many, many patients. And we can only do that by working together as ah as a consortium.
00:08:39
Speaker
Well, that's wonderful. Now, what have been some of the studies that you've done in the past?
Focus on Second-line Therapies and Quality of Life
00:08:45
Speaker
Could you explain those to us? One of our first studies that we did, ICON-1, was looking back at pediatric patients with ITP who had received second-line therapies.
00:09:00
Speaker
um And we were looking at what were the reasons that their physicians chose those particular therapies? How did they respond to those therapies? Because one of the difficulties that we have in treating ITP is that there are a number of different therapies we can choose, but they haven't necessarily been compared directly against one another. And often, i think For families, often it feels like it's a little bit just trial and error and people who are just you trying one thing and maybe it's going to work, maybe it's not, and we try the next one, which we don't necessarily have a crystal ball at the beginning to say this therapy will will work for this patient, right?
00:09:43
Speaker
Ultimately, that would be great if we can have some biologic markers that tell us at the onset this is a patient who's going to respond to this particular type of therapy. But right now we don't. And so ICON1 was to really understand decision-making amongst physicians and why they were choosing different therapies.
00:10:02
Speaker
We learned a number of different things from that study. And one of the things that came out was that the reason pediatric patients are getting started on second-line therapies and quality of life was a really important reason.
00:10:17
Speaker
And so I think that's important for people to keep in mind that, you know obviously, With ITP, one of the biggest things we worry about is bleeding, right? But that's not the only part of the disease. And when we're thinking about treating ITP, we also need to think about the patient's quality of life and how the treatments um can have an effect on that.
00:10:38
Speaker
Absolutely. And I'm so glad to hear you talk about that because years ago, that wasn't a consideration. it was strictly looking at the numbers, you know, what's the platelet count now? And if we try this, will it bring the platelet count up? But it's really a blend of how's the patient presenting themselves? How are they living their life? What do they need to do to make their life more robust? And keeping in mind what their platelet count is and how dangerous it is or how easy it is for them to continue.
Exploring Alternative Medicine for ITP
00:11:10
Speaker
I understand that there was a complementary and alternative medicine study that ICON did. Can you tell us a little bit about that? Yeah, so that was a project actually in collaboration with PDSA using the PDSA registry data.
00:11:29
Speaker
And the goal was to characterize the experience of patients using complementary alternative and integrative medicine in ITP management.
00:11:41
Speaker
That is a project that's still ongoing. i think the um group working on that is hoping to publish findings soon. it looks like a lot of patients, that's something that they're really interested in exploring. And it's an area that there's not a lot of data about. and So I think an area for future research as well.
00:12:02
Speaker
but this is kind of a really helpful first step in terms of just describing how are these therapies being used. And I think it's a needed study to look at because I know patients are always asking, well, have you found that this eating this or eating that or taking these or you know supplements, do they raise your platelet count? And I don't know, my experience we're still very, all of us are so very different and who knows what's going to help and what direction. So I think your study will be very interesting to read when it gets published.
00:12:36
Speaker
So it's nice to know that you worked with the PDSA in gathering this information. And that was from the PDSA's ITP natural history study, correct?
PDSA's Natural History Study Registry
00:12:52
Speaker
Yes. So the PDSA registry, I think, is a really great resource and great study. And I think it's you know really important to, again, help learn more about a rare disease and a way for patients to be involved and give their own data and experience of being treated for ITP.
00:13:15
Speaker
I think that's really great to know that um you've been able to get so much information from the PDSA's ITP Natural History Study Registry, because i I know I've stressed this in other podcasts. It's so important for ITP patients to go on that registry and give their information about their history with ITP, maybe how it started and came about. And for parents of children,
00:13:44
Speaker
um to do the same because that information is very valuable. As you can see, ICON used that information in their there' current study. So um if you haven't gone on the website, which is pdsa.org, please do so and go to the registry because that information is invaluable.
00:14:07
Speaker
And it is anonymous. So you don't have to worry about your your privacy. um Your name is not associated with any of the information that you leave. So that's something I strongly encourage everyone to do.
00:14:21
Speaker
Back to ICON, Dr. Shimano, you did say that you have an annual meeting. Does that happen at the American Society of Hematology Conference or is it another time?
00:14:33
Speaker
Yes, we have a meeting just prior to the start of the American Society of Hematology conference every year. Since any of our members are coming for that conference, we sort of tack on a meeting the day before.
00:14:47
Speaker
We've been really grateful for the support of PDSA to help sponsor that meeting and allow us all to get together in person and share the ongoing work of the consortium and work on new projects in person.
00:15:04
Speaker
Well, that's great. I'm happy to hear the PDSA helps you with that meeting because we're helping you and you're helping us for sure. So the least we can do is help with your meeting.
Joining ICON: How and Why?
00:15:14
Speaker
That sounds terrific.
00:15:15
Speaker
I know that a couple of your ICON members are members of our medical advisory board, which is wonderful. And I've had a few of them on the podcast in the past. And I know you personally have written an article for our e-news when you think it may not be ITP, which I'm sure every parent deals with.
00:15:38
Speaker
With a hit or miss that they go through with treatment, I'm sure they're wondering, is it not? um Question, if a healthcare provider is interested in joining ICON or getting more information, how would they go about doing that?
00:15:57
Speaker
Yeah, so we have a website, icon-itp.org. And on there, there's a section for healthcare providers with contact information if they're interested in learning more about the consortium or um interested in joining There's a memorandum of understanding that new sites sign to become part of ICON. And then, as I mentioned previously, everyone is required to participate in one of the committees.
00:16:28
Speaker
Okay. So there's requirements. It's not just a matter of joining. It's you you need to participate. And that participation pretty much makes sure that they're on the top of their game when it comes to treatment, right? Yeah, exactly.
00:16:42
Speaker
make sure that they're an active participant in helping with the ICON mission and furthering the research efforts. And it takes a lot of work to do ah to do these studies, and it's a you big team effort.
00:16:57
Speaker
That's great. Now, if you're a parent and wondering if there's an ICON physician that they could take their child to is there a way they could find that out?
00:17:10
Speaker
Yeah, the website also has information for patients as well. And it has a list of the different consortium sites. So if you look on there, there's a map and you can see, is there an icon site near you that you could consider going to your child's hematology care?
00:17:27
Speaker
Which is great. And again, your website, tell me again what your website is. It's icon-itp.org.
00:17:38
Speaker
Well, I think you might be getting some hits on your website after after this podcast and um hopefully more participation in the Natural History Study Registry and hopefully you know more information that the PDSA can give you and your organization so that more information is found out about ITP,
00:18:02
Speaker
the treatment, forward movement, et cetera. So thank you again for being with us today. I know you're a very busy physician hematologist, and we really appreciate you taking the time out to spend with us and enlighten us.
00:18:18
Speaker
I'm sure you've alleviated a lot of concerns with parents that think they're standing still, that research is standing still. It's nice to know that there's such a wonderful group of hematologists that collaborate like yourself.
00:18:32
Speaker
and sharing information and looking for answers. So thank you. Great. Thank you so much for having me. It's been a great partnership between ICON and PDSA as well. So I think there's a lot of crosstalk between the the two groups and we we very much appreciate the PDSA support.
00:18:52
Speaker
That's wonderful to hear. I can't speak for the whole organization, but I'm sure... Anything you need, they're happy to supply when it comes to information about their patients with ITP.
00:19:04
Speaker
Thank you again. Keep up the good work. Thank you.
Lifestyle Tips for Living with ITP
00:19:11
Speaker
How do you live your life with a bleeding disorder like ITP? From working in the kitchen with knives to navigating sharp corners in your house, going out to eat in a restaurant, traveling on a plane, attending a sporting event, even dancing at a wedding.
00:19:27
Speaker
ITP patient Barbara Pruitt shares her tips and tricks for moving through life with ITP for more than 60 years. Here's her lifestyle lesson for the day.
00:19:41
Speaker
My message for you today is enjoy your life. I know that's easy to say, but life teaches us lessons. And one thing I've learned is there are things in life you have control over, and there are things in life that you don't have control over.
00:20:00
Speaker
Things like the weather, traffic, even having an autoimmune disease, and the list goes on. But the things we do have control over, like our thoughts and our actions, those are the things that we need to concentrate on to bring enjoyment to our life.
00:20:22
Speaker
We don't get a do-over here. So every day that you have an opportunity to do something that makes you happy, take advantage of it. I know with ITP, we have limitations in what kind of actions we do, but that ends up being a part of your normal life.
00:20:43
Speaker
You know what you shouldn't do. You know what activities are dangerous for you. And depending on your platelet count and how you respond, Sometimes you can go out and ride a bike and enjoy it.
00:20:57
Speaker
And sometimes you're not able to do that. So instead, take a walk if that's something that makes you happy. Think of all the things out there that bring you joy or happiness.
00:21:10
Speaker
Maybe it's having lunch with a friend, taking a vacation, taking a walk in the woods. playing cards, going dancing, whatever it is don't regret not taking the opportunity to do those things.
00:21:28
Speaker
Because you have the control over doing that or not. You can make your life happy or you can make your life sad. So I choose to make it happy.
00:21:42
Speaker
That's it for now. Until next time, wishing you lots of happy, healthy platelets.
Podcast Closing Remarks
00:21:50
Speaker
Thanks for listening to the PDSA podcast, Bruised But Not Broken, Living with ITP.
00:21:56
Speaker
Made possible by our presenting sponsor, Amjet.
00:22:01
Speaker
Special thanks to Gus Majorga for composing our theme music.
00:22:07
Speaker
To see what's coming up, visit our website at pdsa.org and subscribe wherever you get your podcasts. Please share this podcast through social media with anyone who you think might benefit from the information and stories we share with the ITP and other platelet disorders community.
00:22:25
Speaker
As always, please speak with a healthcare professional before making any treatment decisions. But know that PDSA.org is a wealth of information and resources to help you navigate life with ITP and other platelet disorders.
00:22:40
Speaker
Thank
00:22:42
Speaker
Remember, you are not alone.