For People with Bleeding Disorders, Adherence Is Still a Problem

Even as therapies for hemophilia have expanded and improved, the issue of not following treatment regimens as directed remains a major point of concern for patients and physicians.
Author: By Christina Frank

When Sean Pentz was growing up, his mother made sure he received his infusions of clotting factor three times a week.

Although he learned how to self-infuse at age 9, it wasn’t until he went to college that he fully took on the responsibility for his treatment. And as a young adult, he recalls often having “dropped the ball” on his factor infusions.

“The main reason for my nonadherence was immaturity and irresponsibility,” says Pentz, now 38, of Escondido, California. “I didn’t set enough reminders to infuse, and then I’d just completely forget. Plus, my own shame and stubbornness toward seeking help made it tough to make the changes I needed to be more consistent with prophylaxis. As a result, I experienced a bad intramuscular bleed in my arm when I was 25 and multiple knee bleeds at 26 after a partial tear of my meniscus.”

The Dangers of Nonadherence

The problem of not adhering to your prescribed treatment plan — also called noncompliance or “treatment fatigue” — is certainly not exclusive to people with bleeding disorders. Across all chronic conditions, the treatment adherence rate is estimated to be only 50% on average. Yet generally, adherence rates of at least 80% are what prescribers consider necessary to get the full benefits of a treatment therapy. In fact, according to the World Health Organization, adherence to therapy can have a more direct impact on patient outcomes than the specific treatment itself.

For people with hemophilia, staying on top of their treatments not only reduces the number of breakthrough bleeding events but also prevents added complications such as hemophilic arthropathy.

“All these tiny bleeding events can accumulate and lead to long-term damage,” says Robert Sidonio Jr., M.D., M.Sc., director of hemostasis and thrombosis clinical operations at the Aflac Cancer and Blood Disorders Center of Children’s Healthcare of Atlanta. “We’re trying to prevent things now so that 20 years down the road, patients will be in optimal health. We have great therapies in this country, and they work really well when you take them, but they don’t work so well if you don’t take them.”

The Transition to Adulthood

Teenagers and young adults are especially vulnerable to being lax with their treatment plans. Studies have found that nonadherence rates among people with hemophilia are highest among 18- to 24-year-olds.

In addition to the heightened sense of not wanting to be deemed “different” that tends to characterize this age group, young adulthood is typically when people leave home for the first time, start to make their own decisions, and have to take on full responsibility for staying on top of their medical treatment.

“All of a sudden, there’s nobody seeing you every morning at the breakfast table reminding you to infuse,” says Andrew Leavitt, M.D., director of the Adult Hemophilia Treatment Center at the University of California San Francisco.

Some hematologists are concerned that younger patients with hemophilia may not be as compliant with their medications because they’ve never experienced a bleed. “Maybe they’ve been on this treatment for 14 years and they haven’t bled, and then they have a lot of other things to focus on in high school and feel invincible,” says Courtney Thornburg, M.D., M.S., medical director of the Hemophilia and Thrombosis Treatment Center at Rady Children’s Hospital-San Diego. “Adherence with the treatment can go by the wayside, and then there can be bleeding complications.”

Leavitt says UCSF’s pediatric centers work hard to try to make sure that kids take progressive and age-appropriate ownership and responsibility for their care. “The more you are in charge of your care, the better you will be and the happier you’ll be,” he adds.

Do Better Treatments Lead to Complacency?

For many years, the only option for preventing bleeds in people with severe hemophilia was through a standard regimen of clotting factor replacement given intravenously as often as three times a week. Today, we’re in what Sidonio calls the “golden era” of new therapies for hemophilia, including treatments that can be delivered subcutaneously and less often. For some people, gene therapy — a one-time treatment — may now be an option. However, even with all these advancements, bleeds are still possible.

Experts say that as treatments for bleeding disorders have expanded and improved, they are seeing a bigger issue with nonadherence. For instance, people with severe hemophilia who are taking extended half-life products and now consider themselves “free” from intense treatment and management may become less attentive to their treatment.

“With less frequent as well as subcutaneous treatments, you would think that adherence should be better, but there are still challenges with adhering to the new therapies,” Thornburg says.

Even people who have had success with gene therapy may become overconfident and overdo activities that may jeopardize their joint health. While gene therapy may slow down or prevent hemophilic arthropathy progression, it cannot undo the damage that’s already been done, Leavitt says.

“Patients who undergo gene therapy typically experience marked improvement in their joint symptoms, and we see that they then tax their joints well beyond what they did in the past,” he says. “We need to be sure patients understand that underlying arthropathy is not expected to improve, so wise choices are still important for the best possible long-term joint health and function.”

For Sean Pentz, those bad bleeds in his 20s forced him to come to terms with his nonadherence and become much more vigilant about his hemophilia treatment.

“With support from family and doctors, I learned from mistakes, developed better habits, and have minimized bleeds for the past 15 years,” he says. “Now, I enjoy an active life with my daughters — hiking, biking, and playing sports, free from past limitations.”

Tips for Staying Accountable

Keeping on top of your treatment regimen can help prevent serious complications and improve your quality of life. These tips can make it easier to stay compliant.

Respect Routines. 

“Setting routines in place as young as possible will help you be successful with your adherence to your prophylaxis,” Pentz says. “By taking command of giving yourself infusions, ordering your medication, communicating with your hematologist, and setting your schedule, you will definitely reduce the amount of pain and suffering from bleeds that are the result of nonadherence.”

Set reminders. 

Use a written log, a calendar on the refrigerator, or one of the many medication apps to remind you when it’s treatment time. “It doesn’t matter which one you use, just pick one,” Sidonio says. “Use the one that you feel is useful. Some of the apps allow communication with staff at your treatment center, which is helpful as well.”

Get support from community. 

Make it a priority to connect with others who share your condition, Sidonio says. “It’s nice to get encouragement from the staff at your hemophilia treatment center, but I think peer encouragement is helpful, too. You have people who are going through the exact same thing, and sometimes it’s nice just to hear other people saying, ‘I’m struggling with this, too. I missed a dose last week, and then I had a bleed’ or ‘I’m just kind of tired of doing these infusions,’” he says.

“There are lots of virtual communities, lots of groups out there, and lots of opportunities to have get-togethers and go to family camps. These things help reinforce the reasons why you’re doing this. You feel like you’re not the only one.”

Be honest. 

Above all, it’s important to be truthful with your doctors if you’ve missed doses of your medications. Clinicians understand that people miss doses or get off track. They’re there to offer help, not to judge, but they can’t offer suggestions when they do not know the reality of the situation.

“The worst thing that can happen is when a patient tells us that they’re doing their factor prophylaxis when they aren’t,” Sidonio says. “We had a young child get hurt and develop an intracranial hemorrhage. We found out they were not doing the prophylaxis because they had issues with IV access. We could have worked with them and prevented that.”

Pentz says to set aside any shame you may feel and reach out for help. “There is no reason to let shame hold you back from making a positive change for your care,” he says.