Six years ago, when Sonji Wilkes gave birth to a baby boy, she learned very quickly that life with little Thomas was not going to be exactly what she had expected. Shortly after his birth, when her son was circumcised, the uncontrolled bleeding that followed led to a diagnosis of hemophilia. “I was like, hemo-what?” Wilkes recalls. She and her family had just begun to adjust to that diagnosis when, at nine months old, Thomas’s unusual level of bruising revealed that he didn’t just have hemophilia—he had hemophilia with an inhibitor.
An inhibitor develops when a person with hemophilia produces an antibody response to certain factor products, rendering treatment with these products ineffective and making it much more difficult to stop a bleeding episode. There are about 18,000 people in the US today with hemophilia; fewer than 1,000 of them have an inhibitor—making these families a minority among a minority.
“I had no idea what an inhibitor was,” Wilkes says. “And there were only two brochures about inhibitors that the doctors could give me, one of which was about 10 years old. At the time, Thomas was the only pediatric case in Colorado with an inhibitor. It was very isolating.”
So when staff from the National Hemophilia Foundation (NHF) came to Colorado for a meeting, Wilkes took them aside. “Look, we need more information about inhibitors,” she said.
Wilkes wasn’t the only one calling for more focus on inhibitors. Richard Pezzillo, now 26, was diagnosed with an inhibitor at age 17. Formerly a co-chair of the NHF’s National Youth Leadership Institute (NYLI), he says, “I had very different experiences as a young adult, because of my inhibitor, compared to my peers. I faced constant hospitalizations for weeks and months at a time. Hemophilia is manageable in 2010, but with an inhibitor, it can be unmanageable. We bleed the way everyone with hemophilia did in the 1960s. So I talked to all kinds of people, like executive directors of local chapters, telling them that this was an underserved community.”
Staffers took the pleas from patients and families like these to Novo Nordisk. Less than a year later, in November 2005, the first Inhibitor Patient Education Summit was held in Philadelphia.
“It was the first time I was in the room with a group of other inhibitor parents,” says Wilkes. “Someone said, ‘My child can’t walk across a parking lot without getting a bleed,’ and I thought, ‘At last, someone understands what I’m going through’ Knowing that there was a community of people that understood what living with an inhibitor was like—that was as helpful as the content of the summit itself.”
For the past five years, the Inhibitor Summits have focused on combating the isolation that parents, children, families and adults with inhibitors feel, by providing them with the latest medical information, tools for managing life with an inhibitor, and most important of all, creating a sense of community and shared knowledge. Since that first meeting in Philadelphia, there have been a dozen additional summits, with attendance growing at each one.
One of the attendees at that first summit was Rachel Katzman, a Connecticut mother of three whose son Blake, now 7, had been diagnosed with an inhibitor when he was 18 months old. At first he hadn’t had too much trouble with bleeding, but around age 4, his meds stopped working. “At one point he had two elbow bleeds and two calf bleeds, so he didn’t have any use of his extremities. Blake was in and out of the hospital for four months until they could get his bleeding under control,” Katzman says. “Now he’s finally responding to his factor VIII, but it was a difficult three years. And it was lifesaving to have the summits. They felt like home, being with people who understand what you’ve been through.”
The summits proved to be eye-opening not just for patients, but also for the medical professionals, social workers and other leaders in the field who conducted the program. “I’ve been in this field for 15 years, and I still feel like a student myself in this setting with the patients and families, which is so much more different from the clinic,” says Angela Forsyth, PT, DPT, clinical team leader and physical therapist at the University of Pennsylvania Hemophilia and Thrombosis Program in Philadelphia. “I’ll learn about little things that might have helped a patient at the summit—a certain type of shoe, or a particular adjunct pain management tool like a focal compression device combined with cold—and then bring it back into my practice.”
“The biggest eye-opener for me with these summits was how different the care is that patients get, depending on where they are,” says Guy Young, MD, director of the Hemophilia Treatment Center (HTC) at Children’s Hospital Los Angeles and a co-chair of the program series. “Some of it was, frankly, shocking. We discovered that about 50% of patients who really should have been offered immune tolerance therapy, which we consider standard for the vast majority of patients with inhibitors, had never heard about it. I had assumed that everybody was treated in a major HTC, and everybody got top-level care. That’s not how it is, and that fact makes the information we offer at the summits all the more urgent.”
Katzman was one of the parents who got new insights into immune tolerance therapy at the Philadelphia summit. “I learned how much you can really dose your child and that we were allowed to do five times the normal dose,” she says. “There were some very good doctors there, and it gave me a variety of viewpoints to learn from, not just my own doctor. I came back and talked with our doctor about what I had learned, including the possibility of increasing Blake’s dosage, and asked if we could try it. Thanks to the summit, I could talk with our doctor about choices and options, and we both learned together.”
Although families greeted the pilot summit with enthusiasm, they quickly began offering input as to how things could be better. “Typically, we as medical professionals like to put up a PowerPoint slide show and give a presentation, and that’s how we approached things at the beginning,” says Forsyth. “But the families quickly taught us that we have to realize that we are not the teachers. We are the facilitators, and the people in the room are the teachers. We need to bring everyone’s information out and use it to enrich our sessions.”
Charles Gilbert, LCSW, a clinical psychiatric specialist for the Hemophilia Treatment Center of Central Pennsylvania, found that workshops at the pilot summit didn’t end when their time was up. “People would spend the next hour with you, trying to pick your brain. ‘What have you heard about this?’ ‘I have this problem, what can I do?’ They were not eager to participate where there was a gulf between the ‘expert’ presenters and themselves,” he recalls. “They didn’t want the directive lecturing we employed at the beginning; they wanted real solutions and the opportunity to speak.”
The early agendas also ran “hot and cold,” says Katzman. “For some people, the session would be too basic, and for others, it was too over their heads. But during the second and third years, they began to adapt the content to suit both beginners and veterans. They put the basics early on in the agenda, in the ‘Summit Safari’ for new attendees. If you didn’t need it, you didn’t have to go.”
The Summits Evolve
By their third year, the Inhibitor Summits had evolved into an advanced program, with plenaries and concurrent breakout sessions offering a wide variety of educational opportunities—and the audience involvement that participants had been clamoring for. “I’m happy to show just a slide or two with pictures of certain activities, braces, assistive devices and minimal text, and just go where the session takes us,” says Forsyth. “Participants may ask questions about certain things, and I can pull up information to support that, but my sessions have become completely interactive.”
What was often most valuable, says Pezzillo, wasn’t just the medical information, although attendees welcomed that. They soaked up the input from experts and other attendees about real-world, practical matters like assistive devices, coping with pain, traveling (imagine showing up at the airport and going through security with a giant cooler full of factor), and dealing with insurance.
“Insurance is huge for us, because hemophilia is one of the most expensive chronic conditions to manage,” says Pezzillo. “Fortunately, now that healthcare reform has passed, there will be no more lifetime caps, but before that happened, we were stuck. In February 2004, my insurer paid close to half a million dollars for my care. I blew through most people’s lifetime insurance caps in a month.”
That year also marked another milestone: the first program for teens with inhibitors. “It’s hard to put together a program for teens,” says Young. “At my center, I have about 18 inhibitor patients, and that only includes about three or four teens. Most other places have one teen, if any. This is just about the only place where they can all come together and talk with other youth about what matters to them.”
“I know a guy who’s 16 years old and has already had 10 surgeries on his elbows and knees,” says Pezzillo. “He can’t go to school, so he’s being home schooled and is missing out on major aspects of growing up. Kids like him have a whole set of unique needs that need to be met. When you’re in a wheelchair at a regular hemophilia meeting and most guys your age aren’t, you feel isolated even there. But at the inhibitor summits, they’re not the odd ones out. Adolescence is hard enough without this on top of it; these kids need an environment where they feel safe, so they can be open about what they go through.”
Growing Attendance and Changing Content
By 2008 and 2009, the Inhibitor Education Summits had grown even further, with attendance topping more than 550 in 2009. As they grew, says Forsyth, the summits began offering more choices and more faculty covering more disciplines. “We also branched out to use non-hemophilia professionals to bring in information from their specialties,” she adds. “For instance, we have a pain specialist involved. His pain management expertise is perfect for our patients, even though he’s not specifically a hemophilia expert.”
“There’s always something more that we learn,” says Kelly Millette, an Illinois mother whose 12-year-old son Christopher has had his inhibitor since he was 2. “I learned at one of the summits about other patients with inhibitors getting a von Willebrand factor that has proven helpful for some patients with inhibitors, and it’s made a difference for Christopher. We’ve learned different techniques for caring for joints and surgery options. And now that Christopher is older, he loves going. He likes being with other kids like him and knowing he can go to the pool and no one’s going to look at him weird when they see his scars.”
But some participants were concerned that the content of the meetings had begun to grow stale. “I talked to a number of parents after the 2009 summit who were frustrated, because the information had gotten repetitive,” says Wilkes. “So for the 2010 summit, we have a track-based program, with individualized content. We’re doing a better job of making the information pertinent to specific groups within the community.”
The four tracks are:
- Hemophilia with Inhibitors 101: for families new to inhibitors
- Hemophilia with Inhibitors 201: for veteran families seeking more in-depth content
- Young Men with Hemophilia with Inhibitors: focused on the specific psychosocial issues faced by young men with a bleeding disorder as they move from adolescence to adulthood
- Men with Hemophilia with Inhibitors: designed to address the specific needs of adult men with hemophilia, such as issues of aging, pain management and intimacy.
Other changes have come to the summits within the past year as well. Last year’s Los Angeles summit was the first to be filmed and Web cast, so that families who cannot attend the program can still learn from online sessions. On-site Spanish-language translation has been a feature since 2006, although that’s a much more straightforward task in plenary sessions than in breakouts, says Young. “I work with a lot of Spanish-speaking patients, and there is now a pilot Spanish-only summit that we’re working on.” Conferencia Educativa Sobre Inhibidores will be held in Miami, FL, June 18–20, 2010, and will be the first Spanish-language educational activity for families of people with hemophilia with inhibitors.
The summits’ popularity has also helped to fuel a movement for more educational materials aimed at patients with inhibitors and their families. “The summits created a critical mass, and they know they have an audience,” says Wilkes. “Now the NHF, Novo Nordisk, and other companies are putting together materials that respond to our needs—we’ve gone from two brochures to more than a dozen—and they’ve relied on parents and people living with hemophilia with inhibitors to guide what they will look like.”
And inhibitor families have become more visible at general hemophilia-related forums. In 2004, the “inhibitor rap session” at the NHF Annual Meeting drew three participants. By 2009, says Wilkes, “We had a roomful.”
There are a number of challenges that the summits still must take on, though. One, says Young, is finding inhibitor patients who have not yet had a chance to participate. “We’d like to see every patient attend at least one summit. There are a number of patients with inhibitors out there who are cared for by community hematologists, and we still don’t entirely know how to reach them.”
And there’s always the issue of keeping content fresh and relevant for all participants. Katzman says she would like to see more content for other family members. “The bleeding disorder isn’t just about the child with the condition; it’s about the whole family,” she says. “The siblings are often affected the most, because they’re just not thought of. And the spouses of men with hemophilia are rarely thought of—that they might have needs. We need to have more rap sessions that allow groups like these to speak out.”
But whatever new offerings and programmatic changes they may suggest, families and professionals alike agree that the Inhibitor Education Summits have revolutionized the way patients with inhibitors and their families are treated, and the way they see themselves. “The inhibitor community was once the ‘ugly little stepchild’ of the hemophilia world,” says Wilkes. “I truly thought that we were the only ones. We weren’t ignored on purpose, but they just didn’t know what to do with us. But now, the education summits have empowered us, and the sense of community is huge.”
The NHF Inhibitor Education Summits are supported by an educational grant from Novo Nordisk.