For many years, adults with hemophilia had relatively few treatment options, and all of them were fairly similar. But over the last decade, advances in biomedical research have led to several new therapies that work in very different ways, giving people more possibilities to find a treatment that works for them.
In addition to the standard half-life factor VIII and IX products that have been around since the early ’90s, newer hemophilia treatment options include:
- Extended half-life factor products, which enable higher levels of factor to stay in the body for longer, resulting in the need for less frequent infusions.
- The bispecific antibody therapy emicizumab, which mimics the function of factor VIII, allowing for subcutaneous injections rather than intravenous infusions.
- Rebalancing agents such as marstacimab, concizumab and fitusiran, also given as subcutaneous injections, which work to prevent too much bleeding by taking away or blocking proteins that slow down the clotting process.
- Gene therapy, which provides the body with the gene that is not working so that it can make its own factor.
While all of the advances in treatment are welcome and exciting, they can often leave people with hemophilia confused about what’s best for them, says Mark Reding, M.D., director of the M Health Fairview Center for Bleeding and Clotting Disorders at the University of Minnesota in Minneapolis.
“A lot of patients feel pretty overwhelmed by all the choices, because some of these new drugs work so differently, and for patients, it can be hard to get their heads around that,” Reding says.
In the past, when Reding met with patients, the treatment discussion was relatively short.
“It was kind of, ‘Hey, how are you doing? You’re doing great? Awesome. See you next year.’ There wasn’t much to talk about,” he says.
Now, with so many new therapies available, he spends a lot more time talking about treatment options, and he sees just how differently some patients respond during these discussions. “People’s willingness to embrace new things really varies,” Reding says. “We’ve known our patients for a long time, but we haven’t always appreciated how big of a deal this is for them and their families.”
What’s more, when gene therapy was first being developed, it was only being compared with standard half-life factor, “so the bar was not that high.” Now, the options are crowded, and the optimal treatments for each person are not as clear. “The target keeps moving, and it’s moved very rapidly in a very condensed time frame,” he says.
How Doctors Approach Conversations About Hemophilia Treatment Options
According to Reding, the expanded treatment landscape for hemophilia highlights the importance of shared decision-making, an approach to treating diseases in which patients are partners in their own care.
“A big part of these conversations is about gauging a patient’s understanding of what their treatment options are and how their current treatment fits into that menu,” Reding says. “Then the next layer is to talk about their goals, their expectations, what could be better about their current treatment, and if any of these other options could potentially fill those gaps or needs.”
Reding says carving out time to talk about treatment options is especially important since people are getting information from a variety of other sources — including social media and other patients — and some of it may not be accurate. “We want to make sure that patients know they can come to us and that we’re up to date on all of the treatments,” Reding says.
What to Keep in Mind About Hemophilia Treatment Options
To better prepare for your next doctor’s appointment and possible conversations around treatment options, here’s what Reding recommends:
- Have the conversation, even if you’re happy with your current therapy. “When I see a patient in the clinic, even if they're doing fine and I don’t think they need to change, we still have the conversation,” Reding says. “Sometimes it’s fairly brief, but I feel obligated to talk about other options that are out there, just so they’ve heard about them.”
Reding says he’s learned that you can’t really know how patients feel until you ask. Someone may seem as if they’re doing well and never bring up concerns on their own, but once he raises the topic and gives them a little nudge, they might be eager to talk. “And all of a sudden, you have a really interesting and very helpful conversation,” he says.
- Consider real-world use along with clinical trial information. Clinical trials are an integral part of advancing bleeding disorders care, but “data from clinical trials only gets us so far,” Reding says. “Real-world clinical experience is quite a different thing.” Many studies have included large numbers of people outside the U.S. and Europe, often in “resource-poor” settings where people have far fewer treatment options. As a result, he explains, their baseline joint health and expectations about care can be very different from those of patients in the U.S., making it hard to compare experiences across those groups.
In addition, because clinical trials tend to enroll younger people who are otherwise generally healthy, there isn’t a lot of data about how these medications work in older patients, who may have other chronic health conditions.
“We struggle in the clinical trial experience to understand what we’re going to do in unexpected circumstances that don’t happen very often in clinical trials but are going to happen in the real world, such as breakthrough bleeds or an unexpected need for surgery,” Reding says.
- Understand that switching therapies isn’t necessarily straightforward. “What we also don’t learn in clinical trials is how to switch from treatment A to treatment B,” Reding says, adding that some of the newer drugs don’t just disappear when you stop them — they can take months to clear out of your system.
“We’re talking about switching to very different treatments that don’t wear off right away,” he adds. “How much data and experience do we have? The answer is not much.”
- Plan for an ongoing dialogue. “One of the things I’ve realized is it’s not a conversation — it’s a series of conversations,” Reding says. He makes a point of noting what treatments were discussed at each visit so that he can see how the person’s views evolve over time.
Establishing Care at an HTC Is Important
Studies show that the comprehensive care model available at hemophilia treatment centers (HTCs) leads to improved health for people with bleeding disorders.
Reding says for him, one of the benefits of practicing in an HTC is that with a full team of providers supporting him, he has more time to spend on in-depth conversations about treatment options.
“With the treatment landscape looking the way it looks, it’s more important than ever that people with hemophilia get their care at an HTC.”
Click here to learn about a tool that can help with shared decision-making.