More Than a Diagnosis: Finding Your Voice With von Willebrand Disease

For full Prescribing Information for VONVENDI, please visit: [https://takeda.info/3RMfhbp]. A bleeding disorder is part of someone’s story — but it is not the whole story. 

In this episode of the Hope Podcast, Jonathan James talks with Takeda Community Education Specialists Morgan Cook and Maria Rohan about living with von Willebrand disease, the power of self-advocacy, and why sharing personal experiences can create connection within the bleeding disorders community.  The conversation explores finding your voice, communicating with your healthcare team, and learning more about treatment options, including VONVENDI® [von Willebrand factor (Recombinant)]. 

VONVENDI® [von Willebrand factor (Recombinant)] Important Information  

What is VONVENDI? 

VONVENDI is used in adults and children with von Willebrand disease to: 
   • treat and control bleeding episodes 
   • prevent excessive bleeding during and after surgery 
For adult patients only: 
   • reduce the number of bleeding episodes when used regularly (prophylaxis) 

Detailed Important Risk Information 

Who should not use VONVENDI? 
You should not use VONVENDI if you: 
   • Are allergic to any ingredients in VONVENDI. 
   • Are allergic to mice or hamsters. 

Tell your healthcare provider if you are pregnant or breastfeeding because VONVENDI may not be right for you. 

How should I use VONVENDI? Your first dose of VONVENDI for each bleeding episode may be administered with a recombinant factor VIII as instructed by your healthcare provider. Your healthcare provider will instruct you whether additional doses of VONVENDI with or without recombinant factor VIII are needed. 

What should I tell my healthcare provider before I use VONVENDI? 
You should tell your healthcare provider if you: 
   • Have or have had any medical problems. 
   • Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements or herbal remedies. 
   • Have any allergies, including allergies to mice or hamsters. 
   • Are breastfeeding. It is not known if VONVENDI passes into your                  milk and if it can harm your baby. 
   • Are pregnant or planning to become pregnant. It is not known if VONVENDI can harm your unborn baby. 
   • Have been told that you have inhibitors to von Willebrand factor                        (because VONVENDI may not work for you). 
   • Have been told that you have inhibitors to blood coagulation factor VIII. 

What else should I know about VONVENDI and von Willebrand Disease? 
Your body can form inhibitors to von Willebrand factor or factor VIII. An inhibitor is part of the body’s normal defense system. If you form inhibitors, it may stop VONVENDI or factor VIII from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to von Willebrand factor or factor VIII.

What are the possible side effects of VONVENDI? 
You can have an allergic reaction to VONVENDI. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea or fainting. 

Side effects that have been reported with VONVENDI include: headache, nausea, vomiting, tingling or burning at infusion site, chest discomfort, dizziness, hot flashes, itching, high blood pressure, muscle twitching, unusual taste, blood clots and increased heart rate. 

Tell your healthcare provider about any side effects that bother you or do not go away. 

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088. 

Please see VONVENDI full Prescribing Information [https://takeda.info/3RMfhbp]. 

Subscribe, share this episode with someone who needs to hear it, and join us for more conversations from the bleeding disorders community.

Hope Charities is a national nonprofit focused on helping people living with rare and chronic illnesses thrive. Our programs specialize in helping people with genetic bleeding disorders and #hemophilia navigate the challenges of invisible disease by providing emotional, educational, and tangible support. To learn more about our programs, visit our website www.hope-charities.org. 

Transcript

Welcome And Important Safety Info

SPEAKER_04 0:08

Welcome to the Hope Podcast. My name is Jonathan James, and I'll be your host today. I want to thank you so much for taking the time to listen to this podcast about more with people with bleeding disorders. I'm also excited to hear about Takeda's indication for Von Vindi, indications for type 1, type 2, and type 3 for adult prophy, and pediatrics on demand and surgery. This is going to be an exciting conversation today. And I have two community education specialists with me, Morgan and Maria from Takeda, and it's going to be a great episode.

SPEAKER_02 0:43

Von Vendi registered trademark, Von Villabrand Factor, Recombinant. Important information. What is Von Vendi? Von Vendi is used in adults and children with von Vilibrand disease to treat and control bleeding episodes, prevent excessive bleeding during and after surgery. For adult patients only, reduce the number of bleeding episodes when used regularly. Prophylaxis. Who should not use Von Vendi? You should not use Von Vendi if you are allergic to any ingredients in Von Vendi. Are allergic to mice or hamsters. Tell your healthcare provider if you are pregnant or breastfeeding because Von Vendi may not be right for you. Please see detailed important risk information at the end of this video. Talk to your healthcare provider to see if Von Vendi may be right for you. Please find Von Vendi full prescribing information in the description on this platform.

Meet Morgan And Maria

SPEAKER_04 1:30

Well, thank you both for joining us today. It is great to see you, and I'm really excited to dive into this conversation. Start out with both of you just introducing a little bit about yourselves. We'll start with you.

SPEAKER_01 1:40

Yeah. Well, I'm Morgan Cook, and I am a community education specialist for Takeda Pharmaceuticals. And I cover a large territory, including Mississippi, Tennessee, South Carolina, North Carolina, which is also where I live, and West Virginia and Virginia. And so that means I get to work with our local state chapters and specialty pharmacies to bring education direct to patients. I'm also a part of this community, which I'm proud to be a part of this community. I was diagnosed with von Willabrands when I was about 14, um, 97, 98, somewhere around there. Um, and then if not having my own bleeding disorder was enough, I also married into a family of leaders. Um and they have uh von Willebrands and Heme B as well. So bleeding disorders is a huge aspect of my personal and professional life. Yeah. My background is in education, um, which I think fits really great into this role of working with patients on lots of different educational uh resources and topics. Um, and so uh it's really an honor to be here and to be able to help advocate and uh figure out what patients need. And I truly believe that the more people we have who truly understand what it means to have a bleeding disorder and what that everyday looks like and feels like, um the more we're successful at, you know, working with patients and figuring out how they can live their best life. So it's really an honor to be here and to work in this industry and with Takeda and doing the things that I love for the people I love.

SPEAKER_04 3:13

That's so amazing. I fully agree that we need more people who have lived experience in this community to be able to share stories. I I you're surrounded by a 360-degree. You're a little excessive, let's be honest. But no, I'm gonna go. Yeah, I like to go big and go home. So I'm totally, you know, I'm with you there. Uh, but no, that's amazing. That's so great. And Maria, I know that you've been in this community for a long time too. So tell us a little bit just about your background and where you're from.

SPEAKER_00 3:40

So my name is Maria Rohan. I'm a community education specialist. So it's like Morgan. Um, my territories I serve are in the colder part of the nation and Michigan and Ohio. And I've been a nurse for 16 years. So I worked at the HDC in Cleveland for about four years, and I've been with Takeda for about two years. And I really got to see this evolution of a patient when given education. So a patient would get a diagnosis and they almost felt small or overshadowed by the diagnosis. But when given education, they became bigger than it. And so I learned quickly that educating needed to be the way in which I nursed. It was like taking power away from the disease and giving it to the patient. I have to tell you, Jonathan, the bleeding disorder community and the caregivers, they just like they mean a lot to me. Um when you see a patient hurt, when you're the one to collect their tears, you don't you don't forget it. It lives in your bones. And that consistently is what drives me. If we can give education to patients, they have this opportunity to become bigger than a diagnosis. And really live to this theme that I've got Von Willebrand's disease, but it doesn't have to have me.

SPEAKER_04 4:58

That's so good. Oh, yes. Somebody needs to meme that right now. Like that was awesome.

SPEAKER_00 5:04

Please don't.

SPEAKER_04 5:07

Wow, thank you both for sharing your stories. It's so amazing to hear your passion.

Why Self-Advocacy Matters

SPEAKER_04 5:12

Uh, and really in our discussion today about self-advocacy and the importance of other people sharing their stories. Morgan, as a person living with von Rilla Brands disease, um I'm excited to hear your perspective on the importance of self-advocacy and really the tangible ways that people can get involved in sharing their stories.

SPEAKER_01 5:27

Yeah, absolutely. This is something that I'm personally really passionate about, but it lends really to this community. And we are no strangers to advocating. This community has been advocating for decades and will continue to advocate. But I think that we also come across a lot of barriers and face defeat in this community. And that has a lot of implications on how we feel about advocating and when do we speak up and what that looks and feels like because we've been turned away so many times, or, you know, made to feel that our abnormal is okay. And so, you know, I think it's really important for us to be able to take a step back, figure out what works and what doesn't work for our own lives, but also to figure out how to use your voice and the power that can come from using your voice in really purposeful, meaningful ways. And, you know, that's not that's not an easy thing to figure out how to use. We all have a voice, but figuring out how to use it really successfully, you know, there's some barriers to that. And so once we find our voice and figure out what that looks and feels like, I think that it leads to more successful communication, communication around what our needs really are and what it looks like and feels like in our everyday lives. And no matter what bleeding disorder you have, we all bleed individually and we all have these individual needs. And it's not this one size fits all. And so really communicating what our individual needs are is really important. But then we can look at it from a larger perspective as well, you know, from a community base. So finding your voice and really figuring out how to tell those stories that are a part of that everyday journey from, you know, whether, you know, it was before a diagnosis up until where where you are in your life now. There's lots of stories that we can tell to really amplify and hit home the needs that we have. So finding your voice, figuring out how to tell that story and what that journey looks like is a lot of what we focus on in Takeda and getting people comfortable with sharing their voice and story. You know, that puts us in vulnerable states. And so finding, finding out how to do that in a really comfortable space for each person is really important. And then finally, we talk about ways in which we can do that. Um, you know, we've it's one thing to talk about, you know, finding your voice and writing stories down and practicing at your home, but then what do we do with it? And so we we need to have that call of action and figuring out what that looks like, uh, either individually or community-based, and and going out and finding those situations to share your story and share your voice. Um, all of those things lead to self-advocacy for yourself and for our greater community. And as a community, we work really well together. And what works for one uh really helps the larger group. And so the more we do that, I think the better off we are towards getting our needs met.

SPEAKER_04 8:37

That's so good. It really brings up a great question. I mean, how do you think, Morgan, that people can find ways to share their story in their journey?

unknown 8:47

Yeah.

SPEAKER_01 8:48

I think your voice is a really powerful tool and resource that we naturally have built in, but we also don't always know how to use that. So I think getting comfortable with yourself and and figuring out what that looks and feels like for you is really the first step. When we speak up, we are self-advocating. So let's let's put that out there. When we speak up, we are advocating. That's right. And so you're doing the tough part already. You're using your voice to say, this is what I need, this is what I want, this is how I'm feeling. Um, and then we can have actions that hopefully will correct that and help you along the way to make those changes and really to live that quality of life that you're looking for as an individual. And then maybe also someone that you're caring for with a bleeding disorder if you're a caregiver. Um, creating that story and and sharing that is so meaningful and impactful. And that's how we see change and how we get change.

SPEAKER_04 9:48

Which really brings up a great question. I mean, can you talk about some of the things that you think self-advocacy consists of for people?

Assertive Communication And Self-Worth

SPEAKER_01 9:55

Yeah. I struggled with this in the beginning for myself about how to appropriately um vocalize what my needs were because I downplayed a lot of them. We normalize abnormal in this community quite often. And so I really needed to take a step back and think about that. And that's something that we focus here on, Takeda is taking that step back and really evaluating our own self-worth. And I think that's so important. Our self-worth is who we are. And um, when we have faced defeat and have been dismissed, that self-worth dwindles. And so we need to remind ourselves the value that we have to ourselves, our lives, and this community. And once we can wrap our head around that and understand that we are more than just our bleeding disorder, as Maria said earlier, that self-worth begins to grow. And then we can talk and we can communicate and we can use our voice and we can say, you know, this is what I need to be successful. This is what I need to get to towards that quality of life that I'm truly seeking. Gone are the days of being placed in bubbles. And we don't need to live like that anymore. But I think a lot of us are in some ways. And so we need to figure out how to break out of that bubble. And that comes with understanding of our needs and being able to communicate that with our HCPs or in a work or school setting. But I also think there's a fine line when we do that. Um, because we have faced so much defeat, we tend to um be a little aggressive sometimes because we we go into something thinking, oh, this isn't gonna work and no one's gonna listen to me. And so we need to find that fine line between being passive and being aggressive when we're communicating our needs. You know, being passive, we're not gonna get anywhere. There's gonna be a standstill. And when we're overly aggressive, people tend to not listen to us. So we need to find that really fine line to be assertive and saying, these are my my needs. This is my life, this is what I'm looking for. How do I get there? You know, one of my favorite things that I do with my HCPs is I immediately talk about the things that I'm not gonna do. And I think that's because my fears were hindering me from speaking up. And fears can can do that for a lot of people. So I figured that when I go into my HCP, let's get those fears out of the way. Here are the things I'm not gonna do. Here are the things I'm not willing to do, here are the things that I'm not comfortable with. Once I can get all that out of the way and really knock those fears down, then that's a barrier that I'm jumping through. And then we can talk about what it is I'm I can do and what I'm willing to do. And what you're willing to do is super important because if we're not adherent to our, you know, treatment plans, then we're not helping anyone either. So I think it just leads to more success when we advocate, but then also more success towards that quality of life that we're looking for.

SPEAKER_04 13:05

That's so good. Oh, that's so good. I, you know, I still think about when we're in that clinic setting and we're trying to navigate all these things, you know, there's so many things that go through your mind and your head, and it's hard to get clarity of thought sometimes in that. But it makes me think like, what are some strategies maybe for patients to think about when advocating for themselves, uh, or even someone that that they know that's living with a bleeding disorder, like a caregiver. What are some strategies that you think could be helpful? Yeah.

SPEAKER_01 13:34

I think um one of the first things we need to do is take a step back and evaluate our knowledge around our bleeding disorders. When I joined Takeda, I thought that I knew quite a bit about my bleeding disorder and von Willebrands and what that really meant. And I was wrong. There are a lot of things that I had um not wrapped my head around successfully, and there were pieces that I was missing. So I really needed to add to my knowledge base. And so being a lifelong learner around our bleeding disorder is essential. Yeah. And that learning doesn't stop ever. New things come out all the time. I learn new things from my colleagues and I learn new things from patients all of the time. Yeah. And so continuing that education and really understanding what your bleeding disorder means, but more importantly, what it means to you. Because in this community, we know that individuality is just as important as, you know, having a group of von Willebrand's patients. We all bleed differently, we all have different needs. Yeah. And so the more we understand our bleeding disorder and more, and the more we understand our own bleeding disorder and our phenotype, I think it just sets us up for greater success. Yeah. And then we can talk about those needs with our HCPs because we just have a really strong foundation of education and learning. Um, and it just makes it easier to have those conversations and truly verbalize what those needs are and what they mean, and then how we can create actionable items to achieve them together. And I think it's important to remember that your HCP should be a teamwork. It's not just going in and them making decisions for you. It should be a conversation of, you know, you expressing those wants and needs and then talking with your HCP about what that looks like and finding those tangible ways to achieve it and creating those personal goals. I think that just sets us up for that success further on and leads to successful advocating personally for a loved one or the greater community.

SPEAKER_04 15:37

Yeah.

SPEAKER_01 15:37

Um, and so I I think that's a great way to kind of wrap your head around what that looks and feels like.

SPEAKER_04 15:43

That's so good. Yeah, if you've met one patient, you've met one patient. You've met one patient.

SPEAKER_01 15:46

Right. Yeah. I think we all need t-shirts with them.

SPEAKER_04 15:50

Yes. I love that. I love that. You know, throughout the patient journey, there's defining moments. It happens for all of us, right? But I just wonder like, how would you encourage people to incorporate those defining moments really into their story as they're walking it out?

Defining Moments That Shape Your Story

SPEAKER_01 16:06

I'd love a good defining moment. I really do. I think defining moments shape us more than we realize. I also think that we need to remember that our bleeding disorders do not own us, that we are the owners of it. You know, I've said that before, and Maria talks about that all the time. And we talk about that across all of our CES teams, that we are the owners of our bleeding disorder. And so when we think about defining moments, we're looking for moments that have shaped us in one way or another. And that can be a really positive event or it can be a negative event. And both of those have lasting effects. And I think when we talk about those really negative moments, they have such an impact that we can flip the script a little bit and move it from something really negative to something that we've learned a lot from so that we can make changes the next time we come around or the next time we're in that situation. So I took the time to write my story out on a piece of paper and what a long story that was. And I realized that I missed, yeah. I realized that I missed pieces and I and I downplayed a lot of things. So I, you know, I had to go back and add things. And I think when patients like write it out and and and uh you know, draw that picture of what their journey has looked like, there are so many defining moments that have shaped us. But those defining moments are also stories. And those stories are ways that we can um get a point across and give uh, you know, some detail and picture to what that looks like. And so I really encourage everyone to map it out. You know, think about your bleeding disorder from the very beginning until where you are now, but then also where you want to go that long term. Um so I really like looking at it as different chapters of our lives and finding defining moments for different situations to verbalize my needs. Um, and I think that's something that a lot of patients can really focus on for those different defining moments.

SPEAKER_04 18:10

That's so good. Well, what do you think are some ways or platforms where people can share their stories?

Where To Share Your Story

SPEAKER_01 18:16

Well, this is a perfect one right here. I mean, you're doing great. You all have um naturally set up a space that lends itself to feeling comfortable. And once you're comfortable, it's an easy way to kind of bridge that gap of your fears and how to use your voice and what that looks and feels like. But our other national organizations have also created a lot of those platforms where um you uh have a space to be able to talk about those things. Then you have your local state chapters and what wonderful organizations they are to meet new people and to share stories and get out there and meet new people. Social media, talking with your HCP, or maybe it's more um close to home in a school setting or a work setting, or you know, those transitional times of going from one place to another, you're moving. So there's lots of different places that we have naturally built into this community that um are lots of great ways to begin. But I I think that people should start with what they're comfortable with. Start a little bit closer to home and then build that confidence up. And then sky's the limit, really. Yeah. So there's lots of great things, whether it's, you know, legislature with our, you know, state organizations or uh from a national level in DC. You know, there's lots of built-in places that we can advocate naturally.

SPEAKER_04 19:41

You know, I found in my own personal journey that I really needed both, like you said. Like I needed that chapter in engagement. I needed the legislature where they didn't know anything. But I also needed to sometimes tell my neighbor. Like, yeah. And that was a hard hurdle for me. Like just living with my own story was like, but sometimes telling somebody I had no clue what a bleeding disorder was actually helped me get a language that could help really build a more robust way to be able to share my story. You know, so I think I encourage people to do both. Like, you know, like you said, it's just such a great, great way to do that.

SPEAKER_01 20:15

Well, and even your own personal family. You know, when you sit down and tell that story and and you tell those nitty-gritty details, I think your family can also be kind of surprised of, I didn't know that's how you were feeling, or I didn't know that's what that was like.

SPEAKER_04 20:29

That's right.

SPEAKER_01 20:30

And um, you know, it it builds up that um support system when we have a a true understanding.

SPEAKER_04 20:37

There's always more that's going on behind the scenes than what anybody realizes why we need empathy. So good. Well, I mean, this I I could talk about this all day. I know you can too. There's so much more to unpack here, but I I guess just as far as sharing stories and the power of self-advocacy, you have any final thoughts, just encourage people in?

SPEAKER_01 20:56

Yeah. I encourage everyone to pause, stop, and reflect and keep on learning. Reflect on what has helped you grow. Reflect on the areas in which you want to grow, those barriers that have just caused defeat and setbacks, and really reflect on where you want to go and make actionable ways to get there. Talk to your HCP, talk to your chapter, talk to your friends in the community, and continue to grow and reflect and think about ways you can share your story because it's impactful. And you never know what your story can do for someone else. I've learned that firsthand where I fought I thought that I was alone and I felt alone and I realized that I wasn't because someone shared their story and I was like, that's me. That's exactly how I feel. But it took somebody else verbalizing it to know that my feelings were legit and um they're important and they're impactful. So share your story, find ways to find. Find comfort in that um and continue to, you know, find action within that.

SPEAKER_04 22:05

That's so good. That's so good. Well, Morgan, thank you so much for sharing your journey, but also just your passion for our community. I mean, I know it's like, you know, when you're family, you can't really get away from it, but it's at the same time, like I don't want to get away from it. I agree. I'm fully with it. And I have found, you know, I always say the the best thing about being diagnosed with a bleeding disorder is the family that comes along with the community that comes along with it. And and that's so true. And uh I'm glad to to know more about your journey and more about your passion for this community because it's very evident. Yeah, absolutely.

Von Willebrand Disease Basics

SPEAKER_04 22:36

Maria, now I want to talk a little bit about uh Von Vindi and some exciting approval information that's coming out from FDA. But uh but first before we do that, for people who might not be familiar, uh, can you break down a little bit about what von Willebrand's disease is?

SPEAKER_00 22:50

So Von Willebrand's disease can be inherited or a spontaneous mutation, and it's a bleeding disorder where you can't form a blood clot. Blood clot's main job is to stop bleeding. So someone who can't form a blood clot can continue to bleed. And that can lead to prolonged bleeding in different areas of the body.

SPEAKER_04 23:08

Wow. So do we know it causes von Willebrand's in patients?

SPEAKER_00 23:12

So von Willebrand's disease can be caused by two reasons. One is you don't make enough von Willebrand's factor, so you can't form that clot. The second reason is you do make enough von Willebrand's factor, but it doesn't work properly, so you can't form that clot. And this can lead to prolonged bleeding, like I said, in different areas of the body, from bruising to nosebleeds to heavy menstrual bleeding. In fact, nosebleeds and heavy menstrual bleeding were first identified by Eric von Willebrand in 1926 when he I when he noted von Willebrand's disease.

SPEAKER_04 23:45

Right.

SPEAKER_00 23:46

Right. And so um it could range from joint bleeds to GI bleeds, just to name a few. But it's really important for people to know that everyone's bleeding profile is different. And no matter what that profile is, that bleeding matters.

SPEAKER_04 24:02

So true. I want to get into Von Vendi now and talk a little bit about that. But what can you tell me like what makes Von Vendi unique?

How Vonvendi Works And Key Terms

SPEAKER_00 24:10

Yeah. So there are two reasons that make Von Vendi unique. First is that Von Vendi is the first and the only recombinant Von Willebrands factor on the market. So what does recombinant mean? Recombinant means it's manufactured without human or blood plasma. So there is virtually no risk of being exposed to disease carriers that can be shared through blood. Another reason it's unique is something called a half-life. So, what exactly does half-life mean? Half-life describes how long it takes for the body to eliminate half the dose of product. So, let me give you a little bit of an example. Let's say the half-life of a product is 19 hours. So, 19 hours after dosing, there's about 50% of the dose in your body still. So let's talk about Von Vendi. The half-life for Von Vendi for adults is 22.6 hours, and the half-life for pediatrics is 14 hours.

SPEAKER_04 25:07

Wow.

SPEAKER_00 25:07

Yeah.

SPEAKER_04 25:08

That's amazing.

SPEAKER_00 25:09

Yeah, it's exciting.

SPEAKER_04 25:10

So, how exactly does Von Vendi work?

SPEAKER_00 25:12

So the first way it works is attending to the source of the problem. Von Vendi replaces that Von Willebrandt's factor that is defective or that is missing without adding additional factor eight. Someone who has Von Willebrand's disease has a lack of Von Willebrandt's factor or a von Willebrand's factor that's not working. So Von Vendi replaces what you do not have so your body can continue to make a clot naturally. In order to understand Von Bendi a little bit more, we really have to take a deeper look at that von Willebrand's factor and how it works. So Von Willebrand's factor is this complex multifunctional protein. The protein is distributed in the body in different sizes. And that specific protein has many jobs. So it acts like tape. It sticks to the broken blood vessel called collagen. It sticks to platelets, it sticks to factor eight, it carries factor eight, it stabilizes factor eight and protects it from degradation. So all of those things, all of those jobs are done just to stop bleeding. That stickiness of that von Willebrands factor, it depends on the size of the multimer. The sizes of multimers range from low molecular weight multimer to ultra-large multimer. The larger the multimer, the stickier they are. Von Vendi alone enables sustained rise in factor eight levels, which have been observed in our clinical trials. This allows healthcare professionals to create a treatment plan with Von Vendi that would work for the individual patient. And then also determine if extra recombinant factor eight is necessary, really based on the individual's specific situation. Because Von Vendi is made using recombinant technology, that recombinant technology, so it's made in a lab, Von Willebrand's factor is not cut by the enzyme Adams TS-13. So what does that mean? That means all the different sizes of Von Willebrand's factor, including those ultra-large multimers, remain whole when you get your infusion. So once Von Bendi is in your body, your own Adams TS-13 begins to cut von Willebrandt's factor. And that's similar to what happens with natural von Willebrandt's factor. That role of Adams TS-13 and VWD is really complex. So I urge patients to continue the conversation. Continue that conversation with your hematologist, contact your CES, and any additional questions that are needed or wanted about Von Bendi.

SPEAKER_04 27:46

Wow. How are patients able to manage bleeds with Von Vendi?

SPEAKER_00 27:51

So there's a couple different ways patients can utilize Von Bendi for themselves. The first way is prophylactically, which means on a routine basis to prevent bleeding episodes. The second use is for on-demand. When patients taught take Von Vendi as needed for bleeding episodes, and finally, patients can utilize Von Vendi before surgery, during surgery, and after surgery if needed.

SPEAKER_02 28:14

Select important risk information. You can have an allergic reaction to Von Vendi. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or fainting. Please keep watching for additional important safety information.

SPEAKER_04 28:43

Wow. Thank you so much for helping us understand really the role of Von Willebrand's factor and also Von Vendi as a unique treatment

New FDA Approvals And What They Mean

SPEAKER_04 28:51

option. Let's talk about the latest indications that are improved approved by the FDA.

SPEAKER_00 28:56

Let's talk about it. I've been waiting for you to ask this question this whole time. I'm itching. So Takeda's been focused on supporting the Von Willebrands community for the past 10 years. In 2022, Von Vendi was approved for prophylactic use in severe type 3 adult patients who were previously treated on demand. And now we're here. And I get to sit across from you, and I get to be the one that is thrilled to share that Von Vendi has uh has received FDA approval for prophylaxis use in all adults of all types, as well as on demand and surgical usage in both pediatric and adult Von Willebrand's population.

SPEAKER_04 29:36

Wow. Oh my gosh. That's amazing. I know, really.

SPEAKER_00 29:41

It is, it is thrilling.

SPEAKER_04 29:43

Wow. That's truly amazing. It's so exciting. Can you talk a little bit about what it means to be able to expand the uses of Von Vendi?

SPEAKER_00 29:53

I will never say this enough. Every patient matters, every type matters, every bleed matters. And we are just overjoyed that all adults of any type now have this opportunity to trial Von Vendi if appropriate appropriate. We also have this ability to share Von Vendi with our pediatric population. That joy is echoed, especially with the CES team. Because we have this ability to educate the patients, educate the caregivers about the usage of Von Vendi for on-demand and surgical usage for their children.

SPEAKER_04 30:27

Wow. That's so amazing. I have so many thoughts going through my head. Yeah, I want to ask, but I I know, I know it's important for us to just really just be thankful for this moment and I and really excited about the development that's that's unfolding for this community.

SPEAKER_00 30:45

Yeah.

SPEAKER_04 30:45

There's really truly been underserved for a long time.

SPEAKER_00 30:48

Yeah. Oh, yeah.

SPEAKER_04 30:49

You have any final thoughts really about how to wrap all of that

Freedom Of Choice And Closing Thanks

SPEAKER_04 30:53

together?

SPEAKER_00 30:53

So I have a lot of thoughts, and you only want to find. So here's my two cents, right? Um nothing blossoms in the dark. And patients have this ability to break through their own glass ceiling and let the light in. And have the opportunity to grow and embrace their life in full cover color. We've got this freedom of choice program where patients can trial Von Vendi for Profi, for on demand, for surgical use to see if Von Vendi is right for them. And if anyone wants that information about that freedom of choice, if you want to learn more about Von Bendy, if you want to just connect with your CES, please embrace that connection and you can visit us at VonVendi.com.

SPEAKER_04 31:38

That's amazing. Wow. Well, I can't thank you both so much for being here on the Hope podcast today and sharing your story that's been so impactful and the passion that you have for our community. It means so much to me, and I know it means so much to everyone listening. And uh, I'm also so grateful, Maria, for all that you've shared with us about Von Vini. I am gonna be a nerd and go read all about this now because I'm so excited.

SPEAKER_00 32:03

We will never stop being a nerd.

SPEAKER_04 32:05

I'm so glad. And your passion for the development of what's happening and unpacking all of this for our community is just amazing and it's contagious. So thank you both so much for joining us here today. And uh, we look forward to hearing even more great things for our community. So thank you.

SPEAKER_01 32:19

Thank you, Cinq, for having us. It's uh it's a really great honor to be here and be able to talk about Von Mendy and advocating. And so we thank you for your time and the platform to do this.

SPEAKER_00 32:29

You know, I've been wanting to like shout it from the roof rooftops ever since we've got the approval. And this is my rooftop. Hashtag rooftop.

SPEAKER_04 32:40

What is my daughter's deal?

SPEAKER_00 32:41

It's like hashtag I'm not that cool yet, but that's awesome.

SPEAKER_03 32:48

Well, thank you so much.

SPEAKER_00 32:50

Yeah, thanks for having us.

SPEAKER_01 32:50

Absolutely. Thank you for having

Full Patient Safety Information

SPEAKER_01 32:52

us.

SPEAKER_03 32:52

Von Vendi, patient, important information. Von Vendi, von Willebrand factor, recombinant, important information.

SPEAKER_02 33:04

What is von Vendi? Von Vendi is used in adults and children with von Willebrand disease to treat and control bleeding episodes, prevent excessive bleeding during and after surgery. For adult patients only, reduce the number of bleeding episodes when used regularly, prophylaxis. Detailed important risk information. Who should not use von Vendi? You should not use von Vendi if you are allergic to any ingredients in Von Vendi. Are allergic to mice or hamsters. Tell your healthcare provider if you are pregnant or breastfeeding, because von Vendi may not be right for you. How should I use von Vendi? Your first dose of von Vendi for each bleeding episode may be administered with a recombinant factor eight, as instructed by your healthcare provider. Your healthcare provider will instruct you whether additional doses of von Vendi with or without recombinant factor 8 are needed. What should I tell my healthcare provider before I use von Vendi? You should tell your healthcare provider if you have or have had any medical problems. Take any medicines, including prescription and non-prescription medicines, such as over-the-counter medicines, supplements, or herbal remedies. Have any allergies, including allergies to mice or hamsters, are breastfeeding. It is not known if von Vendi passes into your milk and if it can harm your baby. Are you pregnant or planning to become pregnant? It is not known if von Vendi can harm your unborn baby. Have been told that you have inhibitors to von Willebrand factor, because von Vendi may not work for you. Have been told that you have inhibitors to blood coagulation factor 8. What else should I know about von Vendi and Von Willebrand disease? Your body can form inhibitors to Von Willebrand factor or factor VIII. An inhibitor is part of the body's normal defense system. If you form inhibitors, they may stop von Vendi or Factor VIII from working properly. Consult with your healthcare provider to make sure you are carefully monitored with blood tests for the development of inhibitors to von Willebrand factor or factor VIII. What are the possible side effects of von Vendi? You can have an allergic reaction to Von Vendi. Call your healthcare provider right away and stop treatment if you get a rash or hives, itching, tightness of the throat, chest pain or tightness, difficulty breathing, lightheadedness, dizziness, nausea, or fainting. Side effects that have been reported with von Vendi include headache, nausea, vomiting, tingling or burning at infusion site, chest discomfort, dizziness, hot flashes, itching, high blood pressure, muscle twitching, unusual taste, blood clots, and increased heart rate. Tell your healthcare provider about any side effects that bother you or do not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov slash medwatch or call 1-800-FDA 1088. Please keep watching for detailed important risk information and find von Vendi full prescribing information in the description on this platform. US-VON-1038V1.0, April 26th.