Bleeding Disorders Conference 2026
Day 2: Living Well With a Bleeding Disorder
From promising new therapies to conversations about aging, mental wellness, and informed decision-making, Day 2 of the Bleeding Disorders Conference focused on what it means to live well with a bleeding disorder.
As treatment options continue to evolve, attendees explored not only the science driving progress, but also the experiences, perspectives, and practical tools that help individuals and families navigate life today.
A New Era of Treatment Options
“We are in a golden age of new treatments,” Mark Reding, MD, said at the beginning of Advances in Treatment, a session focusing on the pipeline of new therapies for bleeding disorders. One only has to look back about twenty years to see the marked acceleration and innovation in treating hemophilia – and now, thanks to the success of those therapies, treatment possibilities for von Willebrand disease, rare factor disorders, and other rare conditions like Glanzmann’s thrombasthenia are now under investigation.
Dr. Reding explained that these therapies generally fall into three categories: factor replacement therapies, including standard half-life, extended half-life, and ultra-long half-life factor VIII products; non-factor replacement therapies, such as factor VIIIa mimetics like emicizumab and rebalancing agents; and gene therapy. While each approach works differently, they all share the same goal: reducing bleeding episodes and improving outcomes for people with bleeding disorders..
Factor replacement therapies, regardless of half-life – contain a protein that replaces the missing protein from the clotting cascade, either FVIII or FIX. Most factor replacement therapies use synthesized factor molecules to avoid the risk of blood-borne pathogens. These products are infused intravenously, and until the introduction of ultra-long half-life products, had to be administered multiple times per week to reduce bleeds.
Non-factor replacement therapies operate quite differently, Reding said. They improve clotting without replacing the missing factor. Unlike factor replacement therapies, they are only used to prevent bleeds, and cannot be used to treat an active bleed. The non-factor replacement therapies currently on the market or in the pipeline are FVIIIa mimetics – such as emicizumab (Hemlibra) and Mim8, and rebalancing agents, such as marstacimab (Hympavzi), concizumab (Alhemo), and fitursiran (Qfitlia).
“All of these therapies are roughly the same in efficacy and safety,” Dr. Reding said. “But they’re just different.” For example, the rebalancing agents, which work by reducing a body’s natural anticoagulants and allowing more thrombin to be generated, creating a stable clot, provide continuous protection against bleeds, without any of the peaks and troughs of factor replacement therapy. They are equally effective in both hemophilia A and B, regardless of inhibitors. However, they do carry an increased risk of thrombosis, due to coagulation imbalance.
They are also dosed differently – some are based on weight, some on anti-thrombin level, some at a fixed dose. All are administered subcutaneously. And when breakthrough bleeds do occur, the treatment must be done carefully and differently than it would be for a person on factor replacement therapy.
“Most people want to know, ‘Which is the best one?‘” Dr. Reding said. “We cannot answer that question.” Part of the reason is there is no head-to-head comparison. You can’t compare annual bleed rates from clinical trials – there are too many differing variables. And the hemostatic correction itself cannot be measured. For the rebalancing agents, there have been thrombotic events in all the clinical trials, even as the trial design sought to exclude those at heightened risk for thrombosis. “Managing bleeds with a rebalancing agent needs to be done very carefully,” Dr. Reding reiterated. “You really need to understand it. As the healthcare provider, I need to give you all the information and make sure you understand it, and the patients have to take the time to learn.”
While there are risks, there are also rewards. “Thanks to the progress of these drugs, we now have more potential options for von Willebrand disease, rare factor disorders, and Glanzmann’s thrombasthenia,” he said. Several new therapies are currently in clinical trials. HMB-001 (sutacimig) is being investigated for Glanzmann’s thrombasthenia and FVII deficiency. VGA039 (latarcibart) and HMB-002 are being studied as prophylactic treatments for von Willebrand disease (VWD), while HMB-003 is being evaluated for the treatment of heavy menstrual bleeding, regardless of cause..
This emphasis on VWD and the rarer bleeding disorders is welcome news to Dr. Reding. Many of these disorders have few to no treatment options. And VWD should have the same rich tapestry of treatment options that hemophilia does. “It reflects what we see in our clinics,” he said. “We see more people with VWD than with hemophilia.”
Promises Made, Lessons Learned, What’s Next?
Advancements in treatments are welcome news for the younger generation of people with bleeding disorders, but for many of the older folks in the bleeding disorders community, it is something of a bittersweet achievement. The tension between the generations who grew up with hemophilia before safe and effective prophylaxis was one of the discoveries discussed in Promises Made, Lessons Learned, What’s Next, a session focused on a study of aging people with bleeding disorders and their lived experiences.
Sarah Schwartz, PhD, from the University of Southern California; Ellen Kachalsky, LMSW-C, from the Henry Ford Hemophilia and Thrombosis Treatment Center; Misha Ansari, from the University of Michigan; and Tam Perry, PhD, from Wayne State University, are studying the lived experience of multi-racial adult persons with bleeding disorders to account for a diversity of experience in terms of race/ethnicity to characterize select needs and barriers, including emotional, social, physical, and financial. Funded by a Social Work Excellence Fellowship from NBDF, the study of this particular population was in response to an earlier study of aging people with bleeding disorders that focused predominantly on white men.
“I didn’t expect to live to see the 12th grade,” one participant in the study said.
“I didn’t make a lot of future plans. I couldn’t see that far.”
One of the key themes that emerged from the study was self-preservation, the effect of an expectation of a reduced life expectancy. “I didn’t expect to live to see the 12th grade,” one participant in the study said. “I didn’t make a lot of future plans. I couldn’t see that far.” Others planned their lives around their bleeding disorder. Finding jobs that were steady, provided good insurance, and didn’t take too much of a physical toll on the body were also found to be important.
Some of the respondents also discussed discrimination when trying to treat their pain. “Because I’m Black and saying I need pain meds, they see me walking and assume I’m fine,” one participant shared while talking about being assumed to be “med-seeking” rather than seeking treatment for pain.
Another theme that emerged was “medical envy.” Some of the men in the older population are watching the younger generation who are not experiencing the trauma of the previous generation – the repeated bleeds, the joint issues, and the safety of blood products. “They never know what we’ve gone through and I envy that they don’t have the medical issues that we had when we were younger.”
Dr. Schwartz stated that the study was still open, and is accepting adult people with bleeding disorders aged 40 and up. If you are interested, email her at saraschw@usc.edu.
3 Habits for Better Mental Health
As a person with severe hemophilia A, Fernando Reyes, M.Ed., knows something about the stress that managing a bleeding disorder can create. As a mental health professional, he had helpful advice to deal with stress that focused on three key areas: sleep, exercise, and nutrition.
Sleep is critical, said Reyes. “Sleep improves our memory, helps to lower inflammation, improves mental focus, and helps us combat stress. And the restorative role of sleep is a consequence of the enhanced elimination of potentially neurotoxic waste products that accrue in the awake central nervous system.” Reyes recommends at least 8 hours of sleep a night.
“Movement is medicine for your brain,” Reyes continued. “In clinical studies, we have seen that just thirty minutes per day, five times a week, will strengthen two regions of the brain that you need for optimal function.” It can help stimulate the growth of new brain cells and the repair of damaged cells, he said.
Nutrition also plays a role in mental health, he said. He recommends incorporating high fiber foods such as avocado, whole fruits and vegetables, and prebiotics, such as yogurt, and wild blueberries for overall brain health.
Making Tough Decisions With Confidence
People with bleeding disorders are constantly making decisions that could have a huge impact on their lives – what type of treatment should they consider? When making difficult or consequential decisions, the process of how you arrive at the decision is just as important as the choice you make, says Michelle Florendo during “Confident Decision-Making. Every decision has three components: objectives, options, and information. For objectives, one should consider what matters most in the outcome and why, and weigh risks. She recommends looking at a potential decision through a Relief-Regret analysis, where you consider if you would experience relief or regret at the possible outcome of a decision. She also recommends using a decision quality statement: “I can move forward feeling at peace with this decision because I ____”.But let the process happen naturally. “Decision-making happens in a sequence and over time,” she said.
The Bleeding Disorders Conference brings together patients, families, and health care professionals for three days of insightful educational sessions, valuable networking opportunities, and a dynamic exhibit hall featuring leading pharmaceutical partners, organizations, and research poster abstracts.
The National Bleeding Disorders Foundation (NBDF) is dedicated to finding cures for inheritable blood and bleeding disorders and to addressing and preventing the complications of these disorders through research, education, and advocacy, enabling people and families to thrive.
© National Bleeding Disorders Foundation
All rights reserved.
Built with Shorthand