Voices for Change in the Bleeding Disorders Community

Joey Smiles: A Perfect Match

When Joey Smiles, 44, was growing up in the 1980s and ’90s, treatment for hemophilia was “on-demand,” as he puts it. He was using factor only after an injury or after feeling symptoms of a spontaneous bleed. Major bleeds required multiple infusions every 12 to 24 hours to resolve.

Smiles, who lives near Scranton, Pennsylvania, eventually became a biochemist, working in vaccine research and development at a pharmaceutical company. He felt like he had his hemophilia under control at that point. Unfortunately, Smiles went on to experience two major, life-threatening bleeding incidents and continues to have chronic medical problems. He now takes a prophylactic treatment regimen with a prolonged half-life clotting factor. “I’m having no bleeds now,” he says.

His chronic medical problems make it impossible for him to return to the laboratory or even allow him to work a normal schedule. Rather than become depressed or frustrated, Smiles took a different route. “My struggles with hemophilia have allowed me to develop a superpower — adaptability. The reason I’ve been able to lead a semi-normal life is directly related to the miracles of modern medicine,” Smiles says. “So, I wanted to give back. Being no longer able to make my contribution from my lab bench, I knew I needed to adapt, and that’s what ultimately led me to pursuing advocacy.”

Smiles has been advocating on behalf of people with hemophilia and those with other inheritable blood and bleeding disorders on the state and federal levels with the Eastern Pennsylvania Bleeding Disorders Foundation.

“It was a perfect match because I have experiences from some of the darkest times in hemophilia’s history,” he says. “I think that brings a lot of perspective when you’re telling lawmakers and the public about the whole history of hemophilia.”

Thanks to advances in science and medication, Smiles says the problems he’s encountered are never going to happen to others, as long as people continue to have access to medication. “Medicine for blood disorders is extremely expensive. It’s bad enough to just have hemophilia, but not having your lifesaving medication available makes an already bad situation so much worse,” Smiles says. “To us, it matters a great deal what’s going on with health care, and what bills are being introduced on the state and national levels because it could affect us directly.”

Smiles, like many people with bleeding disorders, understands that not everyone is comfortable revealing that they have a bleeding condition, but he urges people to be open about it. “You have a unique voice,” he says, “and telling your story helps all of us in the bleeding disorders community.”

Fabiola Danastorg Cartagena: Finally Understood

Fabiola Danastorg Cartagena, 30, didn’t receive a diagnosis of type 1 von Willebrand disease until she was in her early 20s. Until that point, she had been bleeding excessively during her menstrual periods for more than a decade. “I used to have to wear diapers because the flow was so heavy,” she says.

The bleeding took a toll on her life, leading to absences from school and work. She also had to deal with having her concerns dismissed. She says that in general people don’t consider period problems a legitimate excuse for missing work or school. “It got to a point where I thought it was all in my head,” she says.

Danastorg Cartagena had been to several gynecologists in search of a diagnosis, but it wasn’t until she was hospitalized at one point that a hematologist confirmed she had von Willebrand disease. She now has a Mirena IUD, known to be effective at treating women with bleeding disorders. She says the intrauterine device has diminished her blood loss by 85%.

For the past four years, Danastorg Cartagena has been involved with the Puerto Rico Association of Hemophilia and Bleeding Disorders. She started as a volunteer and is now secretary of her chapter’s board. She also helped create a women’s committee and participates in support groups that specifically address issues that affect women. “In one meeting, all we talked about was Mirena,” she says.

Danastorg Cartagena encourages women with bleeding disorders to attend meetings and support groups and to get involved with their local chapters. “You meet people you can relate to, and you realize you’re not alone. If you’ve been misunderstood, you’ll be understood. It’s a good opportunity to speak up and leave being ashamed at the door,” she says. “You also get to see how important fundraising is and that people are working hard for others not to go through what we’ve been through.”

Bobby Wiseman: A Life-Changing Connection

Bobby Wiseman believes advocacy comes in many forms, from the personal to the political. As such, the 52-year-old has served in many capacities as an advocate and has been involved in numerous bleeding disorders organizations over the years.

In 2018, Wiseman, who has severe hemophilia B, participated in a gene therapy clinical trial he says has changed his life and helped advance the science. His hematologist had been keeping an eye out for an appropriate trial. While attending a hemophilia symposium, Wiseman met with the point of contact for a company doing a gene therapy trial.

“I hopped on the phone, called my doctor, and the two of them connected. The hospital got the paperwork in place, and I was selected for the trial. It was communication, relationship building, and advocacy that made it happen,” he says.

The gene therapy, called Hemgenix, was cleared by the Food and Drug Administration in November 2022. It has been remarkably effective for Wiseman, who hasn’t had to have factor due to a spontaneous bleed in five years. Since starting gene therapy, his factor levels typically range between 52% and 62%.

Wiseman also used the opportunity to make recommendations on how to improve the overall trial selection process to include more diverse study participants and reduce bias. “I am interested in giving the patient perspective as well as the system perspective,” he says.

These days, Wiseman, who lives in Tucson, Arizona, devotes his time to Potential of Ability, a nonprofit he founded to help teens and young adults with the various intersectionality of the social determinants of health, and Rising Beyond, where he provides strategic planning and program enhancement. He has one child, 12 godchildren, nieces and nephews, and he became a grandfather in January 2023.

Briana Reinking: Lobbying for Insurance Reform

Briana Reinking, former chair and current advocacy committee member of the National Bleeding Disorders Foundation’s Colorado chapter, became involved in advocacy after her son was diagnosed with hemophilia. A few years later, her second child was also diagnosed.

Reinking, 45, says her primary focus has been on health insurance issues. “We had several years without any insurance problems,” she says. “Then our plan changed. Trying to understand the plan itself and what the cost of the medications would be was so challenging that it got me interested in the behind-the-scenes policymaking and rules.”

The chapter works with the Chronic Care Collaborative, a coalition of advocacy groups, which Reinking says has allowed them to have a more prominent voice in the Colorado legislature. In 2022, they were able to help pass a bill prohibiting step therapy.

Step therapy is the term used when an insurance company requires people to try the least expensive drug for their condition first; if the drug doesn’t work, they may be approved to try a different and often more expensive medication.

“It doesn’t allow the patient and the doctor to make the decision about the best medication,” Reinking says. “It puts it in the hands of the insurance company and may force someone to try a less effective drug prior to trying one that works. For people with bleeding disorders, taking a less effective drug can cause bleeding episodes that can lead to long-term joint and muscle damage.”

The Colorado chapter and the collaborative also helped support legislation to prohibit copay accumulators. “To have those two bills passed has been a really good outcome for the community on a couple of pretty challenging issues,” Reinking says. “We’ve put in a lot of work over many years to build those relationships at the Capitol.”

She also stresses how important it is to put a human face on the insurance obstacles with which families grapple.

“Families are often unable to obtain the medication they need, or they have extremely large expenses for medication they’re unable to pay for,” she says. “Sometimes they have to make the choice between paying for the medication and paying other bills. Having legislators see how this is actually impacting people in their community is so important.”

Reinking recalls bringing her son with her to a meeting with a legislative aide in Washington, D.C. “I was explaining the challenges we’d had, but I really was not connecting with him,” she says. “Then my son showed the aide pictures of himself playing soccer and learning to rock climb, and the aide kind of lit up and connected with my son. He understood that we’re really just trying to get him medication so he can lead as close to a normal life as possible and just be a kid. Legislators are really just people, and they want to hear your story.”