The New Era of Hemophilia Treatment

We explore how new non‑factor options and high‑sustained factor products are changing hemophilia care and why shared decision making leads to better, more flexible lives. We connect treatment choice to joint health, mental health, aging, and everyday habits that protect long‑term outcomes.

• Non‑factor prophylaxis versus factor replacement and what each does best
• Treating beyond dated trough targets toward durable thrombin generation
• Matching therapy to activity level, joint status, and venous access
• Why shared decision making beats one‑size‑fits‑all regimens
• Practical plans for breakthrough bleeds and procedures
• Aging, comorbidities, and proactive screening for liver cancer and heart disease
• Weight, movement, physical therapy, and joint load reduction
• Vitamin D, bone health, and osteoporosis risk
• Chronic pain, anxiety, and skills to break rumination with breathwork
• Caregiver choices for kids and the importance of evolving plans

Please take some time to look at the link below. We have educational videos that are going to explain all these non‑factor prophylactic options like Alhemo, Qfitlia, Hympavzi, so you can walk into your next appointment prepared. Sponsored by CME Outfitters through an educational grant from Novo Nordisk

Patient education webisodes:  https://bit.ly/HemoOptions

Quick Survey: https://www.surveymonkey.com/r/ZK7693T

Complete the survey to have a chance at winning 1 of 5 $100 gift cards.

Thanks for tuning into the HemoLife Podcast. Be sure to subscribe, leave a review, and share this episode with someone who needs a spark of hope or encouragement. Follow us on Instagram @HemoLife_Podcast and YouTube for updates, guest highlights, and behind-the-scenes content. New episodes drop regularly—your story matters, and this is just the beginning.

Transcript

Welcome And Guest Introduction

SPEAKER_00 0:00

Welcome to the Hemolight Podcast, your gateway to transformation and empowerment. Hosted by LA Guayo, we're diving deep into the world of rare disorders, unlocking the full spectrum of your potential. Each episode, join us as we connect with pioneers, wellness experts, and survivors. They're here to share powerful stories and invaluable insights. From mental resilience to physical health, community news to life-altering strategies. At HemoLife, we provide the tools you need to excel and inspire. Prepare to elevate your life, learn, laugh, and grow with us. Let's embark on this journey together.

Why New Non‑Factor Options Matter

SPEAKER_02 0:54

Hey, how's it going, HemoLife family? And welcome back to the HemoLife Podcast. I'm your host, LA Guaya, and here with my co-host physician and fellow hemophiliac, Dr. Joe Moleschi. Today we're joined by hematology nurse practitioner Maya Bloomberg. She has over 14.6,000 followers on Instagram and over a thousand posts giving extremely valuable information to our community, including hematology, mental health, stress, and pain management. She works hands-on with the hemophilia patients navigating one of the biggest treatment shifts we've ever seen in years.

SPEAKER_03 1:22

Yeah, and in over a year, we have now multiple, actually three non-factor prophylactic options. They've become available to patients that are 12 and older. And the meds are alhemo, cuphitlia, and hempavsy. And welcome, Maya. And just this expanded treatment options for hemophiliax is incredible. What's your thoughts on this?

SPEAKER_01 1:44

It's really exciting. I've been in my current role for over a decade. And when I first started, Loctate was the first extended half-life that had come to market. And just over the past decade, seeing this landscape become very complex, we're having kind of a shift of focus from factor replacement to more thrombin generation, which I'm sure we'll dive in deeper. But I love it because it's allowing us to start taking a more patient-centric, scheduled treatment approach for our patients because it's not a one-size-fits-all with any treatment. I think it allows us to really give the patient the power back and really start thinking of what are your goals, what are your aspirations, what do you want out of your treatment options? And as providers, we now have choices to kind of find the best treatment option based on that.

From Troughs To Thrombin Generation

SPEAKER_02 2:25

So this episode is sponsored by CME Outfitters through an educational grant from Novo Nordisk. And we'll be linking educational videos at the end so that you can dive deeper before your next appointment. So, Maya, let's start with the big picture. Three non-factor prophylaxis options, alhemo, cuphilia, and hempopsy, all becoming available within just over a year. Why is this such a significant moment for the hemophilia community?

SPEAKER_01 2:47

Well, I think we've come a long way when it comes to hemophilia management. When we think of treatments when we first started with just factor replacement, the initial studies were really just trying to see what we can do to help save lives and prevent those life-threatening bleeding episodes. And at the time of the factor studies, that's kind of where we got our 1 to 3% trough level or what your lowest factor level is going to be before each dosage. But when you think about other treatments, when you think about hypertension or you think about diabetes, we're always treating to normalization, to normal levels. So when it comes to chemophilia, treating a 1 to 3% trough level is very dated, especially with all of these advancements in technology and treatment options. So I think we're kind of pushing the boundaries a little bit with these new advancements and these new options that we have available for patients where we're able to get products that are offering really good bleed protection, but also that are more convenient and fitting within the lifestyle for our patients a little bit better.

SPEAKER_03 3:42

Yeah, as someone who grew up with limited options, I'm 38 years old, and really to see these incredible choices now available in the market, how do patients usually react when you're sitting down with them face to face and you talk about we have new therapies?

Patient Reactions And Adoption

Prophylaxis Limits And Breakthrough Care

SPEAKER_01 3:58

I think it varies. We have a huge spectrum of patients from early adopters versus late adopters. I have some patients who just want to stay in the status quo and continue on factor therapy. And I find this very common with my older population, especially those who survived the bad blood era and were literally living through a time where you had to prioritize do I treat a bleed and run the risk of contracting hepatitis or HIV, or do I treat the bleed? So I think it depends on the patient specifically and how open they are. I think it also depends on how well controlled they are. So if I have a patient who is presenting with a lot of bleeding episodes, I like to present what the options are available. So a lot of our hemophilia patients, I see them once a year and I'm very proactive with my dialogue. So I like to be the first one to educate my patients on products on the market before anybody has any potential bias to skew on them. And I kind of give a high-level overview. And I think it's important for patients to determine what are your priorities because my first question is and first information that I really dive into is how we have factor and non-factor therapies. It's important to realize that with any of these non-factor therapies, we mentioned three of them, but MSisimab was the first novel treatment, a factor eight mimetic that was approved back in uh 2017 for our inhibitor patients with hemophilia A and expanded to all patients with and without inhibitors with hemophilia A in 2018. So we have these four novel treatments, and it's important to know that they're only for prophylaxis or only for preventing bleeding episodes. So that means all of these convenient subcutaneous options require a second product for breakthrough bleeds or for surgery, things of that nature. The goal is that your prophylactic regimen is going to work for you and you're not going to have breakthrough bleeds. And we are seeing really great results in real world settings. But I have some patients, they just want an all-in-one product. They want one product for prophylaxis to treat their bleeds for surgery management. And if I have a patient in front of me, what's the point in wasting my energy and trying to, I don't want to say convinced, but educated, spend a lot of time on these novel treatments because there are a lot of additional considerations and education that goes into transitioning patients to these treatments compared to just factor replacement, which has been the standard of care for decades and decades, where we're literally just replacing that missing clotting protein, which then helps uh ensure hemostasis or prevent the bleeding complications.

SPEAKER_02 6:10

Wow, that's amazing. So one of the biggest takeaways is that people can now choose therapies based on their lifestyle. So how do you like help a patient navigate that?

Matching Therapy To Lifestyle

SPEAKER_01 6:19

Sure. So first I want to know what is their joint status. Because we know if somebody has hemophilic authoropathy, they're already at increased risk for developing additional bleeds to that target joint. So first, what is their joint status? Then what is their actual activity level? I'm predominantly an adult provider. I cover my pediatric side when they're on maternity leave, but ultimately I'm an adult provider. So my patients aren't playing school sports and then practices multiple days a week. So it's a little bit different in that scenario. So I want to know what is your activity level, what are the practices like, what are the games like, what type of uh insult and injury is the potential for your body, and from there kind of determine what the best option is. We are, there are a lot of uh clinical trials underway because I think with a lot of these novel treatments, we say MSismab can offer uh an equivalence of having a factor at around 20. Nobody will put it in writing, but we have all of these novel treatments that kind of keep a sustained level of protection in your system. But as providers, we want to know, well, what does that mean for our active patients? And the good news is there's a lot of studies underway that are specifically looking at that bucket of patients. We have studies on mild and moderates on these novel treatments, because that's another bucket of patients. Because my hypothesis and I truly believe that in 10 years from now, standard of care, it's not just going to be our severe patients with hemophilia on prophylaxis. We're gonna have mild and moderates because when we're going more towards the direction of health equity, and again, thinking of the example of hypertension and diabetes treating to normalization, I want my mild and moderates to live as normal of a life and not be limited by their hemophilia. So the expanding landscape, it's really exciting, but the conversations we're having with patients are so much more complex because I need to take into account their joint status, what's their bleeding phenotype, what's their activity level, what's their access? Do they have issues with finding pains to do IV access or are we limited and we don't even have an option really to do factor replacement and need to explore these novel treatments? And once we kind of break through there, then we kind of look and see what is the best option because I do have patients who are very active. They play soccer competitively and they're on novel treatments doing very well. I have other patients who play competitively and they prefer factor because with factor, we have a predictable rise and drop. We have our newest, I call it a super extended half-life product, or they're they're creating another class called a high sustained factor known as altuvia or ethanos octagog, if we want to do the medical term for it. And that has near normal factor levels for the first four days and the trough level by day seven above 15%. So for a patient who wants an all-in-one product, that's something for an active patient where we can have high sustained factor levels and give them the protection. Let's say they have venous access and they can never find a vein, then that's not going to be the most appropriate option. And we then have novel treatments which are effective and we have more studies underway because I think at the end of the day, we have our clinical trials, which are kind of the best case scenario, the not necessarily showing what happens in the real world. So I appreciate these studies and I appreciate the real world evidence so we can give our patients more of that information because there's no way that I can tell a patient, you're going to be on this product, you're not going to have any bleeds. We have to be realistic. I always like to set realistic expectations with our patients. So I am a big utilizer of shared decision making. And that's because we don't have any evidence that says this is the best treatment option. So for me, my role as a provider is to present you with all of the available options, the risks, the benefits, how this works in your lifestyle, and then come to a decision together. And sometimes there's no decision and we decide let's continue on the status quo and we can circle back later on. But shared decision making, in my opinion, is best practice and something that would be revisited at every clinic visit just to make sure that we are optimizing our patients since there are so many treatment options and no one best treatment option out there currently.

Active Patients And High‑Sustained Factor

SPEAKER_03 10:01

Yeah, I mean, I love uh all the tools that we have in uh the tool belt here for hemophilia, severe, mild, moderate. It's beautiful. I mean, for a lot of people listening, the lifestyle and you know, your body as a hemophiliac can change like different target joints as uh an individual grows. Can you talk about why is it important to like revisit these treatment options as a patient ages? You kind of listened to you know, activity levels, uh, you know, whether they can find a vein or not, depending on you know what age the patient is at. I mean, it's it's it's just an incredible thing we have now in in modern medicine that we can actually fit a patient's life into a sub Q shot, a factor shot, an extended half-life. But just go into that a little bit. I mean, you kind of already did, but it's just incredible of what the options we have nowadays.

SPEAKER_01 10:51

And I think where it differs, especially for our aging population, is one thing I always like to emphasize with our patients, because I think a lot of times we're very narrow-sighted. We're just focused on right now, we're not necessarily thinking about the future. But I always make sure my patients with hemophilia know that you have the same life expectancy of the general population. So, because of that, we're gonna start to see age-related risk factors of aging. So when it comes to cardiovascular disease, heart disease, things of that nature. We're also starting to see a bucket of patients who develop cancer independent of hemophilia. There's other risk factors for cancer, but say you have somebody who contracted hepatitis C during the bad blood era, we know that that could potentially convert to liver cancer, even if the hepatitis C was eradicated from the system. So that's why it's really important. Make sure if you have history of hepatitis C, every year you should get an AFP, an alpha-fetoprotein lab marker check. This is a tumor marker for liver cancer. So for my older adults who are cleared, I check this annually. So that way if I see the level uptrending, I can be more proactive doing a CT scan and see if there's any concern of the liver.

SPEAKER_02 11:52

I've never been checked again since I had mine. You know, I I contracted hepatitis C and then went through the treatment plan and then they said it was gone, but I never really went back to have it.

Shared Decision Making In Practice

SPEAKER_01 12:00

As an adult, so I don't want you to think that as in pediatrics they were missing out. It's more when you're 40, 50 and you're older and have the other risk factors, especially if you're an alcohol drinker, taking a lot of Tylenol or have other things that could potentially impact your liver. I definitely checked this. And this was honestly because I had a hemophilia patient who was complaining of really vague symptoms, reflux. He underwent an endoscopy, had imaging. They found um hemangioma, which essentially is like a abnormal blood vessel collection in his liver. And then it ended up being liver cancer and he passed away. So ever this was years ago, but that story just stuck with me. And since then, I'm very proactive because I was like, well, you know what? Maybe if we were checking this, we would have caught it. And so that's just a personal preference. But since we have this opportunity, I'm all about empowering and putting the patient back in the driver's seat. But the point of me bringing up the cancer is that a lot of cancer treatments increase risks of bleeding. So now all of a sudden, I have patients with mild hemophilia who've never received any treatment before. We know patients with mild, you really only need factor if you're having trauma or surgery. So I do have some mild patients who we know their diagnosis based on family history, but they've never once had to take factor replacement at all. Fast forward, they get diagnosed with cancer, they're starting on chemotherapy that increases their bleeding risk, and we're now starting them on factor prophylaxis in order to prevent bleeding risks with cancer. We're also seeing patients with heart disease where when you have certain, if you have atrial fibrillation, which is an abnormal heart condition, you need to be on anticoagulation, otherwise, you're at increased risk of stroke. There's been long debate. Does hemophilia protect you against stroke and blood clots, things of that nature? So, yes, it might be offer some protection, but it doesn't protect you from the age-related risk factors and the black buildup and atherosclerosis that can contribute to stroke and things of that nature. So it's a very interesting time. The aging population I find so interesting, but we don't have guidelines on how to manage the aging population because it's kind of a newer population that we're diving into. So it's different considerations because one, comorbidities or what other health issues are we dealing with. And does that matter? And does that impact what type of treatment options we need, especially if they're on dual antiplatelet therapy or on cancer treatments that could increase their bleeding risk? But then target joint health. We know that hemophilic authoropathy is the number one cause of chronic pain in patients. We can have patients on prophylaxis and you have zero ABR and no annualized bleed rate for the year, but you still have chronic pain. So that kind of removes the target joint definition, which depending on which definition you use, having more than three spontaneous bleeds in that joint is a definition of a target joint. But say you don't have a target joint because you're on a treatment that works really well for you and you haven't had any bleeds, breakthrough bleeds in two years, but you're still having chronic pain, you're dealing with hemophilic authopathy. So sometimes trying to find treatments that kind of capture the box. And this is where it's the patient true. I've said this probably five times now, but the patient is in the driver's seat because I'm not the one who's putting medication in your body. I have some patients who they have such a placebo effect of just the act of infusing, they automatically feel better. Which when you infuse if you have a bleed and you infuse, you're not having improvement in your bleed in that hour. It takes time for it. So if I have a patient who's saying, Yeah, I felt better immediately, it's not I feel worse sometimes when I infuse.

SPEAKER_02 15:08

Interesting. Like if I have a bad bleed going on and I infuse, like I feel like it gets worse before it gets better for some reason. See, I don't know why I'm different.

SPEAKER_03 15:15

I feel like this is something uh the mental health aspect of hemophilia. You're just trained almost, okay. I have a pain, that our fropathy or not, I'm gonna do a shot. Right. It got better.

SPEAKER_01 15:27

And you have the novel treatments where we're now training patients, don't at the instance of having pain because your product might be working. So for consismab, which is alhema, that's a daily one. Oh, that's alHEMO actually. Which is fituceran, that's actually every eight-week injection, which is like, oh my god, you could have six for an entire year of prophylaxis. How great is that? But there's also it's important to realize there's a potential that it could be every four-week dosing. So with that medication, everything is a balance when it comes to bleeding and clotting. So when it comes to hemophilia A, we know that it's a deficiency in factor eight, hemophilia B is a factor deficiency in factor nine, but it's really the resultant thrombin deficiency that causes the prolonged bleeding. So this is why we have scientists who are looking at different ways to improve thrombin production as a method to preventing bleeds and prophylaxis, but it also explains why you can't use it to treat bleeds because we're not replacing factor eight and we're not replacing factor nine.

Aging, Comorbidities, And Screening

SPEAKER_02 16:20

But we're thinking like there might be some kind of placebo effect with doing it every day, being like, man, I'm doing it today. I feel protected. I feel like I can go do productivity now because I'm good.

SPEAKER_01 16:29

But definitely, I think it's very different. I think if we speak to you and we speak to newly diagnosed hemophilia patients who are getting a completely different narrative than what your parents were probably given. So I think there is a placebo effect. When consismab came out or alchemo, in my mind, I'm like, who wants to inject themselves every single day? But at the same time, this is where patient choice is so important because even when uh MSisumab came out, or even when some of the longer acting, I'm like, oh my God, how convenient is this? This is so flexible. It allows your patients to inject in frequently and you give their life back. And then I have a patient say, no, Maya, factor gives me flexibility. It gives me the predictable, I infuse myself, I know what my curve is going to be. And this is why, again, as a provider, I foolheartedly think our job is to just be the ones to provide information in a way that patients understand is most important. Because I think sometimes we have providers who speak in a little bit uh medical jargon and it's hard for patients to empower themselves. And a lot of patients don't they have guilt, I don't know, they just feel uncomfortable asking to repeat or don't want to feel like they aren't smart or whatever that is. But I always encourage ask up, speak up, ask questions. We don't judge you. And at the end of the day, this is your body and you are your biggest advocate. So I think again, we need to present the information to patients and then learn what are your preferences, what are your treatments, what has happened in the past that might impact your future decision and future treatment decisions. Because Dr. Joe, you had shared with me one of your patients who is on factor therapy, or it was actually you who are on factor therapy just because of your friend's experience with one of the novel treatments. So that is an experience that I've never witnessed in my clinical practice. When I look at the real world data and the information that we have, it's not something that um would be a part of my conversation that would steer people away from certain treatment, but it kind of emphasizes how lived experiences and shared experiences really do impact treatment options and treatment decisions for patients. And as providers, we need to respect that. I'm not gonna say, no, Dr. Joe, you're wrong. I don't agree with your friends, but that's just not a way to establish rapport with your patient and trust and make sure that we're gonna have the best options. But it's something that whether it's valid or not, and I think yours is valid. I don't want you to think I'm saying it's not. But as providers, we really do need to take this into account because there's so many options now, and there is not a one best treatment option. And there's luckily with WFH, they came out with a nice shared decision-making tool and kind of walks you through the process as patients. It gives you different goals and aspirations and questions to kind of ponder because if you've never even thought, well, what is my goal of treatment? It's going to be very hard for you to have as involved of a conversation, but it's a very different dynamic, very different conversations. And it's interesting because I'm learning that patients have very different priorities when it comes to selecting their treatment options.

SPEAKER_02 19:10

Yeah. So let's talk about parents and caregivers. So some children aren't old enough yet for these new therapies. So how should caregivers prepare for the future?

Chronic Pain Beyond Target Joints

SPEAKER_01 19:19

Sure. Where I think initially, when we were speaking offline, we were talking about all of the kids are being placed on emosism map or hemlibra when they're born. And I think the conversation starts at the time of birth. Once you are identified as having severe hemophilia, it makes sense. We want to prevent this life-threatening bleeding option. So the conversation is really let's review the SIPAD information that shows the benefits of using a plasma factor therapy. We can also review the MSIMap information, which is has robust. But the important thing that patients have to realize is that you're going to have to play support if you are going that factor route and you're going to be accessing that three times a week, usually. So think about what that traumatic experience does for the patient and for the baby. So a lot of patients do start with that novel. Treatment with MSisma because it doesn't require the port placement and doing it once a week, every two weeks, or even once a month, is a as a new mom, I can only I can't even imagine if I had not gonna feel very fortunate that my kids are doing well and healthy, but I I can never imagine what it is like to have your first child and then you find out that they have severe hemophilia and everything that is going through your mind at that time. So pediatrics infancy, I understand why a lot of them do go the novel route. But then when they start to age and they start to grow, and then maybe they have their first breakthrough bleed, or maybe they're getting more active with sports and in school, we have to continue to have these conversations. And I think back when you guys were born, they were taught like almost like a bubble boy like don't let your kids participate in sports and really do everything that you can to protect. Where now we really are trying to allow our kids to have as near normal a life as possible, but do it safely. So I would say to parents that you don't necessarily need to limit your kids. Yes, we want to do everything safely, but ask your questions. I love when patients come in with a list of questions to doctors' visits because it makes it so much more targeted and so much more relevant. One caveat is do it at the beginning of the appointment. Please do not do it at the end of the entire visit because then I would have totally prioritize time differently. But come with questions. Like there's no such thing as a stupid question, especially as a parent. Like our job is to protect our kids and to do everything in our power to keep our kids healthy, happy, safe, supported, etc. And you only do this by getting information. So I would talk to your providers. If you're not getting the answer you want from your providers, you have that right to get a second opinion. Um, but I really urge anybody with hemophilia or rare disorder to get connected with the hemophilia treatment center because we do this day in and day out. We're very familiar with the expanding treatment options, the nuances, all of the different specialty lab send-ups that we might have to do for that. Where, as for an example, I had a 26-year-old patient refer to me from the community. He'd been followed by a community provider his entire life. He comes to me 26 on demand with factor replacement with all of these target joints. Like, like, I'm glad he got connected. He hasn't had a single breakthrough bleed since we've since he's established care with me. But it makes me think like how different his life could have been if he actually got comprehensive care starting at a young age and what his life would have been like because now he's dealing with chronic pain and joint disease. And yes, it's it's very different. But the the I think the key that I want to emphasize is just you guys are in control. Like, think about what is important for you and your family. There's no right or wrong answer. I take a Switzerland approach when it comes to my treatments and what I'm prescribing my patients. I just want my patients on a treatment that's working for them. I don't care whether it's factor replacement, if it's sub Q, if it's daily treatment, every eight-week treatments, but ultimately I just want my patients to feel empowered and more importantly, trust the product that they're on so they're not having any potential anxiety or stresses and worries with their treatment.

Subcutaneous Schedules And Mindset

SPEAKER_03 22:45

I mean, I really love that message. I mean, because sometimes even that 26-year-old or even a 38-year-old doctor that has hemophilia might not have all the answers. And sometimes I feel like maybe even the older populations or even a new mother that just has a new baby that has chemophilia, they're they feel like, oh my God, I feel like I should have all the answers. And that's not that that's not what you're you're you're alluding to. Hey, we're here, we're gonna guide you, and you know, there's a treatment option for everybody's lifestyle. I just think it's it's really awesome in 2026 that so many people uh can have you know that that conversation, that open conversation with their HTC and prevent future joint damage by getting on a an optimized therapy.

SPEAKER_01 23:30

And sometimes providers don't have all the answers either. Like I'll have patients with me because sometimes this again, these are newer treatments. There is research going on with real world data which hasn't come to publications yet that we're not aware of. So if your provider doesn't know the answer, don't judge your provider for not knowing the answer. Like I'm very I'd rather say, you know, I'm not 100%. Let me find out and I'll get back to you than to potentially misspeak. So there's a lot of new information that's constantly coming out, especially with these newer treatments that were approved in the past year. And we're going to continue to learn more the longer that they're on the market. But have the conversations. And I would say a lot of my patients, they go online, love Dr. Google, but sometimes Dr. Google doesn't give the best advice. Sometimes ChatGPT just makes up advice and whatnot. But when you find your you do your research and you find your information, don't take it that this is 100% accurate. Rather use that to guide your conversation with your provider and be like, you know what? I saw this information online, like I'd love to talk about it further. What do you think? And from that is where you really are making your decisions. Because unfortunately, there's a lot of misinformation out there. So I would take it with a grain of salt, always look at the source you're getting it from, but use it to fuel your conversation. So again, you just knowledge is power at the end of the day. And the more information you have, the calmer you can be, the more confidence you can have, and just the better outcomes you'll have.

SPEAKER_02 24:47

Our next section actually was really to try to inform the listeners on how to get fully prepared for their next clinic visit. What are some like resources or like a checklist of things that people can do to make sure that when they go into their next appointment that they are 100% ready? Um, is it is it social media? Is it reaching out to other hemophiliacs? I mean, is that the best route? I wanna I really don't know what that answer would be either. I mean, I think us older guys, we've always reached out to one another and just like, you know, hey, I I see you're on this. I mean, I have people reach out to me all the time and I'll I'll come posting my medic, you know, my self-infusing and they'll be like, oh, how do you like that medication? You know, and I feel like that's how we, you know, like through social proof, we kind of we just go.

Caregivers, Kids, And Starting Choices

SPEAKER_01 25:26

And I think that's true for treatment options because I'll have patients who they clearly aren't well controlled. They're having a breakthrough bleed once a month, which doesn't sound a lot, but then you quantify that that's 12 breakthrough bleeds a year, and that's not well controlled. So I have patients who clearly aren't well controlled. They should be switching to a different treatment option to optimize their regimen, but they're nervous. And in those cases, I actually do connect them with different uh hemophilia patients who are open to speaking. I'm not given any information, we're not doing any HIPAA violations, but a pre-approved arrangement where they can speak to for patients who are on that treatment to see if it guides. I would recommend checking out the World Federation of Hemophilia. They have a great shared decision-making tool because I think that the most important thing as a patient, and what I would want all of my patients to do is really just take the few minutes to look inward and think, what are my goals? What's working with my treatment? What's not working? How are my IV? What's limiting in my life? How is hemophilia fitting in my life? Because again, I don't want the hemophilia to limit my patients. I have some patients who have zero ABRs, but then I find out they're not bleeding because they're so scared of bleeding that they're sedentary. So it's again, we have to ask the right questions. So if I know that my patient's sedentary and not bleeding, well, let's get you on something that can allow you to be active because there's so many benefits to physical activity. Sometimes people think being active will trigger bleeds, but it's actually the opposite. We know that physical activity is protective for the joints. It can actually reduce the number of bleeds you have. And if you do have a bleed, it'll be faster to recover. So there's so many benefits. But I think the most single most important thing is for a patient to really just start thinking what are my goals? What's important, what's working with.

SPEAKER_02 26:55

I think we need to have like a list of all those high quality questions because as I grew throughout my my life, I started to realize that my quality of life was determined by the quality of questions that I was willing to ask myself. And I've and that takes a lot of reflection time, you know, get going inward and then asking those high quality questions. Like for me, at one point it was, you know, like as at rock bottom, asking myself, you know, why do you why do I want to live? Why would I want to continue? Who could I contribute to? You know, like asking those questions changed my life. And so I I truly believe in that. But I think some people need help with formulating those questions because they're not used to asking themselves those deep questions. So I think it'd be really cool if we had like a list of like high-quality questions to ask yourself before you.

SPEAKER_01 27:34

Shameless plugs for this whole project. Um, we had to create some contents for it. So the content that I had created was specifically on shared decision making and providing guidance to kind of start with those high-level questions to really start looking inward. So if we're doing a shameless plug, hopefully we get it. I love it.

SPEAKER_03 27:52

That'd be awesome. But getting back to your chronic disease, you know, patients that are developing that, you know, getting older, developing heart disease, developing cancer, developing whatever it may be. Yeah. So I mean, really, that should be in that, like you just said, a sedentary. I'm not bleeding, but I'm sedentary and I'm 400 pounds. Like, you know, even though you have hemophilia, like you're still all these other things can catch up with you. Uh, these you know, chronic health conditions that are uh really judged by your metabolism and you know what you're taking in. I think having that on the decision tree is really important because there's other factors, like you just said, if a patient develops a heart failure and it runs in their family or they're hypertensive, and then they have an MI of myocardial infarction, also known as a heart attack, then they're on a plattox and an aspirin, now they're on you know a dual anti-platelet, things change, like things change, and I think it's it should be discussed as the hemophil aging hemophiliac population. You know, it's something I never really thought about, but that that's true. There's there's medications that need to be added to prevent the heart attack, but how is that gonna interact with your uh hemophilia? And if we see you as a 30-year-old, how can we prevent the obesity, prevent the non-alcoholic photophagic liver disease, prevent the diabetes, prevent the hypertension, which is gonna prevent a heart attack, stroke, a limb amputation, and future cancer.

Prepare For Appointments The Right Way

SPEAKER_01 29:17

Exactly. And I think, especially for my adolescents, young adults, et cetera, who are overweight, there's a lot of stigma associated with it. I actually saw a commercial the other day that I was just so moved by it because it was essentially just saying, like, stop judging us. We already judge ourselves type of thing. And I think we need to have a lot more compassion. But I think diet and exercise doesn't work for everybody. And I don't think providers are as proactive as they should be when it comes to being overweight and obese. So again, the patient is in the driver's seat. So if you know that you're overweight, talk to your provider, ask them, like, what do you think I can do for this? Can I you refer me to somebody? We have these GLP ones that from a hemophilia standpoint really shouldn't interact and increase bleeding risk. You might have other health issues, but ultimately talk to your provider. We're not giving medical advice, but we do have treatment options to help with weight loss. Now, this has to be in combination with lifestyle, otherwise, you're just gonna be dragged in on a medication, but it'll help you shed some pounds. So then it's easier for you to start walking or easier to start riding a bike or swimming. A lot of hemophilia treatment centers, we have physical therapists integrated. And I love referring to my PT so I can do a home exercise program and really enable patients to improve their life because I really at the same time, I'm very future oriented. And I think again, like you said, Dr. Joe, there's so many implications of obesity. And even a modest five to 10 pound weight loss can reduce the risk of heart disease, can get you off blood pressure medications, can get you off your heart disease, your diabetes medications. So I think we need to make it into an approachable situation because if you're 400 pounds, that person probably is like, oh, I'm never gonna lose any weight. But I this is where mentality and mindset is so key. And you can probably speak to this so much more than me since that's your your wheel case.

SPEAKER_03 30:51

And the joint implications too, that extra couple pounds of weight on your knee joints, on your hips, on your ankles, on your back. And I can just say, like the GLP1s, obviously, there's clinical trials going on, but they're seeing the studies show that there's some really exciting uh implications for the GLP1s for artropates and joint arthritis that reduce C-reactive protein, they reduce the set rate, they reduce this total body inflammation. So we'll see in the coming years that and maybe maybe an indication for arthritis that you know in 2028 or 2029, whatever, we're gonna see maybe that's an option that, as you know, a nurse practitioner in the hemophilia world or a physician, you know, we're gonna be looking at wow, we have to add a GOP1 to help with their arthropathy that they developed from a hemorrhagic syndromeitis when they were five or six and you know, uncontrolled that the patient was on factor. It's just interesting. All these treatment options we have the non-factor prophylactic options, we have factor options, we have extended life, extended half-life options or targeting outside of that clotting cascade, and then looking to the future, there could be other things for other parts of hemophilia, the comorbidities that we're living with. It's just it's an exciting time. It really is, especially with the GOP1s and peptides that are available.

Movement, Weight, And Joint Protection

SPEAKER_01 32:10

Since we're talking aging, and I feel like I've dropped some pearls for your listeners, I want to just make one more comment of a comorbidity that we do see at higher risk in individuals with hemophilia compared to the general population, and that is osteoporosis or weakening of the bones. And that's another important reason to be physically active because when you're walking or squatting or doing like those bone loading exercises, you're like pushing the calcium back into your bones and you're going to reduce the risk of osteoporosis. The other thing I want all of my hemophilia patients to check is your vitamin D level because you need vitamin D in order to absorb calcium and keep your bone strong. Florida, being the sunny state, has one of the highest prevalence of vitamin D deficiency. And almost all of my patients are on some type of supplementation. So when we're thinking, and just since we've talked about the aging population, we're talking about obesity, weight loss, things of that nature, and what can we do? So our patients with hemophilia are not just living a long life, but a long quality life. Like maintaining a healthy weight, being physically active is so important. And I always tell my patients don't have the goal to be physically active to lose weight. Like be physically active because you want to feel good, because that slight tweakment in your language makes such a difference. Because then when you forget to work out or when you lose or gain some weight, you're not gonna put the shame and make yourself feel bad. And more likely, if you get off of that routine, you're gonna continue with it. That just consistency is key. You don't need a hard workout every single day, just consistent, even five minutes to prove to your body and your brain that, like, hey, I can do this because your brain, our brain is smart, but our brain does anything it can to resist change because change equals danger, even if it's a healthy change. So when you say, you know what, I'm gonna start working out three times a week, that is change to your brain, and your brain is gonna do whatever it can to get you not to move. This is called analysis paralysis. So if you ever want to, like, I know I should work out, but why can't I get up? It's because your brain is literally trying to protect you. So if we know how our brain works, we can overcome that and be like, you know what, my brain's doing that thing again. Let me just do five minutes, right? Or let me go outside for a walk. Just you have to overcome that brain's response. And the more times you do it, you're working a new brain muscle that's neuroplasticity and becomes easier and easier. So a lot of benefits to working out. And my physical therapist told me for every pound that you lose, that actually takes off four pounds from an affected joint. So that means if you lost five pounds of weight, that's almost 20 pounds that's being released from your lower extremity. So anytime I have patients complaining of back, hip or knee or ankle issues, even if they're thin, I always do recommend a modest weight loss, just understanding that statistic. So there's a lot of benefits. I think there's so much more to hemophilia care and really any disease management beyond just treating the hemophilia. I think if we just focus on treating the disease alone, we're gonna miss the mark. Um, but there's so many other factors, no pun intended, um, going into just ensuring health outcomes. And I think again, it all starts with the patient deciding what is most important to you, what's gonna make you live a fulfilling life, what's working, what's not working, and starting to have those meaningful conversations with your provider.

SPEAKER_02 34:58

I do want to ask you for personal reasons. I I've been posting on social media for 10 plus years. For me, when I was growing up, I noticed there was a lot of people suffering from from mental health issues. Um, a lot mostly I would I would feel like due to chronic pain and not being able to manage that correctly. But as I've gone on my journey, I would say over this past year or so, looking at the new generation coming up, I feel like there's like this older generation and then the new generation coming. And I feel like we're all on two completely different spectrums of like almost in a good way. I I don't know really how to word it, but like the new generation's like, what are you guys talking about? Like, we're good. We're good over here, we're chill. Like, so in a way, I'm kind of like, as I'm on my advocacy journey, I'm thinking, is it over? I do I am I done advocating because these guys are good. They don't need me anymore. The older guys were like, hey, I want to relate to you. I want to to know that someone else is going through this with me and that we can overcome this somehow. And that's why I'm really in love with like the whole mindset and and mental health stuff. I think. So I don't know, like what are your thoughts on on this like transition? Or do you see something like that?

Bone Health And Vitamin D

SPEAKER_01 36:05

Well, let's talk mindset because I actually I had um an abstract award at HTRS last year because I presented on comorbidities, race and ethnic differences in comorbidities and bleeding disorders. And the number one self-reported comorbidity among 212 uh individuals, it was 505 total surveyed, but 212 uh participants had chronic pain. So that was almost 42%. But the next two highest self-reported comorbidities were depression and anxiety, being above 30 to 35% of individuals having this. So I think when it comes to mental health, there's such a stigma associated from it that I feel like we're working closer and closer, and every year it's getting a little bit less stigmatized. But I think realizing that there's almost more than a third of patients, and this is probably underreported because patients still are stigmatized and might not self-report, it's very common. So speak to your provider because we know that mental health and physical health go hand in hand and worsening of your mental health can lead to more inflammation inside of your body and can have this whole downstream effect for it. So I think that the newer generation who was born where factor prophylaxis was the standard of care. Maybe they're really early and they were started on emosism up. They haven't had any breakthrough leads, they have a very different chemophilia experience. Like I get nervous for what chemophilia is gonna look like in 10 years from now because now I think inhibitors is gonna be more of an adult problem than a pediatric problem because most of the patients are being started on MSISM app. I think another issue we're seeing is people aren't gonna know how to self-infuse. So self-infusion become, we started teaching home infusion, I think, back in the 50s and it improved autonomy significantly. And now we're losing that skill because the treatments are sub Q. So I'm wondering like what happens when there's breakthrough leads. Are we gonna see more healthcare utilization? So there it's, I think the patients who are saying, What are you talking about? There's no problem, that's because they're blessed and they were born in a time where they had really optimal management. But I think they're probably a little naive and they haven't in 10, 15 years from now. And you ask them again, what was it like growing up with hemophilia? It's you know, it's it's hard to acknowledge because we just want to fit in. You want to be like your peers, you don't want to feel different, you want to live as normal of a life. And the more times you acknowledge that your life is different is a reminder. So my initial thought when you made that comment is these are probably just younger generation.

SPEAKER_02 38:14

So they still need us, they still need us. We do, and I'm like getting chill.

SPEAKER_01 38:17

I'm very into the body sensations. I have chills right now that I do not think what you guys are doing in vain, and I think you're gonna resonate with more and more people. And I think that your experience is going to be invaluable for the younger generation because they might not have a bleed yet. You're gonna have a patient who's like 15 years old and that might be their first bleed that they're experiencing, and they don't understand what's the singling going on inside of my knee, and they don't know what it means to treat early because it's just a completely different uh dialogue that we're having now with patients compared to in you guys. So I think you guys are definitely needed. I think mental health is something that 100% needs to be addressed. So anybody out there, if you feel like you're having more blue days than good days, definitely talk to your provider. I think a lot of us, when we think about the political situation, the turmoil, stress is our baseline. Like we're just in autopilot trying to get through the next day, and that is not a fulfilling life. So I think the more times we can just kind of press the brake on that stress response, it gives your body that stillness it needs because chronic stress, what does it do? It activates your hypocrisy, your HPA access, your hypopitual pituitary adrenal access, and this is what releases cortisol and adrenaline, which short term is very helpful. It helps you survive if the bear is attacking you and you can run and get everything and get out and survive. But chronically, if you have this HPA access chronically on and activated, it's dysregulated, it leads to more inflammation, it affects your immunity, it affects your sleep. There's so much that can affect our body and our physical health beyond just the hemophilia because of stress. That one takeaway is if just start breathing, deep breathing. We're all breathing, but being in the world.

Mental Health, Anxiety, And Data

SPEAKER_02 39:47

I was gonna ask you, how do you break that pattern? If you're living your entire life with chronic pain, they're struggling and they they don't see the light at the end of the tunnel and they don't know how to break this this mental pattern. Yeah. And I I feel like I've been able to do it, but in your like professional opinion, how does somebody break those ruminating thoughts that they can't seem to escape every single day when they're going through that?

SPEAKER_01 40:08

So my professional opinion is honestly based on my own personal experience. So I'm professionally trained in hematology as a nurse practitioner, but my own self-healing journey is kind of what was the catalyst to all of this interest and really understanding the nitty-gritty because I am one where if I can understand what's happening in my brain, it kind of gives me what I need to change uh said behavior. So the most important thing, number one, is the awareness. So the fact that like you can have a lot of ruminating thoughts and be on that hamster wheel and go in and going and have no idea. So at least once you have the awareness of having those negative intrusive thoughts, that's where your power is. You can't, my biggest thing is just in the beginning while you're trying to break that wheel is you can't be positive and negative at the same time. So once you have the awareness that you have that negative feedback loop outbung, think of something you're grateful for. And it does not need to be anything major, it can just be like, you know, I'm grateful for this really comfortable bed, or you know, I'm grateful that today is Saturday. It doesn't need to be deep. But the more times you do that, we spoke about neuroplasticity earlier, but you're working a new, a new muscle in your body. So your body has been thinking negatively, not you, but just generally speaking, people with a lot of ruminating thoughts, these are protective mechanisms. Like for me, I was queen people pleaser, overthinker, overanalyzer. I thought these were personality traits, but through my healing journey, I learned that these were survival patterns that as a child kept me safe. But as an adult, they're holding me back. But I would start to ask my patients, like, what are the themes that you're repeating in your life? What are the repeating patterns in your life? Is it I have patients who it's like a I have some patients who feel like they're everything is always what should I say? Um, but I would say look at what the repeating themes and patterns are in your life. I have some patients who feel like nothing ever happens for them, everything always goes wrong. But if you think that you're gonna speak into existence what you're saying. And this isn't just woo-woo mystical stuff. Like we have a part of our brain, your reticular activating system, your RAS, which I like to explain as Instagram's algorithm. When you're on Instagram and you see information, you like it, Instagram's gonna keep putting more and more information in front of you because the RAS is essentially what scans your environment to pick up any clues or sensations that fit whatever your narrative and what you're speaking into existence. So if you're constantly saying negative things, your RIS is gonna scan your environment and find everything that agrees with that narrative. So the more times that you have the awareness, like, okay, I'm ruminating again, thoughts are not facts. Like when you think what is a belief, a belief is something that you think over and over and over again, but our thoughts aren't necessarily real. So I would start looking inward of what are those beliefs, what are your thoughts? When you have the awareness, you're ruminating again, but something I can be grateful about.

SPEAKER_02 42:39

So like I think like premeditating beforehand, like once you catch on to what that thought is, already come up with like the the alternative thought that you that you want.

SPEAKER_01 42:47

So I like that. Yeah, like already have your your gratitude statement. Like already have it ready.

SPEAKER_02 42:51

So whenever it hits your head, you're like, nope, no, I'm not accepting that. I'm not accepting that thought.

Breaking Rumination With Breathwork

SPEAKER_01 42:55

So you did your gratitude statement, then you're gonna do your breathing because when you're breathing, especially deep breathing, is that's what's gonna activate that parasympathetic nervous system. That's the rest and repair, the opposite of your sympathetic nervous system, which is what's happening when the bear is attacking you. And there's a lot of different breath work exercises. I like to teach my patients box breathing, where you literally you'll inhale for four seconds, hold your breath for four seconds, exhale for four seconds, hold for four. Okay. And if you do two of those cycles, you'll feel a difference. If that's hard to remember, the key is to just exhale or breathe out longer than you're inhaling. And that again will activate the opposite side of your brain. So I always recommend doing your deep breathing when you're feeling well. Like do it every single day. I do it when I brush my teeth. So at least I know in the morning and at night I'm gonna be doing it anyway, brushing my teeth, might as well add deep breathing to it because you don't want to learn a new skill or try a new skill when you're in crisis. So I would just start start breathing, everyone. And that way, when you you do have those ruminating intrusive thoughts, know that we all have it. I'm human too. I've been on this healing journey for a while, but the main difference is my body's not reactive. I have the intrusive thought, I have the awareness within seconds. I shift it real fast, and then I'm on with my day. So the more times you do it, the easier it gets. And I have not always been like this. It's just proof is in the pudding that the awareness is the first step, and then we really have a lot of power and can transform our lives.

SPEAKER_02 44:13

Maya, thank you so much for breaking down what can feel like an overwhelming amount of change going on in the hemophilia landscape. You've helped make this information approachable and empowering. So I really appreciate that.

SPEAKER_03 44:23

To everyone listening, please take some time to look at the link below. We have educational videos that are going to explain all these non-uh-factor prophylactic options like Al Hemo, cuphilia, himpoxy. And so you can walk into your next appointment prepared.

SPEAKER_02 44:40

Sponsored by CME Outfitters through an educational grant from Nova Nordisk. And just remember, as your life changes, your treatment plan should evolve with you. Stay informed, stay empowered, and we'll see you next time on the Hemo Life Podcast.

SPEAKER_00 44:52

Thank you for tuning in to the HEMOLife podcast. Today's episode hopefully inspired you and provided valuable insights to enhance your journey. Join us again to hear more incredible stories and expert advice from our community. Make sure to subscribe and stay connected with the group of extraordinary achievers and pioneers. On behalf of LA Guayo and the entire Emo Life team, keep pushing forward, strive for excellence, and remember you are the architect of your own destiny. Until next time, stay strong, stay inspired, and continue on your path to an elite life.