When 9-year-old Wiley Moran was diagnosed at birth with hemophilia, a disease that prevents blood from clotting properly, it wasn’t a surprise to his mom, Katharine, who found out she was a carrier when her older son, Logan, was diagnosed with it at age 5.
According to Dr. John Fargo, a pediatric hematologist-oncologist in the Showers Family Center for Childhood Cancer and Blood Disorders, clotting helps stop bleeding after a cut, injury or surgery. If clotting doesn’t happen, a wound can bleed too much.
“In order for our body to make a clot and stop ongoing bleeding, there are a number of interactions between blood cells and clotting proteins that need to happen,” he said. “These clotting proteins called factors, and the different factors involved in making a clot, are designated by a number. Individuals with hemophilia A have a low or absent factor 8. The severity of bleeding in a person with hemophilia A depends on how low factor 8 is. For Wiley, who has severe factor 8 deficiency, he can bleed spontaneously without any injury or surgery.”
Wiley experienced his first bleed at age 2 and after that was frequently hospitalized for treatments. A current third grade student at Seiberling CLC in Akron, Wiley missed 76 days of school when he was in kindergarten and 60+ days in first grade. Katharine says frequent blood stream infections from his 4 Mediports and 9 PICC lines contributed to the problem.
“I had to give him daily factor to try and get rid of the inhibitors,” she said. “This involved setting up a sterile field, putting a mask on and having someone help hold him down while he cried and screamed. The whole process could take up to 35 minutes.”
“Since individuals with severe hemophilia A require frequent intravenous infusions with factor we use central venous catheters, like ports, to be able to give the factor medicine regularly and reliably,” said Dr. Fargo. “However, ports and other central venous catheters come with their own risk and increased blood stream infections is one of them.”
Because Wiley has experienced a lot of bleeds in his joints, specifically his ankles, he has developed early arthritis and wears ankle braces to help support him and prevent more bleeds. Katharine says a particularly scary time was when Wiley was 4 years old and experienced a spontaneous epidural bleed in his neck.
“It was scary because he could have become paralyzed or died,” she said. “After consulting with the neurology team it was decided not to pursue surgery, but he had to have neuro checks every 2 hours around-the-clock. After a 29-day hospitalization, the bleed finally went away with the help of factor.”
Akron Children’s became like a second home to the Martins during Wiley’s often week-long stays for bleeds that occurred at least once a month. During this time Katharine was unable to work, and Wiley’s school absences continued to pile up.
Dr. Fargo says Hemlibra is a unique medication because it is not considered a factor protein, but instead an antibody.
“Antibodies are widely used in different medical conditions. In fact, our body makes its own antibodies to fight off infections,” he said. “In Hemlibra’s case, the antibody resembles and can act like the factor 8 protein and can help the body make a clot and stop bleeding – revolutionizing treatment in individuals with hemophilia A and inhibitors.”
Akron Children’s began using Hemlibra in early 2018, and Wiley was the first patient to be placed on it. Katharine admits she was nervous when it was first proposed as a treatment for Wiley.
“It was a brand-new drug, so I did weeks of research before agreeing to start it,” she said.
Dr. Fargo says Hemlibra made sense for Wiley.
“He has hemophilia A with inhibitors and had multiple hospitalizations and complications relating to failure of previous treatments,” he said. “It was time to change his life and not have hemophilia control it anymore.”
It’s been a little over 2 years since Wiley started Hemlibra, and Katharine says he is a different kid.
“He smiles more, laughs more and is happy,” she said. “He used to be depressed and withdrawn.”
Katharine says giving him the medication is also far easier.
“Instead of daily port access, he now gets an injection right under the skin once a week that I can do at home with far less risk of infection.”
This year Wiley has only missed 9 days of school and none of them were due to hemophilia. Katharine was also able to return to work.
“Wiley is on the honor roll at school and gets straight A’s,” she said. “Math is his favorite subject.”
Inspired by Wiley’s journey, Katharine has also been thinking about returning to school.
“I’m not sure yet, but I’m thinking about a career in the health care field.”
Dr. Fargo says the overall prognosis for individuals with hemophilia A is good in the current era of treatment.
“Prior to the 1980s those with hemophilia suffered from debilitating joint disease (from recurrent bleeding into the joints) and, unfortunately, complications related to treatment at that time such as HIV and hepatitis contracted from untreated blood products,” he said. “This is an extremely exciting time for our patients affected by bleeding disorders because treatments available now, or ones that are in the pipeline, are changing the shape of these disorders for the better.”