Future looks bright for boy with rare disease

02/27/2022

By Dave Kurtz, KPC News

This is a happy story … even though it’s about a little boy with severe hemophilia, who is learning to give himself shots in his thigh at a tender age ... his mother assures us.

Paul Herman, now 7, was diagnosed with Type A Hemophilia shortly after birth.

His parents were lucky in one sense. Due to family history, they suspected that Paul might suffer from hemophilia. They were ready to test him right away. They didn’t discover his condition as a nasty surprise in early childhood.

“We knew there would be a chance. … We had a plan in place so we would know quickly” if Paul had hemophilia, his mother, Jaime Herman, said last week from the family’s home in Berne.

The Indiana Hemophilia and Thrombosis Center provided a test kit that Jaime and her husband, Jarrod, could send to its headquarters in Indianapolis right away. Paul has been under the center’s top-notch care since his very first day.

Thanks to his treatments, “At 7 years old, he’s never had a joint bleed, and that’s something, 30 years ago, that a kid at 7 years old couldn’t say,” Jaime Herman said.

Paul is growing up in Adams County, but he represents children with rare diseases all across the state. Tomorrow is Rare Disease Day, calling attention to their needs and to the specialists who are serving them with skill and compassion.

A rare disease is one that affects fewer than 200,000 Americans — but with such a wide variety of rare ailments, the number of patients and affected families becomes significant.

Medical advances already have benefited Paul during his young life. In his early treatment, he received an infusion of a blood-clotting factor every 2-3 days through a port in his chest.

Two years ago, Paul began using a new therapy delivered through an injection he takes only every other week.

Earlier this month, Paul gave himself the shot for the first time.

“Not many adults can do that,” his mother said. “As he grows up, mom’s not always going to be there. … He needs to be able to do this.

“These kids are super brave, they are super tough,” Jaime Herman said about her son and other children with hemophilia. “My son is much tougher than most adults I see.”

Paul’s new therapy gives him an antibody developed through “amazing engineering,” said Dr. Charles Nakar, his pediatric hematologist with the Indiana Hemophilia and Thrombosis Center. The nonprofit clinic for blood disorders is considered one of the best of its kind in the nation.

The genetically engineered antibody mimics a factor needed for blood to coagulate and form clots — a factor that Paul and other hemophilia patients are missing.

“This is a big, big change for kids with hemophilia,” Nakar said. “The goal is to have our kids have completely normal lives. … I want them to feel that the sky’s the limit. I think we are closer to that goal.”

In Paul’s life, that means he can play tee ball, an activity that would have been out of the question for hemophiliac children in the past.

“With Paul’s treatment, he’s able to do these things,” his mother said. “They told him he can actually try basketball. … He wants to try soccer.”

His medication allows Paul to safely climb up into the tractor cab and play on a farm run by his grandfather and great uncle.

This summer, Paul will attend Camp Brave Eagle, sponsored by the IHTC for children with blood disorders. He’ll learn tips about how to manage his hemophilia, but mostly he’ll have fun swimming, sailing and fishing on a lake near North Webster under the watchful eyes of a highly trained staff.

Most young hemophilia patients are living wonderful, active lives thanks to medical advancements, Nakar said.

“One of the goals for the future is to find a cure,” Nakar said. “When we talk about cure, we talk about gene therapy” that will manipulate liver cells to produce clotting factors.

“I believe that in the next few years, we’ll have significant additional improvement” for hemophilia treatment, Nakar said.

The IHTC will be there for Paul and other patients. It conducts regular clinics throughout the state — including sites in Fort Wayne and LaGrange. It employs nurses in the Shipshewana and Berne areas to extend care to Indiana’s Amish communities.

“If we have an issue … all I have to do is pick up the phone and call them” to reach a doctor on staff around the clock, Jaime Herman said.

So, yes, this is a happy story for Paul and children like him.

“He’s going to look like a normal kid,” Paul’s mother says with confidence. “Paul’s getting to do a lot of things that a kid his age wouldn’t have done 30 years ago” with hemophilia. “He’s got a bright future. He’s not experienced horrible bleeds that have caused him not to be able to move his ankle joint … or his knees.

“I wouldn’t wish this on him, but he’s got the right tools,” she said. “He’s got the best doctors that are taking care of him and pushing him in the right direction.”

Source: KPC Media